Inmune Bio, Inc. Stock price
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Key metrics
📘 Market Capitalization
📈 What is it?
Market capitalization shows how much a company is currently worth on the stock market.
🧮 How is it calculated?
🏛️ Why is it important?
It helps classify companies by size (Large, Mid, Small Cap) and indicates their market presence and relative stability.
🧮 Calculation
🎯 What does this mean for investors?
- Large-cap companies tend to be more stable, often pay dividends, but may grow more slowly.
- Smaller firms may offer higher growth potential but come with more volatility.
- Market capitalization is a useful indicator of company size — but not a measure of whether a stock is undervalued or overvalued.
📘 Enterprise Value (EV)
📈 What is it?
Enterprise Value represents the total cost to acquire a company — including its debt and excluding its cash reserves.
🧮 How is it calculated?
(= Market Cap + Net Debt)
🏛️ Why is it important?
EV gives a more complete picture of a company's value than market cap alone and is used in key valuation ratios like EV/FCF or EV/Sales.
🧮 Calculation
🎯 What does this mean for investors?
- Enterprise Value shows the true cost of buying a company, including all financial obligations.
- It is more accurate than just looking at market cap, especially when comparing companies with different levels of debt or cash.
- Professional investors prefer EV-based multiples because they better reflect the company’s full financial footprint.
📘 Net Debt
📈 What is it?
Net Debt shows how much debt remains after subtracting a company’s available cash reserves.
🧮 How is it calculated?
🏛️ Why is it important?
It indicates how dependent a company is on borrowed money and how easily it can service its debt in the short term.
🧮 Calculation
🎯 What does this mean for investors?
- Low or negative net debt signals financial strength and flexibility.
- Companies with strong cash positions are better positioned in crises.
- High net debt increases financial risk — especially in environments with rising interest rates or economic downturns.
📘 Cash
📈 What is it?
Cash represents all liquid assets a company can access immediately — including cash, bank deposits, and short-term investments.
🧮 How is it calculated?
🏛️ Why is it important?
It reflects a company’s financial flexibility and resilience — enabling investments, buybacks, or buffer in downturns.
🧮 Calculation
🎯 What does this mean for investors?
- A strong cash position means greater room for maneuver and crisis resistance.
- Cash-rich companies can invest, pay down debt, or repurchase shares.
- But excess idle cash might indicate a lack of growth opportunities.
📘 Shares Outstanding
📈 What is it?
Shares outstanding represent the total number of a company’s shares currently held by investors — excluding treasury stock.
🧮 How is it calculated?
🏛️ Why is it important?
It’s the basis for key metrics like Earnings Per Share (EPS), Market Capitalization, or the Price/Earnings ratio (P/E).
🧮 Calculation
🎯 What does this mean for investors?
- Fewer shares in circulation typically increase earnings per share — making each share more valuable.
- Share buybacks reduce the number of shares and boost per-share metrics.
- Issuing new shares does the opposite — diluting shareholder value and lowering per-share figures.
📘 Price-to-Earnings Ratio (P/E)
📈 What is it?
The P/E ratio shows how many times a company's earnings per share are reflected in its current share price — in other words, how "expensive" the stock appears relative to its profits.
🧮 How is it calculated?
🏛️ Why is it important?
The P/E ratio is one of the most widely used valuation metrics. It helps investors assess whether a stock appears cheap or expensive compared to its earnings power.
🧮 Calculation
📊 P/E (TTM) = Based on earnings from the last 12 months (Trailing Twelve Months):🎯 What does this mean for investors?
- A low P/E may indicate undervaluation — or signal underlying issues.
- A high P/E may reflect strong growth expectations — or an overvalued stock.
📘 Price-to-Sales Ratio (P/S)
📈 What is it?
The P/S ratio shows how much investors are paying for $1 of the company’s revenue – regardless of profitability.
🧮 How is it calculated?
🏛️ Why is it important?
P/S is especially useful for evaluating growth companies or businesses not yet profitable. It reflects how the market values the company’s sales.
🎯 What does this mean for investors?
- A low P/S may indicate undervaluation — or low profitability.
- A high P/S can reflect strong growth expectations — or excessive optimism.
- Especially helpful when evaluating companies where profits are low, volatile, or negative.
📘 Enterprise Value to Sales (EV/Sales)
📈 What is it?
EV/Sales shows how much investors are paying for $1 of revenue — considering not just equity, but also debt and cash. It’s the capital structure–adjusted version of the P/S ratio.
🧮 How is it calculated?
🏛️ Why is it important?
It’s ideal for comparing companies with different levels of debt. It reflects a company's true cost relative to its revenue.
🎯 What does this mean for investors?
- EV/Sales allows for capital structure–neutral company comparisons.
- A lower ratio may indicate undervaluation; a higher one may signal strong growth expectations or overvaluation.
- Especially helpful when evaluating high-growth companies with low or negative earnings.
📘 Enterprise Value to Free Cash Flow (EV/FCF)
📈 What is it?
EV/FCF shows how many years it would take for a company to "pay back" its enterprise value using its free cash flow.
🧮 How is it calculated?
🏛️ Why is it important?
It focuses on real cash generation, ignoring accounting noise — ideal for assessing profitability and value based on liquidity, not earnings.
🧮 Calculation
🎯 What does this mean for investors?
- A low EV/FCF may signal undervaluation and strong cash generation.
- A high EV/FCF might reflect weak recent cash flow or aggressive growth expectations.
- Best suited for stable, mature businesses with predictable free cash flows.
📘 Price-to-Book Ratio (P/B)
📈 What is it?
The P/B ratio compares a company’s market value to its book value — showing how much investors are paying for each dollar of net assets.
🧮 How is it calculated?
🏛️ Why is it important?
P/B is commonly used for asset-heavy industries like banks or industrials. It helps assess whether a stock is trading above or below its net asset value.
🧮 Calculation
🎯 What does this mean for investors?
- A P/B below 1 may signal undervaluation — or weak profitability.
- A P/B above 1 implies the market expects future value creation (e.g., brand, IP, growth).
- Best used for companies with tangible assets and strong balance sheets.
📘 Equity Ratio
📈 What is it?
The equity ratio indicates what portion of a company’s total assets is financed by shareholders’ equity – in other words, how much it relies on its own capital.
🧮 How is it calculated?
🏛️ Why is it important?
A high equity ratio reflects financial strength and stability, especially during downturns. It’s a key indicator of a company’s solvency and long-term risk profile.
🧮 Calculation
🎯 What does this mean for investors?
- Companies with high equity ratios are generally more resilient and less dependent on external debt.
- Low equity ratios can signal higher risk or aggressive financial strategies.
- Important: Always assess the equity ratio in combination with the return on equity (ROE). This shows not just how stable the company is – but also how efficiently it uses shareholder capital.
📘 Return on Equity (ROE)
📈 What is it?
Return on equity (ROE) shows how efficiently a company uses its shareholders’ equity to generate profit. In other words: how much net income is earned per dollar of equity.
🧮 How is it calculated?
🏛️ Why is it important?
ROE is a core profitability metric. It helps investors understand whether a company delivers attractive returns on the capital provided by its shareholders.
🧮 Calculation
🎯 What does this mean for investors?
- A high ROE indicates that the company is using its capital efficiently and profitably.
- It’s especially meaningful for capital-intensive businesses or firms with high equity bases.
- Important: A very high ROE can also result from high debt levels – always interpret it alongside the equity ratio to assess financial health.
📘 Return on Capital Employed (ROCE)
📈 What is it?
ROCE measures how efficiently a company generates profits from its total capital – including both equity and interest-bearing debt.
🧮 How is it calculated?
It evaluates the return on all capital employed, regardless of how it’s financed.
🏛️ Why is it important?
ROCE is ideal for comparing companies with different financing structures. It shows how well management uses capital to create value for both shareholders and creditors.
🧮 Calculation
🎯 What does this mean for investors?
- A high ROCE means the company uses its capital efficiently – regardless of whether it's funded by debt or equity.
- The higher the ROCE compared to peers, the more value the company creates with its invested capital.
- Especially relevant for capital-intensive sectors like industrials, energy, or infrastructure.
📘 Return on Invested Capital (ROIC)
📈 What is it?
ROIC measures how efficiently a company generates returns from the capital invested in its core operations – regardless of whether the capital comes from equity or debt.
🧮 How is it calculated?
- NOPAT = Net Operating Profit After Taxes
- Invested Capital = Operating assets minus non-interest-bearing liabilities
🏛️ Why is it important?
ROIC is one of the most accurate indicators of capital efficiency. Unlike return on equity, it is not distorted by leverage and shows how much value is created for all capital providers.
🎯 What does this mean for investors?
- A high ROIC shows how effectively a company uses the capital that is truly invested in its core operations.
- Unlike ROCE, ROIC focuses only on the capital that is actively used to run the business – and that requires a return (i.e. interest-bearing).
- Especially useful when comparing companies with large amounts of excess cash or non-interest-bearing liabilities – giving a more realistic picture of capital efficiency.
📘 Leverage Ratio (Debt-to-Equity)
📈 What is it?
The leverage ratio indicates how much a company relies on interest-bearing debt (such as loans and bonds) relative to its shareholders’ equity.
🧮 How is it calculated?
🏛️ Why is it important?
This ratio helps assess a company’s financial structure and risk profile. High leverage can enhance returns – but also increases exposure to interest rate changes and financial stress.
🧮 Calculation
🎯 What does this mean for investors?
- A low leverage ratio signals financial strength and independence.
- A higher ratio can improve returns in good times but increases risk during downturns or rising interest rate periods.
- 👉 Always interpret in the context of industry, capital intensity, and interest rate environment.
📘 Revenue
📈 What is it?
Revenue shows how much a company earns in total from selling its products and services – the gross income before any costs are deducted.
🧮 How is it calculated?
🏛️ Why is it important?
Revenue is one of the key figures to assess a company’s size, market position, and growth potential.
🧮 Calculation
🎯 What does this mean for investors?
- Growing revenue indicates rising demand and can be an early signal of future earnings growth.
- Comparing actual and expected revenue reveals trends in the market environment and analyst sentiment.
- Note: Strong revenue alone isn’t enough – margins and profitability matter just as much.
📘 EBITDA
📈 What is it?
EBITDA stands for “Earnings Before Interest, Taxes, Depreciation, and Amortization.” It reflects a company’s operating profit before the effects of financing, taxes, and accounting depreciation.
🧮 How is it calculated?
🏛️ Why is it important?
EBITDA is widely used to evaluate a company’s operating performance – especially across capital-intensive sectors or international comparisons.
🧮 Calculation
🎯 What does this mean for investors?
- A high or growing EBITDA indicates strong operational profitability – independent of taxes, interest, or accounting methods.
- It’s especially useful for comparing companies across sectors or geographies.
- Important: EBITDA is not a net income figure – it excludes key costs like depreciation and interest.
📘 EBIT
📈 What is it?
EBIT stands for “Earnings Before Interest and Taxes.” It reflects a company’s operating profit after depreciation, but before interest and tax expenses.
🧮 How is it calculated?
🏛️ Why is it important?
EBIT is a core profitability metric that shows how well the company performs in its main business operations – independent of capital structure and tax environment.
🧮 Calculation
🎯 What does this mean for investors?
- A high EBIT indicates strong profitability from the company’s core business – before financial and tax effects.
- It allows better comparison between companies with different debt levels or tax structures.
- Compared to EBITDA, EBIT already accounts for depreciation and reflects capital intensity more clearly.
📘 Net Income
📈 What is it?
Net income is the company’s total profit – the amount left after all expenses, taxes, interest, and depreciation have been deducted.
🧮 How is it calculated?
🏛️ Why is it important?
Net income is the most comprehensive measure of a company’s profitability – showing how much actual profit remains after all business and financing costs.
🧮 Calculation
🎯 What does this mean for investors?
- Growing net income indicates that the company is managing all of its costs efficiently.
- It directly influences valuation metrics like P/E ratio and the company’s dividend capacity.
- Over time, net income trends reveal how resilient and profitable the business model really is.
📘 Free Cash Flow (FCF)
📈 What is it?
Free Cash Flow shows how much actual cash remains after a company covers its operating expenses and capital expenditures.
🧮 How is it calculated?
🏛️ Why is it important?
FCF reflects a company’s real financial strength – regardless of accounting profits. It shows how much flexibility a company has for dividends, share buybacks, or debt reduction.
🧮 Calculation
🎯 What does this mean for investors?
- High free cash flow means the company generates real, usable cash – independent of reported net income.
- It’s often the most reliable base for sustainable dividends and buybacks.
- Declining FCF can be an early warning sign – even when profits appear stable.
📘 Revenue Growth
📈 What is it?
Revenue growth shows how much a company’s sales have changed compared to the previous year – both on a trailing basis (TTM) and based on forward projections.
🧮 How is it calculated?
Forward = (Expected revenue ÷ Revenue in prior year − 1) × 100
Forward growth is based on analyst estimates for the current fiscal year.
🏛️ Why is it important?
Rising revenue signals growing demand, business expansion, and market share gains – especially important for growth-oriented companies.
🧮 Calculation
🎯 What does this mean for investors?
- Growth is the engine of long-term value creation – especially in tech and growth sectors.
- What matters is not just current growth, but its sustainability.
- Forward projections reflect whether analysts expect continued momentum – or a slowdown.
📘 EBITDA Growth
📈 What is it?
EBITDA growth shows how much a company’s operating profit (before interest, taxes, depreciation, and amortization) has increased or decreased compared to the previous year.
🧮 How is it calculated?
Forward = (Expected EBITDA ÷ EBITDA from prior year − 1) × 100
The forward estimate is based on analyst projections for the current fiscal year.
🏛️ Why is it important?
Growing EBITDA indicates improving operational profitability – regardless of financing or accounting effects.
🎯 What does this mean for investors?
- Strong EBITDA growth signals operational efficiency and scalability – especially during growth phases.
- EBITDA growth can be an early indicator of margin and earnings expansion – but should be assessed alongside revenue and EBIT.
📘 EBIT Growth
📈 What is it?
EBIT growth shows how much a company’s operating profit (after depreciation, but before interest and taxes) has increased compared to the previous year.
🧮 How is it calculated?
Forward = (Expected EBIT ÷ EBIT from prior year − 1) × 100
The forward estimate is based on analyst projections for the current fiscal year.
🏛️ Why is it important?
EBIT growth is a direct indicator of a company’s business performance – taking into account capital intensity through depreciation.
🧮 Calculation
🎯 What does this mean for investors?
- Rising EBIT signals improving operating profitability – even after accounting for depreciation.
- It’s especially important for evaluating companies with significant capital expenditures.
- Combined with revenue and EBITDA growth, EBIT growth provides a well-rounded view of operational progress.
📘 Net Income Growth
📈 What is it?
Net income growth shows how much a company’s bottom-line profit has increased or decreased compared to the previous year – both on a trailing basis (TTM) and based on analyst projections.
🧮 How is it calculated?
Forward = (Expected net income ÷ Net income from prior year − 1) × 100
The forward estimate reflects analysts’ expectations for the current fiscal year.
🏛️ Why is it important?
Net income is the ultimate measure of profitability. Growing net income signals stronger efficiency, cost control, and sustainable earnings power.
🧮 Calculation
🎯 What does this mean for investors?
- Stronger net income boosts valuation, dividend potential, and investor confidence.
- If profits stall while revenue grows, it may signal margin pressure.
📘 Free Cash Flow Growth
📈 What is it?
Free cash flow (FCF) growth shows how a company’s available cash – after covering operating expenses and capital expenditures – has changed compared to the previous year.
🧮 How is it calculated?
🏛️ Why is it important?
Free cash flow reflects real financial strength. Growing FCF indicates more flexibility for dividends, share buybacks, and reinvestment.
🧮 Calculation
🎯 What does this mean for investors?
- Declining FCF may point to rising investments, increasing costs, or weaker operating performance.
- Especially for dividend investors, FCF growth is critical – since dividends are paid from actual available cash.
- A negative trend isn't always bad, but it deserves closer attention.
📘 Gross Margin
📈 What is it?
Gross margin shows how much of a company’s revenue remains after deducting the direct costs of goods sold (like materials and production). It represents the company’s “raw profit” before fixed costs, taxes, and interest.
🧮 How is it calculated?
Or simply: Gross Margin = Gross Profit ÷ Revenue × 100
🏛️ Why is it important?
Gross margin indicates how efficiently a company can produce or procure what it sells. It is a key measure of product-level profitability and pricing power.
🎯 What does this mean for investors?
- A high gross margin suggests strong pricing power and efficient production.
- Falling margins may signal rising input costs or competitive pressure.
- Compared to peers, gross margin offers insights into the quality of a business model.
📘 EBITDA Margin
📈 What is it?
The EBITDA margin shows how much of a company’s revenue remains as operating profit before interest, taxes, depreciation, and amortization.It reflects operating efficiency without being distorted by financing or accounting factors.
🧮 How is it calculated?
🏛️ Why is it important?
The EBITDA margin reveals how much operating income a company generates per dollar of revenue – independent of capital structure and tax effects.
🎯 What does this mean for investors?
- A high EBITDA margin reflects strong core profitability – before accounting distortions.
- It allows for effective comparisons across companies and sectors.
- A stable or growing margin signals efficient cost control and business scalability.
📘 EBIT Margin
📈 What is it?
The EBIT margin shows what percentage of revenue remains as operating profit after depreciation but before interest and taxes.
🧮 How is it calculated?
🏛️ Why is it important?
The EBIT margin reflects a company’s core profitability while accounting for capital intensity (e.g. machinery, infrastructure). It’s especially useful for comparing businesses with different levels of depreciation.
🎯 What does this mean for investors?
- A high EBIT margin shows that the company remains efficient even after factoring in depreciation.
- It’s especially relevant for capital-intensive industries.
- Stable or rising EBIT margins over time are a strong indicator of pricing power and business quality.
📘 Net margin
📈 What is it?
Net margin shows how much of a company’s revenue remains as bottom-line profit after deducting all costs, interest, taxes, and depreciation.
🧮 How is it calculated?
🏛️ Why is it important?
Net margin reflects a company’s overall efficiency – across operations, financing, and taxation. It shows how much actual profit is generated from each dollar of revenue.
🎯 What does this mean for investors?
- A high net margin means the company is not only strong operationally but also manages financing and taxes efficiently.
- Peer comparisons reveal business quality and competitiveness.
- Declining margins despite revenue growth can be a red flag for rising costs or inefficiencies.
📘 Free cash flow margin
📈 What is it?
The free cash flow (FCF) margin shows how much of a company’s revenue remains as actual free cash after covering all operating expenses and capital expenditures.
🧮 How is it calculated?
🏛️ Why is it important?
This margin reflects the true liquidity generated by the business – independent of accounting rules or depreciation. It’s especially relevant for dividends, buybacks, and reinvestment decisions.
🎯 What does this mean for investors?
- A high FCF margin means a company consistently generates strong cash flow.
- It’s a positive signal for financial stability and shareholder returns.
- The long-term trend is key – a declining margin may indicate rising investments or weakening operating efficiency.
📘 Earnings per share (EPS)
📈 What is it?
Earnings per Share (EPS) shows how much profit is attributable to a single share – and is one of the most important metrics for evaluating a company's performance.
🧮 How is it calculated?
The diluted share count reflects potential new shares that could be issued through options, convertible bonds, or other rights.
🏛️ Why is it important?
EPS is the basis for many key valuation metrics like P/E ratio, PEG ratio, or payout ratio. It enables comparisons of profitability across companies, regardless of their size.
🧮 Calculation
🎯 What does this mean for investors?
- EPS captures per-share profitability and is especially useful for comparisons over time or with analyst estimates.
- Rising EPS may signal consistent growth or share buybacks.
- Important: Always use diluted EPS for more realistic valuations – especially in companies with stock-based compensation.
📘 Free cash flow per share (FCF per share)
📈 What is it?
Free Cash Flow per Share shows how much free cash flow a company generates per outstanding share – after investments, but before dividends or debt repayments.
🧮 How is it calculated?
Free cash flow is calculated as operating cash flow minus capital expenditures (CapEx).
🏛️ Why is it important?
FCF per Share reveals how much real cash is available per share – useful for dividends, buybacks, or reducing debt. Unlike net income, free cash flow is harder to manipulate and often seen as a more reliable metric.
🧮 Calculation
🎯 What does this mean for investors?
- High FCF per share signals strong financial flexibility.
- It shows how much capital the company can effectively reinvest or return to shareholders.
- Particularly relevant for dividend payers and capital-efficient businesses.
📘 Short interest
📈 What is it?
Short interest indicates how many shares of a company are currently sold short – that is, borrowed and sold by investors who expect the price to decline.
🧮 How is it calculated?
It reflects the percentage of a company’s shares that are being shorted relative to the total shares available.
🏛️ Why is it important?
Short interest serves as a sentiment indicator: A high value may signal skepticism or bearish expectations – but also increases the potential for a short squeeze if prices rise unexpectedly.
🧮 Calculation
🎯 What does this mean for investors?
- Low short interest usually indicates market confidence in the company.
- High short interest can be a warning sign – or an opportunity if sentiment shifts.
- Especially relevant in volatile markets or ahead of key earnings releases.
📘 Employees
📈 What is it?
The employee count shows how many people a company employs worldwide – offering insights into its size, structure, and business model.
🧮 How is it calculated?
🏛️ Why is it important?
It helps assess operational scale, labor intensity, and cost structure. Combined with revenue and profit, it enables key metrics like revenue per employee or productivity.
🧮 Calculation
🎯 What does this mean for investors?
- A high headcount can signal operational complexity – but also significant growth capacity.
