Core Viewpoint - BrainStorm Cell Therapeutics Inc. is committed to advancing its investigational therapy NurOwn for amyotrophic lateral sclerosis (ALS) and welcomes the FDA's review of the data supporting this treatment [1][2][3] Company Commitment and Regulatory Engagement - The company supports the integrity and scientific validity of its data and emphasizes the importance of a comprehensive review of evidence as the regulatory landscape for rare diseases evolves [2][3] - BrainStorm is proceeding with its Phase 3b ENDURANCE trial of NurOwn under an FDA Special Protocol Assessment (SPA) to generate additional data on efficacy and safety for early-stage ALS patients [3][11] Clinical Data and Outcomes - New survival data from 10 participants in the Expanded Access Program (EAP) indicates that they survived more than five years from ALS symptom onset, with a median survival of 6.8 years, significantly higher than the estimated 10% of ALS patients reaching this benchmark [5][6] - In a subgroup with baseline ALSFRS-R scores of 35 and above, NurOwn demonstrated statistically significant functional improvement compared to placebo, with treated participants retaining an average of two more ALSFRS-R points [7] Technology and Research Advancements - NurOwn technology utilizes autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), aimed at delivering therapeutic signals to modulate neuroinflammation and promote neuroprotection [9][10] - The clinical program has provided valuable insights into ALS biology, including pharmacogenomic responses and biomarker data collected over time [11] Community and Advocacy Engagement - The ALS community plays a crucial role in advocating for new treatment approaches, and the company aligns with these efforts to prioritize data-driven decision-making and urgency for patients [4][8]

Core Insights - BrainStorm Cell Therapeutics Inc. announced new survival data from its Expanded Access Program (EAP) for NurOwn® in amyotrophic lateral sclerosis (ALS), highlighting promising long-term survival outcomes for participants [1][4]. Group 1: Survival Data - The EAP included 10 participants who had completed the Phase 3 clinical trial, with survival data analyzed from symptom onset through participation in the trial and EAP [2]. - 90% of participants (9 out of 10) survived more than five years from the onset of ALS symptoms, significantly higher than the approximately 10% survival rate typically expected for ALS patients [7]. - The median survival observed in the EAP cohort was 6.8 years, with a range of 6 to 7 years from symptom onset [7]. Group 2: Clinical Implications - The survival data provide real-world insights into the long-term experience of ALS patients treated with NurOwn, supporting the upcoming Phase 3b clinical trial under FDA Special Protocol Assessment (SPA) [4][8]. - 6 out of 10 patients in the EAP are still alive more than seven years after symptom onset, indicating a clinically meaningful observation [4][7]. - The observed durability of survival underscores the importance of advancing the upcoming registrational Phase 3b controlled clinical trial [4]. Group 3: Technology and Approach - NurOwn® technology utilizes 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 [5][6]. - The platform has received Orphan Drug designation from both the FDA and the European Medicines Agency (EMA) [8]. - BrainStorm is also advancing a proprietary exosome-based platform for delivering therapeutic proteins and nucleic acids, further enhancing its intellectual property portfolio in regenerative medicine [8].

Financial Data and Key Metrics Changes - The company reported significant progress in preparing for the Phase 3b trial of NurOwn, with a focus on securing funding to support trial initiation [24][25] - Financial constraints have been acknowledged, but the company has made strides in regulatory submissions and site selections despite limited resources [13][24] Business Line Data and Key Metrics Changes - The primary focus remains on the clinical development of NurOwn, with the initiation of a pivotal Phase 3b trial aimed at ALS patients [6][10] - The trial design has been agreed upon with the FDA, which is expected to de-risk the regulatory pathway for NurOwn [6][7] Market Data and Key Metrics Changes - The company is actively negotiating with approximately 15 leading clinical centers across the United States for the Phase 3b trial [10] - The trial, named "Endurance," aims to resonate with the ALS community and reflects the company's commitment to developing a therapeutic option for ALS [26] Company Strategy and Development Direction - The company is focused on executing the clinical development plan for NurOwn and is preparing for a significant trial that could lead to regulatory approval [6][12] - There is an emphasis on expanding manufacturing capabilities in the U.S. to support future commercialization and supply chain security [28] Management's Comments on Operating Environment and Future Outlook - Management highlighted the urgency felt by patients and clinicians for innovative therapeutic options in ALS, reinforcing their commitment to executing the trial with scientific rigor [11][12] - The company is pursuing multiple funding avenues, including a promising $15 million non-dilutive grant, to ensure timely trial commencement [25][14] Other Important Information - The company participated in the annual ALF Drug Development Summit, discussing critical areas in ALS therapeutic development [12] - The scientific team is engaged with the academic community to share insights and data, which is crucial for advancing the NurOwn program [12] Q&A Session Summary Question: Can you start the trial without proper funding? - Management emphasized that while significant progress has been made, securing proper funding is essential to commence the trial [24][25] Question: What is the meaning of the trial name "Endurance"? - The name reflects the strength and resilience of the ALS community and the company's commitment to delivering a therapeutic option [26] Question: Will the company also be producing in the U.S.? - The company confirmed plans to expand manufacturing in the U.S. and will announce a letter of intent with a U.S.-based facility soon [28] Question: Can you update on any advances in the exosome program? - The company is encouraged by the progress of its exosome program and is preparing a manuscript detailing its efficacy in preclinical models [32]

