Financial Data and Key Metrics Changes - The company provided forward-looking statements regarding its potential future business operations and performance, particularly in the treatment of neurodegenerative disorders such as ALS [2] - There is an emphasis on the sufficiency of the company's existing capital resources for continuing operations in 2025 and beyond [2] Business Line Data and Key Metrics Changes - Specific financial data and metrics for different business lines were not disclosed in the provided content [1] Market Data and Key Metrics Changes - The company discussed the market potential for the treatment of neurodegenerative disorders, indicating a focus on this area for future growth [2] Company Strategy and Development Direction - The management highlighted the importance of developing strategic collaborations and partnerships to support business planning efforts [2] - The company is focused on the safety and clinical effectiveness of the NurOwn technology platform and its related clinical development programs [2] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the numerous risks and uncertainties that could affect the company's results, which are beyond its control [2] - There was no specific commentary on the current operating environment or future outlook provided in the content [1] Other Important Information - The call included a reminder that forward-looking statements are subject to risks and uncertainties, and the company undertakes no obligation to publicly update these statements [2] Q&A Session Summary - No specific questions or answers from the Q&A session were provided in the content [1]

PART I – FINANCIAL INFORMATION [Item 1. Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) The company's unaudited interim financial statements show a net loss of $5.8 million and substantial doubt about its ability to continue as a going concern [Interim Condensed Consolidated Balance Sheets](index=7&type=section&id=Interim%20Condensed%20Consolidated%20Balance%20Sheets) The balance sheet as of June 30, 2025, shows total assets of $2.6 million and a total stockholders' deficit of $6.1 million Consolidated Balance Sheet Highlights (in thousands) | Balance Sheet Item | June 30, 2025 (Unaudited) | December 31, 2024 (Audited) | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $824 | $187 | | Total current assets | $1,515 | $385 | | Total assets | $2,566 | $1,832 | | **Liabilities & Stockholders' Deficit** | | | | Total current liabilities | $8,528 | $8,978 | | Total liabilities | $8,623 | $9,596 | | Accumulated deficit | $(232,403) | $(226,636) | | Total stockholders' deficit | $(6,057) | $(7,764) | [Interim Condensed Consolidated Statements of Comprehensive Loss](index=9&type=section&id=Interim%20Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) The company recorded a net loss of $5.77 million for the six months ended June 30, 2025, a slight decrease from the prior year Statement of Comprehensive Loss (in thousands, except per share data) | Metric | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Research and development, net | $2,424 | $1,883 | $1,120 | $922 | | General and administrative | $3,238 | $3,573 | $1,453 | $2,060 | | **Operating loss** | **$(5,662)** | **$(5,456)** | **$(2,573)** | **$(2,982)** | | **Net loss** | **$(5,767)** | **$(5,942)** | **$(2,903)** | **$(2,541)** | | Basic and diluted net loss per share | $(0.77) | $(1.35) | $(0.34) | $(0.60) | [Interim Condensed Consolidated Statements of Cash Flows](index=12&type=section&id=Interim%20Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operations was $5.1 million, offset by $5.8 million raised from financing activities during the first half of 2025 Cash Flow Summary (in thousands) | Cash Flow Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(5,133) | $(4,746) | | Net cash provided by financing activities | $5,787 | $6,909 | | Increase (decrease) in cash, cash equivalents and restricted cash | $654 | $2,163 | | Cash, cash equivalents and restricted cash at end of the period | $1,025 | $3,648 | [Notes to Interim Condensed Consolidated Financial Statements](index=13&type=section&id=Notes%20to%20Interim%20Condensed%20Consolidated%20Financial%20Statements) Key notes highlight the company's delisting to the OTCQB market, substantial doubt about its going concern status, and recent capital-raising activities - The company's common stock was **delisted from The Nasdaq Capital Market** and began trading on the OTCQB Venture Market on July 18, 2025, due to failure to meet the minimum shareholder equity requirement[38](index=38&type=chunk)[39](index=39&type=chunk)[82](index=82&type=chunk) - The company has incurred operating losses since inception, with an **accumulated deficit of approximately $232 million** as