Uniqure Stock Price, News & Analysis

The FDA has approved HEMGENIX (etranacogene dezaparvovec-drlb), the first one-time gene therapy for adults with hemophilia B, allowing eligible patients to produce their own factor IX. This treatment reduces the frequency of annual bleeding episodes and eliminates the need for prophylactic therapies. In the pivotal HOPE-B trial, 94% of patients discontinued factor IX prophylaxis post-treatment, with a 54% reduction in the annualized bleeding rate. HEMGENIX represents a significant advancement in hemophilia B treatment, enhancing patient outcomes with a single infusion.

uniQure N.V. (NASDAQ: QURE) announced a Virtual Research & Development Event on November 29, 2022, from 8:30 a.m. to 10:30 a.m. EST, focusing on refractory temporal lobe epilepsy (rTLE) and its preclinical gene therapy candidate AMT-260. The event will feature Dr. Ellen Bubrick, a clinical expert from Brigham and Women’s Hospital, alongside presentations from uniQure’s R&D team and COO Pierre Caloz on advancements in the AAV manufacturing platform. The event will be webcast live and available for replay on uniQure’s website.

VectorY Therapeutics appoints Sander van Deventer as Chief Executive Officer, succeeding Alexander Vos. Van Deventer, a co-founder with 25 years of drug development experience, aims to advance the company's innovative vectorized antibody technology for neurodegenerative diseases, including ALS and Huntington's disease. Other leadership changes include Elena Ritsou as Chief Corporate Officer, Barbara Sanders promoted to Chief Technology Officer, and Frank Walsh joining as an Independent Board Member. These appointments are expected to strengthen VectorY’s strategic direction.

uniQure N.V. (NASDAQ: QURE) reported a net loss of $47.9 million for Q3 2022, with revenues of $1.4 million. The company resumed higher-dose patient enrollment for AMT-130 in Huntington's disease following a DSMB recommendation. The EMA awarded a Good Manufacturing Practice certificate for their Lexington facility, crucial for their gene therapy manufacturing. Cash and equivalents stood at $440.3 million, expected to fund operations until mid-2025. Upcoming events include a virtual investor presentation on November 29, 2022, focused on AMT-260 for refractory temporal lobe epilepsy.

uniQure N.V. (NASDAQ: QURE) announced its participation in several investor and scientific conferences, showcasing its advancements in gene therapy. Key events include Chardan's 6th Annual Genetic Medicines Conference from October 3-4, where a fireside chat with Dr. Ricardo Dolmetsch is scheduled. Additionally, at the European Society of Gene & Cell Therapy from October 11-14, uniQure will present preclinical data on various neurological diseases. Lastly, Dr. Dolmetsch will discuss Huntington's disease at the Latin American Huntington's Disease Conference from October 19-21.

uniQure N.V. (NASDAQ: QURE) announced participation in several investor and scientific conferences this September. Key events include Citi’s 17th Annual BioPharma Conference (Sept. 7-8), Wells Fargo 2022 Healthcare Conference (Sept. 7-9), H.C. Wainwright 24th Annual Global Investment Conference (Sept. 12-14), Cantor Fitzgerald Cell & Gene Therapy Conference (Sept. 15), and European Huntington’s Disease Network (Sept. 16-18). Management, including CEO Matt Kapusta and President of R&D Ricardo Dolmetsch, will engage in investor meetings, fireside chats, and presentations, sharing insights on gene therapies for severe medical needs.

Fulgent Genetics (NASDAQ: FLGT) has appointed Dr. Michael Nohaile and Dr. Leonard Post to its Board of Directors, effective August 1, 2022. Dr. Nohaile, previously at Generate Biomedicines and Amgen, has extensive experience in drug discovery and corporate strategy. Dr. Post, from Vivace Therapeutics, brings over 30 years of drug development experience, particularly in oncology and genetic diseases. Both appointments aim to enhance Fulgent's capabilities as it explores new therapeutic opportunities. The company emphasizes the value these experts will bring to its evolving operations.

uniQure's Phase I/II trial of AMT-130 for Huntington’s disease shows promising results with a 53.8% reduction in mutant HTT protein after 12 months. The treatment was well-tolerated, with no significant safety issues reported. Neurofilament Light Chain levels approached baseline, indicating potential neuronal protection. The company plans to present more data later this year and has completed enrollment for all 26 patients across its U.S. and European trials. CEO Matt Kapusta emphasized the progress in clinical investigations and the commitment to the Huntington's disease community.