- Revenue per employee is a key indicator of efficiency.
- Especially useful for comparing tech, industrial, or service-heavy companies.
📘 Turnover per employee
📈 What is it?
Revenue per employee indicates how much revenue a company generates on average per employee – a key measure of efficiency and productivity.
🧮 How is it calculated?
The employee count is typically taken from the most recent annual report.
🏛️ Why is it important?
This metric helps compare business models – especially between labor-intensive and technology-driven companies. A high value suggests automation, operational efficiency, or strong value creation per head.
🧮 Calculation
🎯 What does this mean for investors?
- A high revenue per employee indicates a scalable and margin-strong business model.
- A low figure may reflect labor-intensive operations or lower value-add.
- Especially helpful when comparing tech companies to industrial or service sectors.
Inmune Bio, Inc. Stock Analysis
Analyst Opinions
10 Analysts have issued a Inmune Bio, Inc. forecast:
Analyst Opinions
10 Analysts have issued a Inmune Bio, Inc. forecast:
Inmune Bio, Inc. Events
Past Events
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AUG
6
Q2 2026 Earnings Call
about 2 months ago
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MAY
7
Q1 2026 Earnings Call
5 months ago
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MAR
30
Q4 2025 Earnings Call
6 months ago
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FEB
27
Special Call - INmune Bio Inc.
7 months ago
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FEB
26
Special Call - INmune Bio Inc.
7 months ago
|
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OCT
30
Q3 2025 Earnings Call
11 months ago
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StocksGuide Free
Inmune Bio, Inc. — Q2 2026 Earnings Call
1. Management Discussion
Welcome to InMuneBio's second quarter 2020. earnings call. At this time, all participants are in a listen-only mode. Following the presentation, there will be a question and answer session. which time you may press star 1 to ask a question. As a reminder, this conference call is being recorded. transcript will be available approximately 24 hours after the call. Before we begin, please note that except for statements of historical fact, statements made by management and responses to questions may constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. of risks and uncertainties that could cause actual results to differ materially from those expressed or implied. reviewed a forward-looking statements disclaimer in today's earnings release and the risk factors described in the company's filings with the SEC, including its most recent quarterly report. forward-looking statements speak only as of the date they are made and accept as required by law. In Yun Bayou undertakes no obligation to update them. It is now my pleasure to turn the call over to In Yun Bayou's Chief Executive Officer, David Ma.
Thank you for joining ImmuneBio's second quarter conference call. The second quarter and the weeks that followed were defined by execution across both of our late stage platforms. I will begin with the investor perspective on the progress we have made. I will then turn the call over to Dr. Mark Liddell, our Chief Scientific Officer and of Chordstrom to discuss Ebstracell and the Chordstrom platform in greater detail. Lori Elsperman will review our financial results and I will return to discuss the milestones ahead before we open the call for questions. For Ebstracell, we secured formal MHRA alignment, received approval of the pediatric investigation plan, completed a commercial manufacturing milestone, and strengthened our long-term supply chain.
Together, these achievements materially reduced regulatory and operational risk ahead of our planned UK marketing authorization application. We now expect to submit EBSTRASELL MAA to the MHRA by the end of Q3 or early Q4 26. The application will seek conditional marketing authorization. in R-DEB and is supported by written MHRA alignment across the CMC, non-clinical, and clinical evidence packages. The agency also recognized the Mission EB data as demonstrating clinical, meaningful, symptomatic benefits, particularly in pain and pruritus. After submitting the MAA in the UK, we plan to submit the MAA to the EMA in early 27 along with a BLA in the US seeking conditional approval. Manufacturing readiness has advanced in parallel. We successfully processed the first commercial ready umbilical cord tissue at the cell and gene therapy catapult facility in Stevenage and transferred the MSC isolation stage used to manufacture master cell banks into the intended commercial facility.
Combined with our expanded Anthony Nolan Agreement, this gives us a scalable supply foundation designed to support UK, EU, and US filings and future commercial supply. We also advanced the Cordstrom platform patent application into the U.S. national phase and established a working scientific advisory board of international recognized MSC and RDEV experts. The SAB will help strengthen EPSTRACELL's late stage development package and prioritize additional disease specific applications of the Cordstrom platform. EXPRO also reached important milestones this quarter. The FDA granted fast-track designation for early Alzheimer's disease, and the Phase 2 mindful study showed a statistically significant treatment effect on white matter myelin MRI biomarkers in the full modified intent-to-treat population. The TEEP The treatment difference was P.0028 with a Cohen's effect size of 0.46. In the biomarker enriched population, the effect size increased further to 0.59.
Expanded analysis presented at AAIC showed concordant treatment related effects across independent white matter and cortical gray matter measures at week 24. These data, together with our successful end of Phase II alignment with the FDA and publication of the mindful results in NPJ dementia, strengthened the clinical and regulatory foundation of the Phase 2b3 program. The peer-reviewed report shows directionally consistent benefit across clinical and biomarker endpoints in the pre-specified inflammation-rich subgroup with no amyloid-related imaging abnormalities observed. Recent TBI, traumatic brain injury, and oncology data also support broader platform optionality, although our clinical priority remains Alzheimer's disease, We continue to evaluate strategic partnership opportunities that could accelerate the program while preserving meaningful value for immune shareholders while we focus all of our attention and resources on getting Epsosol to the R-deb patients who are in great need. With that, I will turn the call over to Dr. Mark Liddell to discuss the Fortstrom program in greater detail. Mark.
Thank you, David. I want to focus on three key areas in the road to bringing Ebtreecel to the market that have been materially de-risked since our last call. First, the regulatory package, the manufacturing and supply chain, and the broader Cordstrom platform from which Ebstratel is the first product to market. So first, the MHRA's official minutes from our May 12th pre-MAA scientific advice meeting confirmed their alignment across every question that we submitted, covering CMC, non-clinical and clinical matters. This is important because it gives us a defined path for the planned conditional marketing authorization application, rather than requiring us to infer what the agency might expect. Second, the MHRA approved the EBSDR-Cell pediatric investigation plan in less than three months. The pediatric strategy incorporates the planned open-label phase three confirmatory study, and the agency's feedback recognized the Mission EB phase two data as demonstrating clinically meaningful improvement in symptoms that matter to patients, mostly pain and pruritus or itch. The feedback also supports evaluating EBSTRASELL as a chronic or intermittent supportive therapy in R-DEP.
Third, we completed a key commercial manufacturing milestone at the Cell and Gene Therapy Catapult Center and the Manufacturing Innovation Center in Stevenage for Ebstracell and for subsequent cell drugs from the Codestron platform. The first commercial compliant core tissues have been processed successfully and the MSC isolation stage for manufacture of the master cell banks was transferred into the facility intended to support registration and subsequently future commercial supply. Our expanded agreement with the Antenodon Core Blood Bank secures long-term access to qualified umbilical cord tissue for the platform for use in the UK, the EU and the US. This matters because the Codestron platform was designed to solve two persistent challenges that we've seen in MSC therapy over the past years, donor variability and manufacturing inconsistency. Our proprietary donor screening, pooling, and expansion processes are intended to produce an off-the-shelf, scalable, batch-to-batch consistent cell medicine, and that is what we have shown the MHRA. The recent manufacturing work brings the initial master cell bank production stage into the commercial ready manufacturing supply chain. We've also strengthened the platform from which the EBSCEL LEAD program is derived.
The Cordstrom patent application entered the US national phase following a favorable international written opinion. and if granted, could provide broad protection into at least 2045. In addition, our newly formed scientific advisory board brings together major leaders in MSC clinical translation, potency assessment, manufacturing, rare pediatric skin disease, and additional therapeutic areas that we can focus on. This is a working advisory board with defined priorities including phase three design, translational biomarker identification, potency and release assays, and selection of these additional indications. But taken together, these achievements give us greater confidence that the scientific, clinical, regulatory and manufacturing components required for a successful filing are now converging. Our immediate objective is to submit the UK MAA by the end of Q3 or early Q4 this year, followed by the planned European and US submissions, while preparing platform for future indications. I'll now turn the call over to Corey for a review of our financial results. Corey?.
Thank you, Mark. I'll provide a brief overview of our financial results for the second quarter. Net loss attributable to common stockholders for the quarter ended June 30th, 2026 was approximately 1.3 million compared to approximately 24.5 million for the quarter ended June 30th, 2025. The prior period included a $16.5 million impairment charge related to acquired in-process research and development intangible assets. Research and development expenses total the benefit of approximately $0.8 million for the quarter end of June 30, 2026, compared to approximately $5.8 million of expense for the quarter end of June 30, 2025. The research and development benefit during the 2026 period was primarily due to the recognition of additional Australian research and development rebate. General and administrative expenses were approximately $2.3 million for each of the quarters ended June 30, 2026 and June 30, 2025. As of June 30, 2026, the company had cash and cash equivalents of approximately $18.4 million.
Subsequent to June 30, 2026, we received approximately $4.2 million in Australian research and development tax rebate, providing non-dilutive capital to support our development programs. Based on our current operating plan, we believe our existing cash resources are sufficient to fund operations into the second quarter of 2027. As of August 6, 2026, the company had approximately 27.8 million shares of common stock outstanding.
I will now turn the call back to David. Thank you, Corey. Before we open the call for questions, I'd like to leave you with a clear view of the value driving milestones ahead. Immunobio now has two differentiated late stage platforms. Ebstracell is approaching global regulatory submissions with a commercial manufacturing and supply foundation in place. EXPRO is supported by FDA fast track designation, end of phase 2 alignment, and statistically significant phase 2 imaging data. We believe this combination provides both a near-term regulatory opportunity and meaningful long-term pipeline value. Based on our current plans and subject to regulatory feedback, investors should watch for four principal milestones.
First, we expect to submit the extra sale marketing authorization application to the UK MHRA this year seeking conditional marketing authorization in our debt. Second, following the UK submission, we plan to submit EBSTR-Cell to the European Medicines Agency, expanding the regulatory strategy to patients across the European Union early next year. Third, we plan to submit EBSTR-Cell Biologics License Application to the U.S. Food and Drug Administration in the first quarter of 27. Fourth, we continue advancing the EXPRO Registrational Strategy and Evaluating Strategic Partnerships supported by FDA Fast-Track designation, end of phase two alignment, and increasingly consistent clinical and imaging evidence from MINDFUL. In parallel, we will continue commercial readiness work for EBSRCEL, including manufacturing, supply chain, market access, and distribution planning. New Cordstrom Scientific Advisory Board will also begin executing against defined priorities for late stage development and platform expansion.
Taken together, these activities provide a clear path to multiple regulatory and strategic value inflection points. Our priority is disciplined execution. completing high-quality submissions, preserving capital, and building the capabilities required to deliver these therapies to patients. I want to thank our employees for their relentless dedication, our investigators and clinical collaborators for their partnerships, Patients and families who have placed their trust in us and our shareholders for their continued confidence and support. Our team is motivated, working tremendously hard, and is always thinking about the patients we serve and are dedicated to improving their lives. We believe the next several quarters can redefine a new bio as we move from clinical development toward regulatory review and potential commercialization. We look forward to updating you as we execute against these milestones. With that, I'd like to move to questions and answers.
Thank you.
Thank you, ladies and gentlemen. We will now begin the question and answer session. If you have a question, please press the star followed by the one in your touchstone phone. Should you wish to cancel your request, please press the star followed by the two. If you are using a speakerphone, please lift the handset before pressing any keys. Once again, that is star one should you wish to ask a question. Your first question is from James Malloy from Alliance Global Partners. Your line is now open.
2. Question Answer
Hi, thank you very much for taking my question. The Open Label Phase III U.S. trial, is that the 12-month Open Label Safety trial we discussed previously or is this a different trial?.
Hey, David, I'm not sure if you're on mute. Sorry about that. Hi, James. It's David here. Because we are submitting AbstraSelf or conditional approval, we have a follow-on trial that'll be running during the approval process. So this is a phase three confirmation trial. It's the same one we spoke about earlier.
I think previously we talked about a 12 month over the level safety trial not needed for filing. Is that this or is the 12 month safe trial also going to be running in addition to this? No. It's one trial. And it's safety and confirmation trial. Okay. You guys had already got to this and that's the one you talked about before. Okay, very good. That's right. Okay. When you look at the UK filing or the EU filing, the next two filings, what do you guys see as the biggest, sort of the biggest thing they'll be looking for that, you know, I think we obviously know the potential benefits of abstra cell. What do you think are the biggest hurdles that you think they'll be looking for against approval and how you've addressed those?.
Yes, so the UK is very straightforward, very clear. We're very far along with the discussions that we've had with them. If you look at the top complaints from ED patients, R-DEP patients, it's number one and two is pain and itch. uh, patient response outcome forum. Uh, I think in 2018, you can find it on YouTube, uh, in the, uh, um, from the FDA's website. And again, the top complaints are itch and pain. And clearly itch is, also related to wounds. You know, if you have an existing wound and you itch it, you introduce bacteria, you keep it from healing.
These patients have such sensitive skin that even if they don't have a wound and they itch, they can very easily open up a wound. And so itch, if you talk to the investigators, they'll clearly tell you that itch is a factor with wound healing and persistent wounds and opening up new wounds. The regulators in all three jurisdictions, realize that. There's published papers around that. So, you know, there have been trials also in itch. So we're moving forward with itch pain as a primary endpoint. So we feel very confident about it. It's It's clear with the investigators, it's clear with the patients.
And in our trial, we also did in the ADASI score, which was not the primary, pick up the wound scores later in the trial, around the six-month mark. Because if you think about it, over a period of time when you're not itching, it takes a while to see those results in terms of wounds, and that's what we saw in the trial.
Okay. And then what does the, just going back to the trial, then I'll get back in the queue. What does the open label confirmatory trial look like and how many people how long I think one twelve months I think is that is the guidance correct and what's the thinking on how long to to enroll is it 12 months from start to finish we start when you have the data and what's the size of the trial.
Yes, so we expect it to be somewhere around 40 to 45 patients. We already have about 33 lined up, ready to go. Most of those are the patients that were on the original, almost all of those actually are the patients that were on the original trial that want to stay on Cordstrom. We expect to enroll about one to two patients We'd like to enroll faster, but the PIs just can't handle that kind of ball of volume. And it'll run a total of 18 months. It's three sessions of infusion that are six So every 10 days or so, they get two infusions, three times a year for a total of six infusions. And every 10 days,.
And we'll have the data right around the middle of 28. Okay, great. And then a final question, I'll hop in the queue. What does a potential approval in the UK, what does a launch look like for you guys in the UK? Yes. Gear for that sort of thing. Okay.
That's a great question, James. So we ran this at the two leading centers that treat the most of the children that have R-DEP in the UK. They're the two leading children's hospitals for EB and R-DEP. It's where a good majority of the patients go. The beautiful thing about it is that the clinical investigators are already very familiar with administering the drug. they're familiar with the results of the drug, and they have the patient population already. My expectation, James, is that within two years of approval in the UK, given the 40-ish odd patients that we'll have on the trial, plus the additional patients that we'll add, I expect that we should very comfortably within two years be right around 100 patients or so in the UK.
Thank you very much for taking my questions.
You're welcome, James. Thank you. I think we also have questions coming from Dan.
Right. Yes, David, so I've gotten a number of questions emailed into me and I'm going to try and compile them for you. You pretty much covered the first question about the size of the opportunity, but I'm There's a lot of people are asking if you can just clarify that the company's focus for the foreseeable future is on the course and program, and then as sort of follow on to the market size, can you talk about the pricing of the drug and what the reimbursement process looks like in the UK?.
Yes, happy to do that. So first of all, let's talk population. When you look at about 10 to 15% of those suffer from the more severe form of R-DEP, in the U.S., it's somewhere around 2,000 to 3,000 patients. In Europe, it's very similar to those numbers. And in the U.K., it's somewhere around 800 or so patients in total. Now, about 60% of those numbers represent children and the rest represent adults. is the breakdown. In terms of pricing, It's quite interesting because one of our competitor's products, Crystals, which is got approved in the UK, they're now going through the price negotiations. It'll be interesting for us to watch that.
But with the recent kind of MFN and what we know about rare diseases, the pricing in the UK should be relatively close to what we expect in the US. We expect it's somewhere around 400,000 to 500,000 pounds per year per child. We will start our pricing negotiations right after we file the MAA will be the timeline for that. And if you look at what Crystal has done is they obviously got approval and now they're in the process of the reimbursement negotiations. Now, you also can get reimbursement before you have the pricing negotiations because the hospitals in the UK have the ability to pay and fund the medication while you're going through that process. There is a budgeting process within the hospitals there to do that.
Did that answer the question, Tim? Great. Yes, that did and brought in to my next question as well, which he took care of nicely. Thank you. So the next question I have here, so basically, in light of recent positive developments, I'm stunned at the current share price. And this is from an investor. I, for one, believe a proper repricing should be in the cards. Would you care to comment?.
Well, you know, I've always kind of beat on the drum that we are, uh, undervalued, right? I mean, it's, it's, I really feel that way, but at the end of the day, I think what we have to do is we have to prove ourselves by getting the MA and getting the product approved. I'll remind everybody that if we get it approved in the US through accelerated approval, it already has orphan drug designation and rare pediatric disease designation. designation. The orphan drug means it's an accelerated review process of about six months. The rare pediatric disease designation means it comes with what's called a priority review voucher. Those priority review vouchers can be sold in the secondary market. The last few went for between $100 and $50 to around $200 million. So we like to say that between 100 and 200 million, which we intend to use to help fund the expansion of the Cordstrom platform and the Expro platform.
If you, in terms of value, I mean, just the PRV alone, obviously, is quite a bit larger than our current market cap. Really what we do is we're just keeping our heads down. We're being very cautious with resources. As you can see, we actually didn't burn much this quarter. And we don't need a huge amount of cash to get to product approval. And so we're going to be very judicious about our spending. And we're going to be very judicious about how we raise money.
I'll remind shareholders that I'm also one of the larger shareholders of this business being, as is Mark, being founders of this company. And so we're highly sensitive to dilution, which I think makes us a little bit different than many other biotech companies. So we're going to be prudent. We don't need a huge amount of cash dilution. to get to where we need to go. And we're going to be doing things like we did this last quarter by being just very special with R&D rebates, negotiating our spending, and being very careful about the amounts of money we raise and how we raise it.
Got you. Last question for you then. You did touch on this, but can you just go over the burn rate as well and sort of capital market strategy?.
Yes, so I'll talk about capital market strategy and then Corey, I'll let you just make a little comment about burn rate. So, you know, our capital market strategy is, is we're, we're really turning our shareholder base over from the previous X pro shareholders into a rare disease shareholders. completely new shareholder base and we've been spending a lot of time going non-deal roadshows, talking to investors. We've been able to get a lot of our expo shareholders back into the business. They understand the value of what we're doing with Cordstrom and we're starting to get some new ones now, especially as we get closer to proving ourselves with the MAA application and eventually approval in the UK. That being said, we're being very careful about spending. Corey, do you want to talk about our last quarter burn rate and the future burn rate?.
Well, in general, I'd say that our burn rate is maybe a million to a million-five per month. It's a little bit unusual what we had in the last six months because we took a lot of R&D rebates in, which is great, but we're not expecting R&D rebates in that amount on a go-forward basis, at least over the next 12 months or so. So, I'd say a million or a million five, and we've got.
cash into Q2 of next year. Yep, exactly. So not a huge burn rate. And the ability to raise that money, our goal is really just to raise money through the end of next year and do it very small, very generously. It's not a huge amount, little bit here, little bit there. And we've got the ATM available to do that if we need to. And, you know, we expect to have approval in the UK sometime, I think, I'm going to say early Q2 next year, but let's just say Q2 the following year, and then we'll start to be able to generate revenue.
Great. That's it for investor questions. Jenny, I'll pass it back to you.
Thank you. That concludes our conference call for today. Thank you everyone for joining. You may all disconnect your lines.
This live transcript is auto-generated without human intervention or review.
[Call has ended.]
Inmune Bio, Inc. — Q1 2026 Earnings Call
1. Management Discussion
Greetings, and welcome to the INmune Bio's 2026 First Quarter Earnings Call. As a reminder, this conference is being recorded. A transcript will follow within 24 hours of this conference call.
At this time, it is my pleasure to introduce Mr. Daniel Carlson, Head of Investor Relations of INmune Bio.
Thank you, operator, and good afternoon, everyone. We thank you for joining us for the call for INmune Bio's 2026 First Quarter Financial Results. Presenting on today's call are David Moss, CEO and Co-Founder of INmune Bio; Dr. Mark Lowdell, Chief Scientific Officer and Co-Founder of INmune Bio; and Cory Ellspermann, INmune Bio's CFO.
Before we begin, I remind everyone that except for statements of historical fact, the statements made by management and responses to questions on this conference call are forward-looking statements under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.
These statements involve risks and uncertainties that can cause actual results to differ materially from those such as forward-looking statements. Please see the forward-looking statements disclaimer on the company's earnings press release as well as risk factors in the company's SEC filings, including our most recent quarterly filings with the SEC.
There is no assurance of any specific outcome. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made as the facts and circumstances underlying these forward-looking statements may change.
Except as required by law, INmune Bio disclaims any obligation to update these forward-looking statements to reflect future information, events or circumstances.
Now my pleasure to turn the call over to INmune Bio's CEO, David Moss.
Thank you, Daniel, and good afternoon, everyone. For our first quarter 2026 earnings call, today, I'll review key takeaways and provide an update on our platform programs. Following my review of recent developments at INmune Bio, I will pass the microphone to Dr. Lowdell, INmune Bio's CSO and inventor of CORDStrom, who will provide an update on our CORDStrom MSC platform and particularly our RDEB program.
