Ra Pharmaceuticals Announces Expansion of Neuromuscular Portfolio with Clearance of IND Application for Zilucoplan for the Treatment of Immune-Mediated Necrotizing Myopathy
Multi-center Phase 2 clinical trial on track to initiate in the second half of 2019
“Patients with IMNM face a severe, chronic, debilitating autoimmune disease with limited treatment options and no approved therapies,” said
IMNM is an autoimmune myopathy characterized by skeletal muscle necrosis, severe proximal limb weakness, and elevated creatine kinase (CK) levels. IMNM is categorized into two subtypes defined by the presence of distinct autoantibodies against 3-hydroxy-3-methylglutaryl-coenzyme A reductase (HMGCR) or signal recognition particle (SRP). In IMNM, these autoantibodies drive complement-mediated necrosis of muscle fibers, resulting in severe, progressive, and debilitating proximal muscle weakness. IMNM affects more than 15,000 patients in
The randomized, double-blind, placebo-controlled, multi-center, Phase 2 trial will evaluate the safety, tolerability, and efficacy of zilucoplan in patients with IMNM who are positive for anti-HMGCR or anti-SRP autoantibodies. Patients will be randomized in a 1:1 ratio to receive daily subcutaneous (SC) doses of 0.3 mg/kg zilucoplan or placebo. Randomization will be stratified based on antibody status (anti-HMGCR+ versus anti-SRP+). The trial is expected to enroll approximately 24 patients with an eight-week treatment period. The primary endpoint will be change in CK from baseline to week eight. Following completion of the trial, patients will have the option to enter into an open-label long-term extension study with zilucoplan.
“The IMNM program represents a meaningful expansion of our neuromuscular pipeline for zilucoplan, as we aim to leverage the unique properties of a small peptide in tissue-based complement-mediated disorders and build on the success of our Phase 2 trial for zilucoplan in gMG,” said
Analyst and Investor Event
Ra Pharma will host an analyst and investor event on
Ra Pharma is developing zilucoplan and zilucoplan extended release (XR) for generalized myasthenia gravis (gMG), immune-mediated necrotizing myopathy (IMNM), 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.
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Ra Pharma's ability to expand patient access to important therapies, the potential, safety, efficacy, and regulatory and clinical progress of Ra Pharma's product candidates, including without limitation zilucoplan and our zilucoplan XR program, beliefs regarding clinical trial data, statements regarding trial design, timeline, and enrollment of Ra Pharma's ongoing and planned clinical programs, including without limitation the Phase 2 trial of zilucoplan for the treatment of IMNM and long-term extension study, and plans to host an analyst and investor event. All such forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Ra Pharma's product candidates, including zilucoplan, will not successfully be developed or commercialized, in the timeframe we expect or at all; as well as the other factors discussed in the “Risk Factors” section in Ra Pharma’s most recently filed Annual Report on Form 10-K, as well as other risks detailed in Ra Pharma’s subsequent filings with the
Ra Pharmaceuticals, Inc.
Natalie Wildenradt, 617-674-9874
David Rosen, 212-600-1902