Ra Pharmaceuticals Initiates Dosing of RA101495 in PNH Patients

Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced that it has initiated dosing in the Company’s global Phase 2 clinical program evaluating RA101495 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). 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 and potent subcutaneously-administered (SC) inhibitor of complement component 5 (C5). Inhibition of C5 is a clinically validated approach for the control of complement-induced hemolysis, the process responsible for the rupture and destruction of red blood cells (RBCs) in patients with PNH.

“Commencement of dosing in our Phase 2 clinical program for RA101495 in PNH is an important step in bringing a potential new therapy to patients with this rare and life-threatening blood disorder,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “Building on our promising Phase 1 data, our Phase 2 program is designed to demonstrate RA101495’s safety and preliminary efficacy in PNH patients. RA101495 is a convenient, once daily, self-administered subcutaneous dose and represents a significant advancement in the treatment of PNH. We look forward to sharing initial data from our Phase 2 program in mid-2017 and additional data by year-end.”

The global Phase 2 program is designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of RA101495 in patients with PNH. The program is comprised of two open-label Phase 2 studies, one conducted outside the U.S. and one conducted in the U.S.

  • Outside of the U.S., the study will evaluate RA101495 in two cohorts. Cohort A is expected to include 8-12 eculizumab-naïve patients and Cohort B is expected to include 6-8 patients switching from eculizumab to RA101495.
  • The U.S. study will evaluate RA101495 in approximately 6-8 patients who are currently treated with eculizumab but have evidence of an inadequate response. An inadequate response is defined as a lactate dehydrogenase (LDH) level equal to or greater than 1.5-fold the upper limit of normal, indicating ongoing hemolysis. LDH is a measure of red blood cell hemolysis and a key disease biomarker.

All patients enrolled in the Phase 2 studies will receive a single, 0.3 mg/kg SC loading dose of RA101495, followed by 0.1 mg/kg SC daily thereafter. Following two weeks of treatment and based on a review of safety and efficacy data, patients will continue with 0.1 mg/kg daily or, if needed, the dose can be increased to 0.3 mg/kg daily to achieve adequate control of hemolysis. 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 LDH from baseline.

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 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 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; 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.


Ra Pharmaceuticals, Inc.
Jennifer Robinson, 617-674-9873
Argot Partners
Eliza Schleifstein, 917-763-8106

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