BOSTON & CAMBRIDGE, Mass.–(BUSINESS WIRE)– 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.