Bio Blast Pharma Explodes on Positive Results

Bio Blast Pharma Ltd. (NASDAQ: ORPN) was blowing away all the other bulls in Wednesday’s session, with its stock up over 150% on positive clinical results. The company announced positive results from its HopeMD Phase 2 six-month open-label clinical study in patients with oculopharyngeal muscular dystrophy (OPMD).

OPMD can be described as 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.

The treatment was observed to be safe and well tolerated with no drug-related serious adverse events reported. Also no patients chose to discontinue the study for reasons related to safety or side effects.



Bernard Brais, M.D., M.Phil., Ph.D., FRCPC, professor, Departments of Neurology and Neurosurgery and Human Genetics, Faculty of Medicine, McGill University, Co-director Rare Neurological Diseases Group at the Montreal Neurological Institute, and principal investigator in the study, commented:

The final study results are consistent with the interim data previously reported and continue to look promising. With no pharmacotherapy available to treat OPMD, trehalose 90mg/mL IV solution may be a step forward in caring for these patients. We look forward to confirming these results in the planned Phase 2b double blind placebo controlled study.

Warren Wasiewski M.D., chief medical officer of BioBlast, added:

We are encouraged by the safety profile observed and the early efficacy signals noted in this relatively small study of OPMD patients. With this knowledge, we can proceed to develop our double blind placebo controlled study to confirm these results and progress our clinical program to registration.

Shares of Bio Blast were trading up about 160% at $6.83, with a consensus analyst price target of $24.00 and a 52-week trading range of $2.58 to $8.50.