Uniqure - QURE STOCK NEWS

uniQure announced the dosing of the first two patients in its European Phase Ib/II clinical trial of AMT-130, a gene therapy for Huntington’s disease. The trial will include 15 patients across Poland, the UK, and Germany. Dr. Ricardo Dolmetsch reported that patient enrollment is expected to finish by year-end, with preliminary data anticipated in Q2 2022. The innovative AMT-130 aims to silence the huntingtin gene using a proprietary AAV5 vector, marking a significant step in gene therapy advancements for neurodegenerative disorders.

CSL Behring released positive long-term results from the Phase 3 HOPE-B trial of etranacogene dezaparvovec (EtranaDez), a gene therapy for hemophilia B. The study showed a stable increase in mean Factor IX (FIX) activity, reaching 36.9 IU/dL at 18 months post-infusion. Participants experienced a 64% reduction in annual bleeding rates and 98% discontinued prophylactic treatment. The therapy was generally well-tolerated, though one unrelated death occurred. This data reinforces the potential of EtranaDez to transform hemophilia B management and may accelerate regulatory reviews in the U.S. and Europe.

uniQure N.V. (NASDAQ: QURE) reported positive preliminary data from its Phase I/II trial of AMT-130 for Huntington's disease. Treatment was well tolerated, with no serious adverse events noted in the first four patients at one year of follow-up. Neurofilament Light Chain levels returned to baseline in treated patients. A total of 19 procedures have been completed, and higher-dose cohort enrollment is progressing. An open-label study in Europe has also commenced, aiming to further assess safety and efficacy. Upcoming data releases are expected in 2022 and beyond.

CSL Behring announced that the European Medicines Agency's CHMP has accepted its request for an accelerated assessment of the etranacogene dezaparvovec Marketing Authorization Application (MAA) for hemophilia B. This gene therapy, currently in Phase 3 trials, could provide a significant long-term treatment option for patients. The accelerated review may cut the evaluation timeline from 210 to 150 days. The company aims to submit the MAA in the first half of 2022, highlighting their commitment to innovative treatments and addressing the unmet needs of hemophilia B patients.

uniQure's etranacogene dezaparvovec gene therapy has achieved significant clinical milestones in the pivotal Phase III HOPE-B trial for hemophilia B. The therapy met its primary endpoint by demonstrating non-inferiority in annualized bleeding rate (ABR) compared to standard FIX prophylactic therapy after 18 months, with an ABR of 1.51 versus 4.19 in the lead-in period. Additionally, it achieved statistical superiority in bleeding rate reduction (p=0.0002). FIX activity remained stable, averaging 36.9% of normal levels at 18 months. CSL Behring plans to file for regulatory approval in the U.S. and EU in 2022.

uniQure (NASDAQ: QURE) announced positive results from the Phase I/II trial of AMT-130 for Huntington’s disease. The Data Safety Monitoring Board (DSMB) recommended proceeding with the trial after reviewing safety data from the first four patients in the higher-dose cohort. Enrollment of the final 12 patients is expected to complete by mid-2022. Currently, eight patients have been treated with AMT-130, with a total of 14 procedures completed. The trial aims to establish safety and efficacy in 26 patients, with a follow-up period of five years.

uniQure N.V. (NASDAQ: QURE) announced its participation in key investor and scientific conferences. The 28th Annual Meeting of the Huntington Study Group will be held virtually from November 4-6, 2021, featuring Dr. David Cooper discussing AMT-130 clinical trials. Additionally, the Virtual Neuroscience 2021 conference takes place from November 8-11, with Dr. Valeria Crepel presenting novel gene therapy for temporal lobe epilepsy. Lastly, the Stifel 2021 Virtual Healthcare Conference is scheduled for November 15-17, including a fireside chat by CEO Matt Kapusta.

uniQure N.V. (NASDAQ: QURE) reported third-quarter 2021 financial results, highlighting significant clinical advancements. The pivotal study for hemophilia B completed 78-week follow-ups, with top-line data expected by year-end 2021. The company has enrolled 14 patients in the Phase 1/2 clinical trial for AMT-130 in Huntington’s Disease, showing no significant safety concerns. Additionally, uniQure acquired Corlieve Therapeutics, enhancing its research pipeline. As of September 30, 2021, the company holds $578.5 million in cash, sufficient to fund operations into 2024.

uniQure N.V. has appointed Rachelle Jacques to its Board of Directors, enhancing its expertise in gene therapy development. With over 25 years in the biotech sector, including leadership roles in major companies, Jacques brings valuable experience in launching rare disease therapies. She will serve on the Audit Committee and assist in advancing uniQure’s clinical programs, including a BLA submission for hemophilia B. Jacques's past achievements highlight her capability to bolster uniQure's pipeline, which addresses severe genetic diseases through innovative gene therapies.