Financial Data and Key Metrics Changes - The company is focused on executing the clinical development plan for NurOwn and has received FDA clearance to initiate a pivotal Phase 3b trial for ALS [6][10] - Financial constraints have been acknowledged, but the company has made significant strides in trial preparations despite limited resources [13][25] Business Line Data and Key Metrics Changes - The NurOwn technology platform is central to the company's strategy, with ongoing clinical trials aimed at demonstrating its therapeutic benefits [6][12] - The company is actively negotiating clinical trial agreements with approximately 15 leading clinical centers across the U.S. for the Phase 3b trial [10][25] Market Data and Key Metrics Changes - The ALS community's strong interest in NurOwn is highlighted by the engagement of renowned ALS clinicians and researchers [10] - The company participated in the annual ALF Drug Development Summit, indicating its active involvement in the ALS therapeutic development landscape [12] Company Strategy and Development Direction - The company aims to secure strategic funding to support the trial, with a focus on partnerships and non-dilutive grants [14][26] - Expansion of manufacturing capabilities in the U.S. is a key strategic objective, with plans to announce a letter of intent with a U.S.-based facility [30] Management's Comments on Operating Environment and Future Outlook - Management emphasized the urgency for innovative therapeutic options in ALS and the commitment to executing the trial with scientific rigor [11][12] - The company is optimistic about the potential of NurOwn and is preparing for various funding opportunities to ensure timely trial commencement [14][26] Other Important Information - The trial, named "Endurance," is designed to resonate with the ALS community, reflecting the strength and resilience of ALS patients [27] - The company is encouraged by progress in its exosome program, which shows potential in treating respiratory and inflammatory diseases [34] Q&A Session Summary Question: Can you start the trial without proper funding? - Management stated that while significant progress has been made, proper funding is essential to commence the trial [25][26] Question: What is the meaning of the trial name "Endurance"? - The name was chosen to resonate with the ALS community and reflects the company's commitment to delivering a therapeutic option for ALS [27] Question: Will the company also be producing in the U.S.? - The company confirmed plans to expand its manufacturing footprint in the U.S. and will announce a letter of intent with a U.S.-based facility [30] Question: Can you update on any advances in the exosome program? - Management expressed encouragement regarding the exosome program's progress and is preparing to advance it towards clinical development [34] Question: Have you had any communications with FDA regarding stratification by UNC13A? - Management indicated that while the FDA has not approved any biomarker as a surrogate, they are excited about the genetic discoveries and will explore them further [39][40] Question: Can the mechanism of action be used as part of the data package for the next BLA? - Management confirmed that the protective effect observed in cell cultures could support the data package for future filings [43] Question: How many clinical trial sites are you looking to open? - Management confirmed plans to open approximately 15 clinical trial sites and is finalizing agreements to begin patient enrollment [48][50]

