Welcome to our dedicated page for Uniqure news (Ticker: QURE), a resource for investors and traders seeking the latest updates and insights on Uniqure stock.
uniQure N.V. (NASDAQ: QURE) is a gene therapy company that regularly issues news on its clinical programs, regulatory interactions, and financing activities. The company describes itself as delivering single‑treatment gene therapies with potentially curative results and has reported that the approvals of its gene therapy for hemophilia B mark a major milestone in genomic medicine. News about uniQure often highlights progress in its pipeline for Huntington’s disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe conditions.
A major focus of QURE news coverage is AMT‑130, uniQure’s investigational gene therapy for Huntington’s disease. Press releases and related SEC filings detail pivotal Phase I/II trial results, including statistically significant slowing of disease progression at 36 months in high‑dose cohorts compared to a propensity score‑matched external control, as well as trends in motor and cognitive endpoints and biomarker changes. Investors also see updates on the company’s regulatory dialogue with the U.S. Food and Drug Administration, including pre‑BLA and Type A meetings and feedback on the adequacy of Phase I/II data for a potential Biologics License Application.
Beyond AMT‑130, uniQure news includes early‑stage data and enrollment updates for AMT‑260 in refractory mesial temporal lobe epilepsy, AMT‑191 in Fabry disease, and AMT‑162 in SOD1 ALS. Corporate news items cover public offerings of ordinary shares and pre‑funded warrants, amendments to loan facilities, and quarterly financial results. This QURE news page aggregates such disclosures so readers can follow clinical milestones, regulatory developments, and capital markets events that shape uniQure’s gene therapy business over time.
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.
uniQure N.V. (NASDAQ: QURE) announced participation in several upcoming virtual conferences in September 2020. Key events include:
- Citi’s 15th Annual BioPharma Virtual Conference (Sept. 9-10) with a live fireside chat on Sept. 9.
- Baird’s Virtual Global Healthcare Conference (Sept. 9-10) featuring one-on-one investor meetings.
- Wells Fargo Global Healthcare Conference (Sept. 9-10) with a fireside chat on Sept. 10.
- H.C. Wainwright 22nd Annual Global Investment Conference (Sept. 14-15) and Cantor Fitzgerald Virtual Global Healthcare Conference (Sept. 15-17).
- Barclays Boston Biotech Bus Trip (Sept. 23-25).
These events aim to provide insights into uniQure's gene therapy advancements.