Patient dosing has started in Ra Pharmaceuticals' (Pending:RARX) Phase 2 program evaluating RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare life-threatening blood disease characterized by the destruction of red blood cells by the complement system. Mid-stage development includes two clinical trials, one in the U.S. and one ex-U.S.
The U.S. study will enroll 6 - 8 subjects who are not responding adequately to Alexion's Soliris (eculizumab).
The company says it expects to report initial results by mid-year. According to ClinicalTrials.gov, the estimated study completion date is February 2018.
RA101495 is a subcutaneously administered inhibitor of complement component 5 (C5).