NEW YORK, April 10, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading biotechnology company developing cutting-edge cell therapies for neurodegenerative diseases, today announced the submission of an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) for NurOwn®, the company's autologous mesenchymal stem cell therapy for amyotrophic lateral sclerosis (ALS). This important milestone sets the stage for the initiation of BrainStorm's highly an ...

Brainstorm Cell Therapeutics Inc. (BCLI) Q4 2024 Earnings Conference Call March 31, 2025 08:30 AM ET Company Participants Operator - Conference OperatorMichael Wood - Conference Call HostChaim Liebowitz - President and CEOAla Patelist - Interim Chief Financial OfficerUnidentified - Unspecified Company RepresentativeDr. Haro Hartonian - EVP and Chief Operating OfficerDr. Bob Dagger - EVP and Chief Medical Officer Conference Call Participants Jason McCarthy - Analyst, Maxim GroupDavid Bouts - Analyst, Zacks S ...

Clinical Trials and Regulatory Status - NurOwn® has completed Phase 3 ALS and Phase 2 PMS clinical trials, with positive top-line data announced on March 24, 2021, for PMS[14]. - The FDA granted a Special Protocol Assessment (SPA) agreement on April 9, 2024, validating the Phase 3b trial design for NurOwn® in ALS[15]. - The FDA's Advisory Committee voted on September 27, 2023, with 17 voting no, indicating that NurOwn® did not demonstrate substantial evidence of effectiveness for mild to moderate ALS[14]. - The BLA for NurOwn® was withdrawn on November 3, 2023, in coordination with the FDA, viewed as a withdrawal without prejudice[14]. - The Phase 3 clinical trial for NurOwn® in ALS did not achieve statistically significant results, with 32.6% of participants meeting the primary endpoint compared to 27.7% for placebo (p=0.453)[47]. - The average change in ALSFRS-R total score from baseline to Week 28 was -5.52 for NurOwn® versus -5.88 for placebo, showing a difference of 0.36 (p=0.693)[47]. - In a pre-specified subgroup with ALSFRS-R baseline scores of 35 or greater, NurOwn® demonstrated a 34.6% responder rate compared to 15.6% for placebo (p=0.305)[47]. - The FDA issued a Refusal to File (RTF) letter on November 10, 2022, stating that the BLA was not sufficiently complete for substantive review, citing issues related to evidence of effectiveness and CMC items[49]. - Following a Type A meeting with the FDA on January 11, 2023, the company requested to file the BLA over Protest on February 6, 2023, which was confirmed by the FDA on February 7, 2023[50]. - An amendment to the BLA was submitted on March 7, 2023, addressing most items in the RTF letter, and the FDA confirmed an Advisory Committee (ADCOM) meeting for the BLA on March 27, 2023[52]. - The ADCOM meeting held on September 27, 2023, resulted in a vote of 17 no, 1 yes, and 1 abstention, indicating that NurOwn® did not demonstrate substantial evidence of effectiveness for mild to moderate ALS[52]. - The BLA for NurOwn® was officially withdrawn on November 3, 2023, in coordination with the FDA, and is considered a withdrawal without prejudice[64]. - A meeting with the FDA on December 6, 2023, focused on discussing plans for a Special Protocol Assessment (SPA) for a planned Phase 3b trial for NurOwn®[65]. - The SPA request for the Phase 3b clinical trial was submitted on February 23, 2024, and written agreement from the FDA was received on April 9, 2024, validating the trial protocol[65]. Manufacturing and Development - The NurOwn® manufacturing process includes harvesting stem cells from the patient's bone marrow and administering MSC-NTF cells intrathecally, which has been shown to be well tolerated[26]. - The company has developed a validated cryopreservation process for MSC, allowing multiple doses of NurOwn® from a single bone marrow harvest, enhancing manufacturing efficiency[53]. - As of November 1, 2023, the company optimized its manufacturing capabilities by leasing a GMP-certified cleanroom manufacturing center, significantly enhancing production capacity for NurOwn®[56]. - The company is actively engaged in research and development of NurOwn® and MSC-NTF derived Exosomes for various neurodegenerative disorders and ARDS, focusing on their unique ability to cross the blood-brain barrier[95]. - The company has improved the efficiency and stability of NurOwn® production, allowing centralized manufacturing and distribution to clinical trial sites[106]. - The company intends to establish fully-equipped cGMP-certified Cell-Processing Centers for NurOwn® production and distribution[203]. Financial and Operational Status - The company currently employs 29 employees, with a focus on recruiting and retaining talent to support the development of novel cell therapies[17]. - The company has not generated any operational revenues for the fiscal years ended December 31, 2021, 2022, 2023, or 2024[217]. - The company expects to incur substantial and increasing operating losses for the foreseeable future as it increases spending on development and commercialization efforts[217]. - The company will need to raise additional