CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ra Pharmaceuticals announced today that it has launched a Phase 1 study of its synthetic peptide C5 inhibitor, RA101495. The Phase 1 trial is a randomized, double-blind study designed to test the safety of single, escalating doses of RA101495 and to establish the pharmacokinetics and pharmacodynamics after subcutaneous administration in healthy volunteers.
“This study is the first step in the clinical development of RA101495 and will allow us to determine a dose suitable for treating patients with a range of complement-mediated disorders including our lead indication, paroxysmal nocturnal hemoglobinuria (PNH), a rare acquired hematologic syndrome in which red blood cells are destroyed by the complement system,” said Debasish Roychowdhury, M.D., Acting Chief Medical Officer of Ra Pharmaceuticals. “In this study we will be monitoring both safety as well as complement inhibitory activity in plasma samples from treated subjects. The data being presented at ASH highlights the novel mechanism of action and the in vivo pharmacokinetics and pharmacodynamics of RA101495. Our in vivo studies have demonstrated that RA101495 can potently and durably suppress complement activity, which is known to be a critical factor in the treatment of PNH”, added Dr. Roychowdhury.
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