Bio Blast Pharma Ltd (NASDAQ:ORPN), a leading clinical-stage biotechnology company, announced final results from HOPEMD Phase 2 open-label clinical study in oculopharyngeal muscular dystrophy patients. The OPMD is a less-known progressive muscle-wasting disease that causes swallowing difficulties and lead to the increased risk of aspiration of food into human lungs. Moreover, it may also result in progressive muscle weakness and weight loss over time.
Insights of Matter
Bio Blast’s senior medical advisor, Prof. Zohar Argov M.D, will present these findings in detail during the plenary session at Myology 2016 in Lyon, France. He will also talk about these conclusions in the month of April at American Academy of Neurology in Canada. The thorough information regarding the timing of these events and presentations can be found on Bio Blast’s official website.
The primary motive behind carrying out HOPEMD Phase 2 open-label study was to access the tolerability and safety of trehalose 90mg/mL IV solution in a hassle-free manner. The team of scientists involved in this study included secondary endpoints in this study to try and see whether trehalose 90mg/mL IV solution could prevent OPMD disease from turning bad to worse.
As part of the Phase 2 study, Bio Blast took into account 25 patients suffering from muscle weakness and clinical dysphagia at two centers in Israel and Canada. So far, these patients have completed the weekly treatment for a continuous duration of 24 weeks with trehalose 90mg/mL IV solution.
The research team found that the drug had no adverse effect on patients. Final results are said to be consistent with what the research team announced during interim result announcement. Going forward, it will continue to take many more such measures to finish this study as soon as possible and start the commercial deployment of trehalose 90mg/mL IV solution in an efficient manner.