Peter Smith
executive
Thank you, Will, and thank you all for joining us today. Today marks a transformational milestone for Remix Therapeutics as we take this step to become a publicly traded company through our merger with Passage Bio. At Remix, our mission is to reprogram RNA processing to address disease drivers at its origin. For decades, drug discovery has focused primarily on proteins, leaving many important disease drivers largely inaccessible by conventional approaches.
We take a fundamentally different approach by targeting RNA processing directly, which could allow us to control gene expression upstream of protein production and unlock new therapeutic possibilities. We believe this represents a meaningful shift in the way medicines can be discovered and developed, enabling new ways to intervene in disease biology. This transaction is structured to accelerate that vision. It provides immediate access to the public markets, enabling us to expand our investor base and scale our efforts.
Importantly, it is supported by an oversubscribed private placement led by new investor Decheng Capital with participation from leading institutional investors that will result in total gross proceeds of over $100 million. Combined with the capital from Passage Bio, we expect to have cash runway into 2028, allowing us to execute against key clinical milestones across our pipeline.
At the core of our company is the REMaster platform, which integrates deep expertise in data science, biomolecular sciences and chemistry to rationally design small molecules that modulate RNA processing. This platform allows us to create highly specific and selective orally available therapies against disease-driving targets that have proven challenging to address with conventional approaches, while also serving as a scalable discovery engine for generating multiple new programs over time.
Our lead program, REM-422, is a clear example of this strategy in action. REM-422 is an orally available mRNA degrader targeting MYB, an oncogenic transcription factor implicated across multiple cancers, starting with our work in adenoid cystic carcinoma or ACC, and acute myeloid leukemia and high-risk myelodysplastic syndromes or AML high-risk MDS. Rather than attempting to directly inhibit the MYB protein, REM-422 induces the inclusion of a poison exon in the MYB mRNA transcript, leading to its degradation through nonsense mediated decay.
Thus, we are inhibiting MYB's function by preventing it from being expressed through an RNA degradation mechanism. This represents a powerful mechanism of action and a new way to approach previously inaccessible targets. REM-422 has received orphan drug designation for AML and ACC as well as Fast Track designation for ACC from the FDA, reinforcing both the unmet medical need in these indications and the potential importance of this therapy. REM-422 is currently being evaluated in a Phase I/II clinical trial in patients with ACC and a Phase I study in AML and high-risk MDS.
In ACC, this includes both a Phase I dose escalation phase in 69 patients designed to determine the maximum tolerated dose and recommended Phase II dose and a Phase II confirmatory cohort targeting 40 to 50 patients, which will further evaluate safety and efficacy in biomarker-positive patients. The Phase I study is completed and our ARIA study is currently enrolling in the Phase II portion, which is an open-label, nonrandomized multicenter trial.
ACC is a solid tumor with over 1,500 new cases seen each year in the U.S. and a prevalent population of 13,000 to 16,000 people. It most commonly arises in the salivary glands and is characterized by frequent recurrence, perineural invasion and dysregulation of the MYB oncogene. Approximately 60% to 65% of ACC patients are MYB poison exon biomarker positive and potential candidates for REM-422 treatment.
Depending on tumor location, patients may experience symptoms such as facial numbness, difficulty swallowing, vision changes or difficulty breathing. Despite multiple therapeutic approaches being explored, including chemotherapy, kinase inhibitors and immunotherapy, clinical outcomes have historically been modest. There are currently no approved treatment options, which underscores the significant unmet need and opportunity for innovation in these patients.
We recently reported encouraging clinical data from the ongoing ARIA study in patients with ACC at the 2026 ASCO Annual Meeting. REM-422 demonstrated dose proportional increases in exposure across all dose levels tested and reduction of mid- mRNA and protein levels in tumor biopsies taken from patients. REM-422 has demonstrated favorable safety and tolerability data with no dose-limiting toxicities observed to date and primarily low-grade adverse events such as anemia, fatigue and epistaxis.
The recommended Phase II dose of 24 milligrams was generally well tolerated with several patients approaching 2 years of treatment and ongoing. In the biomarker positive cohort treated at the recommended Phase II dose, an overall response rate of 43% was observed with durable responses exceeding 1 year and ongoing. We also observed a 100% disease control rate. Responses were observed across molecular subtypes, various histologies and regardless of prior lines of therapy, including in patients with previously treated with antibody drug conjugates, highlighting the potential breadth of activity even in a heavily pretreated patient population.
These results are compelling and unprecedented in this disease, given that there are no systemic treatment options for this life-threatening cancer. Of note, we have aligned with the FDA on the recommended Phase II dose, use of biomarker selection that is an assay designed to identify patients that are MYB poison exon positive performed in collaboration with Tempus and also the design of the Phase II study. Enrollment in the Phase II study is proceeding very well with over 50% of the planned study size already enrolled since the trial opened in late Q4 of 2025.
Looking ahead, we are excited to progress our programs and pipeline, including advancing REM-422 through clinical development and generating key data that we believe can meaningfully inform its potential. We expect to have initial data from our ongoing Phase II cohorts for REM-422 and ACC in mid-2027. We have also started enrollment in the Phase I study of REM-422 in AML high-risk MDS and have observed strong preliminary antitumor activity during dose escalation.
Dose escalation is ongoing to determine the recommended Phase II dose for REM-422 in AML/MDS, and we expect to have top line data in mid-2027. Furthermore, we expect to progress our discovery pipeline with nomination of a development candidate for our mRNA degrader program targeting MYC-dysregulated cancers in 2027. The transaction announced today will fund the company through these milestones with cash runway into 2028. Upon closing of the transaction, I will continue to serve as Chief Executive Officer, working alongside an experienced leadership team and Board of Directors.
Together, this team brings deep expertise in RNA biology, drug development and company building with a shared commitment to translating innovative science into impactful medicines. Taking a step back to reflect, this transaction is transformative as it positions Remix to lead in the new category of RNA-targeted therapeutics, advance a differentiated clinical pipeline and deliver important milestones in the near and midterm. Most importantly, it strengthens our ability to bring meaningful new treatment options to patients with significant unmet medical needs. We are excited about the opportunity ahead and grateful for the continued support of our investors, employees and partners. Thank you for joining us today.
