Ra Pharmaceuticals Announces Clearance of IND Application for the HEALEY ALS Platform Trial

Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today 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.

“We are pleased that the FDA has authorized the initiation of this pioneering platform trial, which, through the pooling of placebo arms, optimizes the chance a patient receives active drug and enables the efficient evaluation of multiple investigational therapies,” said Doug Treco, Ph.D., President and Chief Executive Officer of Ra Pharma. “With substantial data supporting the role of central and peripheral complement activation and membrane attack complex deposition in ALS, we look forward to assessing the therapeutic potential of zilucoplan, an investigational small peptide inhibitor of complement component 5 (C5), in this disease. We’re grateful to the Healey Center for its sponsorship of this trial and share its commitment to improving the lives of patients with ALS, a debilitating disease with limited treatment options.”

The HEALEY ALS Platform trial will be conducted across approximately 54 U.S. centers and is designed to initially evaluate three treatment regimens in parallel. Following enrollment, patients will be randomized to one of three treatment regimens, one of which will be the zilucoplan regimen. The zilucoplan regimen will evaluate the efficacy and safety of zilucoplan versus placebo on ALS disease progression, and the primary endpoint will be change in ALS Functional Rating Scale-Revised (ALSFRS-R) score. Following the initial randomization to this regimen, patients will be randomized in a 3:1 ratio to receive daily subcutaneous doses of 0.3 mg/kg zilucoplan or placebo. The zilucoplan regimen is expected to enroll 160 patients for a 24-week treatment period. Following completion of the trial, patients will have the option to enter into an open-label extension study with zilucoplan.

About the HEALEY ALS Platform Trial

The ALS platform trial is designed to disrupt the standard pace of ALS therapy development by testing and evaluating multiple treatments simultaneously. This model, which has had success in the cancer field, is designed to accelerate development of therapies by allowing investigators to test more drug candidates, increase patient access to trials, and reduce costs by quickly and efficiently evaluating the effectiveness of multiple investigational therapies. Following the Healey Center’s call for the best therapeutic ideas to enter the HEALEY ALS Platform Trial, the first drug candidates were chosen by a group of expert ALS scientists and members of the Healey Center Science Advisory Committee.

About ALS

Amyotrophic lateral sclerosis (ALS) is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 30,000 people in the U.S. and an estimated 500,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in progressive muscle weakness and atrophy that eventually lead to partial or total paralysis. Therapeutic options for ALS are currently limited to two approved products.

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 potential, safety, efficacy, and regulatory and clinical progress of Ra Pharma's product candidates, including without limitation zilucoplan, beliefs regarding clinical trial data, and statements regarding trial design, timeline, and enrollment of Ra Pharma's ongoing and planned clinical programs, including without limitation the Healey Center-sponsored ALS platform trial. 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, 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 be unable to obtain orphan drug designation or to maintain the benefits associated with orphan drug status, including market exclusivity; 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.
Natalie Wildenradt, 617-674-9874

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