Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) Q2 2024 Earnings Conference Call August 14, 2024 8:30 AM ET Company Participants Michael Wood - LifeSci Advisors Chaim Lebovits - President and CEO Alla Patlis - Interim Chief Financial Officer Haro Hartounian - Chief Operating Officer Bob Dagher - Chief Medical Officer Conference Call Participants Joanne Lee - Maxim Group David Bautz - Zacks Small-Cap Research Christopher Nicholson - ACF Equity Research Operator Greetings, and welcome to the Brainstorm Cell T ...

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[95]. - The FDA granted a Special Protocol Assessment (SPA) agreement for the Phase 3b trial of NurOwn® in ALS on April 9, 2024, validating the clinical trial protocol[101]. - 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[95]. - The BLA for NurOwn® was withdrawn on November 3, 2023, in coordination with the FDA, viewed as a withdrawal without prejudice[95]. - The Phase 3 clinical trial of NurOwn® did not achieve statistically significant results, with 32.6% of participants meeting the primary endpoint compared to 27.7% for placebo (p=0.453)[125]. - In a pre-specified subgroup with ALSFRS-R baseline scores of 35 or greater, NurOwn® showed a 34.6% responder rate versus 15.6% for placebo (p=0.305) and an average change of -1.56 on NurOwn® compared to -3.65 on placebo (p=0.050)[126]. - The Phase 2 trial demonstrated a slower rate of disease progression in ALS patients, with significant improvements in ALSFRS-R and FVC[116]. - NurOwn® treatment resulted in statistically significant increases in neurotrophic factors and reductions in inflammatory markers in cerebrospinal fluid samples[120]. - The Phase 3 trial enrolled a broad set of participants, including those with advanced ALS, which may have impacted the sensitivity of the ALSFRS-R[129]. - The company is actively exploring next steps for NurOwn®, including publication of emerging clinical data and development of a protocol for an additional clinical study[95]. - The FDA invited the company for an expedited meeting to discuss the regulatory path forward for NurOwn® in ALS on October 18, 2023[112]. - The company plans to submit a SPA request for a Phase 3b clinical trial for NurOwn® to discuss protocol design with the FDA[112]. - A meeting with the FDA on December 6, 2023, focused on discussing the regulatory path forward for NurOwn® and plans for a Special Protocol Assessment (SPA) for a Phase 3b trial[134]. - The company received written agreement from the FDA on April 9, 2024, under a SPA for the design of the Phase 3b trial of NurOwn® in ALS, validating the clinical trial protocol[134]. Financial Performance and Funding - The company incurred operating costs of approximately $2,982,000 for the three months ended June 30, 2024, a decrease of 45.1% compared to $5,449,000 for the same period in 2023[185]. - Research and development expenditures, net for the three months ended June 30, 2024 were $922,000, a decrease of 67.1% compared to $2,794,000 for the same period in 2023[187]. - The net loss for the three months ended June 30, 2024 was $2,541,000, compared to a net loss of $5,329,000 for the same period in 2023, representing a reduction of 52.3%[191]. - The company had cash, cash equivalents, and restricted cash amounting to $3,648,000 as of June 30, 2024[197]. - Net cash used in operating activities for the six months ended June 30, 2024 was $4,746,000, primarily attributed to clinical trial costs and payroll expenses[197]. - The company received $6,909,000 from financing activities for the six months ended June 30, 2024, primarily from sales of common stock under the ATM program[197]. - The company anticipates needing to raise substantial additional financing in the future to fund operations, including potential debt and equity offerings[210]. - The Company sold an aggregate of 2,446,641 shares of Common Stock at an average price of $9.45 per share, raising gross proceeds of approximately $23.11 million under the March 6, 2020 ATM[201]. - The Company entered into a Securities Purchase Agreement on July 17, 2023, agreeing to sell 4,054,055 shares of Common Stock at a purchase price of $1.85 per share, yielding gross proceeds of approximately $7.5 million[205]. - On June 27, 2024, the Company agreed to sell 7,918,764 registered shares of Common Stock and pre-funded warrants, yielding gross proceeds of approximately $4.0 million[207]. Legal and Compliance Issues - The company is involved in ongoing litigation, including a putative