uniQure Announces Updated Data from Phase 2B Dose Confirmation of AMT-061

uniQure is excited to announce that updated data from their Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B (see attached press release). All patients achieved and sustained therapeutic factor IX activity at up to 51% of normal with a mean factor IX Level of 38% of normal at twelve weeks after administration. None of the patients received factor infusions, reported any bleeding events, or required immunosuppression over a combined 42 weeks of observation.

In addition, they announced that the first patient was treated in the global Phase 3 HOPE-B clinical trial to evaluate the safety and efficacy of AMT-061 (see attached press release). Adult hemophilia B patients classified as severe and moderately-severe are being enrolled in a six-month observational period during which time they continued to use their current standard of care to establish a baseline control. After the six-month lead-in period, patients go onto received a single intravenous administration of AMT-061. the HOPE-B trial is currently enrolling patients interested in learning more. Feel free to write uniQureHOPE0B@uniqure.com for more information.

For more information:

 uniQure Announces Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B

uniQure Announces First Patient Treated in HOPE-B Pivotal Trial of AMT-061 in Patients with Hemophilia B

 uniQure Announces Publications on AAV Gene Therapy Approach to Treating Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) in the Journal Molecular Therapy Nucleic Acids