Ra Pharma To Host Conference Call On Initial Phase II Data For RA101495 In PNH

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that it will host a conference call and webcast on Tuesday, June 27, 2017 to discuss initial data from the Company’s global Phase 2 clinical program evaluating RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Early safety, tolerability, preliminary efficacy and pharmacodynamic data on the first two treatment naïve patients enrolled will be presented.

Ra Pharma is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases and is developing RA101495 as a novel, subcutaneously-administered (SC) inhibitor of complement component 5 (C5).

Details of the conference call and webcast are as follows:

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, once daily SC self-administration. RA101495 is a synthetic, macrocyclic peptide discovered using Ra Pharma’s powerful proprietary drug discovery technology. The peptide binds complement component 5 (C5) with sub-nanomolar affinity and allosterically inhibits its cleavage into C5a and C5b upon activation of the classical, alternative or lectin pathways. By binding to a region of C5 corresponding to C5b, RA101495 also disrupts the interaction between C5b and C6 and prevents assembly of the membrane attack complex (MAC). This activity defines an additional, novel mechanism for the inhibition of C5 function. In Phase 1 studies, dosing of RA101495 was well tolerated in healthy volunteers and demonstrated sustained and near complete suppression of hemolysis and complement activity. To learn more about RA101495, please visit: http://rapharma.com/pipeline/ra101495/.

About RA101495 Phase 2 Clinical Program

The global, dose-finding Phase 2 program is designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of RA101495 in patients with PNH. The study will evaluate RA101495 in three cohorts. Cohort A includes eculizumab-naïve patients, Cohort B includes patients switching from eculizumab to RA101495, and Cohort C includes patients who are currently treated with eculizumab but have evidence of an inadequate response. Patients in all three cohorts will be eligible for a long-term extension study following the completion of the initial 12-week studies. The primary efficacy endpoint is change in lactate dehydrogenase from baseline to the mean level from Week 6 to week 12.

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 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.