FDA agrees to BrainStorm Cell Therapeutics' Phase 3b ALS trial design
Investing.com | Editor Emilio Ghigini
Published Apr 09, 2024 11:18
NEW YORK - BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a pioneer in adult stem cell therapies for neurodegenerative diseases, today announced a significant milestone. The U.S. Food and Drug Administration (FDA) has proposed designing a Phase 3b trial for NurOwn® in amyotrophic lateral sclerosis (ALS) patients.
This agreement, achieved through a Special Protocol Assessment (SPA), solidifies the clinical trial protocol and statistical analysis plan. The upcoming study aims to bolster a future Biologics License Application (BLA).
Dubbed Study BCT-006-US, this Phase 3b trial is a two-part, multicenter endeavor to evaluate NurOwn's efficacy and safety in ALS patients. It targets up to 200 individuals early in their disease progression, specifically those with symptom onset within the last 24 months.
The trial will initiate with a 24-week, double-blind, placebo-controlled phase, succeeded by an open-label extension. During this phase, all participants will receive the innovative treatment.
Chaim Lebovits, President and CEO of BrainStorm, expressed optimism regarding the SPA's potential to de-risk regulatory aspects of the NurOwn clinical program and to advance towards regulatory approval. Stacy Lindborg, co-CEO of the company, highlighted the design's focus on evaluating the treatment's effectiveness and safety based on insights from previous studies.
The primary efficacy endpoint of the trial will be the change in the Revised Amyotrophic Lateral Sclerosis Functional Rating Score (ALSFRS-R) from baseline to Week 24. An independent Data Monitoring Committee (DMC) will oversee participant safety, and cerebrospinal fluid and blood samples will be analyzed for biomarkers related to neurodegeneration and neuroprotection.
BrainStorm plans to start the Phase 3b study in 2024, following protocol reviews with investigators, Institutional Review Board approvals, and engagement with the ALS community. The company hosted a conference call and webcast today to discuss the SPA and the planned Phase 3 program.
This news is based on a press release from BrainStorm Cell Therapeutics Inc. and represents a significant step for the company in its efforts to develop treatments for ALS, a progressive neurodegenerative disease with limited therapeutic options.
h2 InvestingPro Insights/h2As BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) gears up for the critical Phase 3b trial of its ALS treatment, NurOwn®, investors are closely monitoring the company's financial health and market performance. The latest data from InvestingPro reveals a market capitalization of $49.89 million, reflecting the company's current valuation in the market. Despite the promising clinical developments, BrainStorm's financials show a challenging landscape. With a negative P/E ratio of -2.9 for the last twelve months as of Q4 2023, it's evident that the company is not currently generating profits.
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InvestingPro Tips highlight significant challenges for BrainStorm, including a rapid cash burn and weak gross profit margins. Additionally, the company's short-term obligations exceed its liquid assets, and analysts do not expect profitability for this fiscal year. These factors are crucial for investors to consider when evaluating BrainStorm's potential for long-term success.
However, it's not all bleak for BrainStorm. The company has seen a strong return over the last month, with a 121.21% increase, and an even more impressive six-month price total return of 329.41%. This uptick could indicate growing investor confidence in the company's future, especially as it advances its clinical programs.
For those interested in a deeper dive into BrainStorm's financials and future prospects, there are 11 additional InvestingPro Tips available. To explore these insights and make more informed investment decisions, visit InvestingPro. Don't forget to use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription, enhancing your investment research with a comprehensive suite of tools and data.
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Written By: Investing.com
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