The following is a press release from uniQure.
uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, announced it has achieved the targeted dosing of patients in the HOPE-B pivotal trial of etranacogene dezaparvovec (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. The targeted number of patients to be dosed per the clinical trial protocol was 50. In total, 54 patients have received the one-time dose of etranacogene dezaparvovec.
“I am very proud of the entire uniQure team, our investigators, study coordinators and the hemophilia patient communities who helped make this major milestone possible,” stated Matt Kapusta, chief executive officer of uniQure. “With target patient dosing now finished, we are closely monitoring the trial and working within guidance provided by the FDA regarding COVID-19 to minimize any risk or disruption in patient follow-up visits. We continue to expect top-line data from the Phase III trial before the end of this year, which we believe will support a BLA submission in 2021.”
The pivotal Phase III HOPE-B trial follows the Company’s ongoing Phase IIb trial of etranacogene dezaparvovec, in which we have previously reported that, after 52 weeks of follow-up data, all three patients had stabilized and sustained FIX activity at therapeutic levels after the one-time administration of etranacogene dezaparvovec. Additionally, in an ongoing Phase I/II trial of AMT-060, the Company’s first-generation gene therapy for the treatment of hemophilia B, all 10 patients continued to show sustained and stable increases in FIX activity and long-term clinical benefit, including improved disease phenotype and substantial reductions in spontaneous bleeds at up to 4 years of observation.