uniQure N.V. Stock News

uniQure's AMT-130 gene therapy for Huntington's Disease shows promising trial data, leading to significant stock price increases and potential for FDA accelerated approval. QURE's stock has seen volatility but has near-term catalysts like BLA submission and 36-month trial data, suggesting potential for immediate further gains. Despite competition from PTC518, AMT-130's effectiveness and one-tim...

uniQure's AMT-130 gene therapy showed an 80% slowing of Disease progression in Huntington's Disease patients in a phase 1/2 study. Financially strong with $448 million in cash, uniQure can fund operations through H2 2027, reducing immediate financial risk. AMT-191 for Fabry Disease and AMT-162 for ALS are also advancing, diversifying uniQure's pipeline and potential market impact.

~ Breakthrough Therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression ~

~ Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in Huntington's disease; Initiated preparations for a potential Biologics License Application (BLA) submission ~

uniQure's stock surged in December due to positive data around its Huntington's disease candidate AMT-130, clearing the way for potential accelerated FDA approval without another trial. The company's gene therapy Hemgenix, approved for hemophilia B, and three new clinical programs, including AMT-191, AMT-162, and AMT-260, bolster its pipeline. Cost-cutting measures and strategic financing have ...

uniQure's gene therapy AMT-130 for Huntington's Disease shows promising clinical data, with an 80% reduction in disease progression, positioning it for potential FDA approval. Despite past commercial failures, uniQure's current market cap of ~$715m appears undervalued given AMT-130's estimated peak sales potential of ~$2bn. The company has a solid cash position to fund AMT-130's launch, with $2...

94 percent of patients eliminated factor IX prophylaxis and remained free of continuous prophylaxis through four years post-treatment Mean factor IX activity levels were sustained at near normal levels of 37% through four years post-treatment, reinforcing the efficacy of HEMGENIX in the treatment of hemophilia B Phase 3 HOPE-B data showed that a one-time treatment with HEMGENIX provided long-te...

~ Independent Data Monitoring Committee recommends proceeding with dosing of second cohort after planned safety assessment ~