NEW YORK - BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a developer of adult stem cell therapies for neurodegenerative diseases, is set to present the design for a Phase 3b trial of its NurOwn therapy for amyotrophic lateral sclerosis (ALS). The presentation will occur at the MDA Clinical and Scientific Conference on Monday evening at the Hilton Orlando Hotel in Florida.
The planned Phase 3b study is a two-part, multicenter trial aimed at confirming the efficacy and safety of NurOwn in patients with mild-to-moderate ALS. It will begin with a 24-week randomized, double-blind, placebo-controlled period, followed by a 24-week open-label extension. The trial's primary endpoint will utilize the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) for assessment.
BrainStorm has submitted a Special Protocol Assessment (SPA) request to the U.S. Food and Drug Administration (FDA), seeking agreement on the trial design, which is intended to support a future marketing application for NurOwn. The SPA request is currently under review by the FDA, with BrainStorm anticipating feedback to finalize the study details.
Chaim Lebovits, President and CEO of BrainStorm, expressed the company's commitment to making NurOwn available to ALS patients and the intention to generate conclusive data through the new trial. Dr. Bob Dagher added that the design incorporates insights from previous studies to maximize the chances of a successful outcome, focusing on the mild-to-moderate ALS patient population for accurate disease progression measurement.
The company plans to post the presentation poster on its corporate website after the conference. BrainStorm's approach to developing NurOwn leverages autologous adult stem cell technology, and the treatment has already received Orphan Drug designation from the FDA and the European Medicines Agency (EMA) for ALS.
The information for this article is based on a press release statement from BrainStorm Cell Therapeutics Inc.
InvestingPro Insights
As BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) prepares to present its Phase 3b trial design for NurOwn, investors are closely monitoring the company's financial health and market performance. According to InvestingPro data, BrainStorm has a market capitalization of approximately $17.58 million. The company's performance metrics indicate significant challenges, with an adjusted P/E ratio over the last twelve months as of Q3 2023 at -1.06, reflecting investor concerns about the company's profitability.
InvestingPro Tips highlight that BrainStorm is rapidly burning through cash and operates with a moderate level of debt. These factors are crucial for stakeholders considering the costs associated with the development and potential commercialization of therapies like NurOwn. The tips also note that the stock has experienced high price volatility, which could be tied to the speculative nature of biotech investments and the company's recent activities.
Despite these challenges, BrainStorm has seen a strong return over the last three months, with a 54.63% price total return, indicating a potential uptick in investor confidence. This could be related to the anticipation surrounding the company's upcoming trial and the SPA request with the FDA. While analysts do not anticipate the company will be profitable this year, and the stock has taken significant hits over longer periods, the recent positive price momentum may reflect optimism in the company's strategic direction.
For investors looking for a deeper analysis, there are 12 additional InvestingPro Tips available, offering insights that can help in making informed decisions. To explore these tips and gain a comprehensive understanding of BrainStorm's financials and market standing, visit https://www.investing.com/pro/BCLI. Remember to use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
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