“This publication in JAMA Neurology recognizes the impact and significance of the findings from our Phase 2 clinical trial demonstrating zilucoplan’s potential in gMG, a chronic and debilitating neuromuscular disease that affects more than 60,000 patients in the U.S.,” said Doug Treco, Ph.D., President and Chief Executive Officer of Ra Pharma. “These results support our further evaluation of zilucoplan in the ongoing RAISE study, a pivotal Phase 3 trial of zilucoplan in gMG, with top-line results expected in early 2021. If successfully developed and approved, zilucoplan has the potential to expand access to convenient and effective complement inhibition therapy to a broad range of patients with gMG.”

As reported in the JAMA Neurology article, “Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients with Moderate to Severe Generalized Myasthenia Gravis: Results of a Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Clinical Trial,” the study demonstrated that zilucoplan resulted in rapid, clinically meaningful, statistically significant, and sustained improvements in the primary and key secondary endpoints, namely, change from baseline to week 12 in Quantitative Myasthenia Gravis (QMG) and MG Activities of Daily Living (MG-ADL) scores, respectively. Further, near-complete complement inhibition was associated with a faster onset and greater magnitude of benefit than submaximal complement inhibition, with a favorable safety and tolerability profile.

Ra Pharma is currently evaluating zilucoplan in the RAISE study, a single, global, pivotal, randomized, double-blind, placebo-controlled, Phase 3 clinical trial, for the treatment of gMG. The trial, which incorporates feedback from the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), is designed to evaluate the efficacy of a once-daily, subcutaneously (SC) self-administered dose of 0.3 mg/kg of zilucoplan versus placebo. Top-line results from the RAISE study are expected in early 2021.

About Zilucoplan Phase 2 gMG Clinical Trial

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 generalized myasthenia gravis (gMG), regardless of prior therapies, who had an MGFA Disease Class of II-IVa at screening and a Quantitative Myasthenia Gravis (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, subcutaneous (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 Activities of Daily Living (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, 42 (95%) entered a long-term extension to receive active study drug.

About gMG

Myasthenia gravis (MG) is a chronic, autoimmune, neuromuscular disease characterized by weakness and fatigue of skeletal muscles. Patients with MG present with muscle weakness that becomes increasingly severe with repeated use and recovers with rest. Weakness can be localized to specific muscles, such as those responsible for eye movements, but often progresses to affect a broader range, including head, limb, and respiratory muscles. This progression is often described as the generalized, or severe, form of the disease. gMG is estimated to affect approximately 60,000 people in the U.S. alone.

About Zilucoplan

Ra Pharma is developing zilucoplan and zilucoplan extended release (XR) for generalized myasthenia gravis (gMG), immune-mediated necrotizing myopathy (IMNM), amyotrophic lateral sclerosis (ALS), and other tissue-based complement-mediated disorders with high unmet medical need. The product candidate is designed for convenient subcutaneous (SC) self-administration. Zilucoplan is an investigational, synthetic, macrocyclic peptide discovered using Ra Pharma’s powerful proprietary drug discovery technology. The peptide is designed to bind complement component 5 (C5) with sub-nanomolar affinity and allosterically inhibit its cleavage into C5a and C5b upon activation of the classical, alternative, or lectin pathways. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to zilucoplan for the treatment of MG.

About Ra Pharmaceuticals, Inc.

Ra Pharmaceuticals is a clinical-stage biopharmaceutical company focused on leading the field of complement biology to bring innovative and accessible therapies to patients with rare diseases. The Company discovers and develops peptides and small molecules to target key components of the complement cascade. For more information, please visit: www.rapharma.com.