Ra Pharmaceuticals Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Corporate Update

Enrollment ongoing in Phase 3 clinical trial of zilucoplan in gMG, with top-line results expected in early 2021

First patient dosed in Phase 2 clinical trial of zilucoplan in IMNM, with top-line results expected in the second half of 2020

IND cleared by FDA for the HEALEY ALS Platform Trial at Mass General; Zilucoplan selected as one of the first clinical candidates to be evaluated

UCB acquisition of Ra Pharma expected to close by the end of the first quarter of 2020

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced financial results for the fourth quarter and year ended December 31, 2019, and provided an update on recent corporate and clinical developments.

“2019 was a transformational year for Ra Pharma in our efforts to discover, develop, and expand access to important therapies for patients with rare diseases, including the strengthening of our balance sheet with a $150 million financing and the advancement of key clinical and pre-clinical programs. With the initiation of our global, pivotal, Phase 3 RAISE study of zilucoplan in generalized myasthenia gravis (gMG) and our Phase 2 clinical trial of zilucoplan in immune-mediated necrotizing myopathy (IMNM), as well as the selection of zilucoplan for a pioneering platform trial for amyotrophic lateral sclerosis (ALS) sponsored by the Sean M. Healey & AMG Center for ALS at Mass General, we’ve significantly expanded zilucoplan’s pipeline of neurologic, tissue-based, complement-mediated diseases,” said Doug Treco, Ph.D., President and Chief Executive Officer of Ra Pharma.

Dr. Treco added: “Ra Pharma’s pending acquisition by UCB remains on track, and we continue to anticipate the transaction to close by the end of the first quarter of 2020. Sharing our commitment to the rare disease patient community and our goal of developing novel and accessible therapies, UCB is the optimal partner to further advance zilucoplan, our C5 inhibitor life-cycle extension program, and our early-stage discovery pipeline and platform research efforts.”

Fourth Quarter 2019 Highlights and Recent Developments

• In October 2019, Ra Pharma announced the entry into a merger agreement pursuant to which UCB has agreed to acquire Ra Pharma. Under the terms of the agreement, Ra Pharma shareholders will receive $48 in cash for each Ra Pharma share at closing (equity value of approximately $2.5 billion). The Boards of Directors of both companies unanimously approved the transaction, and a majority of Ra Pharma shareholders have voted to approve the transaction, which remains subject to obtaining antitrust clearance and other customary closing conditions. The transaction is expected to close by the end of the first quarter of 2020.
• In February 2020, Ra Pharma announced that full results from its Phase 2 clinical trial of zilucoplan in patients with gMG were published online in JAMA Neurology. The publication in JAMA Neurology recognizes the impact and significance of the trial’s findings demonstrating zilucoplan’s potential for the treatment of gMG. The RAISE study, a pivotal Phase 3 clinical trial evaluating zilucoplan for the treatment of gMG is ongoing, with top-line results expected in early 2021.
• In December 2019, Ra Pharma announced the initiation of dosing in a randomized, double-blind, placebo-controlled, multi-center, Phase 2 clinical trial of zilucoplan for the treatment of IMNM. The trial is designed to evaluate the safety, tolerability, and efficacy of a once-daily, subcutaneously (SC) self-administered dose of 0.3 mg/kg of zilucoplan versus placebo. Top-line results are expected in the second half of 2020.
• In January 2020, Ra Pharma announced the U.S. Food and Drug Administration’s (FDA) clearance of the Investigational New Drug (IND) application for the HEALEY ALS Platform Trial for the treatment of amyotrophic lateral sclerosis (ALS). Zilucoplan was selected as one of the first clinical candidates to be evaluated in this platform trial for ALS led by the Sean M. Healey & AMG Center for ALS at Mass General. The platform trial is designed to accelerate the development of effective treatments for patients with the disease.

Fourth Quarter and Full Year 2019 Financial Results

For the fourth quarter of 2019, the Company reported a net loss of $36.8 million, or a net loss of $0.79 per share (basic and diluted), compared to a net loss of $16.2 million, or a net loss of $0.47 per share, for the same period in 2018. For the full year 2019, Ra Pharma reported a net loss of $102.7 million, or a net loss of $2.31 per share (basic and diluted), compared to a net loss of $64.9 million, or a net loss of $2.06 per share, for the full year 2018.

Research and development (R&D) expenses for the fourth quarter of 2019 were $26.2 million, compared to $15.4 million for the same period in 2018. Research and development expenses for the full year 2019 were $82.6 million, compared to $54.5 million for the full year 2018. The increase in R&D expenses for both the fourth quarter and full year was primarily due to expenses relating to our lead product candidate, zilucoplan, and employee-related costs to support our increased research and development activities.

General and administrative (G&A) expenses for the fourth quarter of 2019 were $11.6 million, compared to $3.8 million for the same period in 2018. G&A expenses for the full year 2019 were $27.3 million, compared to $14.4 million for the full year 2018. The increase in G&A expenses for both the fourth quarter and full year was primarily due to an increase in third-party professional fees related to the merger agreement with UCB, employee-related costs due to the increase in headcount to support the growth of the Company, and increased costs related to pre-commercialization activities.

There was no revenue earned in the fourth quarter of 2019. Total revenue for the year ended December 31, 2019, was $3.0 million, related to the achievement of a clinical development milestone under the Company’s collaboration with Merck. Total revenue earned in the fourth quarter and full year ended December 31, 2018, was $2.5 million, related to the achievement of a pre-clinical development milestone under the Company’s collaboration with Merck.

As of December 31, 2019, Ra Pharma reported total cash and cash equivalents of $265.0 million. The Company expects that its cash and cash equivalents will be sufficient to fund operating expenses and capital expenditures 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), amyotrophic lateral sclerosis (ALS), 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. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to zilucoplan for the treatment of MG.

About Ra Pharmaceuticals

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 safety, efficacy, regulatory and clinical progress, and therapeutic potential of Ra Pharma’s product candidates, including without limitation zilucoplan and zilucoplan XR, 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 HEALEY ALS Platform Trial, plans and timing for the presentation of and beliefs regarding clinical trial data, statements regarding the merger with UCB, including the expected timing of closing, 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; risks relating to the Merger, including: that Ra Pharma may be unable to obtain stockholder approval as required for the Merger; conditions to the closing of the Merger may not be satisfied and required regulatory approvals may be delayed or not be obtained; the Merger may involve unexpected costs, liabilities or delays; the business of Ra Pharma may suffer as a result of uncertainty surrounding the Merger; the outcome of any legal proceedings related to the Merger; Ra Pharma may be adversely affected by other economic, business, and/or competitive factors; the occurrence of any event, change or other circumstances that could give rise to the termination of the Merger Agreement; and other risks to the consummation of the Merger, including the risk that the Merger will not be consummated within the expected time period or at all. If the Merger is consummated, Ra Pharma stockholders will cease to have any equity interest in Ra Pharma and will have no right to participate in its earnings and future growth. Additional factors that may affect the future results of Ra Pharma are 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.

Contacts

Investors:

Ra Pharmaceuticals, Inc.

Natalie Wildenradt, 617-674-9874

[email protected]

Media:

Argot Partners

David Rosen, 212-600-1902

[email protected]