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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 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.
uniQure N.V. (NASDAQ: QURE) announced its participation in several upcoming investor and scientific conferences. Key events include Chardan’s 5th Annual Genetic Medicines Conference on October 4-5, 2021, featuring a fireside chat with CEO Matt Kapusta. Other conferences include the Cell & Gene Meeting on the Mesa (October 12-14), where Joshua Leeman will present, and the ESGCT meeting (October 19-22) for preclinical data presentations. The Jefferies Gene Therapy/Editing Summit will take place on October 27-28, with another fireside chat featuring Kapusta.
uniQure N.V. (NASDAQ: QURE) announced its participation in several investor and scientific conferences from September 8 to September 30, 2021. Key events include Citi’s 16th Annual BioPharma Virtual Conference, the Wells Fargo 2021 Virtual Healthcare Conference with a fireside chat featuring CEO Matt Kapusta, and the H.C. Wainwright 23rd Annual Global Investment Conference. Additionally, Ricardo Dolmetsch will participate in discussions at the SVB Leerink CybeRx Series and the 2021 Cantor Virtual Global Healthcare Conference. uniQure aims to advance gene therapies for severe genetic diseases.
uniQure has completed two additional patient procedures in the higher-dose cohort of its Phase I/II clinical trial for AMT-130, targeting Huntington’s disease. With a total of 14 procedures now done, the independent Data Safety Monitoring Board gave a positive recommendation. The company anticipates completing full study enrollment by mid-2022 and plans to share preliminary imaging and biomarker data later this year. The trial aims to explore the safety and efficacy of AMT-130 in treating early manifest Huntington’s disease.
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