Next, Cory Ellspermann will provide our financial results, after which I'll conclude our prepared remarks. We entered 2026 with clear priorities and strong momentum across our platforms. Most importantly, our CORDStrom platform remains on track, and we are now approaching a key milestone with our regulatory filings.
Based on the progress of our analyses, manufacturing readiness and regulatory preparation, we expect to file for approval beginning in the near term, and we remain confident in the time line that we previously outlined. CORDStrom represents a potential first systemic therapy for RDEB, and we believe the data continue to support both its clinical benefits and its broader platform potential. Execution against this filing is our top priority.
Turning to XPro. While CJ is not speaking today, I want to emphasize that we continue to make meaningful progress. We are advancing additional imaging analysis from the MINDFuL study, including MRI data focused on myelin preservation and structural integrity.
These data sets are important as they further characterize XPro's potential as a disease-modifying therapy. At the same time, we're exploring potential rare disease trials for XPro and potential partners as we define the path forward, including regulatory alignment late-stage development strategies. Naturally, we'll update the markets as these milestones develop.
Overall, we believe we're well positioned across both platforms as we move through a catalyst-rich period for the company and a marked change potentially for the company as we get closer to commercialization. With that, I'll turn the call over to Mark Lowdell to provide more details on CORDStrom. Mark?
Thank you, David, and thank you to everyone that's joined the call. As David said, since our last earnings call, we've moved forward significantly in bringing CORDStrom to market, and it is our central aim. First, we submitted the pediatric investigation plan known as a PIP to the U.K. medicines regulator in February, and we were approved for rapid assessment and receiving their response on the 9th of April.
No substantial issues were raised, and we anticipate submitting our final response in the next few days. The approval of the PIP is an essential step to complete prior to submission of the marketing authorization application in the U.K. and then to the EMA for Europe. We've started the first of the 3 process validation manufacturing runs on time and the remaining 2 are scheduled to meet our MAA submission deadline. Most significantly, we've concluded negotiations with the Anthony Nolan U.K. Cord Blood Bank this month to ensure secure supply of umbilical cords and allow testing by U.S. laboratories to meet the requirements laid down by the FDA in our Type B meeting last year.
This agreement was signed yesterday and is the final step in getting the UCMSC isolation part of manufacturing process validated, ready for commercial manufacture. Facilitating our ability to manufacture consistent batches of CORDStrom, we're pleased to announce that we recently signed an amended material transfer agreement with Anthony Nolan.
This expanded strategic collaboration secures the long-term reliable supply of these high-quality umbilical cord tissues from their world-class cord blood bank to further our CORDStrom platform. Having a consistent supply is essential for us, not only for regulatory authorities, but also to enhance our ability to take the CORDStrom platform forward into other disease indications.
The marketing authorization application submission requires completion of a very significant body of documents in 5 sections. These are now well underway. And as part of the product definition section, we've had to determine the formal names for CORDStrom as applied to RDEB to show it's different to other formulations targeting other diseases in the future.
The active ingredient was named by the World Health Organization as pobistrocel, and we've chosen a commercial drug name of Ebstracel for the formulation to be used in recessive dystrophic EB. In 2 weeks' time, we will meet with the MHRA for further advice about the marketing authorization submission filing in the U.K. and then start to finalize those documents.
Some minor regulatory delays have meant that we expect to submit to the MHRA in early Q3, and we've contracted a U.K. company, TMC Pharma, with expertise in rare disease submissions to run the EMA and the FDA submissions in parallel to meet the end of the year deadline that we described before to you.
Finally, I had the great privilege to speak at the Cure EB Annual General Meeting in London last month, which is one of the largest EB charities in the U.K. I presented our data and our plan was overwhelmed by the response from patients and carers who attended. They're desperate for us to get Ebstracel to the market and to open the next phase of the clinical trial in the U.K. We're doing our utmost to deliver on our promises to them and to you to get into commercial manufacturing and supply in 2027.
I'll hand over to Cory now for an update of the current financials. Cory?
Thank you, Mark. At this time, I'll provide a brief overview of our financial results. Net loss attributable to common stockholders for the quarter ended March 31, 2026, was approximately $5.4 million compared with approximately $9.7 million for the comparable period in 2025.
Research and development expenses totaled approximately $3.6 million for the quarter ended March 31, 2026, compared with approximately $7.6 million for the comparable period in 2025. General and administrative expenses were approximately $2.2 million for the quarter ended March 31, 2026, compared with approximately $2.3 million for the comparable period in 2025.
And at March 31, 2026, the company had cash and cash equivalents of approximately $21.4 million. Based on our current operating plan, we believe our cash is sufficient to fund our operations through Q1 of 2027. And as of May 7, 2026, the company had approximately 26.6 million shares of common stock outstanding.
And now I'll hand the call back to David.
Thank you, Cory. To close, our focus is straightforward. We're executing towards regulatory filings for CORDStrom, which we believe represents a major inflection point for the company.
At the same time, we're continuing to build the case for XPro through additional imaging data, exploring the future rare disease trials and ongoing partnership discussions aimed at advancing the program efficiently.
We believe these efforts position INmune Bio for a significant year ahead with multiple opportunities to create value for both patients and shareholders. Due to travel schedules, we'll not be taking questions, and this concludes our prepared remarks.
If you have further questions, please reach out to the contacts at the end of our press releases, Dan Carlson or myself via those phone numbers or e-mails. Thank you for joining us today.
And thank you, ladies and gentlemen. This concludes today's conference call. We thank you for your participation, and you may now disconnect.
Inmune Bio, Inc. — Q4 2025 Earnings Call
1. Management Discussion
Greetings, and welcome to the INmune Bio's 2025 Fourth Quarter and Year-End Earnings Call. As a reminder, this conference is being recorded. A transcript will follow within 24 hours of this conference call.
At this time, it is now my pleasure to introduce Mr. Daniel Carlson, Head of Investor Relations of INmune Bio. Daniel?
Thank you, Cloey, and good afternoon, everyone. We thank you for joining us on the call for INmune Bio's 2025 Fourth Quarter and Year-end Financial Results.
Presenting on today's call are David Moss, CEO and Co-Founder of INmune Bio; Dr. Mark Lowdell, Chief Scientific Officer and Co-Founder of INmune Bio; Dr. CJ Barnum, Head of Neuroscience; and Cory Ellspermann, INmune Bio's CFO. Before we begin, I remind everyone that except for statements of historical facts, the statements made by management and responses to questions on this conference call are forward-looking statements under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.
These statements involve risks and uncertainties that can cause actual results to differ materially from those such as forward-looking statements. Please see the forward-looking statements disclaimer on the company's earnings press release as well as risk factors in the company's SEC filings, including our most recent And quarterly filings with the SEC. There is no assurance of any specific outcome. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made as the facts and circumstances underlying these forward-looking statements may change. Except as required by law, INmune Bio disclaims any obligation to update these forward-looking statements to reflect future information, events or circumstances.
Now my pleasure to turn the call over to INmune Bio's CEO, David Moss.
Thank you, Dan, and good afternoon, everyone. Thank you for joining us for INmune Bio's Fourth Quarter and Full Year 2025 Earnings Call.
Today, I'll begin with an overview of our progress and strategic priorities across the business. Mark will then provide an update on our CORDStrom platform with a focus on our RDEB program. CJ will follow with an update on XPro and our Alzheimer's disease development efforts. Cory will then review our financial results. After that, I'll return to highlight our key upcoming milestones before we open the call to questions.
2025 was a pivotal year for INmune Bio. We completed our MINDFuL Alzheimer's trial, advanced CORDStrom towards registration, and continue to position each of our platform programs for the next stage of development. As we move into '26, our focus is very clear. Execute against the most important regulatory clinical and strategic milestones across the portfolio.
Starting with CORDStrom, this remains our most advanced program and a major value driver for the company. We recently presented additional patient data that further reinforces the therapeutic profile of CORDStrom in Recessive Dystrophic Epidermolysis Bullosa, or RDEB. These data showed clinical meaningful wound healing, reductions in itch and improvements in quality of life, all with a favorable safety profile.
Based on this progress, we're in the final stages of preparing our regulatory submissions in both the U.K. and the United States, and we remain on track to file the MAA in the U.K. by the end of summer of '26.
As Mark will tell you shortly, CORDStrom has a clear batch-to-batch manufacturing consistency, which makes the product reproducible ensuring commercial grade uniformity. Further, the clinical mechanism of action of CORDStrom for RDEB has been worked out along with the potency assays, which is an important step for regulators. The repeatable reliability -- the repeatable manufacturing reliability with the worked out MOA along with CMC readiness, safety and clinical results is what gives us confidence in the -- in CORDStrom for RDEB. Importantly, we want to highlight that CORDStrom is not simply a single asset opportunity, but as a platform with broader potential.
Beyond RDEB, we believe the biology underlying the program may support development in additional inflammatory and degenerative conditions and over time, may also enable genetically modified applications in oncology and rare disease settings.
Our immediate priority is to bring this therapy to patients with RDEB while also building the foundation for long-term platform expansion. Personally, there is no greater mission in my career than delivering CORDStrom to the children and families living with RDEB. Behind every trial result is a story that I've read and a face that I've seen in the video shared by these incredibly brave families. These images serve as a constant reminder of why we do what we do. Our team is deeply motivated by the human element of this condition, and we are working with an absolute urgency to bring this therapy to patients who need it most.
Further, in our mission to develop medicines to unmet needs, I now turn to XPro for Alzheimer's disease. We believe this program is in the strongest position it has ever been. We completed MINDFuL, we've aligned with the FDA on the development path, and we're now preparing for a Phase III.
This alignment effectively creates a preapproved blueprint for a partner to execute. CJ will give you the full picture shortly. On INKmune we completed our Phase II trial in metastatic castration-resistant prostate cancer ahead of schedule and under budget. The study met its primary endpoint and 2 of its 3 secondary endpoints. Mark will explain more on this later.
Before I hand the call over, I want to thank patients and families who participated in the clinical studies, the investigators and trial sites, who supported this work, and our employees for their continued commitment and execution throughout the years. I also want to thank our shareholders for their continued support. Our strategy of advancing multiple differentiated platforms in parallel continues to create meaningful opportunities for value creation.
We now have 1 platform approaching the regulatory stage, another with a completed Phase II study, an important translational data and 1/3 that has also generated encouraging clinical results. We believe '26 will be an important year for INmune Bios to work to advance CORDStrom towards approval, further clarifying the next steps for the Phase IIb trial for XPro and continue to build the partnerships and resources we need to move our programs forward.
With that, I'll turn the call over to Mark Lowdell for an update on CORDStrom. Mark?
Thank you, David, and thank you to everyone who's joined the call today. Welcome. So as David said, CORDStrom showed great promise in the randomized placebo-controlled trial in RDEB, but it potentially extends way beyond RDEB to other forms of Epidermolysis Bullosa, and indeed to other conditions and indications. CORDStrom remains truly revolutionary in the MSC field. And last year, we reported on the fact that since it's created from mesenchymal stromal cell banks from 4 or more pooled donors, it really has unrivaled stability and reproducibility to compare to all of the other mesenchymal stromal or stem-cell products that are being developed or are on the market.
Moreover, what we found is that the pooling allows us to select the individual mesenchymal stromal cell donor seedstocks, and we can choose those that have the appropriate potency characteristic for each disease indication, which allows us to tailor the final product to target different disease indications and thus have different drugs.
As you know, RDEB is our first disease indication, and over the past 6 months, we've been able to dissect the precise mechanism of action of CORDStrom in RDEB. That's pretty unique for mesenchymal stromal cell product, while the diversity of the product means that working out quite how it delivers its effect is actually very challenging, but we now know that CORDStrom works by secreting an array of chemical messengers called cytokines, which are used by the body to control inflammation.
We know that RDEB is predominantly a disease of inflammation, and it's driven by cells in the skin, which are called Type 1 macrophages or M1 cells. These M1 cells secrete inflammatory cytokines, which drive the itch and lead to the scratching, which causes the skin wounds so prevalent in RDEB patients, in which you'll be familiar with. M1 cells in normal skin also induce itch when provoked, but in RDEB patients, the absence of the protein which binds the skin layers together means that itch scratch cycle causes those very severe lesions that are so famous.
One of the cytokines secreted by CORDStrom drives the M1 cells in the skin to mature into an M2 noninflammatory wound healing cells. We all have these and these M2 cells secrete a cytokine called IL-10, which switches off other INmune cells driving the itch-inducing cytokines.
In parallel, the M2 cells also secrete other chemical messages cytokines, which enhance wound repair. And when we looked at the serum samples from the patients who were treated with CORDStrom on the U.K. trial and compared those to those treated with placebo. The CORDStrom recipients all had in their blood, cytokines that our mechanism of action predicted. And those patients that had the highest concentration reported much less pain, less itch and had better skin scores.
They scored better in all measures of well-being and increased ability to eat. So this is the first RDBE treatment to have such diverse whole body clinical benefits, over and above those, which we see from the skin treatments that are already licensed. The patients, their caregivers and their doctors, all want to continue to have access to CORDStrom, and as David said, we're working tirelessly at present to submit the applications for marketing authorizations in the U.K., and then the European Union, and finally the U.S. before the end of the year.
We are driving forward and they're all completed by the end of this year, and we hope to be supplying CORDStrom to RDEB patients in 2027.
As I said earlier, the fact that CORDStrom is manufactured from a pool of 4 or more donor cell banks means that we can select the best donor cells for specific clinical indications. So while we are progressing with CORDStrom for RDEB and the marketing authorizations, my group of R&D scientists here in the U.K. are working on other broader indications and we're seeking business partnerships to develop those through clinical trials and bring those to market accordingly.
So as a company, we're laser-focused on preparing the marketing authorization application for the U.K. and then the EU and the Biologics License Application, or BLA, for the U.S. by the end of 2026. These are highly aggressive time lines, but so far, we've met all of the deadlines that have been set, and I'm incredibly proud of our team in the U.K. for working so diligently to keep to these time lines to remain on track and to use all the resources that we have in the U.S. office to support.
So I'm happy to take questions that you have, but meanwhile, I'll hand over to CJ for the latest update on XPro. CJ, floor is yours.
Thank you, Mark. I'll give you an update on XPro and where we're headed. MINDFuL was our Phase II trial in Alzheimer's disease. We designed it around a simple question. If we pick patients who have both Alzheimer's pathology, and signs of inflammation in their body, and we treat the inflammation, do they do better? What we saw was very encouraging. The results consistently favored XPro across clinical, behavioral patient-reported and blood and imaging biomarkers. The Phase I identified what works, who it works for and resolved the open questions so that Phase III can be successful.
These results directly inform how we designed the Phase III program. We identified the patient population, those with both Alzheimer's pathology and biomarkers of inflammation. Decades of Alzheimer's research show that cognitive changes come first and functional changes follow with time. That's why the Phase III trial runs 18 months, long enough for the cognitive effects we saw at 6 months to show up on the functional measures the FDA requires for approval. The program is built as an adaptive trial with 2 stages. Phase IIb gives us a decision point at 9 months. a clear go or no go before we commit to the full Phase III investment.
If the data hold, the trial continues seamlessly into the registrational stage with the CDR sum of boxes, the same primary endpoint used to approve lecanemab, and donanemab at 18 months. We presented this program to the FDA at the end of Phase II meeting earlier this year. The agency reviewed our data, our enrichment strategy, and our trial design and aligned with our approach. We are now moving forward on several fronts.
On the development side, we continue to analyze the MINDFul data set to fully understand the impact of XPro treatment. At the same time, we are preparing the Phase III program for initiation, which includes finalizing the protocol based on the FDA's feedback and pursuing the partnerships and funding needed to execute it. There's a lot of work ahead, but the foundation is solid.
I'll hand it back to David. I look forward to your questions. David?
Thanks, CJ. Before I hand the call to Cory to go through our financial results, I want to emphasize from a capital perspective, we remain committed to capital efficiency. Our strategy is built on hitting clear data-driven milestones that allow us to maximize shareholder value while minimizing unnecessary burn. We're focused on maintaining the lean execution-oriented culture that has brought us to this stage. With that, let me pass the call to Cory to go through our financial results. Cory?
Thank you, David. Net loss attributable to common stockholders for the year ended December 31, 2025, was approximately $45.9 million compared to approximately $42.1 million for 2024. Research and development expenses totaled approximately $20.7 million for the year ended December 31, 2025, compared with approximately $33.2 million for 2024, with the decrease due to incurring lower expenses in connection with the Alzheimer's trial in 2025.
G&A expenses was approximately $10.3 million for the year ended December 31, 2025, compared with approximately $9.5 million for 2024. We also recorded a full impairment of our intangible asset of $16.5 million in 2025 following the release of the Phase II results of the Alzheimer's trial, in which the trial did not meet the clinical endpoint.
During 2025, the company sold 3 million shares of common stock for net proceeds of approximately $17.4 million in a registered direct offering. In addition, the company sold approximately 1.3 million shares of common stock for net proceeds of approximately $10.1 million under at-the-market offerings.
At December 31, 2025, the company had cash and cash equivalents of approximately $24.8 million. And as of March 30, 2026, the company had approximately 26.6 million shares of common stock outstanding. Based on the current operating plan, we believe our cash is sufficient to fund our operations through Q1 2027.
And now, I'll hand the call back to David.
Thanks, Cory. Now I'd like to present upcoming milestones for the company, and then we can start with the Q&A. For CORDStrom program, we have several significant milestones ahead, which will really set our track for 2027. As Mark mentioned, we're on track to file the MAA in the U.K. by mid-summer 2026. A few months after the MAA filing, we expect to submit the MAA to the EMA and then the BLA to the FDA towards the end of the year.
We should have feedback from all 3 geographies in '27, if not, approvals by then. I mind investors that it's our belief that a successful BLA application would likely result in the company obtaining a priority review voucher from the FDA, given that the program already has orphan drug designation and rare pediatric disease designation.
For XPro, we continue to make strong progress. We've now received the minutes from our end of Phase II meeting with the FDA, as CJ had mentioned, and we obtained positive initial feedback on the accelerated approval pathways or we're active preparing for next steps. We're advancing partnership and funding discussions to support late-stage development of XPro.
Stepping back, we entered 2026 with a focused set of objectives and multiple meaningful opportunities to create value, while MINDFuL trial did not achieve its top line primary endpoint due to powering the patient population properly, the totality of XPro data set continues to support our conviction in the program's potential in Alzheimer's disease and other neuroinflammatory disorders. At the same time, we believe CORDStrom is advancing towards a potentially transformative regulatory and commercial inflection point with the broader platform still not fully reflected in the market.
We appreciate the continued support of our shareholders and the commitment of our team as we work towards these goals. At this point, Cloey, I'd like you to tell people how they can ask questions and poll for questions.
[Operator Instructions] And we'll take a question from Elmer Piros with Lucid Capital Markets.
2. Question Answer
David, what I'd like to ask, and maybe Mark can help us out here. If there is any anticipated differences between an MAA and an FDA submission. Have you had interactions with the FDA, what might be their requirements different from the European or from the U.K. agency?
Yes, I'll -- that's a very good question. Yes. So we -- the last time we spoke to the FDA specifically was a little bit about 13 months ago? And what they came back with was some -- one of the things that's been at the top of my mindset is, -- all of the work we've done so far in RDEB, the products being made from umbilical cord donors from the U.K. and there is a sensitivity about using U.K. donor materials in the U.S. And so we asked the FDA specifically whether we would be allowed to use U.K. donor cords for the U.S. submission. And they came back and said, absolutely, yes, but we would have to screen the U.K. donors for the standard globally agreed infectious disease markers, but they'd have to be tested in U.S. labs in clear accredited labs.
And so what we're doing at the moment is creating new master seedstock and from donors that we can ethically test in the U.S. So that's the biggest difference. We have to create a new master seedstock, which is ongoing at the moment. But because we -- as I said earlier on, we've worked out the mechanism of action. We now have potency assays. We've been able to demonstrate that we've made 4 different master seedstocks experimentally from U.K. donors, and they've all been consistent. So the next point is that we make for the FDA filing, which you're going through at the moment, will be those that we take through for commercialization globally. So that was the principal question that we had, and it was the principal answer that they came back with The rest of the questions they came back with were identical to those from the MHRA. So yes, we will present exactly the same data set.
Elmer, let me -- if you don't -- Elmer, if you don't mind, let me just add to that. So the plan is what's -- if you -- a few months -- a few weeks ago, we submitted essentially a pre-MAA package to the MHRA, which will -- which really effectively is like a Type B meeting and it kind of -- it kind of smooths the process of the full MAA application speeds the process up that we intend to file midsummer.
Once we get the feedback from the MHRA, and as Mark will tell you, they've already set a face-to-face meeting with us. Once we get that, we'll put that together with the answers to whatever questions they have or whatever feedback they give us, and then we'll submit that as a Type B meeting to the FDA in preparation really like a pre-BLA in preparation of the BLA with the FDA. And so that will be the steps that will take place. I think that might have been a little bit of what you're asking, if I'm correct?
Yes, yes, yes. And just maybe one more detail around this. So would you have to have the samples tested in U.S. labs before you submit or you can have that during the submission or during the evaluation and submit it when you have the results?
So what they will ask is for a confirmation that we will only supply drug into the U.S. from U.S. tested donors. But in point of fact, we're making the master cell batch now. So we will have products that have been made from U.S. tested donors before we submit the BLA.
Yes. And maybe one question about the course -- the XPro program. David, have you had interest, any interactions with potential former partners at ABPD? upon feedback, you get .
