Ra Pharmaceuticals Announces Presentation of Data Supporting Clinical Development of Novel Complement C5 Inhibitor at 2014 ASH Annual Meeting

--Prepares for Novel Peptide Inhibitor to Enter Clinical Evaluation for Paroxysmal Nocturnal Hemoglobinuria--

--Debasish Roychowdhury, M.D., Appointed as Acting Chief Medical Officer--

ASH 2014

CAMBRIDGE, Mass.--()--Ra Pharmaceuticals today announced it will present preclinical data on its novel subcutaneous complement C5 inhibitor, RA101348, at the 56th American Society of Hematology Annual Meeting and Exposition (ASH), taking place December 6-9, 2014. The data presented demonstrate potent C5 inhibition suitable for a broad range of complement-mediated diseases, including paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare acquired hematologic syndrome in which red blood cells are destroyed by the complement system.

“The preclinical data presented at ASH support my view that RA101348 has the potential to provide patients with PNH a more convenient treatment option without compromising efficacy. Further, the mechanism of action of RA101348 opens the door to significant expansion possibilities in other diseases, including many orphan indications.”

“Based on the strength of the RA101348 preclinical data, which demonstrate strong inhibition of C5 and associated hemolysis, along with the fact that PNH offers a well-validated and potentially rapid clinical proof-of-concept for C5 inhibition, I believe that RA101348 is a highly promising candidate for subcutaneous dosing in patients with PNH,” said Doug Treco, Ph.D., President and CEO, Ra Pharma. “With these data in hand, we plan to enter Phase 1 clinical development of RA101348 in early 2015.”

Poster #2936 (to be presented on Sunday December 7) will present in vitro characterization studies and in vivo data in non-human primates. Inhibition of in vivo complement activity was evaluated in non-human primates following single- and multi-dose subcutaneous (SC) administration. RA101348 exhibited high SC bioavailability, and low, single doses inhibited complement activity by 90-100%. Repeat dosing for 7 days was well-tolerated in non-human primates at up to 100-fold above projected human therapeutic doses and resulted in sustained inhibition of complement activity. In addition, an oral presentation on Sunday, December 7 will show that a closely related molecule discovered by Ra reduces complement activation, consumptive coagulopathy, microthrombosis and cytokine production, and protects animals against the sepsis-induced effects on blood pressure and temperature in a baboon model of E. coli-induced sepsis. Notably, organ damage and mortality were dramatically reduced, with no diminution of bacterial clearance.

In addition, Ra Pharma has appointed Debasish Roychowdhury, M.D., as Acting Chief Medical Officer. Dr. Roychowdhury was most recently Acting CMO at Seragon Pharmaceuticals and is President of Nirvan Consultants, LLC.

“I am excited to be a part of Ra. The Company’s Extreme Diversity™ platform has great promise for identifying novel compounds for difficult biological problems and the ability to build compounds with optimized bioavailability, permeability, stability and potency,” commented Dr. Roychowdhury. “The preclinical data presented at ASH support my view that RA101348 has the potential to provide patients with PNH a more convenient treatment option without compromising efficacy. Further, the mechanism of action of RA101348 opens the door to significant expansion possibilities in other diseases, including many orphan indications.”

Dr. Roychowdhury, a hematologist/oncologist, has over 25 years of combined experience in the pharmaceutical industry and patient care. Prior to Seragon, he was Senior Vice President and Head, Global Oncology Division at Sanofi. He previously served in several positions of increasing responsibility at GlaxoSmithKline plc and at Eli Lilly and Company. Prior to his roles in industry, Dr. Roychowdhury served as a faculty member at the University of Cincinnati where he directed a number of clinical programs.

Details on the presentations at ASH are as follows:

Abstract Title: Development of RA101348, a Potent Cyclic Peptide Inhibitor of C5 for Complement-Mediated Diseases
Session: 508. Bone Marrow Failure: Poster II (#2936)
Date: Sunday, December 7, 2014
Time: 6:00-8:00 pm PT
Location: West Building, Level 1, Moscone Center

Abstract Title: A Novel C5 Complement Inhibitor Protects Against Sepsis-Induced Activation of Complement, Coagulation and Inflammation and Provides Survival Benefit in E. coli Sepsis
Session: 331. Pathophysiology of Thrombosis: Novel mechanisms in thrombosis
Date: Sunday, December 7, 2014
Time: 5:15 pm PT (Oral presentation)
Location: West Building, 2006-2009, Moscone Center

About the Extreme Diversity™ Platform

The Company’s proprietary Extreme Diversity™ platform generates highly specific and stable peptide-like molecules with improved cell permeability, the potential for greatly increased bioavailability, and the opportunity to address protein-protein interactions and other previously undruggable targets. The platform is unique in that it combines in vitro display technology with a wide variety of non-natural amino acids. Unlike certain other display technologies, in vitro display does not require the use of a bacterial or yeast host, and it can produce libraries of 10 to 100 trillion members, allowing for the rapid discovery of optimized candidates.

About Ra Pharmaceuticals

Ra Pharma™ is developing a new drug class with the diversity and specificity of antibodies, coupled with the many benefits of small molecules. Ra Pharma’s peptide-like molecules are highly-stable, synthetic products with chemical structures that offer intrinsic cell permeability. Ra Pharma is leveraging its ability to discover and develop its own portfolio of products in oncology and select rare diseases. Ra is also engaged in industry partnerships to rapidly generate drug candidates for broader indications and has developed a strong intellectual property position around its unique capabilities. For more information, please visit: www.rapharma.com.

Contacts

MacDougall Biomedical Communications
Michelle Avery, 781-235-3060
mavery@macbiocom.com

Release Summary

Ra Pharmaceuticals unveils subcutaneous complement C5 inhibitor program at ASH 2014. Phase 1 clinical studies are expected in early 2015. In addition, Ra appoints Debasish Roychowdhury as Acting CMO.

Ra Pharmaceuticals