capital to fund its business objectives and operations, with no assurance that such funding will be available on favorable terms[212]. - The company has a history of losses and anticipates continuing to incur losses, which will adversely impact working capital, total assets, and stockholders' equity[217]. - The company faces risks related to compliance with Nasdaq listing requirements, which could affect stock price and liquidity[210]. - The company is subject to potential securities class action litigation and other stockholder litigation[210]. Intellectual Property and Market Position - The company has a strong intellectual property portfolio and has received Fast Track designation and Orphan Drug status from the FDA for ALS[16]. - The company holds approximately 30 granted patents and is expanding its portfolio to include exosome-based technologies[87]. - The company has a global patent portfolio protecting NurOwn® and exosome-based technology, covering key markets including the U.S., Europe, and Japan[121]. - The company maintains a commercial relationship with Ramot at Tel Aviv University under a Research and License Agreement, granting rights to certain intellectual property developed at the university[127]. - The Israeli subsidiary is required to pay royalties ranging from 3% to 5% of all net sales derived from the licensed intellectual property, and 20% to 25% on revenues from sublicensing[128]. Market and Regulatory Environment - The FDA's approval process for biological products includes multiple phases of clinical trials, with Phase 1 focusing on safety and dosage, Phase 2 on efficacy, and Phase 3 on broader patient populations[143]. - The FDA may issue a Complete Response Letter detailing deficiencies in the BLA, which could delay or refuse approval if regulatory criteria are not met[149]. - The approval process may involve inspections of manufacturing facilities to ensure compliance with cGMP and Good Clinical Practice (GCP) requirements[147]. - The FDA may grant orphan designation to drugs for rare diseases, providing benefits like grant funding and potential seven years of exclusivity upon first approval[152]. - The FDA's Fast Track designation allows for rolling review of marketing applications for products addressing serious conditions, expediting the approval process[155]. - The company intends to pursue regulatory approval for NurOwn® in the U.S., Europe, Japan, and Israel, which requires substantial time and financial resources[134]. - The company is aware of the potential impact of healthcare reform measures on future revenues and pricing pressures[201]. Research and Clinical Findings - Significant increases in neurotrophic factors and reductions in inflammatory markers were observed in cerebrospinal fluid samples post-treatment with NurOwn®[43]. - The Phase 2 study showed a slower rate of disease progression in ALS patients treated with NurOwn®, as measured by ALSFRS-R and Forced Vital Capacity[35]. - Key findings from the Phase 2 PMS trial included a 10% mean improvement in the timed 25-foot walk and a 4.8% improvement in the 9-hole peg test for NurOwn® treated patients[79]. - The company presented new biomarker data at the 3rd Annual ALS Drug Development Summit, suggesting ALS patients may benefit from longer-term treatment with NurOwn®[94]. - Preclinical studies showed that intratracheal administration of NurOwn® derived exosomes resulted in statistically significant improvements in lung parameters, including functional lung recovery and reduction in pro-inflammatory cytokines[98]. - The company presented compelling preclinical data demonstrating the potential of exosome-based technology in treating acute lung injuries and neurodegenerative diseases[112]. Legislative and Compliance Issues - The Act for ALS was signed into law on December 23, 2021, authorizing up to $100 million per year for five years, totaling $500 million for research on neurodegenerative diseases like ALS[157]. - The FDA's Action Plan for Rare Neurodegenerative Diseases, released on June 23, 2022, outlines a five-year strategy to enhance scientific achievement and innovation in ALS drug development[158]. - The FDA and NIH launched the Critical Path for Rare Neurodegenerative Diseases (CP-RND) on September 14, 2022, aimed at advancing understanding and treatment development for ALS[158]. - The federal Anti-Kickback Statute and False Claims Act impose significant compliance requirements on the company’s operations[179]. - The company may face penalties for non-compliance with price reporting metrics required by the government[176]. - The EU GDPR and UK GDPR impose strict data protection requirements that could lead to substantial fines for violations[183]. - Changes in governmental healthcare programs could significantly impact the success of the company's stem cell therapies[173]. Competitive Landscape - The ongoing Phase 3 trial for a competitor's ALS treatment has an estimated completion date of Q2 2026[204]. - Currently, there are four FDA-approved ALS therapies, each showing modest improvements in survival or ALS function[207].

