Ra Pharmaceuticals Reports Third Quarter 2017 Financial Results and Provides Corporate Update

Ra Pharmaceuticals, Inc. (NASDAQ:RARX), a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for the treatment of complement-mediated diseases, today announced financial results for the third quarter ended September 30, 2017, and provided an update on recent corporate and clinical developments, including enrollment in the Phase 2 program for RA101495 SC in paroxysmal nocturnal hemoglobinuria (PNH) and progress toward the initiation of a Phase 2 study in generalized myasthenia gravis (gMG).

“Enthusiasm for a convenient, subcutaneous (SC), self-administered C5 inhibitor continues to be reflected in the rapid recruitment of our Phase 2 PNH study, with 28 patients enrolled to date,” said Doug Treco, PhD, President and Chief Executive Officer of Ra Pharma. “We look forward to reporting data from this program around the end of the year, initiating our Phase 2 study in gMG, and expanding our RA101495 SC clinical program by initiating a Phase 1b renal impairment study to support clinical trials in atypical hemolytic uremic syndrome and lupus nephritis. We believe RA101495 SC holds the potential to become an important and convenient treatment option accessible to a broad spectrum of patients across a number of complement-mediated diseases.”

Recent Developments

  • Progressed enrollment in Phase 2 clinical program evaluating RA101495 SC in PNH, with 28 patients enrolled to date. A total of 27 patients have been dosed with RA101495 SC, including 10 patients in Cohort A (eculizumab-naïve patients), 16 patients in Cohort B (patients switching from eculizumab to RA101495 SC), and one patient in the third cohort (inadequate responders to eculizumab). The Company remains on track to report additional data around the end of the year.
  • Opened an Investigational New Drug (IND) application with the US Food and Drug Administration and activated sites in preparation for the Company’s Phase 2 trial evaluating RA101495 SC for the treatment of gMG. gMG is a complement-mediated autoimmune disease that causes muscle weakness and reduced mobility. Convenient, self-administered RA101495 SC has the potential to address a broader gMG patient population than is practical with intravenous infusions. This target population includes, but is not limited to, patients who have failed multiple immunosuppressants. Ra Pharma anticipates dosing the first patient in this Phase 2 trial in the fourth quarter of this year.
  • Strengthened senior management team with the addition of Steve Caffé, MD, Senior Vice President, Regulatory Affairs and Quality. Dr. Caffé brings significant expertise in the areas of pharmacovigilance and regulatory affairs, having served in senior leadership roles at Sucampo Pharmaceuticals, MedImmune LLC (the global biologics arm of AstraZeneca), Baxter International, Aventis/Sanofi, and Merck & Co. Dr. Caffé obtained his doctorate medical degree from Université Pierre et Marie Curie (Paris 6), Faculté de Médecine Saint-Antoine.
  • Presented data at medical conferences, including the European Meeting on Complement in Human Diseases, the Muscle Study Group, the Macrocyclic and Constrained Peptides Conference, and the Boulder Peptide Symposium. Data presented includes Phase 1 and initial Phase 2 data demonstrating RA101495 SC’s complement C5 inhibition.

Third Quarter 2017 Financial Results

For the third quarter of 2017, the Company reported a net loss of $15.3 million, or a net loss of $0.68 per share (basic and diluted), compared to a net loss of $8.1 million, or a net loss of $14.22 per share for the same period in 2016.

Research and development expenses for the third quarter of 2017 were $13.1 million, compared to $7.1 million for the same period in 2016. The increase in R&D expenses for the third quarter 2017 was primarily due to clinical development costs associated with our lead program, RA101495 SC, for the treatment of PNH.

General and administrative expenses for the third quarter of 2017 were $2.3 million, compared to $1.0 million for the same period in 2016. The increase in G&A expenses for the third quarter 2017 was due primarily to employee-related costs, including salary, benefits, and non-cash stock-based compensation due to the increase in G&A headcount to support the growth of the Company.

There was no revenue earned in the three months ended September 30, 2017 or the three months ended September 30, 2016.

As of September 30, 2017, Ra Pharma reported total cash and equivalents of $84.1 million. The Company expects that its cash and cash equivalents will be sufficient to fund operations through the end 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 (SC) 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 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 in healthy volunteers and as noted in the initial data reported in June 2017 on two eculizumab-naïve patients in Ra Pharma’s Phase 2 program, dosing of RA101495 SC was well tolerated and demonstrated sustained and near complete suppression of hemolysis and complement activity. To learn more about RA101495 SC, please visit: http://rapharma.com/pipeline/ra101495/.

About RA101495 SC Phase 2 PNH Clinical Program

The global, dose-finding Phase 2 program is designed to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetics, and pharmacodynamics of RA101495 SC in patients with PNH. The study will evaluate RA101495 SC in three cohorts. Cohort A includes eculizumab-naïve patients, Cohort B includes patients switching from eculizumab to RA101495 SC, and a third cohort 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 (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 will enroll approximately 36 patients and will include 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: 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 SC, including the timing, designs, plans and announcement of results regarding our ongoing and future studies and programs described in this press release, and our expected cash runway from existing 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, 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.

Ra Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets
(In thousands)
September 30, 2017December 31, 2016
Cash and cash equivalents $ 84,091 $ 117,812
Prepaid expenses and other current assets 1,123 1,690
Property and equipment, net 5,967 5,537
Other noncurrent assets 1,730 1,779
Total assets $ 92,911 $ 126,818
Liabilities and Stockholders’ Equity
Accounts payable and accrued expenses $ 7,371 $ 6,434
Deferred rent 432 303
Noncurrent liabilities 2,531 2,859
Stockholders' equity 82,577 117,222
Total liabilities and stockholders’ equity $ 92,911 $ 126,818
Ra Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations
(in thousands, except per share data)

Three Months Ended
September 30

Nine Months Ended
September 30

Revenue $ - $ - $ - $ 4,928
Operating expenses:
Research and development 13,130 7,079 32,606 18,541
General and administrative 2,284 1,042 7,101 3,418
Total operating expenses 15,414 8,121 39,707 21,959
Loss from operations (15,414 ) (8,121 ) (39,707 ) (17,031 )
Other income (expense), net 139 7 409 (945 )
Net loss $ (15,275 ) $ (8,114 ) $ (39,298 ) $ (17,976 )
Net loss per common share – basic and diluted $ (0.68 ) $ (14.22 ) $ (1.74 ) $ (32.73 )
Weighted average number of common shares outstanding – basic and diluted 22,614 571 22,579 549


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

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