Ra Pharma said topline data from the mid-stage trial of Zilucoplan in patients with generalized myasthenia gravis shows that the study achieved clinically and statistically significant reductions in both primary and key secondary endpoints.
The Phase 2 trial enrolled 44 patients in the U.S. and Canada, and for the 12-week treatment period, patients were randomized in a 1-1-1 ratio to receive subcutaneous doses of 0.1 mg/kg of Zilucoplan, 0.3 mg/kg of Zilucoplan or a matching placebo.
The pipeline candidate also had a favorable safety and tolerability profile, consistent with previously completed Phase 1 and Phase 2 studies, the company said.
Why It's Important
Myasthenia gravis is a chronic autoimmune neuromuscular disease that manifests as weakness and fatigue of skeletal muscles. It's estimated to affect about 60,000 people in the U.S. alone, according to Ra Pharma.
Ra Pharma said it will meet with regulators to review the Phase 2 data and the design of its planned Phase 3 study.
The company plans to present full data from the trial and additional data from the open-label, long-term extension in association with an upcoming major medical meeting.
Ra Pharma shares were rallying 14.4 percent to $18.83 at the time of publication Monday.
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