Exhibit 99.1 BrainStorm Cell Therapeutics Announces Full Year 2024 Financial Results and Provides Corporate Update Conference call and webcast at 8:30 a.m. Eastern Time today NEW YORK, March 31, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the fiscal year ended December 31, 2024, and provided a corporate update. "2024 was a year of resilience and progress for Br ...

Conference call and webcast at 8:30 a.m. Eastern Time today NEW YORK, March 31, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the fiscal year ended December 31, 2024, and provided a corporate update. Fourth Quarter 2024 and Recent Highlights December 30, 2024: Issued a letter to shareholders summarizing key corporate achievements and strategic priorities for 2025 ...

Call Scheduled for March 31, 2025, at 8:30 AM Eastern Time NEW YORK, March 24, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, announced today that it will hold a conference call and webcast to update shareholders on financial results for the fourth quarter and year ended December 31, 2024, at 8.30 a.m. Eastern Time on March 31, 2025. BrainStorm's President and Chief Executive Officer, Chaim Lebovits, ...

Core Insights - The company achieved a significant milestone by securing a Special Protocol Assessment (SPA) with the FDA for the Phase 3b clinical trial of NurOwn, which de-risks the regulatory aspects of its clinical development pathway [2][4] - The company has demonstrated the potential of NurOwn to reduce neurofilament light (NfL) levels, a key biomarker of neurodegeneration in ALS patients, indicating its ability to mitigate neuronal damage [7] - The leadership team has been strengthened with the addition of Dr. Haro Hartounian as COO and the promotion of Bob Dagher, M.D., to CMO, enhancing operational and clinical development capabilities [8] - The company is advancing its exosome platform, which has significant potential for neurodegenerative and respiratory diseases, supported by a recent Notice of Allowance from the U.S. Patent and Trademark Office [9] - The company has raised approximately $8 million since the ADCOM results and is actively pursuing additional funding sources to support the Phase 3b trial and exosome platform development [10] Research and Development - The Phase 3b trial for NurOwn is focused on early-stage ALS patients, aiming to provide robust evidence of the treatment's efficacy [4] - Exploratory analyses have highlighted potential correlations between NurOwn treatment and other biomarkers associated with inflammation and neuroprotection, reinforcing the therapy's multifaceted benefits [3] Corporate Developments - The company entered a Memorandum of Understanding (MOU) with Pluri Inc. for the clinical manufacturing of NurOwn, which will enhance its manufacturing capabilities [17] - The company has maintained financial discipline and operational excellence despite challenges in the biotechnology sector, including a difficult financing environment [10] Future Outlook - The company is entering 2025 with optimism, focusing on the launch of the Phase 3b trial and the advancement of its exosome platform [12][21] - The recent FDA approval of Ryoncil, the first MSC-based therapy, has inspired confidence in the company's own MSC-based therapeutic approach with NurOwn [21]