of June 30, 2025, raising **substantial doubts about its ability to continue as a going concern**[40](index=40&type=chunk)[41](index=41&type=chunk) - During the six months ended June 30, 2025, the company sold **2,311,940 shares for gross proceeds of approximately $4.15 million** under its At-the-Market (ATM) offering[54](index=54&type=chunk) - In March 2025, the company entered into a warrant inducement agreement, **raising approximately $1.64 million** and issuing new warrants to purchase additional shares[57](index=57&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=26&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the regulatory pathway for its NurOwn® cell therapy, its precarious financial condition, and reliance on equity financing [Company Overview and Recent Highlights](index=26&type=section&id=Company%20Overview%20and%20Recent%20Highlights) Recent highlights include the delisting from Nasdaq, FDA clearance for a Phase 3b trial for NurOwn® in ALS, and a new manufacturing agreement - The company's common stock was **delisted from Nasdaq** and began trading on the OTCQB market on July 18, 2025, due to non-compliance with the minimum shareholder equity requirement[90](index=90&type=chunk) - On May 19, 2025, the company announced that the **FDA cleared the initiation of its Phase 3b clinical trial** of NurOwn® for the treatment of ALS[90](index=90&type=chunk) - The company signed a Letter of Intent with Minaris Advanced Therapies on May 27, 2025, to **manufacture NurOwn® for its upcoming clinical trial**[97](index=97&type=chunk) [NurOwn® Clinical Program](index=32&type=section&id=NurOwn%C2%AE%20Clinical%20Program) The company withdrew its BLA for NurOwn® in ALS but subsequently secured an FDA Special Protocol Assessment for a new Phase 3b trial - The company **withdrew its Biologics License Application (BLA) for NurOwn® in ALS** on November 3, 2023, after an Advisory Committee voted against its effectiveness[100](index=100&type=chunk)[123](index=123&type=chunk) - In April 2024, the company received **written agreement from the FDA under a Special Protocol Assessment (SPA)** for the design of a new Phase 3b trial of NurOwn® in ALS[86](index=86&type=chunk)[100](index=100&type=chunk)[123](index=123&type=chunk) - The Phase 2 trial for NurOwn® in Progressive Multiple Sclerosis (PMS) showed positive top-line data, with **38% of patients showing improvement in walking function**[146](index=146&type=chunk) [Research and Development](index=49&type=section&id=Research%20and%20Development) R&D efforts are focused on NurOwn® and its derivative, MSC-NTF derived Exosomes, for various neurodegenerative disorders - R&D efforts are focused on **MSC-NTF derived Exosomes**, which are nano-vesicles that can cross the blood-brain barrier and may treat multiple neurodegenerative diseases[161](index=161&type=chunk) - A preclinical study showed that intratracheal administration of NurOwn® derived exosomes resulted in a **statistically significant improvement in multiple lung parameters** in a mouse model of ARDS[166](index=166&type=chunk) [Results of Operations](index=53&type=section&id=Results%20of%20Operations) The company generated no revenue and incurred a net loss of $5.8 million for the first six months of 2025, with R&D expenses increasing by 28.7% Comparison of Operating Results (in thousands) | Expense Category | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Research and Development, net | $2,424 | $1,883 | $541 | +28.7% | | General and Administrative | $3,238 | $3,573 | $(335) | -9.4% | | **Operating Loss** | **$5,662** | **$5,456** | **$206** | **+3.8%** | | **Net Loss** | **$5,767** | **$5,942** | **$(175)** | **-2.9%** | - The increase in R&D expenses for the first six months of 2025 was primarily due to a **$387,000 increase in payroll** and a **$294,000 increase in clinical activities**[180](index=180&type=chunk) - The decrease in G&A expenses for the first six months of 2025 was mainly due to **lower costs for payroll, PR activities, rent, and consultants**[182](index=182&type=chunk) [Liquidity and Capital Resources](index=55&type=section&id=Liquidity%20and%20Capital%20Resources) The company's ability to continue as a going concern is in doubt, as it requires substantial additional funding which may be hampered by its delisting - As of June 30, 2025, the company had **cash, cash equivalents, and restricted cash of $1,025,000**[190](index=190&type=chunk) - For the six months ended June 30, 2025, the company **raised $5.8 million from financing activities**, including approximately $4.1 million from its ATM stock offering[192](index=192&type=chunk)[193](index=193&type=chunk) - The company anticipates needing to **raise substantial additional financing** to fund operations and the