Ra Pharmaceuticals Presents Data on Oral Small Molecule Complement Inhibitors at the 22nd Congress of the European Hematology Association

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 the presentation of scientific data at the 22nd Congress of the European Hematology Association (EHA), June 22-25, 2017 in Madrid, Spain.

“The data presented at EHA showcase a novel class of orally bioavailable small molecules that bind to C5 with high affinity and inhibit its cleavage into C5a and C5b,” said Alonso Ricardo, Senior Vice President and Head of Research at Ra Pharma. “These findings demonstrate the feasibility of an orally-administered therapy for complement-mediated disorders and support the continued advancement of these molecules.”

Inhibition of C5 is a clinically-validated approach for the therapeutic treatment of complement-mediated disorders. Ra Pharma’s portfolio includes drug candidates for several such disorders, including paroxysmal nocturnal hemoglobinuria (PNH), myasthenia gravis (MG) and lupus nephritis (LN). The Company is pursuing the identification of orally-available inhibitors of C5 with the goal of providing additional options for patients diagnosed with these and other complement-mediated disorders.

“We are encouraged by our scientists’ ability to discover and characterize a small molecule inhibitor of complement C5,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “These data show that C5 inhibition can be accomplished with an orally-bioavailable small molecule, and we hope that compounds from this class can be advanced to provide a new treatment option for a broader spectrum of patients.”

The presentation is available at http://rapharma.com/science/presentations-and-publications/. A summary of the data presented by Ra Pharma at the conference is as follows:

Title: Discovery of Orally Bioavailable Small Molecules for Inhibition of Complement C5
Session Title: Bone marrow failure and PNH (oral presentation)
Presenter: Alonso Ricardo, PhD, Senior Vice President and Head of Research, Ra Pharma
Date/Time: Saturday, June 24, 5:00- 5:15 PM CEST
Location: Room N104
Abstract Code: S500
Summary: These initial studies describe a series of first-in-class, orally bioavailable small molecules that bind to C5, inhibiting the protein’s cleavage into C5a and C5b. These molecules possess favorable pharmacokinetic properties, exhibiting up to 50% oral availability in preclinical species. Molecules from this class were found to bind to a unique site on complement C5 with a 1:1 stoichiometry. Notably, binding to this site also inhibited the cleavage of C5 containing the R885H polymorphism, a mutation that confers resistance to eculizumab, today’s standard of care in PNH. The extensive in vitro and in vivo data, along with a high-resolution crystal structure, support the feasibility of developing alternatives to injectable treatment of disorders of complement regulation.

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 the results of preclinical studies may not be indicative of the results of clinical studies; 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.


Ra Pharmaceuticals, Inc.
Jennifer Robinson, 617-674-9873
Argot Partners
Eliza Schleifstein, 917-763-8106

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