Ra Pharma Announces Publication Of Positive Preclinical Data With RA101295 For The Treatment Of Escherichia Coli Sepsis

In Vitro and In Vivo Data Demonstrate Protection Against Mortality and Multi-Organ Failure

CAMBRIDGE, Mass.--(BUSINESS WIRE)--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 publication of in vitro and in vivo data demonstrating that treatment of Escherichia coli sepsis with RA101295 was associated with significant organ protection and markedly reduced mortality. The article, “Inhibition of Complement C5 Protects Against Organ Failure and Reduces Mortality in a Baboon Model of Escherichia Coli Sepsis” (doi: 10.1073/pnas.1706818114) was published online in the Proceedings of the National Academy of Sciences (PNAS).

“These data indicate that C5 inhibition could play an important role in the treatment of bacterial sepsis and other acute systemic inflammatory conditions, as well as providing further validation for the use of Ra Pharma’s synthetic macrocyclic peptides for the treatment of complement-mediated diseases”

Researchers evaluated the effect of RA101295, a macrocyclic peptide inhibitor of complement component 5 (C5), in baboons receiving lethal doses of E. coli. As compared with placebo, treatment with RA101295 blocked sepsis-induced inflammation, preserved endothelial cell function, decreased leukocyte activation, and reduced overall mortality, while exhibiting no detrimental effects on the clearance of bacteria.

“These data indicate that C5 inhibition could play an important role in the treatment of bacterial sepsis and other acute systemic inflammatory conditions, as well as providing further validation for the use of Ra Pharma’s synthetic macrocyclic peptides for the treatment of complement-mediated diseases,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “While we continue to advance our lead candidate, RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), through its Phase 2 clinical program, we remain committed to exploring the versatility of our proprietary compounds in acute and chronic conditions arising from dysregulation of the complement system.”

Inhibition of C5 is a clinically-validated approach for the treatment of complement-mediated disorders. Ra Pharma’s portfolio includes drug candidates for several such disorders, including PNH, myasthenia gravis (MG) and lupus nephritis (LN).

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, pre-clinical and clinical progress of our product candidates, including RA101295 and 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 RA101295 and 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, 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.