BrainStorm Cell Therapeutics (BCLI, Financial) has disclosed promising survival data from its Expanded Access Program (EAP) for patients with amyotrophic lateral sclerosis (ALS) using its NurOwn therapy. The EAP included 10 participants who had previously completed the Phase 3 clinical trial. Researchers analyzed survival from the onset of ALS symptoms through various stages, including publicly available records.
Participants entered the Phase 3 trial early in their disease progression, with a baseline Mean ALSFRS-R score of 35.8, which decreased to 31.4 at the EAP's start. Key findings indicate that 90% of participants survived over five years from symptom onset, compared to just 10% in the general ALS population. The cohort's median survival was 6.8 years, with a single death occurring due to elective euthanasia. These results suggest that exceptional survival outcomes among the group are not due to chance.
BrainStorm's President and CEO, Chaim Lebovits, emphasized that these findings provide valuable real-world insights and support the forthcoming Phase 3b clinical trial of NurOwn, which will proceed under an FDA Special Protocol Assessment. The overarching aim is to produce robust data confirming NurOwn's benefits for ALS patients.
Wall Street Analysts Forecast
Based on the one-year price targets offered by 3 analysts, the average target price for Brainstorm Cell Therapeutics Inc (BCLI, Financial) is $12.50 with a high estimate of $22.51 and a low estimate of $6.00. The average target implies an upside of 1,036.67% from the current price of $1.10. More detailed estimate data can be found on the Brainstorm Cell Therapeutics Inc (BCLI) Forecast page.
Based on the consensus recommendation from 1 brokerage firms, Brainstorm Cell Therapeutics Inc's (BCLI, Financial) average brokerage recommendation is currently 2.0, indicating "Outperform" status. The rating scale ranges from 1 to 5, where 1 signifies Strong Buy, and 5 denotes Sell.
BCLI Key Business Developments
Release Date: May 19, 2025
- Phase 3b Trial Initiation: FDA clearance received to initiate pivotal Phase 3b trial for NurOwn.
- Manufacturing and Site Selection: Initial manufacturing at Tel Aviv Sourasky Medical Center; technology transfer to Pluri for additional facilities.
- Clinical Trial Agreements: Negotiations with approximately 15 leading clinical centers in the U.S. for Phase 3b trial sites.
- Trial Design: Phase 3b trial includes a 24-week double-blind period followed by a 24-week open-label extension.
- Primary Endpoint: Change from baseline to week 24 in ALSFRS-R total score.
- Biomarker Analysis: NurOwn associated with reduction in neuroinflammatory and neurodegenerative biomarkers.
- Genetic Substudy: UNC13A genotype may influence response to NurOwn therapy.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- The U.S. FDA has cleared Brainstorm Cell Therapeutics Inc (BCLI, Financial) to initiate a pivotal Phase 3b trial for NurOwn, marking a significant milestone in their ALS treatment development.
- The trial design has been agreed upon with the FDA under a special protocol assessment (SPA), which de-risks the regulatory pathway for NurOwn.
- Brainstorm Cell Therapeutics Inc (BCLI) has secured a leading U.S. clinical site that has passed FDA inspection, enhancing their operational readiness for the upcoming trial.
- The company is actively engaged in negotiations with approximately 15 leading clinical centers across the United States for the Phase 3b trial, indicating strong interest from the ALS research community.
- Brainstorm Cell Therapeutics Inc (BCLI) is pursuing multiple funding avenues, including a promising $15 million non-dilutive grant, to ensure the timely commencement of the trial.
Negative Points
- The company faces significant financial constraints, which are common in the biotech industry, and securing proper funding is essential to commence the trial.
- The initiation and successful execution of the clinical trial demand a robust and sustainable cash flow, which is currently a challenge for Brainstorm Cell Therapeutics Inc (BCLI).
- The company has not yet signed clinical trial agreements (CTAs) with the clinical centers, which could delay the trial's start.
- The manufacturing capacity at the Tel Aviv facility is limited, and the company is still in the process of expanding its manufacturing footprint to the United States.
- The exosome program, while promising, is still in the preclinical stage and requires strategic partnerships to advance towards clinical development.
