BioBlast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, announced positive results from its HOPEMD Phase 2 six-month open-label clinical study in patients with oculopharyngeal muscular dystrophy (OPMD), a rare progressive muscle-wasting disease characterized by swallowing difficulties (dysphagia), leading to the risk of aspiration of food into the lungs, weight loss, and generalized progressive muscle weakness. These results will be presented today by Prof. Zohar Argov M.D., Senior Medical Advisor to BioBlast, during the plenary session at Myology 2016, a leading muscle conference in Lyon, France, and, in April, 2016, during two general sessions at the American Academy of Neurology in Vancouver, Canada. See information in this release regarding these scientific symposia.
The primary objective of the HOPEMD Phase 2 open-label study was to assess the safety and tolerability of trehalose 90mg/mL IV solution. Although not powered for efficacy, secondary endpoints were included to explore if trehalose 90mg/mL IV solution could improve or prevent worsening of OPMD disease markers. The study enrolled 25 patients with clinical dysphagia and muscle weakness at two centers in Canada and Israel. All patients have now completed 24 weeks of weekly treatment with trehalose 90mg/mL IV solution and these are the data presented today.