FDA to review citizen petition on BrainStorm’s ALS therapy NurOwn

NEW YORK - BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI), a micro-cap biotech company with a market value of $11.22 million, announced Tuesday that the U.S. Food and Drug Administration will review a Citizen Petition requesting a new assessment of data supporting its investigational ALS therapy NurOwn. According to InvestingPro analysis, the company currently shows a weak financial health score, reflecting the challenging environment for early-stage biotech firms.

The company, which was not involved in drafting or submitting the petition, stated it welcomes the FDA’s willingness to reevaluate existing data. BrainStorm confirmed it will proceed with its planned Phase 3b ENDURANCE trial under an FDA Special Protocol Assessment. Despite the stock’s 78.74% decline over the past year, analysts maintain price targets ranging from $6 to $24.35, suggesting significant potential upside if the therapy succeeds. Discover more detailed analysis and 8 additional key insights with InvestingPro.

"We respect the FDA’s independent review process and welcome its consideration of this request," said Chaim Lebovits, President and Chief Executive Officer of BrainStorm, according to the press release.

The company highlighted previous analyses showing that in participants with baseline ALSFRS-R scores of 35 and above, NurOwn demonstrated statistically significant functional improvement versus placebo. Post-hoc analyses indicated NurOwn-treated participants retained an average of two more ALSFRS-R points than placebo recipients.

BrainStorm also referenced recently announced survival data from 10 participants who completed the Phase 3 trial and entered its Expanded Access Program. These participants survived more than five years from ALS symptom onset, with median survival reaching 6.8 years.

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease that affects motor neurons and severely impairs voluntary muscle movement.

NurOwn uses autologous mesenchymal stem cells that are expanded and differentiated to secrete neurotrophic factors. The therapy has received Orphan Drug designation from both the FDA and European Medicines Agency.

The information in this article is based on a press release statement from BrainStorm Cell Therapeutics. With a current ratio of 0.21 and negative EBITDA of $12.08 million in the last twelve months, investors seeking deeper insights into BCLI’s financial position can access comprehensive analysis through InvestingPro’s detailed research reports, available for over 1,400 US stocks.

In other recent news, BrainStorm Cell Therapeutics has announced a strategic partnership with Minaris Advanced Therapies to manufacture NurOwn® for a Phase 3b clinical trial focused on ALS. This collaboration is expected to enhance BrainStorm’s manufacturing capabilities in the U.S., following the company’s completion of a Phase 3 trial in ALS. Concurrently, BrainStorm is actively pursuing a $15 million non-dilutive grant to address financial constraints and support its clinical development efforts. The company has also begun a technology transfer to Pluri Inc. in Israel to bolster its production network for NurOwn®. Furthermore, BrainStorm is in the process of securing strategic partnerships to improve its financial standing and support ongoing trials. The recent earnings call highlighted the company’s focus on advancing its NurOwn therapy despite financial challenges. Analysts from firms such as Zacks Small Cap have shown interest in the company’s funding strategies and trial progress. These developments underscore BrainStorm’s commitment to advancing therapeutic options for ALS patients.

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