Core Insights - The U.S. FDA has cleared BrainStorm Cell Therapeutics Inc. to initiate a Phase 3b clinical trial of NurOwn® for ALS treatment, marking a significant milestone for the company and the ALS community [1][3] Company Overview - BrainStorm Cell Therapeutics Inc. specializes in developing adult stem cell therapies for neurodegenerative diseases, with its lead investigational therapy being NurOwn® [5][6] - The NurOwn® platform utilizes autologous mesenchymal stem cells to create neurotrophic factor-secreting cells aimed at modulating neuroinflammation and promoting neuroprotection [5] Clinical Trial Details - The Phase 3b trial will enroll approximately 200 participants at leading academic medical centers, featuring a 24-week randomized, double-blind, placebo-controlled phase followed by a 24-week open-label extension [3] - The primary endpoint of the trial is the change from baseline to week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) [3][6] - The trial design has been agreed upon with the FDA under a Special Protocol Assessment (SPA), ensuring the study's endpoints and statistical methodology are suitable for a future Biologics License Application (BLA) submission [2][6] Funding and Development Strategy - The company is actively seeking funding through various channels, including non-dilutive grants, to support the timely launch of the clinical study [3] - BrainStorm has previously completed a Phase 3 trial in ALS and is also advancing a proprietary exosome-based platform for delivering therapeutic proteins and nucleic acids [6]

Clinical Trials and Regulatory Status - NurOwn® has completed Phase 3 ALS and Phase 2 PMS clinical trials, with top-line data announced on November 17, 2020, and March 24, 2021, respectively[83]. - The FDA granted Fast Track designation and Orphan Drug status for NurOwn® for ALS, enhancing its regulatory profile[85]. - On September 27, 2023, the Advisory Committee voted 17 no, 1 yes, and 1 abstention, indicating that NurOwn® did not demonstrate substantial evidence of effectiveness for mild to moderate ALS[83]. - The BLA for NurOwn® was withdrawn on November 3, 2023, in coordination with the FDA, viewed as a withdrawal without prejudice[83]. - The company plans to submit a Special Protocol Assessment request to the FDA for a Phase 3b trial of NurOwn® for ALS[83]. - A productive meeting with the FDA on December 6, 2023, focused on plans for a Special Protocol Assessment (SPA) for a Phase 3b trial of NurOwn®[97]. - The FDA granted a meeting to discuss the regulatory path forward for NurOwn® on November 20, 2023, following the withdrawal of the BLA[97]. - The company submitted a BLA to the FDA for NurOwn® for ALS treatment on September 9, 2022, but received a RTF letter on November 10, 2022, indicating the BLA was not sufficiently complete for review[115]. - The FDA granted a Type A meeting on January 11, 2023, where the company was presented with options to return the BLA to regulatory review, including filing over protest[116]. - The BLA was re-filed on February 7, 2023, after the company requested the FDA to file the NurOwn® BLA for ALS over protest[116]. - An amendment to the BLA was submitted on March 7, 2023, addressing the majority of items in the RTF letter, and the FDA confirmed an ADCOM for the BLA on March 22, 2023[117]. - The Advisory Committee voted on September 27, 2023, with 17 voting no, one voting yes, and one abstention, indicating that NurOwn® did not demonstrate substantial evidence of effectiveness for mild to moderate ALS[117]. - The company announced the withdrawal of the BLA for NurOwn® on November 3, 2023, coordinated with the FDA and viewed as a withdrawal without prejudice[117]. - A meeting with the FDA on December 6, 2023, focused on discussing plans for a SPA for a planned Phase 3b registrational trial for NurOwn®[117]. - 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[118]. Clinical Trial Results - The Phase 2 trial showed that 32.6% of NurOwn® participants met the primary endpoint compared to 27.7% for placebo, but did not reach statistical significance (p=0.453)[112]. - In a pre-specified subgroup with ALSFRS-R baseline scores of 35 or greater, NurOwn® demonstrated a 2.09 point improvement over placebo (p=0.050)[112]. - The Phase 3 trial enrolled rapidly progressing ALS patients but did not achieve statistically significant results, with a responder analysis showing 34.7% for NurOwn® versus 20.5% for placebo (p=0.053)[112]. - The company has completed two Phase 1/2 open-label trials and a Phase 2 double-blind trial, demonstrating tolerability and preliminary signs of activity for NurOwn®[98][99]. - The Phase 2 trial published in JAMA Neurology indicated a slower rate of disease progression in ALS patients treated with NurOwn®[100]. - Key findings from the PMS trial included a 10% mean improvement in the timed 25-foot walk (T25FW) and a 4.8% improvement in the 9-hole peg test (9-HPT) for NurOwn® treated patients compared to historical controls[145]. Financial Overview and Capital Needs - The company aims to raise $2.5 million in stockholders' equity to regain compliance with Nasdaq Listing Rule 5550[87]. - The company reported a net loss of $2,864,000 for the quarter ended March 31, 2025, compared to a net loss of $3,401,000 for the same quarter in 2024, resulting in a net loss per share of $0.45[176]. - Research and development expenditures for the quarter ended March 31, 2025, were $1,304,000, an increase of 35.7% from $961,000 in the same quarter of 2024[172]. - General and administrative expenses increased to $1,785,000 for the quarter ended March 31, 2025, up from $1,513,000 in the prior year, reflecting a rise of 17.9%[174]. - The company had cash, cash equivalents, and restricted cash of $1,826,000 as of March 31, 2025[182]. - Net cash used in operating activities for the quarter ended March 31, 2025, was $1,631,000, primarily due to clinical trial costs and payroll expenses[183]. - The company generated $3,086,000 in net cash from financing activities during the quarter ended March 31, 2025, from sales of common stock and warrants exercise[184]. - The Company anticipates continued losses from clinical development and regulatory activities, necessitating additional capital raises for future trials and commercialization efforts[196]. - Future capital requirements will depend on various factors, including clinical trial costs, regulatory approvals, and commercialization expenses[202]. - The Company expects to incur additional financing needs, potentially through debt or equity, which may dilute existing stockholders[198]. Manufacturing and Production - The NurOwn® manufacturing process is compliant with Good Manufacturing Practice, ensuring quality and safety in production[92]. - The company has developed a validated cryopreservation process for MSC, allowing multiple doses of NurOwn® from a single bone marrow harvest[120]. - The company has contracted with City of Hope and Dana Farber Cancer Institute for the manufacturing of NurOwn® for clinical studies, enhancing production capabilities[121]. - The company has improved the efficiency and stability of NurOwn® production, allowing centralized manufacturing for clinical trials[168]. Intellectual Property and Grants - The company has a comprehensive intellectual property portfolio for NurOwn®, with exclusive commercialization rights held by its Israeli subsidiary[85]. - The Company holds approximately 30 granted patents and is expanding its portfolio to include exosome-based technologies, with a recent patent expected to provide protection until April 10, 2039[150][151]. - The Company was awarded a total of $15,912,390 in grants from the California Institute for Regenerative Medicine to fund the pivotal Phase 3 study of NurOwn® for ALS[147]. Stock and Equity Transactions - The Company entered into a New Distribution Agreement allowing the sale of up to $100 million in Common Stock, with gross proceeds of approximately $706 from 388,059 shares sold under this agreement as of March 31, 2025[191]. - On July 17, 2023, the Company sold 270,270 shares of Common Stock at $27.75 per share, generating gross proceeds of approximately $7.5 million[193]. - A subsequent offering on June 27, 2024, involved the sale of 527,918 registered shares and warrants, yielding gross proceeds of approximately $4.0 million at a purchase price of $5.4 per share[194]. - The Company issued a common warrant to purchase up to 2,762,766 shares of Common Stock on April 1, 2025, subject to stockholder approval[195]. - The exercise price for the Common Warrants from the July 2023 offering is set at $30.00 per share, expiring five years after issuance[193].

