Ra Pharma Receives Orphan Drug Designation From The U.S. FDA For RA101495 For The Treatment Of Paroxysmal Nocturnal Hemoglobinuria

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases. RA101495, the Company’s lead clinical candidate, is a synthetic macrocyclic peptide inhibitor of complement component 5 (C5). The molecule is currently in Phase 2 clinical development as a self-administered subcutaneous (SC) injection for the treatment of PNH, a rare, chronic, life-threatening blood disorder where red blood cells are attacked and destroyed by the complement system.

“There is an urgent need for new treatment options for patients suffering from PNH. The current standard of care requires biweekly intravenous infusions, a dosing regimen that imposes a severe burden on patients, providers, and caregivers,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “We have designed RA101495 for once-daily, subcutaneous self-administration, an approach which has the potential to ease this burden, improve convenience, and provide much-needed dosing flexibility. We are encouraged by our initial Phase 2 data in PNH patients, which showed near-complete inhibition of hemolysis and a favorable safety and tolerability profile. We look forward to advancing our PNH program and providing additional data updates around year-end.”

The FDA grants Orphan Drug Designation status to products that treat rare diseases, providing incentives to sponsors developing drugs or biologics. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States at the time of designation. Orphan Drug Designation qualifies the sponsor certain development incentives, including tax credits for qualified clinical testing.

RA101495 received Orphan Drug Designation for the treatment of PNH in Europe in November 2016.

About RA101495

Ra Pharma is developing RA101495 for paroxysmal nocturnal hemoglobinuria (PNH), refractory generalized myasthenia gravis (rMG), and lupus nephritis (LN). The product is designed for convenient, once daily SC self-administration. RA101495 is a synthetic, macrocyclic peptide discovered using Ra Pharma’s powerful proprietary drug discovery technology. The peptide binds complement 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 also disrupts the interaction between C5b and C6 and prevents assembly of the membrane attack complex (MAC). This activity defines an additional, novel mechanism for the inhibition of C5 function. In Phase 1 studies, dosing of RA101495 was well tolerated in healthy volunteers and demonstrated sustained and near complete suppression of hemolysis and complement activity. To learn more about RA101495, please visit: http://rapharma.com/pipeline/ra101495/.

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. 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 initial data from the Company’s global Phase 2 clinical program evaluating RA101495 for the treatment of PNH may not be indicative of final study results; the risk that initial data from a limited number of patients may not be indicative of results from the fully patient enrollment planned for such study; 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, and 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.