No, good question, Elmer. We have ongoing discussions with some groups. And one of the things that now you have to realize, we've just got the end of Phase II minutes a few weeks ago, 3, 4 weeks ago now. And -- so everything is being factored out. But 1 of the things we intend on doing this is finding a group to help us on the BD perspective because there's just a lot of not just large pharma but midsized companies, that we think the program is very appropriate for because if you think about it, it's a relatively small investment to see the Phase IIb portion for obviously a very large market, potentially one of the largest markets. The Phase IIb portion reads out as we expect with the right patient population from what we've learned from the MINDFuL trial, then it's a very clear path to the registration program as CJ had talked about linking the cognitive aspects of EMAC to the cognitive aspects of CDR and then getting the functional scale of CDR, which comes after cognitive changes over time. So the link is very logical.
The correlation between EMAC and CDR is very logical. And so we think that this package had explained appropriately to the midsized EBITDA biotech companies that have an interest in neurology, all the way up to the large pharma. I think it's going to be a very attractive program.
And it does appear that there are no further questions at this time. I would like to hand it back to David Moss for any additional or closing remarks.
Thank you, Cloey. 2025 was a year of significant progress for INmune Bio. We completed and analyzed the MINDFuL Alzheimer's trial, advanced CORDStrom towards registration in RDEB and positioning commute for its next stage of development in prostate cancer. As we move toward through 2026, our priorities are very clear: advance CORDStrom's towards marketing approval in the U.K., EU and the U.S., secure regulatory clarity on the path forward for XPro and build the partnerships and financial support necessary to bring these programs to patients. On behalf of the entire INmune Bio team, thank you for your continued support and confidence in our mission. We look forward to updating you on our progress in the months ahead. Have a great evening, everybody.
Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.
Inmune Bio, Inc. — Special Call - INmune Bio Inc.
1. Management Discussion
Good morning, everyone. I'm David Moss, CEO of INmune Bio. Thank you for joining us today. INmune Bio is a clinical-stage biotechnology company developing therapies that target neuroinflammation in neurogenerative disease. Our lead program, XPro1595 is a first-in-class selective soluble TNF inhibitor. It works by blocking the form of TNF that drives pathology while preserving the form that supports normal immune function. That distinction is central to everything you'll hear today. The objective of today's webinar is straightforward to demonstrate how clinical results of our Phase II MINDFuL trial directly forged the blueprint for a registration program.
Following a very productive end of Phase II meeting with the FD&A, I am very pleased to report that we reached alignment on the core elements of our Phase III registration program. Today, we'll walk you through the data-driven rationale and the optimized design that will carry XPro through this final stage of clinical derisking. The MINDFuL with strategic lean proof-of-concept designed to solve for 2 critical variables, biologic signal and patient selection. The trial was a first of its kind, a short, small enriched population study in Alzheimer's disease. It was designed to answer 2 specific questions. Does XPro show biologic activity in patients with AD and biomarkers of inflammation and can we identify the right patient population for a registration trial.
The answer to both was yes, providing us with a validated precision medicine road map for our Phase III registration program. The data from MINDFuL directly informed every element of the registration program you'll hear about today, the population, the endpoints, the trial duration and the design, it's the foundation of everything that follows. Was that behind us, now let me introduce our speakers. Dr. Michael Woodward is an Associate Professor and Director of the Memory Clinic at Austin Health in Melbourne, Australia. He's a globally recognized expert in Alzheimer's disease clinical development and served as a site principal investigator in the MINDFuL trial. Dr. Ludwig will walk you through the Phase II data. Many of you know Dr. CJ Barton, who is our Vice President of Neuroscience and has led the AD program since its inception. CJ and his team designed the mindful trial, led the end of Phase II interactions with the FDA and develop the registration program you'll hear about today.
CJ will walk you through how the MINDFuLresults shape the trial design and what the FDA aligned up on. Also, we're delighted to have Dr. Sharon Cohen, who is the Medical Director of the Toronto Memory program in Canada. She's one of the most experienced AD clinical trialist in the world and was also a site PA on MINDFuL. Dr. Cohen will join us live during the Q&A to provide her independent perspective on the trial design, the endpoints and the clinical meaningfulness of our approach. Due to time zone constraints, the presentations have been prerecorded. But Dr. Cohen and CJ and myself will join us live for Q&A.
With that, let me hand webinar over to Dr. Woodward. Dr. Woodward.
Thank you, David, and thank you for the introduction. I'm excited to be a part of this work. Now I was a site principal investigator during the Phase Ib as well as during the Phase II trial. To get going, I'll summarize and share the data from MINDFuL, the global Phase II proof-of-concept clinical trial assessing the treatment of XPro1595 in early Alzheimer's disease. The top line data was shared last July. INmune Bio is set out to test Xpro in Alzheimer's and specifically in a population that match the biology of the drug. To this end, they initially recorded participants positive for amyloid to prove ahead Alzheimer's, but with at least 2 bottle markers of inflammation which is the sweet spot for the mechanism of this medication.
However, it became too hard to initially recruit in the time lines available -- so pragmatically, it led to a dilution of the inclusion criteria. And this study had 200 participants in the modified intent-to-treat population, but 100 in the targeted enriched population that's better matched or completely matched the original entry criteria. We will follow this targeted population, those that had at least 2 markers of inflammation across this Webinar. We also call it the ADI for the Alzheimer's disease with inflammation group. And this is the term used in the manuscript, which is on MDI rxi3 and includes participants with both biologically defined Alzheimer's, i.e., amyloid positive scan and 2 or more biomarkers of inflammation.
Now we set statistically meaningful this at 0.1, which is quite appropriate for such a small population. Due to the loss of nearly half of the sample size when analyzing this AD Alzheimer's disease inflammation population, statistical p values were less likely to be reached. -- instead Immune buyer chose to assess the meaningfulness of the data by means of an effect size and specifically the Cohen's effect size and absolute effect of about 0.2 or higher was set as meaningful in the short study.
And I might say by comparison, drugs that we use are ready for treating Alzheimer's such as donepezil, have effect size of about 0.2 to 0.3. So it's a reasonable choice of effect size. The next slide here shows the population and the subgroup, the ADI population on active drug compared to placebo drug. So there are 100 participants, 28 randomized to placebo and 70 due to expo treatment. And you can see that the general comparison of baseline characteristics between the placebo and the XPro-treated groups pretty much the same. But we do want to highlight 2 characteristics on which there was a slight difference. The CDR global score, which is the clinical dementia rating global score at baseline was slightly worse. In other words, the XPro participants were slightly more affected by the disease with 28% having a score of 1 compared to only 14% in the placebo group that were more mildly affected and this value of 1 basically means that they have significant memory problems and likely some changes in other areas.
Also, the expo participants have a slightly higher average number of biomarkers of inflammation. But this implies that the Expo group may have had slightly more aggressive disease and particularly disease progression at baseline, although by having more markers of inflammation, we also have better opportunity to show the effect of the drug. Anyway, basically, the slide here shows that the XPro group overall had probably slightly more advanced or progressive disease than the placebo group at baseline.
Now on this next slide, you can see the design of the study. We have randomization 2:1. And there's a relatively short treatment duration for this -- it's only 6 months. Many of the Alzheimer's disease-modifying trials that we're doing at the moment, go for 18 months. But remember, this is a proof-of-concept trial. The primary endpoint is the early and mild composite or the EMACC, and it measures cognition in a number of ways. This is a proof-of-concept study in which we allow ourselves to learn by measuring across modalities for a more thorough picture of the drug and its effect in Alzheimer's disease.
Now on this next slide, you'll see the first -- the primary endpoint, the EMACC, -- the dotted line being placebo and the blue line being the treatment group. You'll see that at baseline, there was a suggestion that the placebo group had worsened and the treatment group that actually improved, and there was a difference between the two. The P value was above the 0.1 that had been set. And the Collins effect size was 0.27. I think, however, it's important to see that this is showing the directions that we would like to see because of the treatment with this medication or this investigational product. Now normally over a 6-month period, we'd expect to see the placebo group decline. We did see that -- but we'd also expect to see the treatment -- I mean, a nontreated group maybe stay decline less.
But to actually see a slight improvement, I think it's quite remarkable. In the next slide, we can have a look at a couple of the other end points. This is the clinical dementia rating sum of boxes. And again, you'll see a slightly worse rate of decline, the dotted line in the placebo group than in the investigational product group. This is not unexpected. We don't expect the drug to usually cause improvement. That's why the last results were so remarkable, I think, -- but we do see a difference trending towards improved or less decline, if you like, with the inflammatory subset of the Alzheimer's group.
Now if we actually break it up into dose compliance, by which we mean people who have had at least 21 milligrams per kilogram out of the total 23 milligrams per kilogram over the trial group the trial duration, we see a bigger difference between the treatment and the placebo group. So taking the gives you a greater effect, which is, of course, what we would expect.
On the next slide, we're looking at another endpoint. This is the neuropsychiatric inventory, we see little change, which is not unusual in these sorts of trials in the treatment group, but a worsening in other words, more neuropsychiatric symptoms in the placebo group. Generally, what we're trying to do with treatment is to stop emergence of symptoms, and that's what we see in the line group that there was no change. In other words, no new symptoms emerged or no current symptoms got worse.
But in the treatment group, sorry, in the placebo group, we see that there was a deterioration. Either new symptoms occurred or existing symptoms worsened. And again, P is 2 and the Cohenn's effect size about 0.23 in the dose compliant, 0.27. On the next slide, where we see the -- but the first biomarker change. This is Fosfotelin the plasma [indiscernible] 217 is regarded as a very good indicator of brain amyloid and the neurodegeneration that occurs in Alzheimer's. And again, we see a higher rise, that's a bad thing, a higher rise in the placebo group that we see in the blue line treatment group, with a slightly greater difference in those who are dose compliant. Again, P values as shown on this slide, Cohen's effect size in the dose compliance group, 0.26, which is above the 0.2 that it was set at.
GFAP, which is [indiscernible] protein is a marker of the activity of the inflammatory cells around the amyloid plat and the higher the level -- the greater the level of inflammation. And we see a lesser rise in the XPro group particularly in the treatment compliant group than in the placebo group, which rose higher, indicating that the XPro group seem to be, to some degree, modulating that inflammation, which is exactly the postulated mechanism of action.
In the next slide, we've summarized the results across all these end points and you'll see very reassuringly in terms of hypothesis generating and in terms of going to the next stage of investigation, that most of these end points favored XPro, there's only 1 that did not. But I would say that if we look at these in we look at the FX side, remember we set point to -- many of these were around 0.2 or higher. I think we can see an overall pallet of the effectiveness of this drug in the inflammatory subset of Alzheimer's disease. And not shown here, but probably particularly so in those who are most dose compliant. So I think this is very, very reassuring. And I think it's a good indication that we have a drug that may show the effect in Phase III study, but I'll let CJ talk about that.
One of the concerns with new potentially disease-modifying therapies, particularly the new amyloid targeting therapies is ARIA. ARIA stands for amyloid-related imaging abnormalities. And there's 2 sorts ARIA-Ethe e standing for edema and ARIA-H for heat standing for hemorrhages, particularly microhemorages. These are actually causing a significant number of people who have to withdraw from the current amyloid targeting therapies that are on the market and certainly have to be carefully and regularly monitored with MRIs and other safety assessments. -- it would be great if this drug can be shown to have no risk of area. And indeed, we saw that both in the placebo group and in the treatment group. There were no examples of or no cases of ARIA A or H -- and that was seen over the whole population, 206, not just the ADI population.
But -- it's also important to know that these patients, both in the placebo and in the treatment with the investigational product group had risk factors for ARIA. Some of the risk factors that we recognize are pre-existing cerebral microbleeds, and you'll see that nearly 35% of both groups had these at baseline, the use of any thrombotic agents. These agents were not excluded unlike the protocols for many of the new amyloid targeting therapies were being on particularly an oral anticoagulant is exclusion from the study. This study allows you to be on anticoagulants and antithrombotics. And you'll see that about 25% to 28% were in this particular study. Also, the LOE 4 gene is seen as a risk factor, particularly for ARIA-E and in this group, we had 55% with 1 APOE 4G, and we had 12% to 13% with 1 -- with 2 copies of the APOE4 that's E4 homesites.
These are the ones who are particularly at risk of ARIA-A. And again, despite this risk profile, we do not see ARIA-A or ARIA-H in this study. And I'd like to hand you over to CJ, who I've known for over 5 years. And I cannot tell you the passion, the knowledge and the wisdom of CJ in seeing that the human necrosis factor angle is very important for potential treatments of Alzheimer's disease and not being linked by the amyloid is bad, let's get rid of the amyloid approach but so many other drugs that companies have taken. So CJ, you're up. .
Thank you, Michael. We very much appreciate you taking the time to and your considerable expertise and experience to this webinar. My name is C.J. Barnum and I'm Vice President of Neuroscience at INmune Bio, and I've had the privilege of leading XPro's clinical development in Alzheimer's disease. You just heard what the drug did in Phase II. The key takeaway, consistency, cognition neuropsychiatric symptoms, plasma biomarkers, imaging, patient-reported outcomes every assessed domain favored XPro in the ADI population, that totality of audience is what the FDA saw when they reviewed our data. So where do we go from here? Today, I will show you the road map, walk you through why the FDA agreed with our approach and what gives us confidence it will work.
We recently completed an end of Phase II meeting with the FDA. That's a big milestone. We presented our full scientific and clinical rationale to the agency and had a structured back and forth about the path forward. Before I tell you what they said, let me show you the design and we presented to them. On the left is patient selection. XPro as designed to treat the subset of Alzheimer's patients whose disease is driven by TNF-mediated inflammation. These are patients who carry at least 2 of 4 inflammatory biomarkers.
CVI protein, sedimentation rate, hemoglobin A1C and at least 1 ApoE4. These biomarkers were chosen because they're mechanistically connected to the biology XPro targets. The study design has 2 stages that allow us to replicate the findings of mindful in a Phase IIb and then seamlessly roll into a Phase III registration study if we hit our marks. This approach balances scientific rigor with speed, confirming this signal before scaling to registration. The Phase IIb is a 9-month confirmation stage that will enroll around 300 ADI patients. The co-primary endpoints are EMAC and plasma pTau-217 and mindful, we saw an effect size of 0.27 on EMAC at 6 months and an initial signal on PTL217, suggesting XPro is impacting the underlying disease process.
The additional 3 months and larger sample size gives us more time for placebo decline and the statistical power to detect separation. Advancement to Phase III will require 1 of these 2 primaries to succeed. The trial is statistically designed so that either 1 succeeding as a valid win. The decision gate is a derisking milestone. You get a powered go, no-go signal before the Phase III capital commitment. The FDA also recommended we include about 20% non-enriched patients. These are patients without the inflammatory biomarker profile as an exploratory arm. If XPro shows a signal in that group, the addressable market expands dramatically. If there no signal, those patients don't follow us into Phase III, keeping enrollment focused and cost efficient.
That's upside without obligation. Focus where the drug works and don't spend where it doesn't. Phase III is a registration trial. We'll enroll approximately 1,000 enriched ADI patients with the CDR Sum of Boxes as the primary outcome measure at 18 months. That gives us the statistical power we need for regulatory approval. We're targeting a 2027 start, and David will walk you through the funding strategy. The FDA have validated our approach in 4 important ways: First, they endorsed our biomarker-based enrichment strategy. They were comfortable with the mechanistic link between TNF and each biomarker, the assays, and this was the 1 I expected the most pushback on using a combination of biomarkers rather than a single biomarker to define our patient population.
Second, they endorse the adaptive Phase IIb/III design, including the decision gate with EMAC and PTAO217 as the go-no-go endpoints. Third, they agreed on CDR Sum of boxes as the sole Phase III primary endpoint. It's the registration standard for Alzheimer's because it captures both cognition and function in a single measure. And fourth, they recommended including a nonenriched cohort in Phase IIb. This serves as a built-in validation of our enrichment strategy.
But before I move on, 1 thing -- just last week, the FDA leadership published in the New England oral medicine that a single, well-controlled trial backed by mechanistic coherence and biomarker data is now the default standard for approval. That means a single well-designed Phase III trial with a kind of convergent evidence we already have could be sufficient for registration. So let me address the question we get asked most often -- if the CDR sum of boxes showed no separation in Phase II, why are we confident it will work in the Phase III study.
Two reasons, and both come down to how the CDR Sum of Boxes works and how long it takes to show change. Let me explain. Simply speaking, cognition is the leading indicator, and function is the lagging indicator. Cognitive decline proceeds and predicts functional decline.
In the kind of early-stage patients we enrolled in mindful, functional deficits are barely detectable if they're present at all. More importantly, functional deficits require more time to become measurable because deficits emerge slowly. Cognitive deficits on the other hand, show up early, and as a result, can be detected with sensitive tools like EMAC, the data bear this out. The correlation between cognition and function in early disease where functional deficits haven't emerged is low, approximately 0.41. However, as functional deficits appear, this correlation strengthens considerably, climbing to 0.65 at 18 months.
At this point, the CDR Sum of Boxes is able to catch what EMC has been detecting all along. In clinical trials, cognitive treatment effects consistently propagate into detectable slowing of functional decline and 18 months. Let me make sensitivity and difference concrete. Think of EMAC is a stress test for the brain. It measures some cognitive changes -- for example, your ability to recall a word list or how fast you can process information. Take a word recall. You read a patient 15 words, and they recall as many as they can. And then you repeat this process across several trials. In the early stages of disease, the patient might recall a few words or take longer to recall those words, but the patient can still manage life independently. This is what EMAC captures.
The senior Sum of Boxes is asking a fundamentally different question. It uses broad scoring categories, like none, slight, mild and relies on a caregiver reporting someone who may or may not be attuned to the patient's day-to-day cognitive changes. That subjectivity, combined with broad scoring categories, means a patient has to decline substantially in day-to-day function before the score moves. EMAC detects subtle cognitive changes. CDR Sum of Boxes links for the consequences. EMAC captures at 6 months, the CDR Sum of Boxes captures at 18. What you're looking at here is a conceptual framework, not derived from trial data but it shows the window where each instrument can reliably detect a treatment effect.
The X axis depicts the stage of disease and the y-axis reflects the ability to detect a treatment difference. The purple horizontal band is the detection threshold, the point where a clinical instrument can reliably measure change. Blue curve is EMAC, because it is a sensitive performance-based measure, it crosses that detection threshold early in the disease process. At 6 months, it is sensitive enough to pick up the treatment signal as it did in mindful, at 9 months in Phase IIb with more time and a bigger sample, the likelihood of detection increases. The Magenta curve represents the CDR Sum of Boxes. It uses broad scoring categories. So a longer change and day-to-day function is needed to move from 1 score to the next, which means across the threshold later. Nine months may be a bit too early still, which is exactly why the CDR Sum of Boxes is not a Phase IIb decision-making endpoint.
By 18 months, however, untreated patients who have shown measurable decline in the CDR Sum of Boxes, allowing us to detect treatment differences. That's when it becomes the right instrument, stay in patients, same drug, just different windows on the same underlying biology. The EMAC shows early change that when time becomes measurable by CDR Sum of Boxes. We have the leading indicator now we need the time. Let me bring this all together. We built this program on a simple premise that Alzheimer's patients with TNF-driven inflammation are a definable treatable population. It's not a retrospective discovery -- it's a hypothesis we've been testing from day 1. And that's what the FDA validated in our end of Phase II meeting when they aligned on our enrichment strategy, our endpoints and our trial design.
The Phase II data gave us an early cognitive signal on EMAC at 6 months. The literature tells us that signal will translate into functional outcomes at 18 months. The adaptive design means we'll confirm it at 9 months before we scale to the full 1,000 patient registration trial. A [indiscernible] sequence to answer the next question before committing the next dollar. This Phase III is built on a scientific foundation, the right drug, the right patients, the right measure at the right time to generate a definitive answer efficiently.
I'll hand it over to David to walk through the path to execution. I'm looking forward to taking your questions at the Q&A. David?
Thank you, C.J. Before we open the floor to questions, let me address the question I know many of you have on your mind, how does this program get funded to move forward? The Phase IIb/III design is deliberately staged. The Phase IIb portion, roughly 300 patients and 9 months of treatment is where we will confirm the enrichment population's responsiveness -- the data at 9 months does not meet our threshold and at least 1 of 2 current primary endpoints we will saw. That built-in decision gate limits capital exposure and makes this an attractive structure for partners.
To that end, we are in the final stages of engaging an advisory bank to run a formal global partnership process. This package we bring, Phase II clinical data, FDA alignment on registration design, a well-characterized patient population and a first-in-class mechanism with no signs of ARIA is, we believe, compelling for mid to large pharma partner with interest in neuro inflammation or Alzheimer's disease. I think everybody on the call knows this is a huge unmet need with a very large market. In parallel, we continue to advance CORDStrom, which provides both potential revenue and a priority review voucher.
And these catalysts, as as these catalysts develop, our position in the capital markets should strengthen. On the operational side, we're already moving. Protocol development is underway, and we have begun planning for CMC scale-up and site feasibility assessments. We are building this program so that when the funding is in place, we're ready to go. It's now time to move to Q&A. Let me look for questions, and I'll come right back. .
All right. Thank you, everybody, and CJ and Sharon for joining us. I have a handful of questions, but I'm going to start with my question first, if you don't mind, to you, Sharon. I was very fortunate enough to see you present our MINDFuL Phase II data at AAIC. Thank you so much for doing that in August. And obviously, we had a good sized room there. And I'm wondering if you can share any of the responses that you received from your presentation.
