|Ra Pharmaceuticals Reports Second Quarter 2018 Financial Results and Announces Early Completion of Enrollment in gMG Phase 2 Program|
Surpassed target enrollment in Phase 2 trial of RA101495 SC for gMG
Top-line data now expected around year-end 2018
Regulatory progress on Phase 3 PNH program
“Enthusiasm from neurologists and their patients for a convenient,
self-administered, subcutaneous (SC) C5 inhibitor has resulted in the
rapid recruitment of 44 patients in our Phase 2 clinical trial of
RA101495 SC for the treatment of gMG, not only allowing us to complete
enrollment ahead of schedule, but also to surpass our original
enrollment target of 36 patients. We now expect to report top-line data
around year-end 2018. The acceleration of our gMG Phase 2 program
highlights RA101495 SC’s potential as a convenient and accessible
subcutaneous treatment option designed to deliver everyday complement
control to more patients with gMG,” said
Dr. Treco continued, “We are also pleased to report that we have made
significant progress in discussions with several regulatory authorities
regarding our planned global Phase 3 studies of RA101495 SC for the
treatment of PNH and, to date, have met with the Medicines and
Healthcare products Regulatory Agency in the
“We also completed dosing in our Phase 1b study designed to evaluate the pharmacokinetic (PK) profile of RA101495 SC in patients with renal impairment and expect to report top-line data by the end of the third quarter of 2018. We believe these data will allow us to proceed with the development of RA101495 SC in complement-mediated renal diseases, such as aHUS,” said Ramin Farzaneh-Far, MD, Chief Medical Officer of Ra Pharma.
Second Quarter 2018 Highlights and Recent Developments
Pending the successful outcome of these discussions, Ra Pharma anticipates initiating the Phase 3 clinical trials during the first half of 2019.
Second Quarter 2018 Financial Results
For the second quarter of 2018, the Company reported a net loss of
Research and development (R&D) expenses for the second quarter of 2018
General and administrative (G&A) expenses for the second quarter of 2018
About RA101495 SC
Ra Pharma is developing RA101495 SC for paroxysmal nocturnal hemoglobinuria (PNH), generalized myasthenia gravis (gMG), atypical hemolytic uremic syndrome (aHUS), and lupus nephritis (LN). The product is designed for convenient, once-daily subcutaneous self-administration. RA101495 SC 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 SC is designed to disrupt the interaction between C5b and C6 and prevent assembly of the membrane attack complex (MAC). This activity may define an additional, novel mechanism for the inhibition of C5 function.
About RA101495 SC Phase 2 gMG Clinical Program
The Phase 2, multi-center, randomized, double-blind, placebo-controlled trial is designed to evaluate the safety, tolerability, and preliminary efficacy of RA101495 SC in patients with gMG. The trial has enrolled  patients and will include a screening period of up to four weeks. At the outset of the 12-week treatment period, patients were randomized in a 1:1:1 ratio and will receive daily, subcutaneous doses of 0.1 mg/kg of RA101495 SC, 0.3 mg/kg of RA101495 SC, or matching placebo. The primary efficacy endpoint is change in Quantitative Myasthenia Gravis (QMG) score from baseline to week 12. All patients will have the opportunity to receive RA101495 SC in a long-term extension study.
About RA101495 SC Phase 2 PNH Clinical Program
The global, dose-finding Phase 2 program was designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of RA101495 SC in patients with PNH. The study evaluated RA101495 SC in three cohorts. The first cohort included eculizumab-naïve patients, the second cohort included patients switching from eculizumab to RA101495 SC, and the third cohort included patients who were currently treated with eculizumab but had evidence of an inadequate response. Patients in all three cohorts were eligible for entry into a long-term extension study following the completion of the initial 12-week studies. The primary efficacy endpoint was the change in LDH 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.
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 potential safety, efficacy and regulatory and clinical progress of our product candidates, including without limitation RA101495 SC, planned meetings with regulatory authorities, statements regarding trial design, timeline and enrollment of our ongoing and planned clinical programs, including without limitation our Phase 3 studies of RA101495 SC for the treatment of PNH, and anticipated timelines for the release of clinical data . 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 SC, will not successfully be developed or commercialized, in the timeframe we expect or at all; the risk that topline results as of February 7, 2017 from the Company's global Phase 2 clinical program evaluating RA101495 SC for the treatment of PNH may not be indicative of final study results; 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.