Financial Performance - As of March 31, 2025, cash, cash equivalents, and restricted cash totaled approximately $1.8 million[7] - Research and development expenditures for Q1 2025 were $1.3 million, up from $1.0 million in Q1 2024, representing a 30% increase[7] - General and administrative expenses for Q1 2025 were approximately $1.8 million, compared to approximately $1.5 million in Q1 2024, reflecting a 20% increase[7] - The net loss for Q1 2025 was approximately $2.9 million, an improvement from a net loss of approximately $3.4 million in Q1 2024, indicating a 15% reduction in losses[7] - Net loss per share for Q1 2025 was $0.45, compared to $0.75 for Q1 2024, showing a 40% improvement[7] Clinical Trials and Research - The planned Phase 3b clinical trial for NurOwn is designed to enroll approximately 200 early-stage ALS participants[3] - An IND amendment for NurOwn has been submitted to the FDA, marking a significant milestone for the upcoming Phase 3b trial[3] - NurOwn data was selected for presentation at the ISCT 2025 Meeting, highlighting the impact of the UNC13A genotype on clinical outcomes for ALS patients[3] Assets and Liabilities - Total assets as of March 31, 2025, were $3.571 million, compared to $1.832 million as of December 31, 2024, indicating a significant increase[15] - Total liabilities as of March 31, 2025, were $11.028 million, up from $9.596 million as of December 31, 2024[15]

