uniQure N.V. - QURE STOCK NEWS

uniQure N.V. (NASDAQ: QURE) announced promising clinical outcomes from its Phase IIb study of etranacogene dezaparvovec for hemophilia B. After two years, patients showed a mean FIX activity of 44.2% of normal, with two out of three remaining free from bleeds. Additionally, the Phase I/II trial of AMT-060 demonstrated sustained clinical benefits over five years, including reduced bleeding rates and FIX consumption. The company plans to include these results in upcoming regulatory submissions to the FDA and EMA.

uniQure N.V. (NASDAQ: QURE) announced its participation in several upcoming virtual investor and scientific conferences. Key events include a fireside chat on December 2, 2020, at the Evercore ISI HealthCONx Conference, featuring R&D president Ricardo Dolmetsch. At the American Society of Hematology Annual Meeting, clinical data on etranacogene dezaparvovec in hemophilia B will be presented on December 8, 2020, followed by an investor webcast. Other conferences include the Gene Therapy Conference for Neurological Disorders and the Lega Italiana Ricerca Huntington Annual Conference, with presentations focused on gene therapies.

uniQure (NASDAQ: QURE) announced its participation in the 62nd American Society of Hematology Annual Meeting, highlighting five key presentations, including a late-breaking oral presentation on the HOPE-B pivotal trial of etranacogene dezaparvovec for hemophilia B. Dr. Steven Pipe will present clinical data during a press briefing on December 7, followed by a detailed presentation on December 8. An investor webcast will be held on the same day at 5:00 p.m. ET, providing further insights into their gene therapy advancements.

uniQure announced positive results from its Phase III HOPE-B trial of etranacogene dezaparvovec, a gene therapy for hemophilia B, showcasing a mean Factor IX activity of 37.2% of normal at 26 weeks. 72% of the 54 patients reported no bleeding events, with a 96% reduction in FIX replacement therapy usage. The therapy was well-tolerated, with no serious adverse events. The data will be presented at the upcoming ASH conference. These findings may expand treatment options for patients with pre-existing neutralizing antibodies, enhancing the therapy's accessibility.

uniQure N.V. (NASDAQ: QURE) has announced its participation at the 62nd American Society of Hematology (ASH) Annual Meeting, occurring virtually from December 5-8, 2020. The company will present four data sets, including an oral presentation on the two-year follow-up of the Phase IIb trial of etranacogene dezaparvovec (AMT-061) and a five-year follow-up from the Phase I/II trial of AMT-060. The CEO, Matt Kapusta, confirmed top-line data from the pivotal HOPE-B study will be announced by year-end, potentially at the ASH meeting.

uniQure N.V. (NASDAQ: QURE) has announced participation in several November 2020 conferences, highlighting its focus on advancing gene therapy for severe medical conditions. Key events include the Credit Suisse 29th Annual Virtual Healthcare Conference from November 9-12, featuring CEO Matt Kapusta and R&D President Ricardo Dolmetsch. The Barclay’s Gene Editing & Gene Therapy Summit will take place on November 16, followed by the Stifel 2020 Virtual Healthcare Conference from November 16-18, where the management team will engage in fireside chats and virtual investor meetings.

uniQure N.V. (NASDAQ: QURE) reported Q3 2020 results with revenues of $1.8M, increasing from $1.0M in Q3 2019. The company enrolled four patients in the Phase I/II trial of AMT-130 for Huntington's disease and expects top-line data from the HOPE-B trial in hemophilia B by year-end. A licensing agreement with CSL Behring may yield $450M upfront and up to $1.6B in milestones. However, a net loss of $53.8M was recorded, up from $23.6M a year prior. Cash reserves stand at $279.5M, sufficient to fund operations into 2022, even without CSL Behring's payment.

uniQure (NASDAQ: QURE) announced the completion of two additional procedures in its Phase I/II clinical trial for AMT-130, aimed at treating Huntington’s disease. This follows an independent Data Safety Monitoring Board review, which reported no significant safety concerns. A total of four patients have now been enrolled, with two receiving AMT-130. The trial will assess safety and efficacy in a total of 26 patients. Enrollment is expected to continue after further DSMB reviews, with completion anticipated by mid-2021, aligning with the company's original clinical timeline.

uniQure (NASDAQ: QURE) announced that the independent Data Safety Monitoring Board (DSMB) reviewed 90-day safety data from the first two patients in its Phase I/II trial of AMT-130 for Huntington's disease. No significant safety concerns were noted, allowing the trial to proceed with enrollment of the next two patients. The double-blind study will assess safety and efficacy in 26 patients with early-stage Huntington's disease. The trial is expected to provide insights into the potential of AMT-130 as a treatment option.