Core Points - BrainStorm Cell Therapeutics Inc. has received a Notice of Allowance from the U.S. Patent & Trademark Office for its proprietary exosome technology patent application [1][2] - The patent covers exosomes derived from MSC-NTF cells, which contain neurotrophic factors and other biological molecules, enhancing BrainStorm's intellectual property portfolio [2][3] - The company is focused on advancing its NurOwn therapy for ALS and plans to initiate a Phase 3b registrational trial [3][5] Company Overview - BrainStorm Cell Therapeutics Inc. specializes in developing therapies for neurodegenerative diseases, utilizing both autologous and allogeneic platforms [5] - The company holds exclusive worldwide licensing rights for the NurOwn technology platform, which has received Orphan Drug designation for ALS from the FDA and EMA [5] - BrainStorm's exosome technology represents an allogeneic therapeutic platform with significant potential in treating respiratory and neurodegenerative diseases [4][5] Exosome Technology - Exosomes are nano-sized extracellular vesicles that facilitate intercellular communication and transport biological materials [4] - BrainStorm's proprietary exosomes are developed from MSC-NTF cells and can deliver bio-active molecules non-invasively, targeting specific CNS pathways [4] - The exosomes exhibit unique immunomodulatory properties and customizable cargo, enhancing their therapeutic potential [4][5]