Yes. Thank you, Dave. It was a pleasure to present that data. And it was a pleasure to receive responses from colleagues who had a lot of questions and a lot of interest. They didn't view this as a failed trial because we didn't meet our primary at 6 months. They view this very much as how do we move forward because on so many endpoints as Dr. Woodward showed, we favor XPro in the enriched population. And I think that in a proof-of-concept trial, you want to figure out who's the best group of patients to study.
One comment that was repeatedly made to me is that people were so impressed that at 6 months, we could see so many endpoints show positive effect size. And that's not what we're used to with disease modification in Alzheimer's. We usually have longer Phase II studies and don't expect to see things at 6 months. So I think that was also a takeaway.
No, I appreciate that. Thank you. It was a large room with a lot of people there. It was a great experience. One other question. You've been involved with multiple companies developing many different therapeutics in AD and have a good grasp of the AD development field given your experience. What do you believe is immune bio and/or XPro's greatest differentiator in strength?
No, that's a great question, and I have been involved in clinical trials for 30 years, Phase I to Phase III in Alzheimer's research. And as we move more and more in our field towards precision medicine, really defining the patient population clinically and biologically. I think the XPro program really takes this to the next level, where we are enrolling based on certain biomarkers match to the drug's potential. And looking at this enriched population from the standpoint of cognition function, patient-reported outcomes and biomarkers. That is definitely the way to go.
With the anti-amyloid treatment approaches where we've had some success, it's taken us a long time with a lot of failures to get where we got, and we needed biomarkers. They were essential to the success of those programs. And I think that they will also lead to success here. There is a great appetite for targeting other pathologic abnormalities in Alzheimer's beyond amyloid. And inflammation is such an important target and not only could it be a stand-alone way of treating Alzheimer's, it could augment the effect of anti-amyloid therapies. And furthermore, reduce the risk of ARIA for anti-amyloid therapies by modulating inflammation in the brain. So many ways, there is a unique aspect to XPro. And yes, I'll stop there.
No, I appreciate that. And I think this next question is it kind of dovetails into what you just said about neuroinflammation in the disease, and I'll present this one, I think, to CJ and well, maybe both of you. Treating their own inflammation is important in neurodegenerative disease and specifically Alzheimer's, -- number one, what makes XPro different. And then the second part of that is the ADI or this inflammation rich population, how does it improve the chances of success in the new trial. In other words, this inflammation enriched population, that's probably best for CJ to start. .
Yes. So the first part of that question again, David?
About the uniqueness of XPro in targeting neuroinflammation?
Yes. So I think this is where I get particularly excited about this drug and why I've been working with it for it's nearly 20 years now is because it does something very different. The immune system, and it really -- in my mind, this changes the way we view the immune system and how to target and treat it. Because what we're traditionally doing with targeting inflammation is we are suppressing it. I mean that's how the therapies work and they're very successful when you have diseases where the immune system is extremely strong. But neurodegeneration is a little bit different. Neurodegenerative disease is a little bit different because it appears to be that the immune system just becomes dysfunctional. You get the sort of immune exhaustion that occurs -- and that is a fundamentally different thing.
So it's not just immune system is overactive, which it is, but it's also not doing the homeostatic stuff that it needs to do. And I think we fundamentally underappreciate how important the immune cells are to the brain. I always use this example of neurons being like infants, right Infants, they can't feed themselves. They can't bade themselves. They can't socialize themselves. And they require parents to do that or else they die and they can't thrive. And that's really what the immune cells are. The immune cells provide all those parental needs to the neurons. And I just don't think we appreciate that well enough. So from the perspective of developing therapies to target an immune system that's not functioning properly. You don't want to suppress it because if you suppress it, you reduce some of that inflammation that's driving some of the disease, but you don't get the benefits of repair, restoration and that sort of thing.
And that, in my opinion, is the key difference. We're able to -- XPro is able to normalize the function of the immune system in a way that not only stops it, but allows it to repair and rebuild and restore the things that we've lost. So I think that's what is sort of the key piece, the differentiator with XPro. And I forgot the second question, too.
Second part of the question was the inflammation rich population, how it improves the chances of success in the trial. .
Well, I think the simple explanation is you just need to look at cancer, right? I mean cancer started to become extremely successful, and we started to match the mechanism of action with the biology of the patient, right? So we don't talk about therapy for cancer -- we talk about a therapy for specific types of cancers matched by biology, even within certain cancers like breast cancer, you're matching it within biology, HER2-positive, right? So this is where the success ultimately comes is matching the pathology of the patient with the mechanism of the drug. And to be fair, the reason taken a little bit longer in the neurodegenerative space because it can't really do a biopsy of the brain, right?
So we don't have the same tools that we have with peripheral cancers where we can biopsy and really look at the mechanisms and tie the biology. Just so happens that inflammation is one of the things that we can measure, we can assess because we know a lot about it. We know how inflammation between the periphery and the central nerve system reflect each other. And I think that's a real positive and allows us to be somewhat early in matching pathology with patients because of that.
And maybe I could just add 1 point. I agree with everything that CJ said, but when you have an enriched population with more inflammation in the brain, the untreated group, the placebo group is more likely to decline and you are much more likely to see a separation between treated and untreated.
Yes. And then one last question that I'm going to tag on is why did you see a bigger effect on the neuropsychiatric patients, the neuropsychiatric inventory or the symptoms associated with EXPAREL, -- is that because neuroinflammation is a big driver of neuropsychiatric symptoms.
So do you want me to take that, Sharon?
Sure. I might add on after.
So what's interesting is the literature with inflammation in neuropsychiatric disease is actually longer and has more history than even with neurodegenerative disease. And we know a lot about how inflammation and neuropsychiatric disease sort of go hand in hand. So that's not particularly surprising. The other thing is from a neuropsychiatric disease perspective versus neurodegeneration, we're typically talking about cells that are losing connections, right, as opposed to losing cells in neurodegenerative disease.
So if you restore the immune system or they can repair the biology that currently exists, you should be able to change those neuropsychiatric diseases or those symptoms pretty early, now it's not entirely as sometimes the neuropsychiatric symptoms are a result of the degeneration that occurs over time. But we know in depression studies, for example, those changes occur quite rapidly when you give -- for example, TNF inhibitors to patients with neuropsychiatric disease, you can see changes very quickly because you're able to restore the balance pretty quickly, if that makes sense.
Yes. It's so interesting that neuroinflammation has potentially a direct impact on behavior and neuropsychiatric symptoms. And I think there's another possible explanation that could go hand in hand. If XPro is reducing cognitive decline, then as a downstream effect, you have less frustration less agitation or just the way cognition drives function cognition drives behavior as well. So I think both explanations fit hand-in-hand.
That's great. Thank you. I have a question here from Tony. He says, "Hello, David, with the Phase II trial being successful, why do you think it's not reflected in your share price." That's a really frustrating question that we deal with. I think twofold -- I'm going to answer that twofold. Number 1 is most people think the trial was not successful. They don't realize that in a Phase II study, -- if we would have had the right population in their all 200 patients or close to 200, it probably would have been a great success. We're amongst the first ever do an enriched neuroinflammation trial. And when you're the first, there's just certain things that you don't know. And really, this trial was an R&D experiment and it defines the patient population for a successful trial.
Second thing I going to say is that when you're the first doing something novel and there is nothing to look at a proxy or an example of -- for example, there were many anti-amyloid trials that were run that failed and they refined, they refine, they've refined and they got better and better and better, and they finally got it approved. We don't have that luxury in what we were doing. And so we're really writing the playbook for this trial, and we're learning along the way to make this trial successful. And in biotech development, my experience has been that the companies that are doing, let's say, a cancer drug that follows the pathway of many other successful cancer drugs that have been approved -- it's very easy for investors to jump on board. But when they're going after something for the first time, think of like a CAR-T, for example, the initial CAR-T companies until they had success -- really good data it took a while for them to get valued really appropriately.
And then once they had the data and got it approved, they were widely valued. And we had hundreds of CAR-T companies after that instead of just a few. I kind of feel like we're in that avenue, where we're pioneering an approach. We're writing the playbook as we go. We're doing a very novel approach in Alzheimer's disease, which can expand into neuroinflammation, and we're paying the price -- over time, we'll figure out a way to get there. I think we've come with a very sophisticated and rational strategy to get there. It's going to take us longer than we had hoped, but I think it's a prudent way to go.
Okay. Next question. I'm going to send this 1 to you, Sharon. What would a potential future approval of a drug like XPro mean for people living with Alzheimer's disease and their families.
Well, this would be huge. This would offer another choice in therapy to slow down disease, potentially stabilize symptoms, cognition, function, neuropsychiatric symptoms and get at an important underlying pathologic mechanism without -- as CJ said, without suppressing the immune system. We don't want to damage the ability of immune system to do its job to provide terrific factors and everything else. We want to shut off the pro-inflammatory aspect. So for patients, for families, this would be a welcome addition, whether it's taken alone or in combination with anti-amyloid therapies, and the safety profile is really important here, and it's a mode of administration.
People can take the drug as a subcutaneous injection once a week at home, they're not tied to infusion centers, they can travel. We're talking about mild patients who have busy lives, some of them still in the workplace or busy traveling around the world, and they can self-administer the drug -- and they don't have to be tied to MRI facilities for ARIA monitoring. There is no ARIA -- so again, this would be a very welcome addition to the treatment armamentarium for patients and for clinicians, the ease of use would be also welcome.
Yes. And just tagging on to the question. We have 2 platforms, 1 that's in a pediatric and then obviously, this 1 is to treat Alzheimer's patients. And when you see the families, my father suffered from Alzheimer's disease and you see that patients you get very motivated to try and do something for them. And we've got 2 great platforms at this company, and I can tell you that everybody in the company is extremely motivated to figure out a way to bring these products to the patients and their families. We always like to say that you don't really treat the patient, you treat the family, and it's very, very true. Next question, CJ, the FDA end of Phase II meeting and alignment is a large milestone for XPros development. What makes you excited for the next steps of XPro of this recent end of Phase II meeting with the FDA. .
I mean there's a lot of things to like. But I think one of the things that was really surprising to me was the FDA got it. They understood it. And -- and the conversation was so easy. That was really exciting. And what it told me was that we have a partner in this -- so sometimes people will talk about the FDA as sort of butting heads and trying to figure out a way how to work with them and get them to understand what they're doing. I think they understand they get it. They were -- they were very accommodating. They -- and so I think as we move forward, our ability to engage with them in a meaningful way is better than I would have expected.
And I think I'm really excited for that because that potentially is a major roadblock. You can have a drug that works well and people just don't understand it, right, for whatever reason. And that regulatory hurdle is pretty big. So I think that being able to get past that is a huge milestone for us. And really, it's just a matter of let's run the study. Let's get it done. And that really excites me.
Great. Thank you, question here from Tim, can you speak to the partnership path in previous discussions with pharma and their key issues, concerns, pushbacks.
I'm not going to speak to what we have currently going on. I'll tell you that we recently just got the minutes from the FDA -- it took us a while to go to get that. And now that we have that in hand, we have a real complete package as CJ has outlined to you. We have a clear path on how EMAC at the 9 months [indiscernible] the 9-month period drive the go/no-go decision to go to registration part of the program or not. It's not a huge costly expense for really, frankly, a multibillion-dollar opportunity, treating millions of patients that are in need of additional therapies. We think the value proposition with the complete package is very attractive. And really what we want to do now that we have those minutes in hand, which are just a few weeks old now. is due a very blanketed approach to the mid- to large-sized groups out there t`hat would have an interest in neurology.
And we do have some ongoing discussions, and we're going to continue those. We're sharing this information. It is not a quick process. pharma, by nature are very conservative, and it takes a lot of time to educate them, just like we had to educate the FDA and we have to educate the investor market. we also have to educate partners especially in this is a new modality of an approach. And I'd say concerns or pushbacks I don't think we've really had a lot of pushback. I mean, -- it's also a timing issue, finding the partners have to be in a position where they're ready to do something. There's a lot of external factors where they may or may not be ready at a particular time. So timing is the issue, but we haven't had any real -- I mean, what would you say is any -- have we had any negative real pushbacks, CJ? .
No, I don't -- I wouldn't characterize it as negative. I think you're right. I mean pharma -- the way pharma makes decisions is quite interesting if you get a peek behind the curtain. And it's not always clear why and how they decide things. But I think to David's point, the -- they were really waiting to see what the FDA said about the data and the path forward. I mean, that's a huge decision-making point. Being able to come to them and say, "Look, here's what we discussed with the FDA here the minutes." They'll be able to see that there's alignment that there is a path to a commercial path for this, where I think if you -- you're doing a completely different mechanism, which, to be perfectly honest, I don't think -- generally speaking, the scientific community understands inflammation. I think they know it's important, but I don't think they understand it. And so if you've got a new mechanism and you've got a new strategy, we're using biomarkers to enrich -- and we're using biomarkers in a different way that they are, they want to understand how does the regulatory agencies view this?
Do you really have a program where there's a path to registration -- and now I think once with this alignment, we'll be able to put this together, we'll be able to have discussions that I think will be meaningful. But to David's point, it does take time. I mean there's multiple -- these big companies have lots of different divisions and they sort of go back and forth. So we'll get there. But I think this is really -- I wouldn't call it a push back they just wanted to see how the regulatory agency was going to respond.
Yes. And I'll just add 1 last thing is that if we don't get a partner or we don't like the terms of what a partnership look like, where next year should have CORDStrom approved in 2, maybe 3 jurisdictions: U.K., Europe and the U.S. If we get it approved in the U.S., it comes with a priority review voucher. -- and we should be in a much better capital situation, having a product approved. There's partnership potential there and obviously revenue generation and potential -- that's money that at that point, we'll be able to turn around and complete the Phase IIb portion ourselves. So we're going to run this company prudently -- we're going to -- our primary goal is to get expert over the -- sorry, to get courtroom over the line first.
That's where our growth is happening, that's where our resources are going. Meanwhile, in the background, we're getting extra ready for the start of the Phase III program or an everything we can with the limited resources we have. It's a wonderful program. It has a real potential meaning in patients' lives. It's a product that needs to see patients, and we're dedicated to figuring out a way -- so I don't have any other questions. I'll give 1 minute for people to see if they're going to type anything. Anything Sharon, that you want to say or CJ, you want to say before I -- if I don't get any more questions before I read the conclusion and sign off.
Maybe I could just comment for a moment on the unmet need. Yes, we have antibiotic antibodies. And that's great. And the landscape is evolving, and many of us are adapting to patients being on these treatments. But they're not for everybody. In many jurisdictions, APOE4 homozygous are excluded people on anticoagulants are excluded. And I think having an alternative option for people who have this high unmet need is so important and tackling inflammation, I think for all patients with Alzheimer's is important. But if you enrich for this and you don't have the exclusions that we have for monoclonal antibodies, either on the market or any trials, you will recruit well for your Phase IIb/III study. I think that I, as an investigator at a clinical trial site would have no problem enrolling patients in this upcoming study. I look forward to that. .
Can I ask a follow-up question to that, Sharon, because I'm very interested in your thoughts as a CPI. If or as the anti-amyloid therapies become more standard of care and people are doing clinical trials. My take is there is real I don't want to say unrest, but reservation about how do we incorporate new therapies where anti-amyloid are standard of care, right? It changes -- somewhat changes the calculus of how -- who you enroll and how you enroll your studies and from a regulatory safety perspective, are these things going to play well together. How do you -- without putting words in your mouth, how do you see XPro in that regard as it relates to would you have any reservations of treating someone with XPro if they were on an anti amyloid therapy outside of the general context of this is a new therapy. But from a mechanistic point of view, what are your thoughts?
So I think combination therapy makes good sense and how do you proceed with that when you've got 1 approved therapy and 1 still in the development stage. So assuming anti-animal antibodies are standard of care. They're getting there, but in Canada, we don't have reimbursement yet. So there'll be lots of people who won't be able to afford or access the drug. So you'll still have a population of treatment-naive patients just on symptomatic treatment. But if you enrolled patients on the background of anti-amyloid therapy in your trial, you'd be looking for enhanced benefit over what you would get from the anti-amyloid approach alone. And so that complicates trial design. Nevertheless, it's doable. What I would love to see is a trial where we have patients naive to both therapies enrolled in roughly the same time frame on both treatments. And why do I say that?
The risk of ARIA from anti-amyloid monoclonal antibodies is in the first 6 months or so. That's where it's highest. And so if you're past that window of RIA, you're not going to be able to demonstrate that XPro potentially reduces the risk of ARIA. So if you have people who are already on [indiscernible] and have been on for 9 months a year, and then you add XPro. Hopefully, you'll see the increased benefit, increased efficacy, but you won't see the mitigation of the safety profile. So it's complicated. I sort of ramble here, but you can see there's lots of different ways that one could think about designing combinations.
That's great. Well, I really appreciate everyone joining. And I certainly, from the bottom of my heart, really want to thank Dr. Woodwork and Dr. Cohen for their time today and for their continued partnership as investigators. Their experience and their willingness to share their independent perspective is invaluable. And we're very fortunate to work with them. I also want to thank each of you for joining us. We understand that confidence is earned and our job is to earn it with data, with execution and with transparency. -- today was 1 step in that process. If you have follow-up questions, I didn't get to us, our Investor Relations teams available -- you can find the contact information on our website or at the bottom of any of our press releases. We look forward to updating you as the program advances.
Thank you for your time today and your continued interest and support in INmune Bio.
Thank you. Thanks, Sharon.
Thank you.
Inmune Bio, Inc. — Special Call - INmune Bio Inc.
1. Management Discussion
Good afternoon, everyone, and thank you for joining us. I'm David Moss, CEO of INmune Bio. Today, we're proud to share pivotal updates regarding our MissionEB Phase III clinical trial and the impact of CORDStrom on Recessive Dystrophic Epidermolysis Bullosa (RDEB), as you'll hear it in this presentation.
Today's event is being recorded and will be available on our YouTube channel later. As we begin, I remind everyone of our forward-looking statements, which is on the screen.
Now before we dive into the data, I want to introduce two individuals whose dedication has been instrumental in reaching this milestone. First, I'm pleased to introduce Dr. Mark Lowdell. Many of you know Mark and is the inventor of CORDStrom and a pioneer in the field of cellular therapy, with a distinguished background in developing advanced medicinal products, his vision for using pooled umbilical cord-derived mesenchymal stromal cells, has paved the way for a potential new standard of care in treating systemic inflammation.
I'm also thrilled to introduce Dr. Anna Martinez, the investigator of the MissionEB trial. Dr. Martinez is the clinical lead at Great Ormond Street Hospital, or GOSH in London and a leading expert within the Epidermolysis Bullosa field, bringing years of clinical expertise in pediatric dermatology to this program. Dr. Anna Martinis is the Specialty Lead of the Pediatric Dermatology Service at Great Ormond Street Hospital and Honorary Senior lecture at the Institute of Child Health, University College London. Anna trained at St. Bartholomew's Hospital and was appointed as consultant in Pediatric Dermatology at GOSH Hospital in January of '03. Since her appointment, Anna has led the National Commission Highly specialized service for Epidermolysis Bullosa and is a world leader in management of skin fragility diseases. Anna runs the pediatric dermatology services at GOSH and manages children with many skin conditions. Most importantly, as the Chief Investigator who designed and led and was the key enzyme in this trial, she has been at the forefront of shifting the treatment paradigm for RDEB from simple wound management to systemic disease modification.
At this time, I'll hand the call over to Dr. Mark Lowdell to discuss CORDStrom, how CORDStrom is addressing the critical window of opportunity for these patients, before passing the webinar to Dr. Martinez. Mark, the floor is yours.
Thank you, David, and thank you, and welcome to everybody who's joined the webinar. So as David said, we're going to concentrate this evening on -- or this afternoon on CORDStrom, which now has an INN name, as you can see, and its first indication, which is in recessive dystrophic epidermolysis bullosa or RDEB. And as you've seen, and I hope we're going to demonstrate to you this evening, this really is a disease-modifying therapy for this horrible disease. And I'm really excited that Anna is here to give you her observations from the trial and recount the clinical impact.
So what is CORDStrom for those of you that might be new to this? It's a mesenchymal stromal cell product, but uniquely, it's a pooled product from 4 donor umbilical cords. Its mechanism of action are very well understood now, and I'll go through that in some detail towards the end of the webinar after Anna. But we know it can suppress inflammation in RDEB and other diseases. It can enhance wound healing in RDEB and other diseases. And in the right setting, it can suppress fibrosis. We've got global IP, and we've already established large volume manufacturer, which you'll see later on in the webinar.
So Anna, it's a delight to have you on, and thank you for fitting this into your busy clinical schedule. The floor is yours. Please go ahead and tell us recount the trial and where you hope us to take that -- expect this to go. Thank you.
Brilliant. Thank you very much, Mark. When I say our next slide, I'll let you move them, but thanks. I'm delighted to be here. Thank you for that introduction. So -- and I'm going to talk to you about RDEB processor dystrophic EB. So next slide, please.
So obviously, this is what it looks like from the outside, and this is what families and children have to live with every day. But actually, next slide, this is a multisystemic disease. And by that, I mean that it affects most parts of the body, as you can see here. And importantly, next slide, please, it's a disease that gets worse and worse over time. And we published the natural history of this particular type of EB about 3 years ago.
Next slide. And why that's relevant is because the disease changes with time. And when babies are born, they often have absent skin. And this can take several months to heal, but it does heal. And generally, the first few months of life are relatively stable.
Next slide. After that, from age 18 months onwards, we see this increased burden of disease. And although wounds open and close, they're not chronic, we start to see first signs of internal involvement, this multisystemic disease. This inflammation starts boiling -- the weight starts to fall, anemia starts, and we start to see esophageal strictures and scarring in the esophagus.
