Update on uniQure's Hemophilia B Gene Therapy Program

The following is an excerpt from a press release from uniQure. Read the press release in its entirety here.

uniQure announces the initial topline data from our Phase 2B dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. All patients achieved and sustained therapeutic factor IX activity, with a mean FIX Level of 31 percent of normal at six weeks after administration. None of the patients received factor infusions, experienced any reported bleeding events, or required immunosuppression over a combined 24 weeks of observation.
In addition, patient enrollment is underway in the global Phase 3 HOPE-B clinical trial to evaluate the safety and efficacy of AMT-061.  Adult hemophilia B patients classified as severe and 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 baseline control.  After the six-month lead-in period, patients will go onto receive a single intravenous administration of AMT-061.  Treatment of patients in the HOPE-B pivotal trial is expected to start in the first quarter of 2019. “AMT-061 has the potential to be a major advancement in gene therapy for patients affected by hemophilia B,” stated Steven Pipe, M.D., Professor of Pediatrics and Pathology and Pediatric Medical Director of the Hemophilia and Coagulation Disorders Program at the University of Michigan and principal investigator of the HOPE-B clinical trial.  “A one-time treatment, such as AMT-061, could be life-changing for these patients, many of whom struggle to manage ongoing challenges, including compliance with frequent infusions and recurrent episodes of bleeding.”
 
Read the press release in its entirety here.