Ra Pharmaceuticals 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, MA, USA I July 19, 2017 I 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).

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.

SOURCE: Ra Pharmaceuticals

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