Next slide. Now why this is important is this is a really fantastic window opportunity for anti-inflammatory therapies, which we can see. So under the age of 10, we know that this has an opportunity that we will lose if we don't try and treat early.
Next slide, please. So from the age of 10 to 20, we see this increased skin involvement. Wounds start to become chronic. By that, I mean that they don't close within 21 days. We see bacterial colonization and what we call multiple comorbidities, many other complications aside the skin, and this dramatic reduction in the ability to heal.
Next slide, please. Now also because of this phenomenal levels of inflammation, which we know has been monitored, this inflammation affects how the body works in these major pathways in the body, leading to pubertal delay, growth failure, osteoporosis, fractures, anemia. And we know if we wait, the longer we wait, anti-inflammatory therapies, although they will help, they're likely to become a little less effective.
Next slide, please. Now from the age of 20 onwards, we see that the disease now just escalates. There's an exponential increase in inflammation. Wounds at this point very, very rarely heal.
Next slide. And tragically, these patients are at high risk of developing cancer, squamous cell carcinoma. This is the life-threatening complication of this type of EB. So these tumors grow rapidly. They're often on the extremities where these children have had absent skin from birth and chronic inflammation and trauma. And the mean survival is just 2.4 years when the tumor develops. And most of these children, young people will die in their late 30s or early 40s.
Anti-inflammatory therapies, antifibrotic therapies at this point are likely to be less effective. The longer we wait, the harder it is to put out the fire. So what do people living with EB right now actually want? What are their priorities to get better? And we -- I was on the steering group of this prioritization survey of just under 800 -- well, 900 members of the global community worldwide. And for dystrophic -- for this particular type of EB, what people living with recessive dystrophic EB want now is treatments to improve pain, itch, mental health, reduce inflammation and to help their esophageal issues. So they want anti-inflammatory therapies.
Next slide, please. And what therapies do we actually have at the moment? We have two. And this one was called Fucibet, which was approved in the U.K. a couple of years ago. And it's a cream, in fact, it's quite greasy. Most of its sunflower oil, 10% is Birch bark, it's meant to be anti-inflammatory and it's for children over the age of 6 months, you're meant to put it on all wounds. It's expensive. And unfortunately, most of our patients don't want it. It's uneffective and so they stop.
The second treatment that we have available, but not in the U.K. is gene therapy. And this is called B-VEC, and it's a topically applied gel -- that is used -- that has a nonintegrated herpes simplex virus, which carries the collagen 7 to the skin. Now the issues with this is you have to apply it under the age of 3, you only can use 1 ml at a time. And so it really only covers about 1.75 of about less than 2 credit size wounds, okay? So you're talking about very -- you're only allowed to treat a very small area. And when you're over the age of 3, you're only allowed to use just under 4 credit card worth of body surface area to treat at a time. So you can imagine when you have a child with a back like this that it's really not going to go very far. And importantly, it's really not going to have this systemic anti-inflammatory effect. It's just not going to do that.
So next slide. The other thing about B-VEC is it's extremely expensive, it's approved in the States. Actually, they've been using it now for 2.5 years, and it's approved in Europe last year. It's not approved in the U.K. And the cost per patient per week is about GBP 25,000. So the estimated global lifetime cost of this drug on its own is about $15 million per patient. So those are the 2 treatments that we have.
So there's an urgent need for early systemic anti-inflammatory therapy. And we knew this, and in 2012, Cure EB, which is a charity, funded a study called EBSTEM. And this was an open-label study treating 10 children at Great Ormond Street with recessive dystrophic EB. And these children received 3 infusions of bone marrow-derived stem cells. And we really saw very, very promising results, so promising that we went to the NHS and we said, we want to start giving this treatment to our patients. And on the back of that, they said, no, you can't unless you run a double-blinded placebo-controlled study.
Next slide, please. So that took us 11 years to finally open. I was awarded a big grant from NHS England, which stipulated we have to unfortunately give placebo intravenously to these children, which you imagine was very complicated. And we ran the study at Great Ormond Street and the other National EB Center for Children at Birmingham Children's Hospital.
Next slide, please. So the study was called MissionEB, and this is a lovely logo that Gabriel, our first patient on the study designed, and this is my core team.
Next slide, please. This is the cartoon of the study. So essentially, patients were divided into 2 groups. They either had stem cells, CORDStrom to start 2 doses, 2 weeks apart and then a 9-month washout period and then placebo or they had the opposite. They would have to have 2 doses of intravenous infusions of placebo, a 9-month washout period and then CORDStrom. And throughout the study, we collected a whole load of outcome measures.
So the aim of the study was obviously, to assess if they were safe, but also do they benefit our patients. And importantly, we really wanted to explore the views of parents and families on the acceptability of the treatment, and we did this through blinded interviews.
So the first patient was treated Gabriel, the boy you saw there in 2021, and we completed the trial with no toxicity events throughout the study related to the drug; 37 children were recruited. That is the largest study yet to date globally, 6 withdrew, so we had results on 30, and we gave a total of 124 infusions. So the results from 30 patients, 16 with the most -- with intermediate type and 14 with the most severe type and 21 of our patients were under the age of 10. And we saw a total itch reduction at 3 months of 21% and at 6 months of 26%. And remember, itch is actually the top 2 priorities of treatments that these families want globally.
Next slide, please. And we also saw benefits across all patients receiving CORDStrom for itch. Under the age of 10, we saw an improvement not only in itch though, but with their skin scores. So the improvement of wound healing and their itch improved by 17%. So the younger ones had an itch improvement of 17% and skin 7%.
The intermediate patients that have more severe disease also saw a pain reduction. So the average pain improved by 22% and the worst pain by 28%. When I'm talking about the most severe patients in this cohort, 9 of the 30, they didn't see an improvement in their skin at 3 months, but they had this massive reduction in itch of 25% even larger than the intermediate group.
So if we look at the 6 months data, so remember, this is 6 months now after receiving treatment, the 2 infusions. Itch reduction is maintained even 6 months after the cells across all the cohorts. And fascinating, the largest itch reduction was in the most severe patients and their itch improved even further and was reduced by 27.5% at 6 months. And importantly, we also saw in this cohort now 6 months after the infusion that not only was their itch reduced further, but we started to see a big improvement in their iscorEB, the skin scores, the patient report outcomes. And this, we didn't see at 3 months. And the thought most likely is with this sustained improvement of itch, we started to see this really improvement in wound healing in these patients. Thank you.
So conclusions from the data is that cells #1 are safe across the whole cohort, it was phenomenal. We saw no serious adverse events. Itch reductions maintained 6 months post 2 infusions. So imagine if we give them more frequently. And the largest itch reduction was seen in the most severe cohort of these patients.
We also saw a big improvement in these scores, iscorEB scores in the most severe group, which we didn't detect at 3 months. And this improvement that we saw and the sustained wound closure is likely to be, in my opinion, disease-modifying, improving pain and quality of life and it should reduce their long-term risk of squamous cell carcinoma.
We published this in the eClinical Journal of Medicine to the end of last year. And I'd really like to end a couple of minutes just talking about the qualitative data. So these are interviews that parents and children had blinded after they had either the cells, 3 months after cells or 3 months after placebo. So the person who was interviewing the children and adults didn't know that they -- what treatment order they had and neither did the patient. And these are the results.
There was clear evidence that people, adults and children knew. 10 out of the 13 said they absolutely knew when they had CORDStrom as opposed to the placebo because they simply felt better. In fact, all the intermediate patients in children and one parent from most of the severe cohort said that they had a large impact on the quality of life of their child. And many families talked about the difficulty of symptoms coming back when they could no longer have the cells. And a quote from a parent said, when she's been getting ill, she's just been recovering in the same way as her brother.
Normally, illnesses affected much more because it causes inflammation. We've just been able to cope with everything so much better and have lots of energy, and I suppose life has just felt normal. And another family said, I think it's been lovely to see them having energy and having an appetite. I suppose actually, that's one thing we've noticed well, she's put on weight. And the child said, it was less painful. And when I itch, it's painful. So obviously, that's not comfortable, but I had less pain, my skin was less itchy, my hands were less hot. And when they're hot, I itch, and that has not been happening so much. And another child, normally it would hurt when have a bath, but lately, it hasn't been hurting. So these are quotes genuinely families expressing the quality of life improvements that they saw with this treatment.
So the next stage is our INmune Bio are going to support an open-label study here at Great Ormond Street Birmingham children MissionEB Deliver. And we're going to treat all children that took part in the study plus others that weren't able to with the diagnosis of this type of EB. The study will be over 12 months, and each child will be offered 2 infusions every 4 months for 12 months, and we will be collecting again a lot of data.
So I wanted to say that CORDStrom has proven to be beneficial with no safety signals in all children with recessive dystrophic EB from the age of 6 months. The intermediate and younger patients over the age of 10 saw effects more quickly with improvement in their pain and itch. But the most severe patients and older ones, although they didn't show skin changes at 3 months, by 6 months, their itch has continued to improve, and we also saw big improvements in their skin scores.
So treatment with CORDStrom in recessive dystrophic EB patients is likely, in my opinion, to be disease-modifying, improving itch and quality of life and it should reduce the risk of squamous cell carcinoma, the life-limiting complication of this disease. So thank you very much for listening.
Thank you, Anna that was an excellent master class in RDEB. And I know it was really painful to have a situation where patients had to be treated with placebo. But ultimately, it strengthens your data enormously to have children and parents able to identify blindly in which order they were treated is really quite remarkable in the absence of most of these studies are not done double blinded.
So I'm just going to give some details now about how the why CORDStrom works, how it works and how we're going to deliver it coming as we move forward. So those of you that have been familiar with mesenchymal stromal cells therapies and the difficulty in delivering them, it's largely been due to the heterogeneity, not just of where the source of the cells, whether they come from bone marrow or adipose tissue or in our case, umbilical cords, but also within the individual donors, there's donor-donor heterogeneity, which gets worse as we get older.
Also within a mesenchymal stromal cells product, there are multiple subsets of mesenchymal stromal cells. And so many, many, many trials, I'd say 99.9% of trials of MSCs have assumed that all MSCs are the same, and we knew that wasn't the case. So we invented CORDStrom back in 2014, not within the company. It was an academic invention that we bought into the company. And I chose to use umbilical cord because it was an easy tissue and they're much more homogeneous. But we also decided that we would pull donors. And so that heterogeneity, if you make a product from a single donor, when you come back and make it from another single donor, it's inevitably different. And by pooling donors, that heterogeneity of 4 donors is reflected in the next 4 donors. And so you end up with a much more homogeneous product, and that's proven to be the case. And although I won't share today, we have a lot of data we've shared with regulatory agencies to confirm that.
Most importantly, we can define our product on potency. I think that's the biggest difference between CORDStrom and all of the other MSC products. We actually have a mechanism of action, which I'll share with you, and we have potency assays to support that. So these are some of the numbers behind the -- or graphs behind the numbers that Anna just shared with you.
If we look at CORDStrom versus placebo at 3 months and 6 months and looking at improvement scores, I believe these are going up as the patients improve, we can see significant improvements at 3 months in red and 6 months in green, in patients treated with -- when they were treated with CORDStrom compared to the patients when they were treated with placebo.
If we look at the actual pain score, so a high number here is more severe pain, you can see that those on the placebo treatments remained stable in terms of pain, whereas those on CORDStrom on average, their pain score reduced over 12 weeks. And so you've heard that, that is the most important indication for these patients in terms of management of their disease. And we think it's a key differentiation factor in terms of their quality of life. But itch was the second most important pathology as far as the children were concerned. And you know that there is this scratch or itch scratch and then skin wounding cycle, which just gets worse and worse and worse. And you can see some of these quotes from a study done by Deborah in the U.S. I don't sleep. I can't wait for the sun to come up, so I can start being bandaged because of the damage I did while I was asleep, and it's just a different form of pain.
These children were assessed with an itch score called the itchman score, which has a 0 to 5. But the patients in our trial were mostly Level 2 and Level 3 and Level 2, sometimes this is described as sometimes interfering activity in children scored themselves. And Level 3, itch is a lot difficult to be still and concentrate. So this is where the quality of life starts to get really impacted. And if we look at these categorical scales and the percentage of patients in these groups, we can see that severity level 2, mild itch, sometimes interfering with activity, 21% of the patients were at severity level 2 before treatment. And a similar 21% of course, treated patients were at Level 3.
If we look after treatment, those patients, 67% of those patients at Level 3 fell to Level 2, and this is this increase here. So a 67% reduction in the children with Level 3, the severe form of age. Whereas in placebo, the severity level -- the patients in the severity level 3 actually increased after following placebo with no reduction in disease.
What that means in terms of other quality of life issues, we look at eating and you saw that quote -- "My daughter was able to eat." And you can see here improvements in eating at 3 months and sustained at 6 months on CORDStrom and not with the placebo. And bowel performance, we have a high placebo effect here at 3 months. But the story here is that CORDStrom showed effects at 3 months that were contained or continued at 6 months. And if we look at other well-being scores, general well-being, sleep and pathology, their ability to interact with their siblings and to go to school in all aspects CORDStrom at 3 months and at 6 months outperforms the placebo treatment.
If we look at skin scores and obviously, the picture here that everybody thinks of are those pictures that Anna showed you of topical wounds. And although those can be treated, there's still obviously a major issue for these children. So anything that can be done that can improve skin scores is important. And what you can see here is that looking at the number of patients describing when we've analyzed the data and seen how these scores go, we've got 18% of patients under placebo getting worse, a small number, no change and a small number improving.
When they were treated with CORDStrom, a much lower number of patients got worse, a small number of patients got worse and a significantly greater number of patients improved. And that translates -- these data are also seen using the iscorEB skin score, where you can see a moderate, if any improvement at 3 months on placebo a worsening. So this is increasing skin score is worsening disease, a worsening at 6 months. Whereas at 3 months, the patients treated with CORDStrom at 3 months, you can see improvement in that skin score, which was maintained and there was no worsening at 6 months. And as I showed before, that's what translates into these improvements in well-being.
One of the problems of stromal cell therapies has been the inability to understand how they deliver the effects that we see. And in fact, those of you have been familiar with the length of time it took for Remestemcel to be approved in the U.S. and Europe. And that was most entirely due to their inability to convince the regulatory agencies that they understood the mechanism of action of the drug.
We know that CORDStrom and other MSCs secrete a number of cytokines, and these are the cytokines that we have measured CORDStrom. We know they're secreted and we can hypothesize how CORDStrom might be having these effects. So itch and pain are driven by release of interleukin-31 and TSLP from Th2, Th17 cells in the wound site and elsewhere in the body.
And we know that pro-inflammatory M1 macrophages are present at high numbers in the topical wounds, and we imagine elsewhere in pro-inflammatory sites throughout the body. And we know that these M1 macrophages secrete IL-6 and drive this cell population, but they also secrete IL-31 in their own right. And so what we would want to do, we would hypothesize is that the PGE2 secreted by CORDStrom would downregulate this through downregulating TNF-alpha, gamma interferon and IL-6. And PGE2 should also induce these M1 macrophages through the release of IL-13 to turn into an M2 macrophage.
Similarly, the IDO secreted by CORDStrom should activate these M2 macrophages and induce them to migrate into the wounds. And these M2 cells will secrete these wound healing cytokines as well as immunosuppressive IL-10, which switches off the Th2, Th17 cells.
CORDStrom can also secrete wound healing and cytokines, TGF-beta VEGF and HGF, and we would expect these to go up in patients treated with CORDStrom. And interestingly, and Anna spoke about this earlier on, the increased problem with wound infections as these children age. And the IL-8 secreted by CORDStrom activates neutrophils, and we would expect that to have a positive benefit on infection.
So this was our hypothesis and we had to start trying to find our evidence to see if it was true. And first off, we look to see whether this PGE2 IDO access could induce this M1 to M2 transition. And that we could only do in vitro. And what we did was use a cell line, which can be differentiated from arresting an undifferentiated M0 cell. By treating it with cytokines, we can drive it to an M1 phenotype or we can drive it to an M2 phenotype. And looking at these variety of surface antigens here, you can see the intensity of expression of these is different on M1 cells than it is on M2 cells. And what we did was we took the M1 cells that we generated, we treated them with superlatives from CORDStrom. And you can see that within 3 days, these cells change from the M1 phenotype to the anti-inflammatory M2 phenotype with a reduction in CD8, the pro-inflammatory CD80, CD86 molecules.
So we demonstrated in vitro that this could happen. But plainly, we didn't have skin biopsies from the children to see if it would happen in vivo, but we could measure other aspects. And so what we did next was look to see whether these cytokines from M2 cells actually went up. And you can see, if we look at the changes after treatment with placebo compared to changes after treatment with CORDStrom, we can see that we increase this IL-13, which is indicative of M1 to M2 transition. We increased the amount of IL-10 secreted, which is suggesting that these M2 cells in the patients are being activated and are secreting more IL-10. And interestingly, also, we saw a small uptick in IL-8 suggesting that they may well be having a systemic effect in IL-8.
If we look at wound healing, and you saw that these children did show an improvement in skin integrity, we can see that TGF-beta, VEGF, PIGF, all of these cytokines involved in wound healing are increased after CORDStrom and most of them either reduced or are unchanged after placebo treatment. And what was really interesting from our perspective was the change in TNF-alpha, this driver of inflammation. And as a company, we're interested in TNF-alpha for other reasons. But we now know that we know from publications that TNF-alpha drives this IL-6 response and drives the inflammation. And we can see a significant reduction in TNF-alpha. These are all serum samples from patients pre and post treatment either with CORDStrom or with placebo.
So really uniquely, I think, for a mesenchymal stromal cell product, we already know its mechanism of action, and we have evidence from the patient population that this mechanism of action is actually happening. It now means, of course, that we have ways of testing batches of CORDStrom so that we are able to have these as our release assays for the regulatory agencies, which tie in directly with our mechanism of action. They need to make this into a commercial product to be able to treat patients, we need to be able to make the product at scale. And we work with, as I said, 4 umbilical cords. We select cords with specific characteristics based upon that mechanism of action to have the right cords for the right product. And indeed, we can select different cords for different products, and that's a different story. But we take these into bioreactors. We produce master cell banks from the optimized cords and those master cell banks then are stored down for expansion into working cell banks and these working cell banks give us the opportunity either to scale out with multiple 3-liter bioreactors or as we're doing at the moment, scaling up into 15 liters. Either scale up or scale out, we can end up in a single run manufacturing around about 200 doses, which is enough to treat 30 patients for an annual run. So we already have the capacity to deliver these cells at the scale needed for commercialization.
So this is our path to market, which we're planning. So as many of you know, we started this back in December '24. So we're now at 14 months into the process, where we got approval from the FDA for RDD and we got ODD approval a month later. We've tech transferred into a commercial manufacturing space in the U.K., which we rent from a U.K. government facility called the Catapult. This is a very efficient and cost-efficient and staff-efficient way of doing this. And we already have the engineering runs underway and plan to have that license in place by the end of Q1 this year.
We have -- we've submitted the pediatric investigation plan and the response from the MHRA will be on the 9th of March, so promised of that. And we have a face-to-face pre-MAA meeting with the MHRA already scheduled for the second week of May, which aligns almost very nicely with the pre-IND meeting for the open-label trial that Anna described with the FDA, so we can run that in the U.K. regulated by both regulators.
That will lead to the breakthrough designation submission at the end of Q2 this year and then either at the end of Q2 or very early in Q3, probably in July, we will submit to marketing authorization to the MHRA, and they are fully aware of that time line from this premeeting. And then by the end of the year, when we've done a few more experiments that the FDA have asked us to do, we will be submitting our BLA to the FDA.
So this is where we are at the moment in terms of the alternatives as you've heard, Vyjuvek and Zevaskyn are already out there and placebos as well. Vyjuvek is a topical skin cream from Krystal Biotech. They have a $400 million turnover this year alone. And Anna identified some of the costs associated with that.
The topical skin graft, I don't even know how many patients have been treated. None of these addressed the systemic nature of the disease, as Anna pointed out. And so I believe that we have a great market opportunity to get into this disease and to provide the treatment that these children so desperately need. But as I alluded to earlier on, we have a little bit of way to go. But -- we believe this really is the first treatment that's beyond topical care. And these IV infusions have already had multiple systemic effects in the children that you've seen in terms of EBDASI, IScore, itch, pain and quality of life. And as Anna said, it's been a remarkable safety profile even in these children with very severe disease, having an intravenous infusion is not an easy thing to deliver. And yet -- it was completely safely delivered and has been formulated for that.
So commercial readiness, as you've seen, we're ready to scale up and scale out. We've organized and produced clinician-friendly packaging and a supply chain that fits in with hospital activities. The CMC is now locked down and our potency assays that you've seen are simple and are validated. And we've been engaging with regulators throughout and that engagement is underway and is currently on time for that schedule that you saw.
So we are now moving from being a clinical development company to a commercial provider, which I'm extremely excited about, and we really look forward to being able to provide something for these children globally as soon as possible.
So this, I think, is this goes as a quote from Anna from one of her parents, one of the parents in the trial. We look at these changes and you go, well, it was 27% or it was 9% or it was 7%. That's not 90% or 100% improvements. What these children actually ask for is a 1% difference, a 1% difference and the parents are happy to come into London with their child to be treated in a clinical trial. It's a lot for him, 1% less pain is 1% more real life. And I think that's a tremendous quote and should be the company logo.
So with that, I'll hand this back to David, and very happy to take any questions, and I'm sure Anna will join in. Thank you very much indeed.
