Ra's zilucoplan meets in Phase II for generalized myasthenia gravis, company raises $130M

Ra Pharmaceuticals Inc. (NASDAQ:RARX) said on Dec. 10 that zilucoplan (formerly RA101495 SC) met the primary and key secondary endpoints in a Phase II trial to treat generalized myasthenia gravis. The company plans to meet with regulatory authorities, including FDA, in 1H19 to discuss the design of a Phase III trial to evaluate a 0.3 mg/kg dose of zilucoplan in the indication.

The double-blind, U.S. and Canadian trial enrolled 44 patients to receive placebo or once-daily 0.1 or 0.3 mg/kg subcutaneous doses of the peptide inhibitor of complement C5 (C5) for 12 weeks. On the primary endpoint, both doses of zilucoplan significantly improved Quantitative Myasthenia Gravis (QMG) score from baseline to week 12 vs. placebo (placebo-adjusted improvement of 2.8 points for high-dose zilucoplan, p=0.05). Significance in the trial was prespecified at a one-sided p-value of 0.1. Both doses of zilucoplan also met the secondary endpoint of improving Myasthenia Gravis-Activities of Daily Living (MG-ADL) score from baseline to week 12 vs. placebo (placebo-adjusted improvement of 2.3 points for high-dose zilucoplan, p=0.04). The company plans to present full data at a medical meeting...