QURE Stock Recent News

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to uniQure N.V.'s QURE AMT-130 for Huntington's disease on Thursday

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

UniQure (QURE) came out with a quarterly loss of $1.50 per share versus the Zacks Consensus Estimate of a loss of $0.44. This compares to loss of $1.53 per share a year ago.

~ 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 extended uniQure's operating runway through 2Q28, with significant analyst support and price targets up to $70 a share.

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 $251m in cash and $183m in securities, despite a $100m net loss in the first 9m of 2025.

CSL Limited CSLLY and uniQure Inc QURE announced the four-year results from the pivotal HOPE-B study confirming the long-term durability and safety of a one-time infusion of Hemgenix (etranacogene dezaparvovec-drlb) for adults hemophilia B patients.

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-term bleed protection as mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by approximately 90% from lead-in as compared to year four KING OF PRUSSIA, Pa. , Feb. 7, 2025 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced the four-year results from the pivotal HOPE-B study confirming the long-term durability and safety of a one-time infusion of HEMGENIX® (etranacogene dezaparvovec-drlb) for adults living with hemophilia B.

~ 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 ~