BrainStorm advances ALS therapy with FDA trial submission

Published 10/04/2025, 12:34
BrainStorm advances ALS therapy with FDA trial submission

NEW YORK - BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a biotech firm focused on neurodegenerative diseases, has submitted an Investigational New Drug (IND) amendment for its Phase 3b clinical trial of NurOwn® to the U.S. Food and Drug Administration (FDA). The trial will investigate the autologous mesenchymal stem cell therapy in patients with amyotrophic lateral sclerosis (ALS). The company, currently valued at $7.27 million, has seen its stock decline by nearly 88% over the past year, according to InvestingPro data.

The submission follows a Special Protocol Assessment (SPA) agreement with the FDA, confirming that the trial design and statistical analysis plan align with regulatory standards for a potential Biologics License Application (BLA). This development could expedite the review process and the trial's initiation. InvestingPro analysis reveals that the company faces significant financial challenges, with an overall Financial Health Score of 1.65, labeled as 'WEAK', and is currently burning through cash rapidly.

The upcoming trial will enroll roughly 200 ALS patients and consist of two parts. Initially, participants will be randomly assigned to receive either NurOwn or a placebo during a 24-week double-blind period. Subsequently, all participants, including those from the placebo group, will be offered the actual therapy in an open-label extension for a further 24 weeks. The primary goal is to measure changes in ALS functional rating scale scores at week 24 to evaluate disease progression.

NurOwn technology involves harvesting patient-derived mesenchymal stem cells, which are then differentiated to secrete neurotrophic factors (NTFs). These cells are intended to deliver NTFs and immunomodulatory cytokines to damaged areas, potentially slowing disease progression.

BrainStorm's CEO, Chaim Lebovits, expressed optimism for a rapid review by the FDA and the subsequent launch of the Phase 3b study. The company is committed to addressing the unmet needs in ALS treatment by working with the FDA, clinical investigators, and the ALS community.

The company has previously conducted a Phase 3 pivotal trial and a Phase 2 open-label multicenter trial for multiple sclerosis under the IND application, with support from grants. NurOwn has also received Orphan Drug designation from the FDA and the European Medicines Agency (EMA) for ALS treatment. With the next earnings report scheduled for May 12, 2025, investors seeking deeper insights into BrainStorm's financial health and growth prospects can access comprehensive analysis through InvestingPro's detailed Research Report, which is part of their coverage of over 1,400 US stocks.

The information in this article is based on a press release statement from BrainStorm Cell Therapeutics Inc.

In other recent news, Brainstorm Cell Therapeutics Inc. reported its financial results for the fourth quarter of 2024, focusing on significant cost reductions. The company reduced its research and development expenses from $10.7 million in 2023 to $4.7 million in 2024, while its net loss decreased to $11.6 million from $17.2 million in the previous year. Despite these cost-cutting measures, cash reserves fell to $400,000 by the end of 2024, down from $1.5 million the previous year. Brainstorm Cell Therapeutics is advancing its Neuron ALS treatment, having secured a Special Protocol Assessment with the FDA. The company plans to initiate a Phase 3b trial for Neuron in 2025, targeting early-stage ALS patients, and anticipates needing $20-30 million annually to fund the trial. They are exploring non-dilutive financing options, such as grants, to support this initiative. Additionally, Brainstorm Cell Therapeutics announced a warrant inducement agreement expected to raise approximately $1.64 million, demonstrating investor confidence in their strategy. The company is also considering licensing non-core assets to secure further funding.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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