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Ra Pharma Announces Completion of End-of-Phase 2 Interactions with FDA and Design of Phase 3 PNH Program

Plans to initiate a global, pivotal, single-arm Phase 3 trial in treatment-naïve PNH patients

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 5, 2018-- Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced the completion of End-of-Phase 2 interactions with the U.S. Food and Drug Administration (FDA) for its global Phase 3 program of RA101495 SC for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Based on feedback provided by FDA during End-of-Phase 2 discussions regarding the Company’s planned global Phase 3 PNH program, as well as advice previously provided by the Medicines and Healthcare products Regulatory Agency in the United Kingdom (MHRA) and Health Canada, Ra Pharma plans to initiate a global, pivotal, single-arm Phase 3 trial to evaluate the safety and efficacy of RA101495 SC in approximately 40 treatment-naïve PNH patients. The co-primary endpoints will be hemoglobin stabilization and reduction in lactate dehydrogenase (LDH) levels from baseline.

Ra Pharma anticipates the data from the planned global Phase 3 registrational trials, if positive, will serve as the basis to support submission of a New Drug Application (NDA) for RA101495 SC for the treatment of PNH. Ra Pharma also plans to meet with the European Medicines Agency (EMA) in the fourth quarter of 2018.

In addition, Ra Pharma expects to initiate a supportive trial in approximately 40 transfusion-independent patients switching from eculizumab to RA101495 SC. The primary endpoint for this study will be maintenance of transfusion-independence after switching.

Furthermore, in a separate CMC Type C meeting, Ra Pharma has also reached agreement with the FDA on the pharmaceutical development plans for RA101495 SC required to support the Phase 3 program.

“With feedback from FDA, MHRA, and Health Canada now incorporated into the design of our global Phase 3 PNH program, we’re looking forward to meeting with the EMA in the fourth quarter of 2018 and, pending the successful outcome of those discussions, anticipate initiating our Phase 3 clinical trials during the first half of 2019,” said Doug Treco, PhD, Chief Executive Officer of Ra Pharma. “Our recent regulatory progress brings us one step closer to being able to provide a more convenient and readily accessible C5 inhibitor to patients with PNH, generalized myasthenia gravis (gMG), and other complement-mediated disorders.”

About RA101495 SC

Ra Pharma is developing RA101495 SC for paroxysmal nocturnal hemoglobinuria (PNH)generalized myasthenia gravis (gMG), and other complement-mediated disorders. 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 potential safety, efficacy and regulatory and clinical progress of our product candidates, including without limitation RA101495 SC, planned meetings with regulatory authorities, statements regarding trial design, timeline and enrollment of our ongoing and planned clinical programs, including without limitation our Phase 3 studies of RA101495 SC for the treatment of PNH, and our expectation that Phase 3 data, if positive, will serve as the basis to support submission of an NDA. 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 SC, will not successfully be developed or commercialized, in the timeframe we expect or at all; the risk that topline results as of February 7, 2017 from the Company's global Phase 2 clinical program evaluating RA101495 SC 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.

Argot Partners
Natalie Wildenradt, 212-600-1902
David Rosen, 212-600-1902