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Ra Pharmaceuticals Announces Presentation of RA101495 SC Phase 2 gMG Clinical Trial Design at the 70th Annual AAN Meeting

Phase 2 gMG Patient Enrollment on Target

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apr. 25, 2018-- Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that the design of its Phase 2 clinical trial of RA101495 SC as a treatment for generalized myasthenia gravis (gMG) will be presented in an oral platform presentation at the 70th Annual American Academy of Neurology (AAN), taking place April 21-27, 2018 in Los Angeles.

“Despite current treatments, patients with gMG suffer from the progressive and debilitating burden of complement-mediated neuromuscular damage,” said James F. Howard, MD, Distinguished Professor of Neuromuscular Disease, Department of Neurology, University of North Carolina School of Medicine. “RA101495 SC is a unique and potent C5 inhibitor differentiated by convenient, once-daily, subcutaneous (SC) self-administration. I am encouraged by the rapid rate of patient enrollment in this Phase 2 clinical trial and look forward to its outcome.”

Initiated in December 2017, the Phase 2, 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. At the outset of a 12-week treatment period, patients are randomized in a 1:1:1 ratio and receive daily, SC doses of 0.1 mg/kg of RA101495, 0.3 mg/kg of RA101495, or matching placebo. The primary efficacy endpoint is change in Quantitative Myasthenia Gravis (QMG) score from baseline to week 12. All patients will have the opportunity to receive RA101495 SC in a long-term extension study.

To date, 13 patients have been dosed in the trial. The Company remains on track to report data from the study in the first half of 2019.

“We are motivated by patient and investigator enthusiasm for RA101495 SC in gMG,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “We continue to make important progress toward our goal of delivering more convenient and accessible treatment options to patients living with complement-mediated diseases.”

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: www.rapharma.com.

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, statements regarding trial design, timeline and enrollment of our ongoing and planned clinical programs.; statements regarding the timing of the release of clinical trial data; and statements regarding our goal of delivering more convenient and accessible treatment options to patients 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 risk that Ra Pharma's product candidates, including RA101495, will not successfully be developed or commercialized; the risk that we may fail to obtain additional financing; the risk that we may fail to enroll patients in our clinical trials, which may cause delays or other adverse effects; the risk that our product candidates may have undesirable side effects that may delay or prevent their regulatory approval or limit their commercial success; and 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.

Investors:
Ra Pharmaceuticals, Inc.
Jennifer Robinson, 617-674-9873
jrobinson@rapharma.com
or
Media:
Argot Partners
David Rosen, 212-600-1902
david.rosen@argotpartners.com