Ra Pharma Announces Clearance Of IND Application For The HEALEY ALS

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.-Bio Space