planned Phase 3b trial, but its ability to do so is uncertain[187](index=187&type=chunk)[189](index=189&type=chunk)[205](index=205&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=61&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section is omitted as the company qualifies as a smaller reporting company - The company is a **smaller reporting company** and is not required to provide the information under this item[211](index=211&type=chunk) [Item 4. Controls and Procedures](index=63&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls were not effective due to a material weakness in internal controls over short-term loans - Management concluded that **disclosure controls and procedures were not effective** as of June 30, 2025, due to a material weakness in internal control over financial reporting[213](index=213&type=chunk) - The material weakness was related to controls over the **initiation, review, authorization, and execution of short-term loans** but did not result in a misstatement[216](index=216&type=chunk) - A **remediation plan has been adopted** to address the material weakness, though the weakness is not yet considered fully remediated[217](index=217&type=chunk) PART II – OTHER INFORMATION [Item 1. Legal Proceedings](index=63&type=section&id=Item%201.%20Legal%20Proceedings) The company is involved in several lawsuits, including a securities class action and shareholder derivative actions related to FDA communications - A **putative securities class action** was filed against the company, alleging violations related to communications with the FDA about NurOwn® for ALS[220](index=220&type=chunk)[221](index=221&type=chunk) - **Four shareholder derivative actions** have been filed, asserting claims for breach of fiduciary duty and unjust enrichment against officers and directors[222](index=222&type=chunk) - 3D Communications, LLC filed a **breach of contract lawsuit** against the company in November 2024, alleging non-payment for consulting services[223](index=223&type=chunk) [Item 1A. Risk Factors](index=65&type=section&id=Item%201A.%20Risk%20Factors) Key risks include the consequences of delisting from Nasdaq, adverse macroeconomic conditions, and a material weakness in internal controls - The **delisting from Nasdaq** could lead to limited liquidity, reduced trading activity, less analyst coverage, and a decreased ability to raise future financing[228](index=228&type=chunk)[229](index=229&type=chunk) - The company faces risks from **adverse macroeconomic conditions**, including inflation and rising interest rates, which could impair its ability to raise additional funding[232](index=232&type=chunk) - The company has incurred **unsecured short-term debt**, which increases financial obligations and could adversely affect its financial condition[234](index=234&type=chunk) - The identified **material weakness in internal controls**, if not remediated, could result in material misstatements in future financial statements[235](index=235&type=chunk)[236](index=236&type=chunk) [Item 5. Other Information](index=69&type=section&id=Item%205.%20Other%20Information) No directors or officers adopted, terminated, or modified a Rule 10b5-1 trading arrangement during the quarter - No directors or officers adopted, terminated, or modified a **Rule 10b5-1 trading plan** during the second quarter of 2025[240](index=240&type=chunk) [Item 6. Exhibits](index=71&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including certifications and XBRL data files

Corporate Update and Highlights [Management Commentary](index=1&type=section&id=Management%20Commentary) The CEO highlights FDA clearance for the NurOwn Phase 3b trial as a key milestone and affirms the company's clinical execution focus - Reached a key milestone in Q2 with **FDA clearance** to initiate the Phase 3b trial for NurOwn, designed to support a potential Biologics License Application (BLA) submission[2](index=2&type=chunk) - Advancing operational activities, including discussions with clinical sites and engagement with CDMO partners to ensure readiness for clinical drug supply[2](index=2&type=chunk) - Supports the FDA's consideration of the Citizen Petition, viewing it as a potential opportunity for a fresh, objective evaluation of NurOwn's scientific evidence[2](index=2&type=chunk) [Recent Highlights](index=1&type=section&id=Recent%20Highlights) The company received FDA clearance for its Phase 3b trial, saw a Citizen Petition filed, and reported positive EAP survival data - The FDA has cleared the company to initiate the Phase 3b clinical trial of NurOwn, known as ENDURANCE, expected to enroll **approximately 200 participants**[3](index=3&type=chunk) - A Citizen Petition was submitted to the FDA by the ALS Community, requesting a new