Latest news with #BCLI
Yahoo
08-07-2025
- Health
- Yahoo
FDA Review of Citizen Petition Offers a Fresh Look at NurOwn®'s Evidence of Treatment Effectiveness
BrainStorm to Continue with Planned Phase 3b Trial and Remains Committed to Advancing Access for People Living with ALS NEW YORK, July 8, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today acknowledged that the U.S. Food and Drug Administration's (FDA) consideration of a Citizen Petition requesting a new review of the data supporting NurOwn will provide a critical opportunity to reaffirm its potential as therapy for amyotrophic lateral sclerosis (ALS). A Citizen Petition, submitted to the FDA under the Federal Food, Drug, and Cosmetic Act (21 CFR § 10.30), is a regulatory process that allows any interested party to formally request the Agency to take action on specific matters, such as reviewing data, issuing new guidance, or taking enforcement action. In this case, the Citizen Petition is requesting that the FDA review the data supporting NurOwn. BrainStorm has consistently supported the integrity and scientific validity of its data and believes a comprehensive review of all evidence is essential, especially as the regulatory landscape for rare diseases with unmet needs continues to evolve. Although BrainStorm was not involved in drafting or submitting this petition or its contents, the company welcomes the FDA's willingness to reevaluate existing data. It endorses any process that thoroughly assesses the scientific evidence related to potential treatment options for people living with ALS. "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. "We continue to stand firmly behind the scientific integrity of BrainStorm's data and remain committed to working collaboratively with the FDA and the ALS community to advance the development of safe and effective therapies." BrainStorm is committed to continuing alignment with FDA and will proceed with its Phase 3b ENDURANCE trial of NurOwn under an FDA Special Protocol Assessment (SPA), designed to generate additional robust data and confirm the product's efficacy and safety in early stage ALS patients. In parallel, the company supports exploration of potential regulatory pathways that may allow appropriate access to NurOwn for individuals living with ALS today. Bob Dagher, M.D., Chief Medical Officer at BrainStorm, stated, "A new review of the NurOwn data offers an important chance to ensure all available evidence is fully considered. We remain dedicated to rigorous science and to increasing access for ALS patients in need." Mary Kay Turner, Senior Vice President, Advocacy and Public Affairs at BrainStorm, added, "The ALS community has been a powerful voice in advocating for new approaches to treatment. We stand with advocates in supporting efforts that prioritize both data-driven decision-making and urgency for patients facing this devastating disease." ALS is a rapidly progressive neurodegenerative disease that leads to the loss of motor neurons, severely impairing voluntary muscle movement. Despite recent advances in care, effective disease-modifying treatment options remain limited. Peer-reviewed analyses have demonstrated clinically meaningful benefits in individuals with less-advanced ALS: In the subgroup of participants with baseline ALSFRS-R scores of 35 and above, NurOwn showed statistically significant functional improvement versus placebo. Post-hoc analyses revealed NurOwn-treated participants retained an average of two more ALSFRS-R points than placebo recipients - representing meaningful functional preservation. BrainStorm recently announced new survival data from 10 participants who had previously completed the Phase 3 clinical trial and subsequently entered its Expanded Access Program (EAP). Notably, participants survived more than five years from ALS symptom onset – well above published estimates suggesting that only 10% of people diagnosed with ALS reach that benchmark. The median survival in the group was 6.8 years. These results strongly suggest that the observed survival outcomes in the EAP patients are unlikely to be due to chance alone and further underscore the need for continued scientific exploration. BrainStorm will continue to engage transparently with the FDA, clinicians, advocacy groups, and the broader ALS community, and will provide updates as appropriate. About NurOwn® The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. About BrainStorm Cell Therapeutics Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a second Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTS Investors: Michael Wood Phone: +1 646-597-6983 mwood@ Media: Uri Yablonka, Chief Business Officer Phone: +1 917-284-2911 uri@ Logo - View original content: SOURCE BrainStorm Cell Therapeutics Inc.
Business Insider
26-06-2025
- Business
- Business Insider
BrainStorm files to sell 2.76M shares of common stock for holders
16:41 EDT BrainStorm (BCLI) files to sell 2.76M shares of common stock for holders Confident Investing Starts Here: Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>
Yahoo
20-05-2025
- Business
- Yahoo
Brainstorm Cell Therapeutics Inc (BCLI) Q1 2025 Earnings Call Highlights: Pivotal Phase 3b ...
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. Warning! GuruFocus has detected 2 Warning Signs with BCLI. Release Date: May 19, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. The U.S. FDA has cleared Brainstorm Cell Therapeutics Inc (NASDAQ:BCLI) 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 (NASDAQ: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 (NASDAQ:BCLI) is pursuing multiple funding avenues, including a promising $15 million non-dilutive grant, to ensure the timely commencement of the trial. 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 (NASDAQ: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. Q: Can you start the trial without proper funding? A: Chaim Lebovits, President and CEO, stated that while significant progress has been made in preparing for the trial, initiating and executing it requires robust funding. They are actively pursuing multiple funding avenues, including a $15 million non-dilutive grant and strategic partnerships, to ensure the trial's commencement. Q: What is the significance of naming the trial ENDURANCE? A: Chaim Lebovits explained that the name ENDURANCE resonates with the ALS community, symbolizing the strength and resilience of individuals with ALS and their families. It also reflects Brainstorm's commitment to generating robust data for NurOwn's regulatory approval. Q: Will the company also be producing in the U.S.? A: Hartoun Hartounian, COO, confirmed that expanding manufacturing to the U.S. is a strategic objective. They plan to announce a letter of intent with a U.S.-based facility that has passed FDA inspection, which is crucial for future commercialization and supply chain security. Q: Can you update on any advances in the exosome program? A: Netta Blondheim-Shraga, SVP of R&D, shared that the exosome program is progressing well, showing potential in treating respiratory and inflammatory diseases. They are preparing a manuscript on its efficacy in a COPD model and are pursuing strategic partnerships for clinical development. Q: Have you discussed stratifying by UNC13A with the FDA for the Phase 3b trial? A: Ibrahim Dagher, CMO, noted that while the FDA has not approved any biomarker as a surrogate, the UNC13A findings are exploratory. The trial design under the SPA is agreed upon, but they will continue to explore genetic factors in post hoc analyses. Q: How does the hypoxic stress co-culture with neuron cells support NurOwn's mechanism of action? A: Netta Blondheim-Shraga explained that the enriched media from NurOwn cells showed a protective effect on cells under hypoxic conditions, restoring them to near-normal levels. This supports NurOwn's mechanism and was included in their IND. Q: What are the plans for clinical trial sites and patient enrollment? A: Chaim Lebovits mentioned plans to open approximately 15 clinical trial sites, with details on They aim to enroll 200 patients over two to three years, with the BLA filing contingent on significant results from the first part of the trial. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
