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Ra Pharmaceuticals Announces Selection for the First ALS Platform Trial by the Sean M. Healey & AMG Center for ALS at Mass General

Multi-center platform trial sponsored by the Healey Center will simultaneously assess multiple drug regimens

Zilucoplan selected by leading experts in ALS as one of the first therapeutic candidates to be tested

BOSTON & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 18, 2019-- Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced the selection of zilucoplan as one of the first clinical candidates to be evaluated in a pioneering platform trial for amyotrophic lateral sclerosis (ALS), led by the Sean M. Healey & AMG Center for ALS at Mass General. The Healey Center has agreed to provide funding for the execution of the platform trial to support its goal of accelerating the development of effective treatments for patients with ALS.

“The Healey Center brought together top leaders from around the world to dramatically re-think how we design ALS trials and create a groundbreaking platform trial program,” says Merit Cudkowicz, M.D., M.Sc., director of the Sean M. Healey & AMG Center for ALS at Mass General. “With research suggesting a key role for central and peripheral complement activation and deposition in ALS, we look forward to improving patient access to promising therapies and contributing important knowledge to the ALS community.”

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, aims to greatly accelerate development of therapies by allowing investigators to test more drugs, increase patient access to trials, and reduce costs by quickly and efficiently evaluating the effectiveness of multiple therapies. The Healey Center issued a call for the best therapeutic ideas to enter the HEALEY ALS Platform Trial, and the first drug candidates have been chosen by a group of expert ALS scientists and members of the Healey Center Science Advisory Committee.

“Robust pre-clinical data support the evaluation of a C5 inhibitor in ALS. Zilucoplan is a small peptide inhibitor with enhanced tissue distribution as compared with therapeutic monoclonal antibodies and has demonstrated efficacy in gMG, a complement-mediated disease of the neuromuscular junction,” said Simon Read, Ph.D., Chief Scientific Officer of Ra Pharma. “The ALS platform trial design, together with a close relationship with the ALS patient community, has the potential to enable rapid recruitment and robust assessment of the therapeutic effect of zilucoplan in this disease.”

“We’re grateful to the Healey Center for its sponsorship of this trial and are honored to participate in this important and innovative study. We look forward to working closely together in this collaboration,” said Doug Treco, Ph.D., President and Chief Executive Officer of Ra Pharma. “Participation in the ALS platform trial represents a further expansion of our neurologic pipeline for zilucoplan, which leverages the unique properties of a small peptide C5 inhibitor in complement-mediated neurologic diseases.”

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 candidates are 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

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-led 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.

Source: Ra Pharmaceuticals, Inc.

Investors:
Ra Pharmaceuticals, Inc.
Natalie Wildenradt, 617-674-9874
nwildenradt@rapharma.com

Media:
Argot Partners
David Rosen, 212-600-1902
david.rosen@argotpartners.com