Ra Pharmaceuticals to Present Overview of RA101495 and Oral C5 Inhibitor at the International PNH Interest Group Annual Scientific Assembly

Ra Pharmaceuticals (NASDAQ:RARX), a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for diseases of complement dysregulation, today announced that the Company will present an overview of its lead product candidate, RA101495, a potent subcutaneously-administered macrocyclic peptide inhibitor of C5, and an update on its oral small molecule C5 inhibitor program, at the International PNH Interest Group Annual Scientific Assembly on Friday, December 2, 2016 in San Diego, CA. The presentation will include a summary of Phase 1 study results and a description of the planned Phase 2 study design for RA101495 in paroxysmal nocturnal hemoglobinuria (PNH) patients, as well as new pre-clinical data from Ra’s oral small molecule C5 inhibitor program. A copy of the presentation slides will be made available on the News and Investors section of the Company’s website, www.rapharma.com, at the start of the meeting.

“Our Phase 1 study in healthy volunteers demonstrated that RA101495 is a potent inhibitor of C5, provides rapid, complete and sustained inhibition of hemolysis, was well tolerated and can be conveniently and effectively delivered by subcutaneous injection,” said Doug Treco, PhD, Co-Founder, President and CEO of Ra Pharma. “We are eager to initiate our Phase 2 program in PNH patients in the first quarter of 2017, and we also look forward to advancing our oral small molecule C5 inhibitor program through early development and toward an IND. PNH is a rare and life threatening blood disorder for which new treatment options are needed. We believe RA101495, as well as molecules discovered through our oral small molecule C5 inhibitor program, represent attractive alternatives to monoclonal antibody-based treatment options, which require regular intravenous infusions.”

Date: Friday, December 2, 2016
Time: 6:00 p.m. PT
Location: Manchester Grand Hyatt, San Diego

For more information about the IPIG Annual Scientific Assembly, visit:

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, subcutaneous self-administration. RA101495 is a synthetic, macrocyclic peptide discovered using Ra’s Proprietary Extreme Diversity™ platform. The peptide binds complement C5 with subnanomolar affinity and allosterically inhibits its cleavage into C5a and C5b upon activation of the classical, alternative or lectin pathways. RA101495 also directly binds to C5b, disrupting the interaction between C5b and C6 and preventing assembly of the membrane attack complex (MAC). This activity defines an additional and novel mechanism for the inhibition of C5 function. Dosing of RA101495 was well tolerated in healthy volunteers and demonstrated sustained and near complete suppression of hemolysis and complement activity.

About the Extreme Diversity™ Platform

Ra Pharma’s proprietary Extreme Diversity™ platform allows us to produce synthetic macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small molecules. We utilize a process called "mRNA display" to produce extremely large and diverse libraries of peptides from which to select potential product candidates. The platform generates highly specific and stable peptide-like molecules with the potential for greatly increased bioavailability, improved cell permeability, and the opportunity to address protein-protein interactions including previously undruggable targets. It can produce libraries of 10 to 100 trillion members, allowing for the rapid discovery of highly potent candidate molecules.

About Ra Pharmaceuticals

Ra Pharmaceuticals is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for diseases of complement dysregulation and other orphan indications. 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; as well as the other factors discussed in the “Risk Factors” and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections in Ra Pharma’s Registration Statement on Form S-1 (File No. 333-214242), as amended, which is on file with the Securities and Exchange Commission and was declared effective on October 25, 2016, 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.


Argot Partners
Susan Kim, 212-600-1902
Eliza Schleifstein, 917-763-8106

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