review of the NurOwn data, which the company believes provides a new opportunity to reaffirm NurOwn's potential[7](index=7&type=chunk) - New survival data from the NurOwn Expanded Access Program (EAP) showed that **100% of participants (10/10) survived more than 5 years** from the onset of ALS symptoms[7](index=7&type=chunk) - BrainStorm signed a Letter of Intent (LOI) with Minaris Advanced Therapies, a global CDMO, to manufacture NurOwn for the upcoming Phase 3b clinical trial[7](index=7&type=chunk) - New pharmacogenomic data highlighting the impact of the UNC13A genotype on clinical outcomes were selected for presentation at the ISCT 2025 Annual Meeting[7](index=7&type=chunk) Financial Performance [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) The company reported a net loss of $2.9 million for Q2 2025, with increased R&D expenses and decreased G&A expenses Q2 2025 Financial Highlights | Financial Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Cash, cash equivalents, and restricted cash | ~$1.03 million | N/A | | Research and development expenditures, net | $1.1 million | $0.9 million | | General and administrative expenses | ~$1.4 million | ~$2.1 million | | Net loss | ~$2.9 million | ~$2.5 million | | Net loss per share | $0.34 | $0.60 | [Consolidated Financial Statements](index=5&type=section&id=Consolidated%20Financial%20Statements) The statements show total assets of $2.57 million, a stockholders' deficit of $6.06 million, and a Q2 net loss of $2.90 million [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) As of June 30, 2025, total assets increased to $2.57 million while the total stockholders' deficit improved to $6.06 million Financial Position Comparison | Balance Sheet Item (in thousands) | June 30, 2025 (Unaudited) | December 31, 2024 (Audited) | | :--- | :--- | :--- | | Cash and cash equivalents | $824 | $187 | | Total current assets | $1,515 | $385 | | Total assets | $2,566 | $1,832 | | Total current liabilities | $8,528 | $8,978 | | Total liabilities | $8,623 | $9,596 | | Total stockholders' deficit | $(6,057) | $(7,764) | [Consolidated Statements of Comprehensive Loss](index=6&type=section&id=Consolidated%20Statements%20of%20Comprehensive%20Loss) The company recorded a net loss of $2.90 million for Q2 2025 and $5.77 million for the six months ended June 30, 2025 Three Months Ended June 30 | Statement of Loss (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Research and development, net | $1,120 | $922 | | General and administrative | $1,453 | $2,060 | | Operating loss | $(2,573) | $(2,982) | | Net loss | $(2,903) | $(2,541) | | Basic and diluted net loss per share | $(0.34) | $(0.60) | Six Months Ended June 30 | Statement of Loss (in thousands) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Research and development, net | $2,424 | $1,883 | | General and administrative | $3,238 | $3,573 | | Operating loss | $(5,662) | $(5,456) | | Net loss | $(5,767) | $(5,942) | | Basic and diluted net loss per share | $(0.77) | $(1.35) | Company and Product Overview [About NurOwn®](index=3&type=section&id=About%20NurOwn%C2%AE) NurOwn® is an investigational autologous stem cell therapy designed to deliver neurotrophic factors to sites of damage - The NurOwn® technology platform uses **autologous MSC-NTF cells** produced from a patient's own bone marrow-derived mesenchymal stem cells (MSCs)[8](index=8&type=chunk) - MSCs are converted into MSC-NTF cells, which are induced to secrete high levels of **neurotrophic factors (NTFs)**[8](index=8&type=chunk) - The therapy aims to deliver NTFs and immunomodulatory cytokines to the site of damage to slow or stabilize disease progression[8](index=8&type=chunk) [About BrainStorm Cell Therapeutics Inc.](index=3&type=section&id=About%20BrainStorm%20Cell%20Therapeutics%20Inc.) BrainStorm is a clinical-stage biotech company developing autologous stem cell therapies for neurodegenerative diseases - BrainStorm is a leading developer of **autologous adult stem cell therapies** for debilitating neurodegenerative diseases[9](index=9&type=chunk) - The lead investigational therapy, NurOwn®, has completed a Phase 3 trial in ALS and is set to launch a **Phase 3b trial** under a Special Protocol Assessment (SPA) with the FDA[10](index=10&type=chunk) - The company is also advancing a proprietary, **allogeneic exosome-based platform** and recently received a Notice of Allowance for a foundational patent[10](index=10&type=chunk) Other Information [Conference Call Information](index=2&type=section&id=Conference%20Call%20Information) The company held a conference call on August 14, 2025, to discuss Q2 results, with a replay available until August 28 - A conference call and webcast were scheduled for **8:30 a.m. Eastern Time on Thursday, August 14, 