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uniQure N.V. (NASDAQ: QURE) is a leading gene therapy company focused on developing transformative therapies for patients suffering from severe genetic and other devastating diseases. Based on its proprietary technology platform, uniQure aims to deliver single-treatment solutions with potentially curative results. The company is dedicated to addressing unmet medical needs in the areas of hemophilia, Huntington's disease, and cardiovascular diseases.
uniQure's core business revolves around advancing a robust pipeline of gene therapies, including its collaboration with Bristol Myers Squibb to develop treatments for cardiovascular diseases. The company’s most notable product, HEMGENIX®, is the first and only gene therapy approved for the treatment of adults with hemophilia B. Recent data from the HOPE-B study presented at the American Society of Hematology Annual Meeting confirmed the long-term efficacy and safety of HEMGENIX®, highlighting its ability to offer elevated and sustained factor IX activity levels for years post-treatment.
The company's financial highlights and strong partnerships underscore its commitment to innovation and leadership in the gene therapy field. uniQure continues to make significant strides with current projects and patient enrollment for new trials expected to begin in the first half of 2024.
With its validated modular technology platform and a dedicated focus on severe genetic diseases, uniQure’s work holds significant promise for transforming patient care and outcomes. The company's latest developments and financial performance are followed closely by investors and the biotechnology community, reflecting its pivotal role in advancing gene therapy solutions.
CSL Behring's Marketing Authorization Application (MAA) for etranacogene dezaparvovec has been accepted for accelerated assessment by the EMA, potentially making it the first gene therapy for hemophilia B. Supported by the phase 3 HOPE-B study, which showed a 64% reduction in annual bleeds after a single infusion, this breakthrough therapy aims to significantly improve patients' lives. Etranacogene dezaparvovec addresses the underlying genetic cause of hemophilia B, offering hope for a transformative treatment option in the EU and EEA.
uniQure N.V. (NASDAQ: QURE) announced the completion of enrollment for the first two cohorts in its Phase I/II clinical trial of AMT-130, targeting early-stage Huntington's disease. A total of 26 patients were enrolled across two cohorts, with initial results expected in Q2. This trial aims to evaluate the safety and efficacy of AMT-130, a gene therapy designed to reduce harmful protein levels associated with the disease. A third cohort is planned to refine the administration procedure. The trial advances uniQure's commitment to innovative gene therapies addressing critical medical needs.
uniQure N.V. (QURE) reported a significant revenue increase to $524 million for 2021 from $37.5 million in 2020, driven primarily by a $462.4 million license revenue from its CSL Behring agreement. The company ended 2021 with $556 million in cash, sufficient to fund operations into 2025. Positive clinical data from the HOPE-B trial demonstrated the efficacy of etranacogene dezaparvovec for hemophilia B, with a 64% reduction in annualized bleeding rate. Ongoing studies for Huntington’s Disease and new therapies for refractory temporal lobe epilepsy and Fabry disease are advancing, maintaining a strong pipeline.
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.
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