Ra Pharmaceuticals, Inc. (RARX) reported initial data from its phase 2 clinical program evaluating RA101495 to treat paroxysmal nocturnal hemoglobinuria (PNH). Notable findings included no safety or tolerability concerns identified, and no injection site reactions; near-complete inhibition of hemolytic activity; rapid declines in lactate dehydrogenase (LDH); 100% compliance with once daily, subcutaneous self-administration.
One patient reported recurrence of PNH symptoms associated with hemoglobinuria, and an elevated LDH level. The episode of breakthrough hemolysis occurred in the setting of an intercurrent illness, judged by the investigator to be most likely due to a viral infection. “These data will help inform dose selection for pivotal trials and will help refine our clinical development strategy,” said Doug Treco, CEO of Ra Pharma.