Core Viewpoint - BrainStorm Cell Therapeutics Inc. has submitted an Investigational New Drug (IND) amendment for NurOwn®, an autologous mesenchymal stem cell therapy for amyotrophic lateral sclerosis (ALS), paving the way for a Phase 3b clinical trial in collaboration with the FDA under a Special Protocol Assessment (SPA) [1][2] Group 1: IND Submission and Trial Design - The IND amendment submission and FDA's agreement on the trial design under the SPA represent a significant advancement towards making NurOwn available to ALS patients [2] - The Phase 3b trial will enroll approximately 200 ALS participants and will consist of two parts: a 24-week double-blind period followed by an open-label extension [2] - The primary efficacy endpoint will evaluate changes in ALSFRS-R scores from baseline to week 24, comparing the disease progression between NurOwn and placebo groups [2] Group 2: NurOwn Technology and Company Background - NurOwn® technology involves autologous MSC-NTF cells derived from bone marrow, designed to deliver neurotrophic factors and immunomodulatory cytokines to slow or stabilize disease progression in neurodegenerative disorders [4] - BrainStorm Cell Therapeutics is a leading developer of autologous adult stem cell therapeutics for neurodegenerative diseases and holds exclusive rights to the NurOwn® technology platform [5] - The company has received Orphan Drug designation from the FDA and EMA for ALS treatment and has completed a Phase 3 pivotal trial investigating the safety and efficacy of autologous MSC-NTF cells [5]

Financial Data and Key Metrics Changes - Research and development expenditures for the year ended December 31, 2024, were approximately $4.7 million, a decrease from $10.7 million in 2023 [22] - General and administrative expenses for the same period were about $7 million, down from $10.7 million in 2023 [22] - The net loss for the year ended December 31, 2024, was approximately $11.6 million, or $2.31 per share, compared to a net loss of approximately $17.2 million, or $6 per share, for 2023 [22][23] - Cash equivalents and restricted cash were approximately $0.4 million at the end of December 2024, down from $1.5 million at the end of December 2023 [23] Business Line Data and Key Metrics Changes - The company is focused on advancing the Phase 3B trial of Neuron, its investigational treatment for ALS, with preparations for patient enrollment underway [7][8] - The company has established partnerships with leading clinical centers and a clinical research organization to support trial execution [12][13] Market Data and Key Metrics Changes - The company is actively pursuing multiple funding avenues, including licensing non-core assets and exploring non-dilutive financing opportunities such as grants [15][34] - The anticipated funding requirement for the trial is approximately $20-30 million annually, with a focus on achieving key milestones to improve market valuation [18][35] Company Strategy and Development Direction - The primary focus for 2025 is on clear execution of the Phase 3B trial, with a strong belief in Neuron's potential to significantly impact ALS patients [8][20] - The company is committed to transparency and rigorous regulatory processes, emphasizing the importance of robust data to validate Neuron's efficacy [17][20] Management's Comments on Operating Environment and Future Outlook - Management acknowledges the urgency and frustration regarding trial timelines but emphasizes the complexity of regulatory processes as a reason for perceived delays [29] - The company is confident in its ability to navigate financial challenges and is committed to ensuring that the team's dedication will be recognized as milestones are achieved [51] Other Important Information - The company has entered into a warrant-inducing agreement expected to raise approximately $1.64 million in gross proceeds [23] - Management has implemented cost-saving measures, including salary reductions, to prioritize the trial's progress [50] Q&A Session Summary Question: Concerns about delays in initiating the Phase 3B trial - Management understands the frustration regarding the trial timeline, attributing delays to the complexity of regulatory processes and negotiations with clinical sites [29][30] Question: Financial situation and funding plans - The company is actively pursuing multiple funding avenues, including a recent warrant inducement agreement and exploring non-dilutive financing through grants [34][35] Question: Addressing skepticism about Neuron's efficacy - Management acknowledges skepticism but emphasizes the strong support from key opinion leaders and the commitment to providing robust data to validate Neuron's potential [41][42] Question: Status of manufacturing facilities - The company has contracted with a clinical manufacturing partner and is planning to establish a U.S.-based manufacturing center to ensure a consistent supply of Neuron [46] Question: Ensuring operational continuity amid financial constraints - Management has implemented strategic cost-saving measures and remains focused on advancing the trial despite financial challenges [50][51] Question: Why not refile the original BLA - Management believes that generating robust data through the Phase IIIb trial is the most strategic path forward to address specific concerns raised previously [53]

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].

Financial Performance - For the fiscal year ended December 31, 2024, BrainStorm reported a net loss of approximately $11.6 million, a decrease from a net loss of approximately $17.2 million in 2023, representing a 32% improvement[9]. - Research and development expenditures for 2024 were $4.7 million, down from $10.7 million in 2023, indicating a 56% reduction in R&D spending[9]. - General and administrative expenses decreased to approximately $7.0 million in 2024 from $10.7 million in 2023, reflecting a 35% reduction[9]. - Cash and cash equivalents were approximately $0.4 million as of December 31, 2024, down from $1.5 million at the end of 2023[9]. Clinical Trials and Partnerships - The company is preparing for a Phase 3b trial of NurOwn, which will involve approximately 200 participants with ALS, having secured a Special Protocol Assessment (SPA) agreement with the FDA[2]. - A strategic partnership with Pluri was announced to support manufacturing for the Phase 3b NurOwn trial, enhancing operational readiness[8]. Funding and Compliance - BrainStorm has entered into a warrant inducement agreement expected to raise approximately $1.64 million, anticipated to close on or about April 1, 2025[6]. - The company regained compliance with Nasdaq's minimum bid price requirement on October 30, 2024, ensuring continued listing on the Nasdaq Capital Market[8]. Intellectual Property and Research Findings - BrainStorm received a Notice of Allowance for a new patent covering exosome platform technology, strengthening its intellectual property portfolio[5]. - The company presented findings at the 2024 NEALS Meeting, showing a statistically significant survival benefit for ALS patients treated with NurOwn[8].

Core Insights - BrainStorm Cell Therapeutics Inc. reported financial results for the fiscal year ended December 31, 2024, highlighting resilience and progress in developing therapies for neurodegenerative diseases, particularly ALS [2][7]. Financial Performance - Cash, cash equivalents, and short-term bank deposits were approximately $0.4 million as of December 31, 2024, down from $1.5 million as of December 31, 2023 [10]. - Research and development expenditures for the year were $4.7 million, a decrease from $10.7 million in 2023 [10][17]. - General and administrative expenses were approximately $7.0 million for 2024, compared to $10.7 million in 2023 [10][17]. - The net loss for the year was approximately $11.6 million, an improvement from a net loss of $17.2 million in 2023 [10][17]. - Net loss per share for 2024 was $2.31, compared to $6.00 in 2023 [10][17]. Corporate Developments - The company is preparing for a Phase 3b trial of NurOwn in ALS, with an agreement on a Special Protocol Assessment (SPA) from the FDA, which reduces regulatory risks [2]. - A new patent covering exosome platform technology was allowed by the U.S. Patent and Trademark Office, enhancing the company's intellectual property portfolio [5]. - A warrant inducement agreement is expected to raise approximately $1.64 million, closing around April 1, 2025 [6][10]. Clinical and Regulatory Updates - NurOwn technology, which utilizes autologous MSC-NTF cells, aims to target pathways in neurodegenerative disorders and has received Orphan Drug designation from the FDA and EMA for ALS treatment [12][13]. - The company has completed a Phase 3 pivotal trial in ALS and is actively working on the regulatory approval process for NurOwn [13].

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]