Clinical Trials and Regulatory Status - NurOwn® has completed Phase 3 ALS and Phase 2 PMS clinical trials, with positive top-line data announced on March 24, 2021, for PMS[90]. - The FDA granted Fast Track designation and Orphan Drug status for NurOwn® for ALS, indicating its potential significance in treating this condition[93]. - On September 27, 2023, the Advisory Committee voted 17 no, 1 yes, and 1 abstention, stating that NurOwn® did not demonstrate substantial evidence of effectiveness for mild to moderate ALS[90]. - The BLA for NurOwn® was withdrawn on November 3, 2023, in coordination with the FDA, viewed as a withdrawal without prejudice[90]. - A Special Protocol Assessment (SPA) request for a planned Phase 3b clinical trial of NurOwn® was submitted to the FDA on February 23, 2024, and agreement was received on April 9, 2024[95][97]. - The company has reached alignment with the FDA on Chemistry, Manufacturing, and Controls (CMC) aspects of the Phase 3b clinical trial for NurOwn®[102]. - The FDA granted a meeting to discuss the regulatory path forward for NurOwn® on December 6, 2023, focusing on a planned Phase 3b registrational trial[114]. - The FDA granted written agreement on the design for a Phase 3b trial of NurOwn® on April 9, 2024, validating the clinical trial protocol and statistical analysis[140]. - The company plans to submit a Special Protocol Assessment (SPA) request for a Phase 3b clinical trial of NurOwn® for ALS, with the goal of securing FDA agreement on the trial design[150]. Clinical Data and Efficacy - The Phase 2 U.S. study showed a responder analysis of 32.6% for NurOwn® versus 27.7% for placebo, but did not reach statistical significance (p=0.453)[129]. - In a pre-specified subgroup with ALSFRS-R baseline score of 35 or greater, NurOwn® showed a 34.6% responder rate compared to 15.6% for placebo (p=0.305)[131]. - The average change in ALSFRS-R total score from baseline to Week 28 was -1.56 for NurOwn® versus -3.65 for placebo, indicating a statistically significant improvement (p=0.050)[131]. - The Phase 3 trial enrolled rapidly progressing ALS patients, but did not achieve statistically significant results despite showing numerical improvements[128]. - Clinical data from Phase 1/2 trials indicated a slower rate of disease progression post-treatment, as measured by ALSFRS-R and Forced Vital Capacity[117]. - The treatment effects were more pronounced in the rapid progressor subgroup, with significant increases in neurotrophic factors and reductions in inflammatory markers observed post-treatment[124]. - NurOwn® treatment outcomes can be predicted by baseline ALS function and key CSF biomarkers, showing decreases in neuroinflammatory and neurodegenerative markers in treated participants compared to placebo[134]. - A statistically significant treatment difference of more than 2 points (p=0.050) was observed in the average change from baseline in ALSFRS-R for participants with a baseline score of at least 35[135]. - The company presented new biomarker data suggesting that ALS patients may benefit from longer-term treatment with NurOwn® at the 3rd Annual ALS Drug Development Summit[187]. Financial Performance and Funding - For the three months ended September 30, 2024, the company incurred operating costs and expenses of approximately $3,048,000, a decrease of 49.5% compared to $6,035,000 for the same period in 2023[201]. - Research and development expenditures, net for the three months ended September 30, 2024 were $1,045,000, a decrease of 68.6% compared to $3,330,000 for the same period in 2023[202]. - General and administrative expenses for the three months ended September 30, 2024 were $2,003,000, a decrease of 25.9% compared to $2,705,000 for the same period in 2023[205]. - Net loss for the three months ended September 30, 2024 was $2,708,000, compared to a net loss of $1,226,000 for the same period in 2023, resulting in a net loss per share of $0.51[209]. - Net loss for the nine months ended September 30, 2024 was $8,650,000, an improvement compared to a net loss of $11,614,000 for the same period in 2023, with a net loss per share of $1.80[210]. - Cash provided by financing activities for the nine months ended September 30, 2024, was $6,909,000 from sales of common stock under the August 9, 2021 ATM program and June 2024 sales of unregistered securities[218]. - The Company anticipates needing to raise substantial additional financing to fund operations, including conducting a Phase 3b trial for NurOwn®[228]. - The Company has entered into multiple funding alternatives, including public and private sales of Common Stock and warrants, to meet its capital needs[213]. - The Company may not be able to continue as a going concern if it cannot raise additional capital[215]. - Net cash used in operating activities for the nine months ended September 30, 2024, was $8,045,000, primarily due to clinical trial costs, rent, payroll, and legal expenses[217]. Legal Matters - Five lawsuits were filed in the U.S. District Court for the Southern District of New York by purported shareholders between November 1, 2023, and April 12, 2024[237]. - A Securities Complaint was filed against the Company alleging violations related to NurOwn® for the treatment of ALS, seeking damages for an allegedly inflated stock price between February 18, 2020, and September 27, 2023[238]. - The Derivative Complaints assert state law claims for breach of fiduciary duty and unjust enrichment against certain officers and directors, relating to the same issues as the Securities Complaint[239]. - The Company intends to vigorously defend against the lawsuits filed against it[240]. Company Operations and Strategy - The company currently employs 29 employees across the United States and Israel, with a focus on recruiting and retaining talent to support its objectives[94]. - A Memorandum of Understanding (MOU) was entered into with Pluri Inc. for the manufacturing of NurOwn® for the planned Phase 3b trial[106]. - The company is actively exploring the next steps for NurOwn®, including publication of emerging clinical data and development of a protocol for an additional clinical study[90]. - The proprietary technology of NurOwn® involves a multi-step process that includes harvesting stem cells from the patient's bone marrow and administering MSC-NTF cells intrathecally[108]. - The company has optimized its manufacturing capabilities for NurOwn® by leveraging partnerships and operational resources, including a GMP-certified cleanroom facility[144]. - A partnership with Catalent for manufacturing commercial quantities of NurOwn® was established, with successful technology transfer completed by December 7, 2021[145]. - The company is actively engaged in research and development for clinical applications of NurOwn® and MSC-NTF derived exosomes in various neurodegenerative disorders and ARDS[189]. - The company is focused on developing and optimizing large-scale cell culture processes for MSC-NTF exosomes, which may have clinical potential for treating ARDS[190]. - The company aims to advance NurOwn® as a treatment for Alzheimer's Disease, focusing on its capability to target multiple biological pathways[166]. - The company has established a broad portfolio of 27 granted patents and 23 patent applications globally to protect its proprietary technologies[171]. - The company has received patents for NurOwn® in multiple jurisdictions, including the United States, Canada, Japan, Europe, Hong Kong, Brazil, and Israel[185].

Exhibit 99.1 BrainStorm Cell Therapeutics Announces Third Quarter 2024 Financial Results and Provides Corporate Update Conference call planned for later in Q4 2024 to provide updates on NurOwn® program NEW YORK, November 14, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the third quarter ended September 30, 2024 and provided a corporate update. "BrainStorm's prim ...