BioBlast Pharma Announces Cabaletta Phase 3 Study In OPMD Patients - Quick Facts

(RTTNews) - BioBlast Pharma Ltd. (ORPN) announced the start of a Phase 3 pivotal study in the USA and Canada for Cabaletta (trehalose) in Oculopharyngeal Muscular Dystrophy. The company plans to initiate a 12 month, placebo-controlled, multi-center Phase 3 study in the next quarter that will begin enrolling approximately 60 OPMD patients in the USA and Canada.

Colin Foster, BioBlast's President and CEO said, "We see positive signals in a consequential number of the 25 treated patients currently enrolled in our open label HOPEMD study, with respect to various muscle and other performance tests. These positive signals lead us to believe that the clinical study duration required to demonstrate efficacy may be considerably shorter than we initially predicted."

The company expects that the Phase 3 study will be completed by the end of 2016, and because of its shorter duration, will provide results at the same time as the HOPEMD study.