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Ra Pharmaceuticals to Present Design of RA101495 SC Phase 2 Clinical Trial in gMG at the 70th Annual AAN Meeting

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 1, 2018-- Ra Pharmaceuticals, Inc. (NASDAQ:RARX), a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases, today announced that the design of its Phase 2 clinical trial of RA101495 SC for the treatment of generalized myasthenia gravis (gMG) will be presented in an oral platform presentation at the 70th Annual American Academy of Neurology (AAN) Meeting, taking place April 21-27, 2018 in Los Angeles. The study will be presented along with previously detailed Phase 1 clinical trial results evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of RA101495 SC in healthy volunteers.

In December 2017, Ra Pharma announced that it had commenced dosing in the Phase 2 trial of RA101495 SC in gMG. gMG is a rare, complement-mediated, autoimmune disease that causes weakness in skeletal muscles. The multicenter, randomized, double-blind, placebo-controlled trial is designed to evaluate the safety, tolerability, and preliminary efficacy of RA101495 SC in approximately 36 patients with gMG over a 12-week treatment period. All patients completing the 12-week study will have the opportunity to receive RA101495 SC in a long-term extension study. Ra Pharma is developing RA101495 as a novel, subcutaneously-administered (SC) inhibitor of complement component 5 (C5) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), gMG, and severe, debilitating renal diseases, such as atypical hemolytic uremic syndrome (aHUS) and lupus nephritis (LN).

“gMG is a disabling, chronic, autoimmune, neuromuscular disease that presents a significant and lifelong burden on patients’ quality of life. There is high unmet need for effective, disease-modifying treatment options,” said Ramin Farzaneh-Far, M.D., Chief Medical Officer of Ra Pharma. “RA101495 SC potently inhibits C5, a validated therapeutic target in myasthenia, and offers a convenient, once-daily, subcutaneous, self-administration regimen that has the potential to benefit a broad population of gMG patients. We continue to work toward rapid enrollment of this Phase 2 trial, with the goal of reporting top-line data in the first half of 2019, and to advance RA101495 SC into Phase 3 in PNH in the second half of 2018.”

Details of the presentation are as follows:

Title: RA101495, A Subcutaneously Administered Peptide Inhibitor of Complement Component 5 (C5) for the Treatment of Generalized Myasthenia Gravis (gMG): Phase 1 Results and Phase 2 Design
Session Title: S31: Therapeutics, Phenotypes, and Biomarkers in Neuromuscular Disorders
Presenter: James F. Howard Jr., M.D., Department of Neurology, University of North Carolina, Chapel Hill
Date/Time: Wednesday, April 25, 2:00 PM PT
Location: Platform Presentation Number 006
Abstract Number: 2035

About RA101495 SC

Ra Pharma is developing RA101495 SC for paroxysmal nocturnal hemoglobinuria (PNH)generalized myasthenia gravis (gMG), atypical hemolytic uremic syndrome (aHUS), and lupus nephritis (LN). The product is designed for convenient, once-daily subcutaneous self-administration. RA101495 SC is a synthetic, macrocyclic peptide discovered using Ra Pharma's powerful proprietary drug discovery technology. The peptide binds complement component 5 (C5) with sub-nanomolar affinity and allosterically inhibits its cleavage into C5a and C5b upon activation of the classical, alternative, or lectin pathways. By binding to a region of C5 corresponding to C5b, RA101495 SC is designed to disrupt the interaction between C5b and C6 and prevent assembly of the membrane attack complex (MAC). This activity may define an additional, novel mechanism for the inhibition of C5 function.

About Ra Pharmaceuticals

Ra Pharmaceuticals is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for complement-mediated diseases. The Company discovers and develops peptides and small molecules to target key components of the complement cascade. For more information, please visit:

Forward-Looking Statement

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 the safety, efficacy and regulatory and clinical progress of our product candidates, including RA101495, and statements regarding trial design, timeline and enrollment of our ongoing and planned clinical programs . 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 RA101495, will not successfully be developed or commercialized; the risk that topline results as of February 7, 2017 from the Company's global Phase 2 clinical program evaluating RA101495 for the treatment of PNH may not be indicative of final study results; 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 Securities and Exchange Commission . There can be no assurance that the actual results or developments anticipated by Ra Pharma will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Ra Pharma. All information in this press release is as of the date of the release, and Ra Pharma undertakes no duty to update this information unless required by law.

Source: Ra Pharmaceuticals, Inc.

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