securities class action complaint alleging violations related to NurOwn® for the treatment of ALS[220]. - The company is facing multiple derivative action complaints alleging breach of fiduciary duty and unjust enrichment against certain officers and directors, with claims related to the internal controls and FDA submissions for NurOwn® for ALS treatment[221]. - The company received a letter from Nasdaq on November 1, 2023, indicating non-compliance with the $1.00 minimum bid price requirement, with a compliance period until April 29, 2024[226]. - Nasdaq determined to delist the company's common stock on May 1, 2024, due to continued non-compliance with the bid price requirement[226]. - The company submitted a hearing request to Nasdaq on May 2, 2024, and was granted a temporary exception to regain compliance by October 21, 2024[227]. - The company received a notice on July 18, 2024, indicating it did not maintain the minimum market value of listed securities of $35 million, with a compliance period until January 14, 2025[228]. - Failure to regain compliance with either the bid price or market value requirements could lead to delisting from Nasdaq, negatively impacting trading and liquidity[229]. - The company is subject to securities class action litigation and other stockholder litigation, which could result in substantial costs and divert management's attention[230]. Company Operations and Workforce - The company currently employs 29 employees in the United States and Israel, with a focus on recruiting and retaining talent to support its objectives[99]. - The company has a GMP manufacturing center in Tel Aviv, enhancing its capacity to manufacture and ship NurOwn® into the EU and local Israeli markets[99]. - The company has developed a validated cryopreservation process for MSC, allowing multiple doses of NurOwn® from a single bone marrow harvest, enhancing manufacturing efficiency[135]. - 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®[138]. - The company has improved the efficiency of NurOwn® production and its stability, allowing centralized manufacturing and distribution to clinical trial sites[183]. Research and Development - 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 in May 2024[103]. - The NurOwn® technology has received Fast Track designation and Orphan Drug status from the FDA and EMA for ALS, indicating its potential significance in treating this condition[98]. - The FDA granted Fast Track designation and Orphan Drug Status for NurOwn® in ALS, providing potential for extended exclusivity[112]. - The company is considering advancing NurOwn® as a treatment option for Alzheimer's Disease, focusing on its capability to target multiple biological pathways[161]. - The company received a total of $15,912,390 in grant funding from the California Institute for Regenerative Medicine (CIRM) for the Phase 3 study of NurOwn® for ALS[162]. - The company holds 27 granted patents and 23 patent applications worldwide, protecting its proprietary technologies and products[164]. - The company announced the European grant for NurOwn® and the acceptance of applications for exosomes derived from MSC-NTF cells in Australia and Israel[171]. - The Phase 2 PMS clinical trial compared clinical efficacy outcomes with a matched clinical cohort from the CLIMB Study, demonstrating significant improvements in various measures of function and cognition[156]. - In the Phase 2 study for Progressive Multiple Sclerosis (PMS), 80% of the 20 enrolled patients completed the study, with no deaths or serious adverse events reported[155]. - 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[158].

[Executive Summary & Corporate Update](index=1&type=section&id=Executive%20Summary%20%26%20Corporate%20Update) BrainStorm provided Q2 2024 financial results and corporate updates, highlighting NurOwn Phase 3b trial preparation [Second Quarter 2024 and Recent Highlights](index=1&type=section&id=Second%20Quarter%202024%20and%20Recent%20Highlights) BrainStorm announced Q2 2024 results, emphasizing regulatory alignment and trial site establishment for NurOwn Phase 3b trial - CEO Chaim Lebovits stated that regulatory aspects of the program have been substantially derisked, having secured agreement with the FDA on a Special Protocol Assessment and also reached alignment on the CMC aspects[2](index=2&type=chunk) - A leading Clinical Research Organization (CRO) has been selected, and the company is actively working to establish multiple trial sites for the Phase 3b