Core Viewpoint - BrainStorm Cell Therapeutics Inc. has been granted an extension until June 30, 2025, by the Nasdaq Hearings Panel to regain compliance with certain continued listing standards of The Nasdaq Capital Market [1][2]. Group 1: Compliance and Listing - The company presented its compliance plan to the Nasdaq Hearings Panel on February 25, 2025, which resulted in the granted extension [2]. - The extension reflects the company's commitment to executing its compliance plan within the specified timeframe [2]. Group 2: Company Initiatives - The company is focused on launching a Phase 3b ALS study for its NurOwn® therapy, reducing overall debt, and pursuing strategic partnerships to enhance its pipeline [3]. - BrainStorm is dedicated to maintaining transparency and will provide updates as necessary during this process [3]. Group 3: Technology and Development - BrainStorm is a leading developer of therapies for neurodegenerative diseases, holding exclusive worldwide licensing rights for the NurOwn® technology platform, which produces autologous MSC-NTF cells [4]. - The NurOwn® cells have received Orphan Drug designation from the FDA and EMA for ALS, and the company is planning a confirmatory Phase 3b trial to evaluate safety and efficacy [4]. - The company has also completed a Phase 3 trial for NurOwn in ALS and is advancing studies in progressive multiple sclerosis, supported by grants from CIRM and NMSS [4].

Company Overview - BrainStorm Cell Therapeutics Inc. is a leading developer of adult stem cell therapeutics for neurodegenerative diseases, focusing on innovative therapies leveraging both autologous and allogeneic platforms [5] - The company holds exclusive worldwide licensing rights for the NurOwn® technology platform, which produces autologous MSC-NTF cells, designated as Orphan Drug by the FDA and EMA for amyotrophic lateral sclerosis (ALS) [5] - BrainStorm is planning a confirmatory Phase 3b trial in ALS with NurOwn to further evaluate its safety and efficacy [5] Upcoming Conference Call - A conference call and webcast will be held on March 31, 2025, at 8:30 AM Eastern Time to update shareholders on financial results for the fourth quarter and year ended December 31, 2024 [1] - Key executives participating in the call include Chaim Lebovits (President and CEO), Hartoun Hartounian (COO), Bob Dagher (CMO), and Alla Patlis (interim CFO) [2] - Participants can submit questions in advance by March 28, 2025, and the call will include a Q&A session with the investment community [3][2] Clinical Development - The company has completed a Phase 3 trial assessing NurOwn in ALS and is advancing clinical studies of NurOwn in progressive multiple sclerosis (MS), supported by grants from the California Institute for Regenerative Medicine (CIRM) and the National MS Society (NMSS) [5] - BrainStorm's exosome technology, derived from MSC-NTF cells, offers customizable, nano-carrier-based solutions for targeted delivery of bioactive molecules, addressing critical pathways in neurodegenerative and respiratory diseases [5]

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].

Financial Performance - Cash, cash equivalents, and restricted cash amounted to approximately $0.35 million as of September 30, 2024, down from $1.3 million as of December 31, 2023[11] - Net loss for Q3 2024 was $2.7 million, compared to a net loss of $1.2 million in Q3 2023, indicating an increase in losses of 125%[12] - Net loss per share for Q3 2024 was $0.51, compared to $0.45 for Q3 2023, reflecting a 13.3% increase in loss per share[13] - The net loss for the nine months ended September 30, 2024, was $(8,650,000), compared to $(11,614,000) in 2023, representing a reduction of about 25.5%[18] - The operating loss for the nine months ended September 30, 2024, was $(8,504,000), an improvement from $(16,635,000) in 2023, showing a decrease in losses of approximately 48.9%[18] - The operating loss for the three months ended September 30, 2024, was $(3,048,000), compared to $(6,035,000) in 2023, indicating a decrease of approximately 49.6%[18] - The net loss for the three months ended September 30, 2024, was $(2,708,000), compared to $(1,226,000) in 2023, reflecting an increase in losses of about 121.5%[18] Expenses - Research and development expenses for Q3 2024 were $1 million, a decrease of 69.7% compared to $3.3 million in Q3 2023[12] - General and administrative expenses for Q3 2024 were $2 million, down from $2.7 million in Q3 2023, representing a 25.9% reduction[12] - Research and development expenses for the nine months ended September 30, 2024, were $2,928,000, compared to $9,048,000 for the same period in 2023, indicating a decrease of approximately 67.7%[18] - General and administrative expenses for the nine months ended September 30, 2024, were $5,576,000, down from $7,587,000 in 2023, reflecting a reduction of about 26.5%[18] - Financial expenses for the nine months ended September 30, 2024, were $(11,000), a significant decrease from $91,000 in 2023[18] Liabilities - Total liabilities as of September 30, 2024, were $8.08 million, a decrease from $9.07 million as of December 31, 2023[17] Clinical Trials - The Phase 3b trial of NurOwn is expected to enroll approximately 200 participants with ALS, receiving 3 doses over 24 weeks[5] - The primary efficacy endpoint of the Phase 3b trial will be the change in ALSFRS-R from baseline to week 24 for NurOwn versus placebo[6] - Promising long-term survival benefits were observed in NurOwn treated patients, with a median survival time of 46.6 months compared to 41.1 months for matched controls[10] - BrainStorm entered into a Memorandum of Understanding with Pluri Inc. for the manufacturing of NurOwn for the Phase 3b trial[3] Share Information - Basic and diluted net loss per share from continuing operations for the nine months ended September 30, 2024, was $(1.80), improved from $(4.35) in 2023[18] - The weighted average number of shares outstanding used in computing basic and diluted net loss per share for the nine months ended September 30, 2024, was 4,793,026, compared to 2,683,700 in 2023[18] Warrants Liability - The gain (loss) on change in fair value of Warrants liability for the nine months ended September 30, 2024, was $(135,000), compared to a gain of $4,930,000 in 2023[18]

