uniQure Announces First Patient Treated in Pivotal Trial

uniQure N.V. recently announced it treated the first patient in its HOPE-B pivotal trial of AMT-061, an investigational AAV5-based gene therapy incorporating the patent-protected FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. AMT-061 has been granted Breakthrough Therapy Designation by the United States Food and Drug Administration and access to Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.

“We are very pleased to have successfully administered AMT-061 to the first patient enrolled in the HOPE-B pivotal trial and mark this as a milestone for the field in advancing a potential one-time treatment for patients with hemophilia B,” said Robert Gut, MD, PhD, Chief Medical Officer of uniQure. “I am extremely proud of the efforts made by the uniQure team to advance this study, and we plan to complete full patient enrollment in this study before the end of this year.”

“Our mission in hemophilia B has always been to be first to market with a best-in-class gene therapy,” said Matt Kapusta, chief executive officer. “With the initiation of the dosing phase of our pivotal study for AMT-061 and the data generated to date in our Phase IIb study, we believe that we are on track toward our goal of developing the first gene therapy that provides durable, functionally-curative benefits to nearly all patients with hemophilia B, without the complications associated with capsid-related immune responses or the need for immunosuppression therapy.”

The global HOPE-B Phase III clinical trial will evaluate the efficacy and safety of AMT-061. Approximately 50 adult patients with hemophilia B classified as severe or moderately severe will be enrolled in a six-month observational period, during which time they will continue to use their current standard of care to establish a prospective comparator. After the six-month lead-in period, patients will receive a single intravenous administration of AMT-061. The primary outcome measure is the assessment of Factor IX activity 26 weeks after AMT-061 dosing. Secondary outcome measures include annualized bleeding rate (ABR) and usage of Factor IX replacement therapy over a 52-week time frame, as well as other efficacy and safety aspects. Post-treatment, patients will be followed for 5 years.

AMT-061 consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). uniQure holds multiple issued patents in the United States and Canada broadly covering methods of treating bleeding disorders, including hemophilia B, using AAV gene therapy with the FIX-Padua variant. Additional patents are pending in the European Union.

AAV5-based gene therapies have been demonstrated to be safe and well-tolerated in a multitude of clinical trials, including four uniQure trials conducted in 25 patients in hemophilia B and other indications. No patient treated in clinical trials with the Company’s AAV5 gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid. Additionally, preclinical and clinical data show that AAV5-based gene therapies may be clinically effective in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates.

uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and other severe genetic diseases. www.uniQure.com