Uniqure Stock Price, News & Analysis

uniQure N.V. (NASDAQ: QURE) announced it will present eight studies at the ISTH Virtual Congress from July 17-21, 2021, focusing on gene therapy for hemophilia B. Highlights include five-year data on FIX expression stability and outcomes from the Phase 3 HOPE-B trial. Notable presentations include the clinical outcomes of patients with pre-existing AAV5 antibodies and a case report on liver safety. The company continues to advance gene therapies for severe conditions, emphasizing its commitment to innovative treatment solutions.

uniQure N.V. (QURE) announced promising results from its Phase III HOPE-B trial of etranacogene dezaparvovec, showing sustained Factor IX (FIX) activity at an average of 41.5% of normal after one year. The treatment reduced bleeding episodes by 80% and allowed 96% of patients to discontinue FIX replacement therapy. No severe adverse events were reported. Following a meeting with the FDA, uniQure will submit a BLA by Q1 2022, focusing on non-inferiority in annualized bleeding rates.

uniQure (NASDAQ: QURE) has announced a definitive agreement to acquire Corlieve Therapeutics for €46.3 million. This acquisition expands uniQure's gene therapy pipeline targeting temporal lobe epilepsy (TLE), which affects 1.3 million individuals in the U.S. and Europe, including 800,000 drug-resistant patients. The lead program, AMT-260, leverages miRNA silencing technology to address chronic spontaneous seizures. The deal may involve additional milestone payments totaling up to €203.7 million, pending clinical progression and regulatory approvals.

uniQure N.V. (NASDAQ: QURE) has announced the completion of the first patient procedures in the Phase I/II clinical trial for AMT-130, targeting Huntington's disease. This trial's second cohort will involve 16 patients at a higher dose, following a review by the Data Safety Monitoring Board. So far, seven patients have been treated with AMT-130, with five receiving imitation surgeries. The company plans to enroll more patients and expects to share initial imaging and biomarker data before year-end.

uniQure N.V. (NASDAQ: QURE) will host a virtual Research & Development Day on June 22, 2021, from 8:30 to 11:30 a.m. ET, focusing on its expanding gene therapy pipeline for CNS and liver-directed disorders. The company will also participate in multiple upcoming virtual conferences including the Jefferies Virtual Healthcare Conference (June 1-4), European Hematology Association Virtual 2021 (June 7-13), and Goldman Sachs 42nd Annual Global Healthcare Conference (June 8-11). Notably, results from the pivotal Phase 3 HOPE-B trial will be presented during the EHA conference.

uniQure, a leader in gene therapy, announced its upcoming participation in several virtual investor and scientific conferences, including the Jefferies Virtual Healthcare Conference and the European Hematology Association Virtual 2021. Key highlights include a Research & Development Day on June 22, 2021, focusing on advancements in gene therapy for CNS and rare liver-directed disorders. Additionally, the company will present pivotal data from the HOPE-B trial for etranacogene dezaparvovec targeting hemophilia B.

uniQure has announced the independent Data Safety Monitoring Board (DSMB) has recommended proceeding with the second, higher-dose cohort of its Phase I/II clinical trial for AMT-130, aimed at treating Huntington’s disease. This decision follows a positive review of safety data from the first cohort of ten patients. Moving forward, uniQure plans to enroll patients in the higher-dose cohort and initiate clinical development in Europe later this year. The trial aims to establish the safety and efficacy of AMT-130 in patients with early-stage Huntington's disease.

uniQure presented pivotal Phase III HOPE-B trial data for etranacogene dezaparvovec at the ASGCT Annual Meeting. The study, involving 54 hemophilia B patients, showed that pre-existing neutralizing antibodies (NAbs) to AAV5 did not significantly correlate with FIX activity. Participants with NAbs achieved similar treatment results compared to those without. Notably, 57.4% of subjects had no pre-existing NAbs, and all but one participant achieved stable FIX levels, suggesting etranacogene dezaparvovec could be a viable therapy for a broader patient base.

uniQure N.V. (NASDAQ: QURE) reported its Q1 2021 results, highlighting its advancements in gene therapy for hemophilia B and Huntington's disease. The company closed a $450 million licensing deal with CSL Behring and completed patient enrollment for a Phase I/II trial of AMT-130 for Huntington's. Cash reserves increased to $260.8 million, with funding expected until 2024. However, the net loss rose to $41.6 million, with R&D expenses reaching $32.7 million. Upcoming catalysts include 52-week follow-up data from the HOPE-B trial and a planned R&D Day.