Core Viewpoint - BrainStorm Cell Therapeutics Inc. is advancing its NurOwn® program for ALS, focusing on a Phase 3b registration trial, with updates expected in Q4 2024 [1][6]. Company Updates - The company has refined the Phase 3b trial design based on previous study learnings to enhance the likelihood of demonstrating treatment efficacy [2]. - A Memorandum of Understanding (MOU) was signed with Pluri Inc. for the manufacturing of NurOwn® for the upcoming trial [3]. - The Phase 3b trial is expected to enroll approximately 200 ALS participants, with a two-part structure involving both placebo and NurOwn treatments [4]. Clinical Trial Details - The trial will consist of a 24-week Part A with three doses of either NurOwn or placebo, followed by a 24-week open-label Part B where all participants will receive NurOwn [4]. - Primary efficacy will be measured using the ALSFRS-R score, with additional assessments including survival rates and various clinical measures [4]. Financial Performance - As of September 30, 2024, the company reported cash and cash equivalents of approximately $0.35 million, a decrease from $1.3 million year-over-year [7]. - Research and development expenses for Q3 2024 were $1 million, down from $3.3 million in Q3 2023, while general and administrative expenses decreased to $2 million from $2.7 million [7]. - The net loss for Q3 2024 was $2.7 million, compared to a net loss of $1.2 million in the same quarter of 2023 [7]. Long-Term Benefits and Biomarkers - Promising long-term survival benefits were observed in patients treated with NurOwn, with a median survival time of 46.6 months compared to 41.1 months for matched controls [5]. - Consistent reductions in neurofilament light (NfL) levels were noted, indicating ongoing benefits from extended treatment, which aligns with the understanding of NurOwn's mechanism of action [5].

Core Insights - BrainStorm Cell Therapeutics Inc. presented promising data on NurOwn® at the 2024 NEALS Meeting, indicating potential long-term benefits for ALS patients from extended treatment with debamestrocel [1][3] Group 1: NurOwn and ALS Treatment - The Expanded Access Program (EAP) for NurOwn involved 10 ALS participants, with 9 out of 10 remaining alive at the last visit, showing a median survival time of 46.6 months for the debamestrocel group compared to 41.1 months for the matched control [2][3] - A significant reduction in neurofilament light (NfL), a biomarker for neurodegeneration, was observed in participants who received debamestrocel, while those on placebo showed a 37% worsening in NfL levels [2][3] Group 2: Upcoming Phase 3b Trial - The upcoming Phase 3b trial aims to enroll approximately 200 ALS participants, with a two-part design involving 3 doses of either debamestrocel or placebo over 24 weeks, followed by an open-label period [4][6] - Key entry criteria for the trial include ages 18 to 75, ALS diagnosis within 24 months of screening, and specific ALSFRS-R scores [4][6] Group 3: Efficacy and Regulatory Aspects - The primary efficacy endpoint will compare changes in ALSFRS-R scores from baseline to week 24 between the debamestrocel and placebo groups [4] - The company has secured a Special Protocol Assessment (SPA) agreement with the FDA, which reduces regulatory risks associated with the trial [6] Group 4: NurOwn Technology Overview - NurOwn technology utilizes autologous MSC-NTF cells, which are designed to secrete neurotrophic factors targeting neurodegenerative diseases, with a focus on ALS [7][9] - The clinical program has provided insights into ALS pathology and treatment, including pharmacogenomic analysis of clinical outcomes [8]

Company Overview - BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for neurodegenerative diseases, specifically targeting amyotrophic lateral sclerosis (ALS) through its NurOwn® technology platform [5] - The company has received Orphan Drug designation status from the U.S. FDA and the European Medicines Agency for the treatment of ALS [5] Upcoming Events - BrainStorm will participate in the 2024 Maxim Healthcare Virtual Summit from October 15 to 17, where CEO Chaim Lebovits will discuss the planned Phase 3b clinical trial for NurOwn® [1][2] - The Fireside Chat is scheduled for October 15 at 9:30 a.m. ET, providing an opportunity to engage with the investment community [1] Clinical Development - The NurOwn® technology platform involves harvesting MSC-NTF cells from ALS patients, which are then manufactured to secrete neurotrophic factors targeting neurodegenerative diseases [3] - The clinical program has provided insights into ALS pathology, disease progression, and treatment, including a comprehensive analysis of biomarkers and genetic data [4] Research and Funding - BrainStorm has completed a Phase 3 trial for ALS, supported by grants from the California Institute for Regenerative Medicine and the ALS Association [5] - The company has also conducted a Phase 2 open-label multicenter trial for progressive multiple sclerosis, backed by the National MS Society [5]

NEW YORK, Sept. 24, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, today announced the acceptance of two abstracts featuring NurOwn® (MSC-NTF or Debamestrocel) at the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis Consortium (NEALS) Meeting, to take place October 21 - 24, in-person in Clearwater, Florida and virtually."We look forward to sharing the latest update on NurOwn at this year's NEALS meeting. ...