Ra Pharmaceuticals announced positive results from the Company’s Phase 1b pharmacokinetic study evaluating zilucoplan in patients with renal impairment. The Company has received International Nonproprietary Names clearance for use of the name zilucoplan from the World Health Organization. The Phase 1b, multi-center, open-label trial was designed to evaluate the PK profile of zilucoplan in patients with severe renal impairment as a lead-in to studying zilucoplan in complement-mediated renal disorders. The trial enrolled 16 subjects, including eight patients with severe renal impairment matched with eight healthy control subjects with normal renal function. Each patient received a single, subcutaneous dose of 0.3 mg/kg of zilucoplan. The PK profile of zilucoplan was consistent across both groups, with exposures similar in renally-impaired patients and healthy volunteers. There were no adverse events reported. Overall, the data indicate that zilucoplan can be used in clinical studies of patients with renal impairment without any need for dose adjustment. Zilucoplan is currently being studied in a Phase 2 study in generalized myasthenia gravis with a top-line data read-out expected around year-end 2018. The Company also recently completed interactions with regulators, including the U.S. Food and Drug Administration, for its global Phase 3 program of zilucoplan for the treatment of paroxysmal nocturnal hemoglobinuria. Based on these interactions, Ra Pharma plans to initiate a global, pivotal, single-arm Phase 3 trial in treatment-naive PNH patients during the first half of 2019.
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