Ra Pharmaceuticals, Inc. (NASDAQ:RARX) rose Monday after the company disclosed that its Phase 2 trial of zilucoplan met its primary endpoint and 'key' secondary endpoints.
The company, out of Cambridge, Mass., announced positive top-line results from the Company’s Phase 2 clinical trial evaluating zilucoplan for the treatment of generalized myasthenia gravis (gMG), achieving clinically meaningful and statistically significant reductions in both the primary and key secondary endpoints for both zilucoplan dose groups tested versus placebo at 12 weeks.
The Phase 2, multi-center, randomized, double-blind, placebo-controlled trial was designed to evaluate the safety, tolerability, and preliminary efficacy of zilucoplan in patients with gMG, regardless of prior therapies, who had a MGFA Disease Class of II-IVa at screening and a QMG score, a physician-administered assessment of MG-related muscle weakness, of ≥ 12 at screening and randomization.
The trial enrolled 44 patients in the U.S. and Canada. At the outset of the 12-week treatment period, patients were randomized in a 1:1:1 ratio to receive daily, SC doses of 0.1 mg/kg of zilucoplan, 0.3 mg/kg of zilucoplan, or matching placebo.
The pre-specified primary efficacy endpoint was the change in QMG score from baseline to week 12. The key secondary efficacy endpoint was the change in MG-ADL score, a patient-reported outcome measure, from baseline to week 12.
Significance testing was pre-specified at a 1-sided alpha of 0.1. All 44 patients completed the 12-week study and, of these, 43 (98%) elected to enter a long-term extension to receive active study drug.
Ra shares gained 26 cents, or 1.6%, to $16.72
