RARX - Ra Pharmaceuticals Reports Second Quarter 2019 Financial Results and Provides Corporate Update

Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced financial results for the second quarter ended June 30, 2019, and provided an update on recent corporate and clinical developments.

“We expanded our neuromuscular portfolio for zilucoplan with the addition of immune-mediated necrotizing myopathy (IMNM), a severe, chronic, and debilitating autoimmune disease with limited treatment options,” said Doug Treco, Ph.D., President and Chief Executive Officer of Ra Pharma. “With both the Phase 2 clinical trial in IMNM and the Phase 3 clinical trial in generalized myasthenia gravis (gMG) on track to initiate in the second half of 2019, we’ve continued to a build a foundation for leveraging the properties of a small peptide in tissue-based complement-mediated neurologic diseases with significant unmet need. With a recent follow-on offering raising gross proceeds of $149.5 million, we are well-positioned to build meaningful value through the advancement of this neurologic pipeline.”

Dr. Treco continued: “This quarter was also marked by significant progress in our life-cycle extension program. With pre-clinical data for two zilucoplan extended release (XR) formulations supporting the potential for once-weekly or less frequent dosing, the XR program is on track to enter the clinic in the first half of 2020, an opportunity to further advance our mission of developing and expanding patient access to convenient treatment options.”

Second Quarter 2019 Highlights

• In April 2019, Ra Pharma announced the successful completion of End-of-Phase 2 interactions with the U.S. Food and Drug Administration (FDA) for its Phase 3 clinical trial of zilucoplan in gMG. Based on FDA feedback and advice provided by the European Medicines Agency (EMA) in the second quarter of 2019, Ra Pharma plans to initiate a single, pivotal, 12-week, Phase 3, randomized, double-blind, placebo-controlled trial evaluating the efficacy of zilucoplan in patients with gMG in the second half of 2019.

• In June 2019, Ra Pharma expanded the neuromuscular pipeline for zilucoplan with the FDA’s clearance of the Company’s Investigational New Drug (IND) application for zilucoplan for the treatment of IMNM. The Company is on track to initiate a Phase 2, randomized, double-blind, placebo-controlled trial to evaluate the safety, tolerability, and efficacy of zilucoplan in patients with IMNM in the second half of 2019.

• In July 2019, Ra Pharma entered into an exclusive, worldwide license agreement for the use of Camurus AB’s (Nasdaq STO:CAMX; Lund, Sweden) proprietary FluidCrystal® (FC) technology to develop, manufacture, and commercialize a long-acting formulation of zilucoplan. In July 2019, Ra Pharma reported pre-clinical data for the FC XR formulation of zilucoplan, in which non-human primates receiving a single dose of the FC XR formulation of zilucoplan rapidly achieved and maintained target levels of complement inhibition for at least seven days without the need for intravenous loading.

• In July 2019, Ra Pharma strengthened its balance sheet with a follow-on offering, raising $149.5 million in gross proceeds.

Second Quarter 2019 Financial Results

For the second quarter of 2019, the Company reported a net loss of $21.9 million, or a net loss of $0.52 per share (basic and diluted), compared to a net loss of $15.7 million, or a net loss of $0.49 per share, for the same period in 2018.

Research and development (R&D) expenses for the second quarter of 2019 were $17.9 million, compared to $12.3 million for the same period in 2018. The increase in R&D expenses for the second quarter was primarily due to expenses for non-clinical studies and clinical trials and increased headcount and employee-related costs to support increased research and development activities.

General and administrative (G&A) expenses for the second quarter of 2019 were $4.9 million, compared to $3.8 million for the same period in 2018. The increase in G&A expenses for the second quarter was primarily due to increased headcount and employee-related costs and increased expenses related to pre-commercial activities.

There was no revenue earned in the second quarter of 2019 or the same period in 2018.

As of June 30, 2019, Ra Pharma reported total cash and cash equivalents of $173.6 million. The Company expects that its cash and cash equivalents, along with the net proceeds of $140.2 million from the follow-on offering closed in July 2019, will be sufficient to fund operating expenses and capital expenditure requirements through at least the end of 2021.

About Zilucoplan

Ra Pharma is developing zilucoplan and zilucoplan extended release (XR) for generalized myasthenia gravis (gMG), immune-mediated necrotizing myopathy (IMNM), and other tissue-based complement-mediated disorders with high unmet medical need. The product candidate is designed for convenient subcutaneous (SC) self-administration. Zilucoplan is an investigational, synthetic, macrocyclic peptide discovered using Ra Pharma's powerful proprietary drug discovery technology. The peptide is designed to bind complement component 5 (C5) with sub-nanomolar affinity and allosterically inhibit its cleavage into C5a and C5b upon activation of the classical, alternative, or lectin pathways.

About Ra Pharmaceuticals, Inc.

Ra Pharmaceuticals is a clinical-stage biopharmaceutical company focused on leading the field of complement biology to bring innovative and accessible therapies to patients with rare 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 Statements

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 Ra Pharma's ability to expand patient access to important therapies, the potential, safety, efficacy, and regulatory and clinical progress of Ra Pharma's product candidates, including without limitation zilucoplan and zilucoplan XR, beliefs regarding clinical trial data, statements regarding trial design, timeline, and enrollment of Ra Pharma's ongoing and planned clinical programs, including without limitation the Phase 3 trial of zilucoplan for the treatment of gMG, the Phase 2 trial of zilucoplan for the treatment of IMNM, and the planned clinical studies for the XR program, and the expectation that Ra Pharma's cash and cash equivalents will be sufficient to fund operating expenses and capital expenditures through at least the end of 2021. 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 zilucoplan and zilucoplan XR, will not successfully be developed or commercialized, in the timeframe we expect or at all; the risk that Ra Pharma may fail to enroll patients in its clinical trials, which may cause delays or other adverse effects; the risk that Ra Pharma may fail to obtain additional financing on favorable terms or at all; 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.

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