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Ra Pharmaceuticals Reports First Quarter 2018 Financial Results and Provides Corporate Update

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 9, 2018-- Ra Pharmaceuticals, Inc. (NASDAQ:RARX) today announced financial results for the first quarter ended March 31, 2018 and provided an update on recent corporate and clinical developments.

“We continue to make important progress in advancing our lead clinical candidate RA101495 SC in multiple, complement-mediated indications,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “While first generation treatments may address the basic mechanism of these diseases, the challenges of lifelong treatment access, convenience, and patient independence all contribute to restricting their broader use. RA101495 SC, a unique and potent C5 inhibitor differentiated by its convenient, once-daily, subcutaneous (SC) self-administration, has the potential to address these limitations, positioning it as a possible first-line therapy of choice.”

Dr. Treco added, “We are excited by the recent appointment of John C. King as Chief Commercial Officer, given his background in commercializing Soliris® for generalized myasthenia gravis (gMG) in the U.S. and growth of paroxysmal nocturnal hemoglobinuria (PNH) in markets around the world. We look forward to initiating a Phase 3 clinical trial in PNH in the second half of this year, and to completing enrollment in our Phase 2 clinical trial in gMG, followed by data in the first half of 2019.”

Recent Developments

  • Appointed John C. King as Chief Commercial Officer. Mr. King joined Ra Pharma in April 2018, bringing more than 20 years of senior, global, commercial leadership experience within the biotechnology rare disease space. Mr. King most recently served as Vice President, U.S. Neurology Business Unit at Alexion Pharmaceuticals, a role in which he was responsible for building and leading the team launching Soliris® (eculizumab) for gMG in the U.S. Prior roles include Vice President, Head of Global LAL-D Franchise and Vice President, Head of Global PNH Franchise, at Alexion, as well as commercial roles of increasing responsibility at Wyeth Pharmaceuticals.
  • Presented an overview of the Phase 2 clinical trial design evaluating RA101495 SC for the treatment of gMG at the American Academy of Neurology 70th Annual Meeting, April 21-27, 2018 in Los Angeles. 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 approximately 36 patients with gMG. The oral presentation also highlighted Phase 1 healthy volunteer data for RA101495 SC. To date, fourteen patients have been dosed in the Phase 2 clinical study, and data is expected in the first half of 2019.
  • Initiated dosing in the Company’s Phase 1b clinical trial evaluating RA101495 SC in patients with renal impairment. This trial is designed to characterize the pharmacokinetics (PK) of RA101495 SC in these patients, thereby enabling the evaluation of RA101495 SC in complement-mediated renal diseases, such as atypical hemolytic uremic disorder (aHUS) and lupus nephritis (LN). To date, eleven patients have been dosed in the Phase 1b study, and study results are expected mid-2018.

First Quarter 2018 Financial Results

For the first quarter of 2018, the Company reported a net loss of $16.5 million, or a net loss of $0.61 per share (basic and diluted), compared to net loss of $11.4 million, or a net loss of $0.50 per share for the same period in 2017.

Research and development expenses for the first quarter of 2018 were $13.4 million compared to $9.0 million for the same period in 2017. The increase in R&D expenses for the first quarter 2018 was related to clinical development costs associated with our lead program, RA101495 SC.

General and administrative expenses for the first quarter of 2018 were $3.3 million, compared to $2.5 million for the same period in 2017. The increase in G&A expenses for the first quarter of 2018 was related to employee-related costs due to the increase in G&A headcount to support the growth of the Company.

As of March 31, 2018, Ra Pharma reported total cash and equivalents of $109.1 million. The Company expects that its cash and cash equivalents at March 31, 2018 will be sufficient to fund operations through the end of 2019.

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 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 RA101495 SC Phase 2 gMG Clinical Program

The Phase 2, multicenter, 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 is designed to enroll approximately 36 patients and includes a screening period of up to four weeks. At the outset of the 12-week treatment period, patients will be 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 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:

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 SC, statements regarding trial design, timeline and enrollment of our ongoing and planned clinical programs, statements regarding the timing of the release of clinical trial data; statements regarding the potential of RA101495 SC to be a first-line therapy of choice, and expectations regarding the sufficiency of our cash and cash equivalents. 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; the risk that we may fail to obtain additional financing; the risk that we may fail to enroll patients in our clinical trials, which may cause delays or other adverse effects; the risk that our product candidates may have undesirable side effects that may delay or prevent their regulatory approval or limit their commercial success; 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.

Ra Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations
(in thousands, except per share data)

Three Months Ended
March 31,

2018 2017
Operating expenses:
Research and development $ 13,412 $ 9,012
General and administrative   3,312     2,469  
Total operating expenses   16,724     11,481  
Loss from operations (16,724 ) (11,481 )
Other income (expense), net   226     121  
Net loss $ (16,498 ) $ (11,360 )
Net loss per common share – basic and diluted $ (0.61 ) $ (0.50 )
Weighted average number of common shares outstanding – basic and diluted 27,242 22,549
Ra Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets
(In thousands)
March 31, 2018 December 31, 2017
Cash and cash equivalents $ 109,122 $ 70,381
Prepaid expenses and other current assets 1,736 2,496
Property and equipment, net 5,621 5,606
Other noncurrent assets   1,697   1,714
Total assets $ 118,176 $ 80,197
Liabilities and Stockholders’ Equity
Accounts payable and accrued expenses $ 6,765 $ 8,285
Deferred rent 450 329
Noncurrent liabilities 2,286 2,399
Stockholders' equity   108,675   69,184
Total liabilities and stockholders’ equity $ 118,176 $ 80,197

Source: Ra Pharmaceuticals, Inc.

Ra Pharmaceuticals, Inc.
Jennifer Robinson, 617-674-9873
Argot Partners
David Rosen, 212-600-1902