RA Pharmaceuticals to present overview of RA101495, oral C5 inhibitor
Ra Pharmaceuticals announced that the company will present an overview of its lead product candidate, RA101495, a potent subcutaneously-administered macrocyclic peptide inhibitor of C5, and an update on its oral small molecule C5 inhibitor program, at the International PNH Interest Group Annual Scientific Assembly on Friday, December 2, 2016 in San Diego, CA. The presentation will include a summary of Phase 1 study results and a description of the planned Phase 2 study design for RA101495 in paroxysmal nocturnal hemoglobinuria patients, as well as new pre-clinical data from Ra's oral small molecule C5 inhibitor program. "Our Phase 1 study in healthy volunteers demonstrated that RA101495 is a potent inhibitor of C5, provides rapid, complete and sustained inhibition of hemolysis, was well tolerated and can be conveniently and effectively delivered by subcutaneous injection," said Doug Treco, PhD, Co-Founder, President and CEO of Ra Pharma. "We are eager to initiate our Phase 2 program in PNH patients in the first quarter of 2017, and we also look forward to advancing our oral small molecule C5 inhibitor program through early development and toward an IND. PNH is a rare and life threatening blood disorder for which new treatment options are needed. We believe RA101495, as well as molecules discovered through our oral small molecule C5 inhibitor program, represent attractive alternatives to monoclonal antibody-based treatment options, which require regular intravenous infusions."