2025**[1](index=1&type=chunk)[5](index=5&type=chunk) - A replay of the conference call will be available until **August 28**[5](index=5&type=chunk)[6](index=6&type=chunk) [Forward-Looking Statements](index=3&type=section&id=Forward-Looking%20Statements) This section cautions that the press release contains forward-looking statements subject to substantial risks and uncertainties - The press release contains forward-looking statements regarding the clinical development of NurOwn, potential regulatory approval, and the future success of the company[11](index=11&type=chunk) - These statements are subject to inherent uncertainties, risks, and assumptions that are difficult to predict, including the ability to **raise additional capital** and continue as a going concern[11](index=11&type=chunk)

Core Insights - BrainStorm Cell Therapeutics Inc. reported financial results for Q2 2025 and provided updates on its clinical development plan for NurOwn, a therapy for neurodegenerative diseases, particularly ALS [1][2] Clinical Development - The FDA has granted clearance for BrainStorm to initiate a Phase 3b clinical trial of NurOwn, named ENDURANCE, which aims to enroll approximately 200 participants at leading academic medical centers [4] - The trial will consist of a 24-week randomized, double-blind, placebo-controlled phase followed by a 24-week open-label extension, with the primary endpoint being the change in the ALS Functional Rating Scale-Revised (ALSFRS-R) from baseline to week 24 [4] - A Citizen Petition has been submitted to the FDA by the ALS community for a new review of NurOwn data, which the company supports as an opportunity to reaffirm the therapy's potential [4] Financial Performance - As of June 30, 2025, BrainStorm reported cash, cash equivalents, and restricted cash of approximately $1.03 million [4] - Research and development expenditures for Q2 2025 were $1.1 million, an increase from $0.9 million in Q2 2024 [4] - General and administrative expenses decreased to approximately $1.4 million in Q2 2025 from approximately $2.1 million in Q2 2024 [4] Survival Data - New survival data from the NurOwn Expanded Access Program indicated that 100% of participants (10 out of 10) survived more than 5 years from the onset of ALS symptoms, compared to an estimated 10% survival rate for ALS patients [4] - The median survival observed in the EAP cohort was 6.8 years from symptom onset, with a range of 6 to 7 years [4] Manufacturing and Partnerships - BrainStorm has signed a Letter of Intent with Minaris Advanced Therapies for the manufacturing of NurOwn for the upcoming Phase 3b clinical trial [4] - The NurOwn data was selected for presentation at the ISCT 2025 Meeting, highlighting the impact of the UNC13A genotype on clinical outcomes for ALS patients treated with NurOwn [4] Company Overview - BrainStorm Cell Therapeutics Inc. specializes in developing autologous adult stem cell therapies for neurodegenerative diseases, with its proprietary NurOwn platform utilizing mesenchymal stem cells to produce neurotrophic factor-secreting cells [6][7]

Company Overview - BrainStorm Cell Therapeutics Inc. is a leading developer of autologous adult stem cell therapies for neurodegenerative diseases, utilizing its proprietary NurOwn® platform to produce neurotrophic factor-secreting cells [5] - The company focuses on therapies for conditions such as amyotrophic lateral sclerosis (ALS) and progressive multiple sclerosis (MS) [6] Upcoming Conference Call - A conference call is scheduled for August 14, 2025, at 8:30 AM Eastern Time to update shareholders on the financial results for the second quarter of 2025 [1] - Key executives participating in the call include President and CEO Chaim Lebovits, COO Hartoun Hartounian, CMO Bob Dagher, and Interim CFO Alla Patlis [2] Clinical Development - NurOwn® has received Orphan Drug designation from both the U.S. FDA and the European Medicines Agency (EMA) for ALS treatment [6] - A Phase 3 trial for ALS has been completed, and a second Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA [6] - The clinical program has provided insights into ALS disease biology, including pharmacogenomic responses and biomarker data [6] Intellectual Property - BrainStorm has received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, enhancing its intellectual property portfolio in regenerative medicine [6]

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]

Investor call and webcast scheduled for today at 8:30 a.m. ET NEW YORK, May 19, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the company to initiate its Phase 3b clinical trial of NurOwn® (autologous MSC-NTF cells) for the treatment of amyotrophic lateral sclerosis (ALS). The trial design was previously agreed upon with the FD ...