#United States #New York #BrainStorm #ALS #NurOwn

BrainStorm Cell Therapeutics Advances ALS Treatment with IND Amendment

In a significant development for neurodegenerative disease treatments, BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), renowned for its innovative cell therapies, has announced the submission of an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) regarding its promising autologous mesenchymal stem cell therapy, NurOwn®, specifically targeting amyotrophic lateral sclerosis (ALS). This amendment bolsters the company's efforts as it gears up for its highly anticipated Phase 3b clinical trial.

The filing represents a crucial milestone not just for BrainStorm, but also for ALS patients who have limited therapeutic options. This amendment is poised to facilitate the swift progression into the Phase 3b trial, designed in collaboration with the FDA under a Special Protocol Assessment (SPA). The SPA indicates that the trial's design, along with its statistical analysis plan, meets the expectations set forth by the FDA, confirming a streamlined path for a future Biologics License Application (BLA).

Chaim Lebovits, CEO of BrainStorm, expressed optimism over the IND's approval process, stating, "The submission of this IND amendment, coupled with the FDA's agreement on our trial design, marks a major step toward making NurOwn accessible to ALS patients. We are hopeful for a rapid review and eager to kick off our Phase 3b study to gain regulatory approval."

Overview of the Phase 3b Trial

Anticipated to consist of around 200 ALS participants, the Phase 3b trial will unfold in two distinct parts. During Part A, participants will receive a total of three doses of either NurOwn or placebo over a 24-week double-blind period. Post this phase, everyone, including those who received the placebo, will transition to an open-label extension (Part B), where they will receive three additional doses of NurOwn over another 24 weeks. The trial's primary goal is to evaluate changes in ALS Functional Rating Scale-Revised (ALSFRS-R) scores from baseline to week 24, comparing disease progression between the NurOwn and placebo cohorts. A successful outcome from Part A would set BrainStorm on track for a BLA submission, expediting NurOwn’s journey toward potential market approval.

The urgency surrounding ALS treatments cannot be overstated, given the devastating nature of the disease and the current lack of effective therapies. BrainStorm is firmly committed to collaborating closely with regulatory bodies, clinical researchers, and the greater ALS community to ensure a rigorous and prompt evaluation of NurOwn. Updates regarding the FDA's review process will be shared as they become available.

About NurOwn®

The NurOwn® platform is an innovative approach that centers around autologous MSC-NTF (mesenchymal stem cell neurotrophic factor) cells, designed to target critical disease pathways associated with neurodegenerative disorders. This advanced technology utilizes bone marrow-derived mesenchymal stem cells, which are expanded and differentiated outside the body. They are then transformed into MSC-NTF cells through proprietary conditions, prompting these cells to release significant amounts of neurotrophic factors (NTFs). The ultimate intention is to ensure that the derived MSC-NTF cells can deliver multiple NTFs and immunomodulatory cytokines to the injury site directly, fostering a desired biological response that may slow or stabilize disease progression.

About Brainstorm Cell Therapeutics Inc.

As a leader in the biotechnology space, BrainStorm's mission revolves around developing cutting-edge autologous adult stem cell therapies aimed at incapacitating neurodegenerative illnesses. Holding exclusive rights for the clinical development and commercialization of the NurOwn® technology platform, the company has secured Orphan Drug designation status from both the FDA and the European Medicines Agency (EMA) for ALS treatment. BrainStorm has previously completed a Phase 3 pivotal trial for ALS and continues to investigate the potential of autologous MSC-NTF cells in conditions such as progressive multiple sclerosis (MS).

In conclusion, the recent IND amendment submission emphasizes BrainStorm's unwavering commitment to enhancing treatment options for ALS and instills hope for those affected by this challenging condition. As the company embarks on this next phase of clinical trials, the eyes of the medical and scientific community will be closely watching its progress in the quest for effective ALS therapies.