uniQure N.V. Stock News

~ 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 ~

~ Independent Data Monitoring Committee Recommends Proceeding with Dose Escalation After Planned Safety Assessment of First Dose Cohort ~

LEXINGTON, Mass. and AMSTERDAM, Jan. 08, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the pricing of its underwritten public offering of 4,411,764 of its ordinary shares at a public offering price of $17.00 per share. The aggregate gross proceeds to uniQure from the...

LEXINGTON, Mass. and AMSTERDAM, Jan. 07, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it has commenced an underwritten public offering of its ordinary shares and pre-funded warrants to purchase its ordinary shares. All securities to be sold in the offering will...