Thank you, Mark and Anna. It was great. And if anyone has any questions, just type them in the chat. We have a few already. The first is, do you believe that clear evidence of systemic wound improvement, i.e., measured by imaging, et cetera, would be required for approval?
We've not had that feedback from the MHRA and indeed, it wasn't a comment that the FDA made in the Type B meeting that we had last year. So I'm -- these skin scores, and Anna is better positioned to answer this than I am, but these skin scores are internationally recognized. So -- and they -- one of the things that we measured in the upcoming trial is the total body area that's affected, which is a numerical value rather than imaging.
And I can answer it's extremely difficult to take photographs because it's very, very painful. And so you can't do total body photographs. You have to do it sort of limb by limb and body-by-body area. So -- and actually, it's been shown to be very unreliable because these wounds open and close.
So the pictures that we use from MissionEB with a company called Canfield, in fact, were not helpful. What was helpful were the quotes and the scoring systems that are validated, as Mark said. So I wouldn't imagine that they would need that.
Yes. Let me just add, Deborah did a panel with RDEB patients in 2018 that's recorded on YouTube with the FDA. And similar to what you mentioned, Anna, that the patients rank itch and pain far above their wounds -- anyhow. And I think part of the history is, and maybe, Anna, you could add to this is that a long time ago, they looked at RDEB as a topical disease because it presents itself with the external wounds, but it clearly is a systemic disease, right, with no systemic treatments on the market.
All right. Question two, can you elaborate on the few more experiments that the FDA has required you to do? This is the CMC mark.
Yes, that's easy. So when we went to the FDA, one of the things that I was interested, currently, all of the batches of CORDStrom we've made have been made using umbilical cord donors from the U.K. cord blood banks. So these are cords from which the cord blood has been removed and the discarded tissues come to us for isolation of mesenchymal stromal cells.
Now I was interested to see whether the FDA would allow us to use U.K. donor cords to treat U.S. patients. And the FDA came back and said, yes, but the cords will have to be -- or the cord donors will have to be tested for infectious diseases, disease markers in U.S. accredited laboratories. They wouldn't use European tests. So that's not uncommon. It's the same for umbilical cord blood. And this means that we have to go and get new cords, which we're doing all the time anyway, but we'll have to create a new master cell bank and that new master cell bank will have to be from donors who've been tested both in the U.K. and in the U.S. And so those are awaited. We're just sorting out the donors.
It's simple things like these ladies have been -- have given consent to have their blood tested in the U.K. for infectious diseases. They haven't given consent for that to be done in the U.S. and for their data to go to the U.S. So we had to change consent forms. We had to work with the cord blood bank to make that happen. And that's what we're waiting for.
So I was doing the audit yesterday at the cord blood bank. We are ready now to move forward with that. And once we get the new cords in and we selected those that are optimal for CORDStrom and EB and manufacture the master cell bank, then we'll have the data that we can take to the FDA. But it's just that slow process.
There are just additional CMC steps that the FDA has that are different from what the other regulatory agencies have.
They're just -- yes, they're screening.
Exactly. Next question. Might CORDStrom have any applicability to other types of EB?
That I leave to Anna as the expert in EB.
Well, yes, a brilliant question. I don't know who asked that. So yes, definitely. So junctional EB, which again is systemic, very inflammatory, we would really -- in fact, I'd like -- we have discussed such compassionate grounds whether we can start treating the junctional children. And so that's another cohort. It's not such a large -- it's a much smaller group. But yes, there are other types, which will be junctional type.
Great. Thank you, Anna. Next question. Can you talk about the increase in itch on placebo? It's not an increase in itch. It's just that they stayed on Level 2. Is that progression of the disease or just fluctuation without treatment to help? I'll let you answer that, Mark.
I think these are small numbers. I think it's fluctuation without treatment.
Yes. And second question, when thinking about labeling, we have seen cell therapies approved in patients as young as 2 months of age. Would you be seeking a similar age range? And does this position you to target pretty much the entire spectrum of RDEB?
So I can answer that from a regulatory perspective and Anna can answer it from a clinical perspective. So the MissionEB trial was performed in children over the age of 6 months. And there were good reasons for that because -- not least because of the placebo treatment.
Now when one submits a pediatric investigation plan in the U.S. or the U.K., you have to say what ages you're going to treat, and it's assumed that you would treat from birth. We didn't have any data to support treating from birth. And so we have submitted our PIP on the basis from 6 months. But Anna, we discussed this only on Monday about how we might deal with this in the open label. And there are pros and cons for going to be below 6 months, but Anna can talk about the technical difficulties. I have no problem in supplying this drug for children less than 6 months, but there are technical difficulties, which Anna will give details of.
Yes. Great. Thanks. So I mean, I want to treat it as soon as possible from birth. But we said 4 weeks because anyway, it takes a couple of weeks, sometimes that long to get the diagnosis anyway. So we need to confirm with the skin biopsy and genetics. And -- but as early as possible, you saw the first phase where the CORDStrom plays in the absent skin. Now that area is the risk for squamous cell carcinoma. So the quicker we can heal that area, the better. So the sooner we can give treatment, the better is the answer.
Thank you. Have you noticed any increased interest or acceptance for cell therapies for rare pediatric diseases, now that Mesoblast seems to be gaining traction for its therapy at gene therapy price levels?
I think the answer there is certainly speaking to regulators, there's enthusiasm. In terms of funding, that's not an area that I can really speak to apart from the fact we know that some very expensive therapies are already approved and are being used in the U.S. And for rare pediatric diseases, only today, I was talking to NHS England, and they were proposing that we go and speak to the National Institute for Clinical Effect, NICE, and clinical evaluation rather as early as possible. So we will open those negotiations.
Then with the topical agents of Vyjuvek, Filsuvez, do you see CORDStrom as competitive or something that would be used in combination?
No. So these are really good questions. You should come. So it's combination therapy. But anti-inflammatory therapy is the most critical of all the therapies. I mean if you -- there will be other therapies that will be needed. Filsuvez, it does not work. I mean, so not that particular one. B-VEC, you have to apply weekly. There should be -- there will be better gene therapies that can last longer, hopefully, permanently. But there will also always be a need for systemic anti-inflammatory therapy for the older patients where you can't genetically correct.
So the treatment is going to be individualized and combination therapies for EB, but with a key role of anti-inflammatory, in my opinion.
We also have to remember that the 2 licensed products, 3 licensed products, all 3 of them have itch as a declared side effect. So a solution to the itch problem is something that we would like to believe we can provide even for patients who are on alternative therapies.
Do you have a timeline in mind for including junctional EB patients?
Well, I can answer that because it's -- I'll have to make the cells. So in the -- so globally, patients either get treated with clinical trial products and investigational medicine or they get treated with a licensed medicine or they get treated compassionately. And in the U.K., drugs are actually manufactured under a different license for compassionate use, and we are waiting for that license to be awarded to the manufacturing facility where we're making CORDStrom.
As soon as that happens, then yes, if Anna has patients with junctional disease, she can prescribe it and we can supply it. So yes, that's high up on our priority list.
What do you think is the optimal dosing regimen duration of CORDStrom? And the -- and I'm going to add to that, we saw sustained durability over -- from 3 months to 6 months on the single course of therapy. What do you think the multiple course of therapy over a year would look like?
So Anna can talk better to this than I can, but at least I will say that her design of giving the drug once every 4 months or 2 doses, 2 weeks apart and then repeating it every 4 months, I think, is extremely sensible given the data that she's already derived from MissionEB. And there's been one small study in South Korea where a similar dose of umbilical cord MSCs, albeit from a single donor rather than a pool product did have considerable effect on statistically significant improvement in skin performance after 3 doses. So this was 3 months apart.
So I think there's good evidence that the design that Anna has come up with will be the one that's successful and we go forward with. And then it will just be constant treatment throughout many, many years. But Anna, what's your thought?
Yes. So I think the -- first of all, the logistics of doing it more frequently is not sustainable because it's really difficult to put cannulas in drips in these children. So we went for the balance between wanting to really have a sustained improvement globally in their health, but not too many interventions. So if we made it 6 monthly, I didn't think that we would really get on top of the systemic inflammation that much. But if we did it every 3 months, there's too many cannulas.
So I imagine that the younger children, if we start early, we could space out the interval between the infusion somewhat, I would imagine, over time. But the older children over the age of 10, I suspect that they will need it roughly 4 monthly. But I think the beauty is that it's safe -- and so we can adapt it over time if we need to. But that's really the reason why we chose the 4 monthly infusions.
I think we also have an opportunity for some data which we didn't show. None of these patients, even though they had multiple infusions, made any immune response to the product. And so this -- although this is a completely allogeneic product, we didn't see any T cell immunity or antibody immunity to these products. So I'm confident that we can give multiple infusions.
So we have a few more minutes left unless I get more questions. But the last one I have is you mentioned that CORDStrom is likely disease-modifying therapy. Can you elaborate on the long-term potential to reduce the risk of squamous cell carcinoma, which currently has a median survival of only 2.5 years for this EB subtype.
Yes, sure. So we know that we've published 25 years of data on cancer in these patients and where the cancers are, and they are predominantly in the extremities in areas where they have absent skin. And so we know that repeated trauma and open wounds and then an itch, which keeps opening the wounds if this puts the risk -- these areas at risk over time, increasing risk. So the longer the skin is open and the longer wounds are chronic, the higher the risk that they will turn into squamous cell cancers.
So if we can cure early the dysplasia, so they don't have chronic wounds there and make sure that the wounds, although they will be there, they won't be chronic, it will for certain reduce because they know the biology of these tumors -- so -- but obviously, we'll know that over time. But there is now very good evidence of the drive is trauma and chronic wound and inflammation in these cancers.
Then the very last question before I give the concluding remarks is can children with severe RDEB who are not based in the U.K. participate in the upcoming study?
So the current plan, and this is due to funding issues more than anything else is that the trial will run solely in the U.K. and will be registered with the FDA as well. So the data from the U.K. trial will be usable in the submission for the BLA. But no, at the moment, we have no plans to open clinical trial sites in the U.S.
Thank you, Mark. And I do want to note, we put these children and parents first. We're doing all we can to accelerate this product to get it to market. We really believe in what we're doing here, and we put the patient and their families first. So as we conclude today's session, the data from the MissionEB trial reinforce our belief that early intervention with CORDStrom can fundamentally change the disease trajectory for these children living with RDEB.
We've seen encouraging results, including a 20% reduction -- 26% reduction in itch at 6 months and significant beneficial effects in patients under the age of 10. We're now moving aggressively toward making CORDStrom the first systemic disease-modifying treatment available for the RDEB community. Our regulatory road map is clear. We've already submitted our pre-submission package to the U.K. MHRA, and we intend to file a full marketing authorization application by mid-summer of this year.
Following this, we're targeting regulatory submissions in Europe at the EMA and the U.S. FDA in the fourth quarter of this year. Furthermore, as Mark highlighted earlier, the truly exciting aspects of our platform is the tunability nature of CORDStrom. This flexibility allows us to optimize the therapy's anti-inflammatory and regenerative medicine properties, specifically for unique challenges of this disease.
We hope to leverage this capability to develop more advanced versions of CORDStrom RDEB, which we believe will allow us to expand the use across broader EB spectrums in the future. By targeting this window of opportunity in early childhood before the onset of life-threatening complications like squamous cell carcinoma, we aim to provide these families with more than just wound care.
We aim to provide a better quality of life. I want to thank Dr. Lowdell for his groundbreaking invention and Dr. Martinez, the entire clinical team for their dedication to these patients. We remain relentlessly committed to delivering transformative solutions for these patients suffering from the devastating effects of RDEB. Our mission is to move beyond temporary relief and provide a systemic disease-modifying therapy that fundamentally improves the lives of these children and their families. Thank you for your continued support of INmune Bio.
Inmune Bio, Inc. — Q3 2025 Earnings Call
1. Management Discussion
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2. Question Answer
" Raymond James Ltd., Research Division
" Maxim Group LLC, Research Division
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Greetings, and welcome to the INmune Bio Third Quarter 2025 Earnings Call. [Operator Instructions] As a reminder, this conference is being recorded. A transcript will follow within 24 hours of this conference call. At this time, it is my pleasure to introduce Mr. Daniel Carlson, Head of Investor Relations of INmune Bio. Daniel?
Thank you, operator, and good afternoon, everyone. We thank you for joining us for the call for INmune Bio's Third Quarter 2025 Financial Results. Presenting on today's call are David Moss, CEO and Co-Founder of INmune Bio; Dr. Mark Lowdell, Chief Scientific Officer and Co-Founder of INmune Bio; Dr. CJ Barnum, Head of Neuroscience; and Cory Ellspermann, INmune Bio's CFO.
Before we begin, I remind everyone that except for statements of historical fact, the statements made by management and responses to questions on this conference call are forward-looking statements under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements involve risks and uncertainties that can cause actual results to differ materially from those such as forward-looking statements.
Please see the forward-looking statements disclaimer on the company's earnings press release as well as risk factors in the company's SEC filings, including our most recent quarterly filings with the SEC. There is no assurance of any specific outcome. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made as the facts and circumstances underlying these forward-looking statements may change.
Except as required by law, INmune Bio disclaims any obligation to update these forward-looking statements to reflect future information, events or circumstances. It's now my pleasure to turn the call over to INmune Bio's CEO, David Moss. David?
Thank you, Dan, and good afternoon, everyone. For our third quarter 2025 call, today, I will review key takeaways and provide an update on our platform programs. Following my review of the recent developments at INmune Bio, I will pass the microphone to Dr. Lowdell, INmune Bio's Chief Scientific Officer and inventor of CORDStrom, who will provide an update on our CORDStrom MSC platform and particularly our RDEB program, along with INKmune.
Next, Dr. CJ Barnum, who leads our CNS drug development efforts, will provide an update on the Alzheimer's program; and then Cory Ellspermann, our CFO, will present our financial results, after which I'll conclude our prepared remarks with a review of our upcoming catalysts, and then we'll be happy to take your questions.
Since taking over the role of CEO at INmune Bio in July, it's been a time of transition for the company. Having spent the last 2 years with our main focus on Alzheimer's trial, which ended in late June, we're now able to direct our attention to the next stage of development for our platform drug programs.
And we're highly optimistic that the next couple of years will demonstrate the success of our efforts as we take our programs through key development milestones, which we expect could lead to benefits not only for investors, but for patients suffering from diseases with limited therapeutic options available at this time.
CORDStrom is our most advanced program as we are now in the process of preparing for submission for marketing approval to the regulatory bodies in both the U.K. and the U.S.
We believe CORDStrom has demonstrated a clear and safe benefit to patients suffering from recessive dystrophic epidermolysis bullosa, also referred to as RDEB, a very debilitating disease. Patients on the drug had a significantly reduced itch, which not only affects wound healing in these young sufferers, but also reduces the itch-scratch wound cycle. While RDEB primarily manifests as a genetic condition causing skin fragility, blistering and scarring due to mutations of the collagen VII gene, it also involves mucus membrane and internal organs leading to multisystem complications. While RDEB has traditionally been treated topically, RDEB is a systemic disease that has blisters and scarring in the mouth, esophagus, eyes, urethra and anal area causing nutritional deficiencies, emotional stress and other problems.
We believe CORDStrom is potentially one of the first systemic treatments for RDEB and made an improvement in the quality of life of the patients treated in our trial.
Looking beyond our initial indications of RDEB, we believe CORDStrom is a platform opportunity for INmune Bio as it has the potential to accomplish all sorts of things in many different diseases.
For example, we can genetically modify it to treat cancer cells, which is what it was originally developed for, and there are many other modifications you will hear about in the near future. So there's a lot of things that we can do with CORDStrom to improve its targeting in a variety of rare diseases and indeed, less rare or more common diseases.
We'll be excited to share these modifications in the future as well as expansions towards other indications.
Turning to the XPro platform. We remain confident in its potential to treat neuroinflammation in Alzheimer's disease. In September, we submitted a manuscript detailing the results of the Phase II MINDFuL trial for peer-review publication. As we analyze the complete data set, we're gaining deeper insight into the drug's activity. Dr. Barnum will -- will elaborate, but our findings indicate positive results in patients with higher baseline inflammation.
We're actively pursuing an accelerated regulatory pathway and preparing for our end of Phase II meeting with the FDA.
Defining a clear path forward for XPro is a primary objective and may be crucial for strategic funding and partnership discussions.
2025 also saw us complete the Phase II trial of INKmune in prostate cancer ahead of schedule. With the primary endpoint and 2 of the 3 secondary endpoints met, I'm excited to have Mark share more on INKmune later.
To conclude my remarks before I turn the call over to the team to discuss the individual programs, I'd like to thank the patients that participated in our trials, the clinical trial sites and our dedicated team for helping us execute these very complex trials. I also want to thank our investors for their continued belief in our novel platforms and support.
Our decision to develop 3 very different drug platforms in parallel to provide strength and opportunity has borne fruit. INmune now has 2 later-stage platform therapeutics that have demonstrated success in clinical trials and are ready to advance to the next stage of development and a third which completed a Phase II trial successfully.
Our value proposition to shareholders and patients is clear. First is to get CORDStrom from to MAA in the U.K., followed by a BLA in the U.S. Meanwhile, for XPro, we await regulatory alignment with the end of Phase II study to determine next steps. We anticipate all of this will happen in '26, an exciting year for the company.
Now I'll turn the call over to Mark Lowdell for more color on CORDStrom and INKmune. Mark?
Good afternoon, everybody, and thank you, David, for the introduction. As you've heard, we're progressing towards drug registration, firstly in the U.K. and then the U.S. with CORDStrom in RDEB as our initial indication, whilst developing other indications for the platform at the same time. This is a truly debilitating disease, which presents itself in the first months of life and for which there is no cure currently. The median survival for those with severe disease is fewer than 30 years. And although skin wounds are the most apparent manifestations of the disease, the lesions, as David has told you, are present throughout the gastrointestinal tract inside the nose and behind the eyes. The disease is driven by inflammation and CORDStrom provides systemic suppression of inflammation.
Most importantly, CORDStrom is most effective when activated by the inflammatory cytokines at the sites, so its effect is somewhat targeted. During the MissionEB randomized placebo-controlled trial in the U.K., over 120 infusions of CORDStrom were administered to over 30 children without any severe adverse reactions or adverse events.
As David has said, reduction in systemic itch was a major reported benefit by patients some as young as 2 years old who used a cartoon depiction of whole body itch to demonstrate their experience severity. Itch control is important because the resulting scratch initiates new skin wounds and increases risk of infection, which is part of the disease cycle. And I can't emphasize how much important -- how very important itch is to these children as it drives a vicious itch scratch wound cycle but impairs wound healing by separating skin layers and forming new blisters. These rupture easily creating open wounds or exacerbating existing wounds, delaying healing and creating this terrible feedback loop. It's painful for these children with intense itch causing a poor quality of life with distress, sleep loss and emotional burden.
Breaking this itch-scratch wound habit is difficult and highlights one of the special aspects of CORDStrom.
MissionEB was an investigator-led trial. It wasn't sponsored or funded by INmune. We secured access to the entire trial data pack in August and have appointed an independent group of clinical statisticians to analyze all of the data in depth. This is critical for our submission to regulatory agencies and is well underway.
In the trial, all patients received both CORDStrom and placebo, separated by 6 months, some treated first with placebo and then CORDStrom and the other half treated with CORDStrom first and then placebo. These in-depth analysis of these data show improvements in disease activity scores in all patient subgroups after CORDStrom compared to their previous placebo treatment.
In preparation for registration filing, INmune in the U.K. has now relocated into rented CGMP manufacturing space, which is compliant with commercial production as a licensed medicine.
We successfully completed the technology transfer earlier this month, and we're on track to be ready for U.K. filing at the end of Q2 next year.
But alongside the CGMP work, we're confirming the complex mechanisms of action of CORDStrom in RDEB and validating assays to test drug batches at the end of manufacture. This work is very critical since FDA and other agencies require robust tests of drug potency and failures to get these right with cellular drugs have delayed other drug approvals for many years.
This year also saw the completion of the Phase II aspect of our trial of INKmune in patients with castration-resistant prostate cancer. As David said, we met the primary endpoint of the trial in Q1 of this year, and analysis of the first 9 patients showed evidence of NK cell proliferation in vivo and generation of the functional memory-like NK cells that we understand INKmune generates in 4 of the 6 patients treated at the lowest and intermediate dose levels.
The data from the patients in the highest dose cohort are awaiting analysis, but the team is tied up with CORDStrom at the moment. Thus 2 of the secondary endpoints were met. The final secondary endpoint was reduction in tumor load, and our primary measure was PSMA PET scans. But it was obvious from the analysis of the first 6 subjects that the patients being enrolled had very high disease burden beyond that, which would respond to immunotherapy. We thus decided that since we've met the primary and 2 of the secondary endpoints at both low and intermediate doses, we had identified the dose to take forward to a randomized Phase II trial and so we could close the current trial to recruitment.
We're analyzing the blood samples and the PET scans from the final 3 patients at present, and we'll report them to you as soon as they become available. Now we plan to work on the design of the randomized trial during 2026 as resources become available.
So 2025 has been incredibly busy for the U.K. team in supporting both INKmune and CORDStrom, but all the staff remain fully dedicated to delivering the goals we've set, and we look forward to providing more good news as the data become available.
Now I'll hand over to CJ to report on the company's progress with XPro and look forward to any questions later in the call. CJ?
Thank you, Mark, and good afternoon, everyone. We have established 4 strategic priorities for XPro. First, to secure regulatory alignment with the FDA at our forthcoming end of Phase II meeting; second, to pursue an accelerated approval pathway. third, to publish comprehensive insights from our Phase II MINDFuL trial; and fourth, to advance discussions with potential partners to support late-stage clinical development.