Core Viewpoint - BrainStorm Cell Therapeutics Inc. is making significant progress in the development of its NurOwn® therapy for ALS, with plans for a Phase 3b trial aimed at confirming efficacy in early-stage ALS patients and supporting a new Biologics License Application (BLA) [2][4]. Financial Results - As of March 31, 2025, the company reported cash, cash equivalents, and restricted cash of approximately $1.8 million [4]. - Research and development expenditures for Q1 2025 were $1.3 million, an increase from $1.0 million in Q1 2024 [4]. - General and administrative expenses for Q1 2025 were approximately $1.8 million, compared to $1.5 million in Q1 2024 [4]. - The net loss for Q1 2025 was approximately $2.9 million, an improvement from a net loss of approximately $3.4 million in Q1 2024 [4][14]. - Net loss per share for Q1 2025 was $0.45, compared to $0.75 for Q1 2024 [4][14]. Corporate Updates - The company submitted an IND amendment for NurOwn to the FDA, which is a critical step for the upcoming Phase 3b trial [4]. - The Phase 3b trial, named ENDURANCE, is designed to enroll approximately 200 early-stage ALS participants and will assess changes in ALSFRS-R scores [4]. - NurOwn data was selected for presentation at the ISCT 2025 Meeting, highlighting the impact of the UNC13A genotype on clinical outcomes for ALS patients [4]. - Insights on biomarkers related to NurOwn treatment were presented at the 2025 ALS Drug Development Summit [4]. About NurOwn® - NurOwn® is an investigational therapy utilizing autologous MSC-NTF cells, which are derived from bone marrow and designed to deliver neurotrophic factors to slow or stabilize neurodegenerative diseases [6][8]. - The therapy has received Orphan Drug designation from both the FDA and EMA, and a Phase 3 trial has been completed [8]. Company Overview - BrainStorm Cell Therapeutics Inc. focuses on developing autologous adult stem cell therapies for neurodegenerative diseases, with its proprietary NurOwn® platform aimed at modulating neuroinflammation and promoting neuroprotection [7].

Core Viewpoint - BrainStorm Cell Therapeutics Inc. has rescheduled the release of its financial results for Q1 2025 and an investor conference call, indicating ongoing engagement with investors and stakeholders [1][2]. Company Overview - BrainStorm Cell Therapeutics Inc. is a leading developer of adult stem cell therapies targeting neurodegenerative diseases, utilizing its proprietary NurOwn® platform to produce neurotrophic factor-secreting cells [5]. - The company's lead investigational therapy, NurOwn®, has received Orphan Drug designation from both the FDA and EMA, and a Phase 3 trial for ALS has been completed [6]. Upcoming Events - The financial results will be released post-market on May 15, 2025, and the investor conference call is scheduled for May 19, 2025, at 8:30 a.m. Eastern Time [1][2]. - Participants are encouraged to submit questions in advance, with specific contact details provided for access to the conference call [3]. Clinical Development - BrainStorm has completed a Phase 2 trial of MSC-NTF cells in progressive multiple sclerosis, supported by a grant from the National MS Society [6]. - The company is advancing an allogeneic exosome-based platform for delivering therapeutic proteins and nucleic acids, with a foundational patent recently allowed by the U.S. Patent and Trademark Office [6].

Company Overview - BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of adult stem cell therapeutics aimed at treating neurodegenerative diseases [3][4] - The company's proprietary NurOwn® platform utilizes autologous mesenchymal stem cells (MSCs) to create neurotrophic factor-secreting cells (MSC-NTF cells) [3][4] Upcoming Conference Call - A conference call is scheduled for May 15, 2025, at 8:30 a.m. Eastern Time to discuss the financial results for Q1 2025 and provide a corporate update [1][2] - The call will feature BrainStorm's President & CEO Chaim Lebovits, along with Dr. Bob Dagher, Chief Medical Officer, and Alla Patlis, Interim CFO [2] Clinical Development - NurOwn® is the lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the FDA and EMA [4] - A Phase 3 trial for ALS has been completed, and a confirmatory Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA [4] - The clinical program has provided insights into ALS disease biology, including pharmacogenomic responses and biomarker data [4] Additional Research and Development - BrainStorm has completed a Phase 2 open-label multicenter trial of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society [4] - The company is also advancing an allogeneic exosome-based platform for delivering therapeutic proteins and nucleic acids, with a foundational patent recently allowed by the U.S. Patent and Trademark Office [4]