BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced results from the recently completed U.S. randomized, double-blind, placebo-controlled phase 2 Study of NurOwn® in Patients with ALS. The study achieved its primary objective, demonstrating that NurOwn was safe and well tolerated. NurOwn also achieved multiple secondary efficacy endpoints, showing clear evidence of a clinically meaningful benefit. Notably, response rates were higher for NurOwn-treated subjects compared to placebo at all time points in the study out to 24 weeks.
“We are very encouraged by these Phase 2 data that show that NurOwn cell therapy is safe and that a single administration produces a transient and clinically meaningful beneficial response in terms of both the ALS-FRS-R rating scale and CSF biomarkers,” said Dr. Robert H. Brown, Professor and Chair of Neurology at theUniversity of Massachusetts Medical Center and Medical School, and investigator in the trial. “These exciting findings clearly indicate that it is appropriate to conduct a longer study with repetitive dosing.”
“We are excited about the trial results which provide important information on safety, preliminary efficacy and biological effects of NurOwn,” said Merit E. Cudkowicz, MD, MSc, Professor of Neurology at Harvard Medical School and Chief of Neurology at Massachusetts General Hospital. “More participants in the treated group had slowing of progression and there were no safety concerns. The increased levels of growth factors in the cerebrospinal fluid and decreased inflammatory markers observed after two weeks are encouraging evidence for a biological effect. Based on these results, repeat dosing at 8 to 12 weeks and a larger confirmatory trial are warranted. I look forward to continuing to work closely with Brainstorm on the development of NurOwn for ALS.”
“After almost thirty years of conducting clinical studies in ALS I am personally very encouraged and excited by the trial results.” stated Anthony J. Windebank M.D, Professor of Neurology and Director for Discovery, Mayo Clinic Center for Regenerative Medicine. “There are positive efficacy signals with clear indications to proceed to the next phase. We at Mayo clinic will be eager to move ahead with that as soon as possible.”
“Patients in the Brainstorm study tolerated treatment extremely well and there were no serious adverse events related to therapy. The safety profile certainly provides the opportunity to continue to study this approach to ALS treatment.” stated Carlayne E. Jackson, MD, FAAN, Professor of Neurology and Otolaryngology, Chief Medical Officer – UT Medicine San Antonio, University of Texas Health Science Center who served as the chair of the Data Safety Monitoring Board on this study.
“I would like to thank the patients who participated in this clinical trial as well as the investigators, DSMB members and all those who contributed to the successful completion of this trial. These data give us valuable insights into the beneficial treatment effect with NurOwn” stated Chaim Lebovits, CEO of BrainStorm. “This study met its objectives, demonstrating both the safety of NurOwn and its ability to provide clinical benefit to ALS patients, and most importantly, will help us to determine the study population and design for a pivotal study of multiple doses of NurOwn in ALS.” – PRNewswire