During the third quarter, we achieved an important milestone by submitting the Phase II MINDFuL trial results for peer-reviewed publication. A preprint manuscript is now available on MedRx, providing the scientific community and our stakeholders with expanded insights. While much of the data has been previously presented, this manuscript introduces new analyses from the dose-compliant patient set. These are patients who received at least 21 of the 23 scheduled doses. This population reflects the impact of sustained therapy and has been recognized by the FDA as a potential indicator of disease modifications.
The findings demonstrate that longer treatment durations with XPro are associated with greater improvements in neuropsychiatric symptoms and biomarkers, including pTau217 and GFAP.
We continue to build a robust evidence base to advanced analysis of neuroimaging endpoints. These focus on white matter integrity, an indicator of myelin preservation and gray matter metrics related to neurodegeneration. Emerging results are expected to further substantiate XPro's potential as a differentiated disease-modifying therapy, and we plan to share these findings as they become available.
There remains substantial unmet need beyond existing anti-amyloid treatments, particularly for patients with a strong inflammatory profile or those unable to receive anti-amyloid therapies due to safety concerns such as ARIA.
XPro is uniquely positioned to address this gap as no ARIA-related safety signals were observed even among high-risk individuals.
Despite the challenges inherent in Alzheimer's drug development, XPro continues to distinguish itself through its targeted patient selection, compelling safety profile and growing body of evidence. Our disciplined data-driven approach, which prioritizes scientific rigor, regulatory engagement and financial responsibility supports the long-term goal of establishing XPro as a transformative therapy and delivering sustained value to patients, partners and shareholders.
We look forward to providing ongoing updates as we advance toward key clinical and regulatory milestones. I will now turn it over to Cory for the financial update.
Thank you, CJ. At this time, I'll provide a brief overview of our financial results. Net loss attributable to common stockholders for the quarter ended September 30, 2025, was approximately $6.5 million compared with approximately $12.1 million for the comparable period in 2024.
Research and development expenses totaled approximately $4.9 million for the quarter ended September 30, 2025, compared with approximately $10.1 million for the comparable period in 2024.
General and administrative expenses were approximately $2.5 million for the quarter ended September 30, 2025, compared with approximately $2.2 million for the comparable period in 2024.
At September 30, 2025, the company had cash and cash equivalents of approximately $27.7 million. And based on our current operating plan, we believe our cash is sufficient to fund our operations into Q4 2026.
As of October 30, 2025, the company had approximately 26.6 million shares of common stock outstanding. Now I'll pass it back to David.
Thank you, Corey. Now I'd like to present upcoming milestones for the company, and then we can start the Q&A session.
For our CORDStrom program, we have a number of significant events in front of us. In Q4, we'll present additional data from the trial. In mid-'26, we expect to file a marketing authorization application in the U.K. A few months after filing the marketing authorization application, we expect to file a BLA, biologics licensing application with the FDA.
We'd expect to hear back from the FDA sometime by the middle of '27 or later.
For XPro, we expect to accomplish a lot in the next few quarters. In Q4, we anticipate getting more MRI data conducted during the MINDFuL trial. With this data, we hope to show improvements in myelin, gray matter and white matter, which would support the cognitive and biomarker findings of XPro's benefits that CJ spoke about earlier.
We expect to hear from the FDA on accelerated pathway sometime in Q1 of '26, and we anticipate having the minutes from the end of the Phase II meeting sometime in Q1 of '26. So a lot happening in '26.
At this point, I'd like to hand the call back to the operator to poll for questions.
[Operator Instructions] We'll take our first question from Gary Nachman with Raymond James.
This is Denis Reznik on for Gary Nachman. So just a couple from us. So on XPro, as you're preparing for this end of Phase II meeting with the FDA, can you just walk us through what some of the biggest questions or discussion topics that you're hoping to get more clarity on? And then I believe you were previously saying that the meeting could occur before the year-end, and now you're guiding to the meeting occurring in 1Q. So could you just speak as to why the slight delay occurred? And I've got one follow-up.
Yes. No, I appreciate it, Denis. Let me start with the second part of your question, and then I'll let CJ jump to the first part. We anticipated getting everything together, but we weren't able to get enough of the data in time to really get it to the FDA to have the meeting at the end of Q4. I will say, though, that we're very close. It still could happen. We're going by the end of the date that the FDA puts forward. I don't know what that's going to look like.
And then keep in mind that you don't get the minutes for the meeting until approximately 30 days after. Our experience with the FDA in the past has been that we usually get it on day 30 or very close to day 30. So we're being relatively conservative saying in Q1, which is the -- not only the period of time you have the response from the FDA, but the 30 minutes -- the 30 days to get the written minutes. CJ, I'll let you respond to the type of questions. I'm not sure we're going to publicly disclose them, but I'll tell you that they relate around setting up a registration study. CJ?
Yes. I mean I think there's a few obvious ones out there. One of them is that we talked about quite a bit is EMACC, right? We want to understand the agency's view on EMACC. Another one of the key questions is the enrichment biomarkers. It's clear from our data that the patients that had greater inflammation were the ones that are more likely to respond. And this is somewhat novel in this space, enriching for these biomarkers. So these are the sorts of questions that we need to ask.
And as David said, because our goal is to really get alignment on how we move this into a registration trial, another key question is the safety database, right? The agency usually requires a certain number of patients to be treated prior to -- before the drug could be commercialized. So I think those are sort of the obvious ones. There's some obvious or some other nuanced ones as well, but those are sort of the big questions that I think that we're -- that we can discuss at this point.
That's super helpful. And then sticking with XPro on the partnership conversations, can you provide some color as to how those are progressing or at least maybe compare the tone of the conversations as to where they were at the end of last quarter? And then separately, on INKmune, can you just talk a little bit more about how you view the future of that asset? Is this something that you're going to take through development yourself? Or would you consider partnering with?
Appreciate it, Denis. So I'll let Mark jump in on INKmune. Let me just jump in a little bit further. I think that before we really have aggressive partnering discussions, there's been very top-level discussions with a handful of groups. I think like our investors, they want to see what the regulatory feedback and alignment looks like. And on top of that, they want to see more of the dataset. I think if we're able to provide imaging data, white and gray matter that aligns with what we saw in our highly inflamed patient group, I mean, that's going to be, I think, very compelling.
So we're still gathering all the package together. We want to deliver a complete package where we have really serious discussions.
Mark, do you want to comment about INKmune?
Yes. Thanks for the question. So as you know, we've published data on INKmune potentiating NK responses to a number of different tumors, both hematological and solid. So there are plenty of opportunities to take INKmune into Phase II trials now that we've completed one Phase II trial in other disorders and indeed going now into prostate and looking at patients with better risk disease. I'm always very keen to talk to potential partners and others that would want to invest in or buy the asset. But I think from our perspective at the moment is to get more Phase II data in randomized trials in at least prostate and then maybe other diseases as funds become available.
And then, yes, indeed, look for partnership opportunities in the first instance and potentially keeping it within the company depending upon funding and taking it through to commercial in the way that we're hoping to do with or expecting to do with CORDStrom.
Next question please.
We will move next with Jason McCarthy with Maxim Group.
I'm going to concentrate them on the CORDStrom activity. First, has there been any feedback from European regulators with any specifics you could provide us for potential MAA filing? And do you think if you could file the MAA, that will be further supportive with regulators here in the States?
So another good question. So we are waiting for our scientific meeting with the MHRA. I sit on the British Pharmacopoeia, which is part of the MHRA. So I do get unofficial conversations with colleagues at the agency. And they have been very supportive of us taking this through for scientific advice before the end of the year. So we're putting that package together, and we will submit it as soon as we've got the data from the enhanced statistical analyses that are being done at the moment.
So I'm expecting early next month to submit the data to the agency for a scientific advice meeting, which we may well get before the end of the year, but it's probably going to be early next year and then move ahead with our program.
We have contracted a specialist advisory group that work or advisory company called TMC Pharma that works with rare diseases and has taken a lot of these through the agency. And so they're giving us a lot of daily feedback really. They're doing all of our paperwork for that filing to the MHRA and then for the MAA. So as David said, we are still on track to deliver the MAA submission to the MHRA by the end -- by Q2 next year or in Q2 next year.
And then the regulatory agencies talk to each other a lot, as I'm sure you know, and none likes to gain say another. So I'm confident that if we address the subtle differences for U.S. use compared to European use and get those data ready in the months after our MAA filing, we'll be in a good position to file a BLA by the end of the year, by which time the MHRA will have had their first opportunity, they have their 80-day line to come back to us with comments, and we can -- any improvements that they come up with, we can put into the FDA document.
So I think if we have got an MAA being considered by the U.K. agency that will feed into the FDA's opinion of the same data. But obviously, these are agencies, so we can't really comment on what they -- on their activities and the way in which they review other people's data.
It's part of that discussion -- thank you, Mark. It's part of that discussion to include at least conversation or anecdotal thoughts around limitations of gene therapy, given that they are topical, there's now 2 that are on the market, as you know, and potential use in the setting of some of these gene therapies because you'd imagine many of these kids going forward are going to probably try this.
Yes, absolutely. But the -- neither of those provide a systemic solution. So this is what we believe makes CORDStrom unique. But also the side effects associated with those therapies, one of the major side effects that was reported is itch. So even if it was an additional therapy to address the itch that isn't addressed by these topical therapies, there's a rationale there for CORDStrom's use in patients who are potentially being treated with the gene therapies. But as I say, the data we're getting at the moment, which once we've got them audited and good enough to share or secure enough to share, are already demonstrating changes in systemic cytokines associated with CORDStrom treatment.
So we believe that the strength of this drug will be its ability to have a systemic effect on inflammatory cytokines and therefore, downstream as the patients get treated for longer. in terms of the overall disease pathway.
And just one mechanistic question, sort of a 2-part question. First, you had mentioned, can you talk a little bit about the uniqueness of cytokine-based activation for CORDStrom once it is given systemically, at least in a setting of inflammation like in RDEB and also the ability to use the CORDStrom platform and tailor it to specific disease types. Obviously, that would be beyond RDEB and how that separates itself from other MSC therapies that are out there.
Absolutely. I could talk for hours on that. But the first question was about inflammation. And we know that for some functions of some MSCs, they require what's called licensing. So they need to be activated by inflammatory cytokines. And indeed, we have evidence in vitro that cause there's a ton of things that CORDStrom cells do without being activated by inflammatory cytokines. And then there are additional cytokines and functions that they perform in the presence of inflammatory cytokines.
Now as I'm sure you know, cytokines don't work -- don't really work systemically in vivo. They are signaling molecules between cells, and they normally operate over sort of nano distances. But -- so what we know is that these patients have inflammatory cytokines being generated at the site of itch actually and then the wound. And the itch response is driven by a particular T cell called Th2 releasing a cytokine called IL-31.
And we know that the CORDStrom suppresses those Th2 cells and suppresses the myeloid cells that are also producing inflammatory cytokines, and they are induced by the inflammatory cytokines that are there.
So yes, we think that these cells have a targeted effect in vivo. But your second question was about CORDStrom being used in other indications. And what makes CORDStrom truly unique? There are, as you say, many MSCs have been put into trial and there are at least 3 products, which are now licensed around the world. But all of -- none of those use MSCs from 4 pooled donors. So what we do is we take our [ molecical ]cords, we get them from cord blood banks in the U.K.
And we isolate the MSCs from those individual cords and then we test them for their various different potencies. So if we know the mechanism of action we want in a particular disease, we choose 4 cords that have that particular strength in their mechanism of action, and we pool them.
So CORDStrom for EB is from 4 pooled donors that we have characterized very well, and we have other donors with the same characteristics, which we've also pooled and shown we make the same product. So CORDStrom is one -- CORDStrom EB is one derivation of CORDStrom, but we can select donors cords with different characteristics that might -- some of them are specific to chondrocyte interactions and anti-inflammatory responses that could be targeted to osteoarthritis, for example. We've used another pool to treat SLE patients in in a trial in France, which is published.
So we can -- the reason it's a platform is because by selecting different cords, it becomes a different drug because the potency data, the potency selection are different, and therefore, the potency assays and release assays are different. So that's why we're really excited about it as a platform for multiple different indications.
Sorry, David, a quick one for you. Just we discussed this, you and I at length a couple of weeks back, but just some high-level thoughts of the regulatory environment here in the States, particularly around MSCs and cell therapy in general because as you guys both know, there is one approved -- the first one in the United States was approved last year for GVHD. It could be another filing for heart failure soon, and you know where Capricor is in DMD.
And then here, you guys are coming with Well, that's not an MSC, but Capricor cell therapy potentially for RDEB next year?
Sure. I think the beauty of Mark and his team and when Mark comes up with an idea, he wouldn't pursue it if he knew that he couldn't manufacture it consistently in large scale to deliver not dozens of doses or hundreds of doses, but tens of thousands of doses at a commercial scale cost for a drug. Whereas a lot of companies, they get in love with the science and they develop a drug and then they go back if they get approval to figure out how to manufacture it and the manufacturing cost ends up being so high. A lot of it has to do with staff and facilities and time.
And CORDStrom and INKmune both solve that problem, right? I mean we can get the cost down dramatically. We can provide a repeatable batch and so on. So I think that what Mark has done is exactly what the agencies want to see. They want to see a product that batch to batch consistency. It's the same. They know that if Mark produces a batch today or produces it 10 years from now, it's not a different product.
And then from an end user standpoint, an insurance standpoint and a payment standpoint, yes, these are ultra-rare diseases, so the prices are high because the volume is low, but still the margins are there. Typically, cell therapies, oftentimes, the margins are a little bit tight. We've seen some companies that have some approvals with some cell therapies, and they charge very high prices for it. But what they're making net is a challenge. They've got a lot of work to streamline their manufacturing. Those are not things we have to worry too much about with CORDStrom because that's the way Mark has built it. Truly kind of this process engineering mindset from the start. big advantage.
So from a regulatory standpoint, it's nice to see that they're approving products like this. We've -- I like to think that CORDStrom is the most advanced mesenchymal stromal cell program out there, at least as far as I have seen. And I think it's designed with the regulators in mind from the very beginning.
We will move next with James Molloy with AGP Alliance Global Partners.
Matt on for Jim today. First, on CORDStrom, I wanted to ask about the treatment paradigm, the current treatment paradigm for RDEB in the U.K. and how that looks and where CORDStrom might slot in there? I understand Krystal VYJUVEK is approved there, but not Abeona's EB?
Yes. So it is approved. It's not approved by NICE for reimbursement through the NHS. So there is no RDEB-specific treatment available to be prescribed and paid for by the U.K. government for -- in our health care system. So it's only available for self-payers in the U.K. and I'm not aware of it being widely used, if at all. Certainly, the largest center for pediatric RDEB in the U.K. is Great Ormond Street, where the trial was led from. And they are weekly calling me up and saying, how can we open the next phase of the trial for the patients who were treated.
So there is a big demand for this. I think alluding to what David was saying, the challenge here with the therapies that are available in RDEB is the price, the price point, and we have a drug here that can come in well below those current price points and have a systemic effect.
So we already have -- this trial was driven by NIHR. It was a publicly funded trial, and we were paid to supply the drug. We didn't run the trial, as I said. And that -- the NIHR, which is part of NHS England, specifically said they wanted this drug to be developed as a commercial product if the trials were successful. So we have a lot of push in the U.K. to make this drug available to patients as soon as possible. And I don't see a problem in terms of competing drugs at the moment.
What will happen in the U.S., I'm uncertain because obviously, those drugs are already licensed and are being used in the U.S. But it going to come down to efficacy and cost at the end of the day in terms of which ones survive and which ones and where CORDStrom comes within that.
No, sorry to interrupt. I just want to add, if you don't mind. We cheer on all the competitor products. When you see this disease, it's a huge unmet need in these children, it's just heartbreaking to see -- and so we cheer them on. But I think that the one thing to really keep in mind is that it's typically looked at as a dermatologic disease from a topical nature, but the systemic system is overlooked.
But a byproduct of all of the 3 approved products in the United States is an increase in itch. If you look at the label, it will say itch. And that's really part of the process of healing, right? So the more healing you have the itch.
And again, if you're going to itch, you're going to itch this cream off, you've got to cover it up, tremendously painful. And if you look at the list of the top complaints from RDEB EB patients, usually #1 is itch, even before pain. It's been described to us as like being bitten by mosquito 1,000 times a day. It's just chronic itch. And so it should slot nicely with the competitors.
And again, keep in mind, we're conscious on the margins of the product from day 0 before we even started the trial.
Got it. And in terms of data points suggesting CORDStrom has a systemic effect, I know you mentioned the cytokines, but do you have any anecdotal or quantitative data suggesting symptomatic relief for patients in terms of systemic like wounds behind the eyelid and stuff like that?
Yes. So the data that are published from the trial by Great Ormond Street that were published a few months ago demonstrated the systemic effects that are associated with itch is a systemic disease effectively. But yes, there are systemic data reports from that. What we're looking at now are getting into those data much more acutely, and that's being done independently and blinded from us to guarantee that when we have the data to take to the agencies, there's no question that we have manipulated it.
So we'll be getting those data, but I haven't seen them, so I'm unable to comment on them. But when they come through, we will be sharing them.
Yes. And I'll just add from an anecdotal standpoint, because a very good question. The patients on the trial could pretty much identify whether they're on drug or placebo. And one of the patients to us ended up speaking to the BBC and the BBC published an article about it, and he talked about his massive improvement in quality of life, being able to do things he wasn't able to do before.
And this is something that we heard from some of the PIs as well. And visually, they saw an improvement in the quality of life of these children and their wounds look different to them as well. But if you type in BBC and RDEB into Google, I think it's the first thing that pops up, but that is a child that describes his experience on the MissionEB trial from CORDStrom.
Got it. And then just lastly, on cash runway. Where does your current cash position get you out to in terms of milestones with CORDStrom and also with XPro as well?
Yes. So Q1 is a big timing period for us for XPro. We should have very clear clarity on an accelerated pathway, end of Phase II meeting and certainly the imaging data by then.
Our cash runway, as we've reported in the quarter is really to the end of next year in Q4.
And then obviously, we've got -- we're getting close to the MAA, which will be around the middle of next year.
On top of that, as Mark had alluded earlier, we'll have -- we should have some of the cytokine data and additional data around the MissionEB program towards the end of this year as well. So a number of milestones before we run out of cash.
And this does conclude our Q&A session. I will now turn the call back to David for closing remarks.
Appreciate it. I'd like to wrap up our prepared remarks by saying that we're as excited as ever about the future of INmune Bio. Despite the setbacks of missing the top line on the MINDFuL trial, we're convinced in the prospects for XPro in the Alzheimer's disease and in other diseases.
Meanwhile, we're very optimistic about filing an MAA and BLA in course from next year, and we believe that platform has far greater potential than the market is giving it credit for.
We've had a number of attainable goals in front of us, and we appreciate your support as we go about achieving them.
As always, we thank our stakeholders for your continued support and look forward to updating you on our progress on the discussion milestones. Thank you, everybody.
Thank you. And this does conclude today's program. Thank you for your participation. You may disconnect at any time.
Financial data from Inmune Bio, Inc.
Revenue
Revenue is the sum of all sales generated by a company, e.g. for its products or services.
Revenue (TTM) metric explainedDirect Costs
Direct costs are the costs incurred directly in connection with the manufacture of the product or service.
Gross Profit
Gross Profit indicates how much of the revenue remains in the company after deducting direct production costs. If the percentage share of sales is calculated, this is referred to as the gross margin.
Gross Profit metric explainedSelling and Administrative Expenses
Selling, general and administrative expenses (SG&A) include all expenses for marketing and sales as well as the general administration of the company.
Research and Development Expense
Research and development costs (R&D) provide information on how much the company invests in the research and development of its products. The costs are particularly interesting as a percentage of revenue and in comparison to direct competitors.
EBITDA
EBITDA (Earnings Before Interest, Taxes, Depreciation and Amortization) is the company's earnings before interest, taxes, depreciation and amortization. The EBITDA margin is calculated as a percentage of sales.
Depreciation and Amortization
Depreciation represents reductions in the value of the company's assets (e.g. due to wear and tear on machinery).
EBIT (Operating Income)
EBIT (Earnings Before Interest and Taxes) is the company's profit before interest and taxes, also known as the operating income. The EBIT Margin is calculated as a percentage of sales at
.
Net Profit
Net Profit represents the profit or loss after deduction of all costs.
Net Profit metric explainedStocksGuide Premium
| Jun '26 |
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| Revenue | - - |
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100%
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| - Direct Costs | - - |
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| Gross Profit | - - |
-
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| - Selling and Administrative Expenses | 10 10 |
14%
14%
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| - Research and Development Expense | 10 10 |
67%
67%
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| EBITDA | -20 -20 |
-
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| - Depreciation and Amortization | 0.20 0.20 |
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| EBIT (Operating Income) EBIT | -20 -20 |
49%
49%
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| Net Profit | -18 -18 |
67%
67%
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In millions USD.
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Inmune Bio, Inc. Stock News
Company Profile
INmune Bio, Inc. focuses on controlling components of the immune system to activate an immune response against cancer and Alzheimer's disease. Its product pipeline INKMUNE-Cancer, INB03-Cancer, XPRO1595-Alzheimer, and LIVNATE. The company was founded by Mark Lowdell in September 2015 and is headquartered in La Jolla, CA.
StocksGuide Premium
| Head office | United States |
| CEO | Mr. Moss |
| Employees | 21 |
| Founded | 2015 |
| Website | www.inmunebio.com |


