Uniqure Stock Price, News & Analysis

uniQure (NASDAQ: QURE) announced positive progress in its Phase I/II clinical trial of AMT-130 for Huntington's disease. An independent Data Safety Monitoring Board confirmed no significant safety concerns after reviewing six-month safety data from the first two patients and 90-day data from two others. All remaining patients in the first cohort are cleared for enrollment, targeting full enrollment by mid-2021. The trial aims to assess safety, tolerability, and efficacy in 26 patients, with initial biomarker and imaging data expected by year-end 2021.

uniQure N.V. (NASDAQ: QURE) has announced its participation in several key virtual investor and scientific conferences in February 2021. Highlighted events include the 14th Annual EAHAD Congress, where uniQure will present data on their gene therapy candidate, etranacogene dezaparvovec, for hemophilia B. Other presentations will focus on medical affairs and healthcare economics. The company will also engage in a fireside chat during the SVB Leerink Global Healthcare Conference. Additional details on their presentations will be disclosed closer to the event dates.

uniQure N.V. (NASDAQ: QURE) has announced that its hemophilia B gene therapy program, including the pivotal Phase III HOPE-B study, is on clinical hold by the FDA after a serious adverse event linked to liver cancer was reported in a patient. Although patient dosing is complete, no new patients will be treated. A full surgical resection of the liver lesion is scheduled, with no other cases of liver cancer reported in over 100 patients. The company is investigating the event and plans to monitor patients without impacting regulatory timelines.

uniQure (NASDAQ: QURE) announced encouraging results from its HOPE-B Phase III trial of etranacogene dezaparvovec for hemophilia B. The study met its primary endpoint, showing a mean Factor IX activity of 37.2% at 26 weeks. Patients experienced a 91% reduction in bleeds requiring treatment, with 87% reporting no such bleeds. Etranacogene dezaparvovec was well-tolerated, with no serious adverse events. This trial includes the largest sample of patients (54) receiving a single investigational gene therapy, indicating a potential breakthrough for hemophilia B treatment.

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.