BrainStorm Announces NurOwn Expanded Access Program

BrainStorm Cell Therapeutics has announced the initiation of a NurOwn® (MSC-NTF cells) Expanded Access Program (EAP) for patients with amyotrophic lateral sclerosis (ALS) who completed the Company's recently concluded pivotal Phase 3 clinical trial and meet specific eligibility requirements.

The protocol for the EAP was developed in partnership with the United States Food and Drug Administration (FDA) to provide access to NurOwn for Phase 3 clinical trial participants who meet specific eligibility criteria. Initially, patients less severely affected by ALS, as measured by the Revised ALS Functional Rating Scale (ALSFRS-R), will be the first to receive treatment. This approach is informed by recently announced topline data from the Company's Phase 3 clinical trial. According to the FDA, EAPs, alternatively known as "compassionate use" programs, provide a pathway for patients to receive an investigational medicine for a serious disease or condition outside of a clinical trial.

"This expanded access program is an appropriate and welcome next step in following up the exciting results of the Phase 3 study; it is widely anticipated and deeply appreciated by our ALS patients," said Robert Brown, MD DPhil, Leo P. and Theresa M. LaChance Chair in Medical Research, and chair, department of neurology, University of Massachusetts Medical School and UMass Memorial Medical Center, and one of the Principal Investigators in the NurOwn® Phase 3 study.

Through the newly announced EAP, the six clinical centers of excellence participating in the Phase 3 NurOwn® trial will each have the opportunity to treat ALS patients who completed the trial. These six centers are: University of California, Irvine; Cedars-Sinai Medical Center; California Pacific Medical Center; Massachusetts General Hospital; University of Massachusetts Medical School; and Mayo Clinic. EAP treatment of ALS patients who have completed the Phase 3 clinical trial will not interfere with data or regulatory timelines. The Dana Farber Cancer Institute will initially manufacture the investigational therapy, assisted by BrainStorm personnel who will be on-site.

"We are pleased to have the opportunity to treat additional patients with NurOwn through this Expanded Access Program, which was strongly advocated for by members of the ALS advocacy community. We recognize the urgency with which people with ALS want and need access to new potential treatments. This EAP is an important next step to providing an immediate option for some patients. We remain committed to rapidly advancing NurOwn through clinical development and regulatory review in the hope that the greatest number of people living with ALS may benefit," Chaim Lebovits, CEO of BrainStorm, said.