trial[2](index=2&type=chunk) [Clinical and Regulatory Milestones](index=1&type=section&id=Clinical%20and%20Regulatory%20Milestones) BrainStorm achieved key regulatory milestones for NurOwn, including FDA alignment on CMC and SPA for the Phase 3b trial - In June 2024, BrainStorm reached alignment with the U.S. Food and Drug Administration (FDA) on the Chemistry, Manufacturing, and Controls (CMC) aspects of its planned Phase 3b clinical trial for NurOwn® in ALS[3](index=3&type=chunk) - In April 2024, the FDA granted BrainStorm a Special Protocol Assessment (SPA) agreement for the Phase 3b trial, validating the clinical trial protocol and statistical analysis for a future BLA[3](index=3&type=chunk) - New biomarker data on NurOwn from the prior Phase 3 study and Expanded Access Program (EAP) were featured at the 3rd Annual ALS Drug Development Summit in May 2024, suggesting continued benefits for patients with extended treatment[5](index=5&type=chunk) [Corporate Developments](index=2&type=section&id=Corporate%20Developments) BrainStorm completed a $4.0 million registered direct offering and strengthened its leadership with key appointments - In June 2024, BrainStorm completed a successful registered direct offering, raising gross proceeds of **$4.0 million** from a single institutional investor[6](index=6&type=chunk) - In June 2024, Hartoun Hartounian Ph.D. was appointed as EVP and Chief Operating Officer, bringing over **32 years of experience** in the biopharmaceutical industry, with a focus on cell and gene therapy[6](index=6&type=chunk) - In April 2024, Dr. Bob Dagher was promoted to Executive Vice President and Chief Medical Officer, with over **20 years of experience** in clinical research and development[6](index=6&type=chunk) [Financial Performance](index=2&type=section&id=Financial%20Performance) BrainStorm's Q2 2024 financial performance showed reduced net loss and improved cash, driven by decreased operating expenses [Second Quarter 2024 Financial Summary](index=2&type=section&id=Second%20Quarter%202024%20Financial%20Summary) BrainStorm reported a Q2 2024 net loss of $2.5 million, a significant reduction from $5.3 million in Q2 2023 due to decreased expenses - Cash, cash equivalents, and restricted cash amounted to approximately **$3.65 million** as of June 30, 2024[6](index=6&type=chunk) Key Financials (Q2 2024 vs Q2 2023) | Metric | Q2 2024 (USD millions) | Q2 2023 (USD millions) | Change (YoY) | | :-------------------------- | :--------------------- | :--------------------- | :----------- | | Research & Development, net | $0.9 | $2.8 | -67.9% | | General & Administrative | $2.0 | $2.7 | -25.9% | | Net Loss | $2.5 | $5.3 | -52.8% | | Net Loss Per Share | $0.04 | $0.13 | -69.2% | [Interim Condensed Consolidated Balance Sheets](index=5&type=section&id=Interim%20Condensed%20Consolidated%20Balance%20Sheets) As of June 30, 2024, BrainStorm's total assets increased to $5.683 million from $4.208 million at year-end 2023, driven by cash increase Balance Sheet Highlights (USD thousands) | Metric | June 30, 2024 (Unaudited) | December 31, 2023 (Audited) | Change | | :----------------------------------- | :------------------------ | :-------------------------- | :------ | | Cash and cash equivalents | $3,469 | $1,300 | +$2,169 | | Total current assets | $3,816 | $1,899 | +$1,917 | | Total assets | $5,683 | $4,208 | +$1,475 | | Total current liabilities | $7,691 | $7,800 | -$109 | | Total liabilities | $9,210 | $9,066 | +$144 | | Total stockholders' deficit | $(3,527) | $(4,858) | +$1,331 | [Interim Condensed Consolidated Statements of Comprehensive Loss](index=6&type=section&id=Interim%20Condensed%20Consolidated%20Statements%20of%20Comprehensive%20Loss) For Q2 2024, BrainStorm reported a net loss of $2.541 million, a substantial improvement from $5.329 million in the prior year Comprehensive Loss Highlights (USD thousands) | Metric | Three months ended June 30, 2024 | Three months ended June 30, 2023 | Six months ended June 30, 2024 | Six months ended June 30, 2023 | | :----------------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development, net | $922 | $2,794 | $1,883 | $5,718 | | General and administrative | $2,060 | $2,655 | $3,573 | $4,882 | | Operating loss | $(2,982) | $(5,449) | $(5,456) | $(10,600) | | Net loss | $(2,541) | $(5,329) | $(5,942) | $(10,388) | | Basic and diluted net loss per share | $(0.04) | $(0.13) | $(0.09) | $(0.27) | | Weighted average shares outstanding | 71,215,481 | 39,696,665 | 67,977,012 | 38,224,230 | [Conference Call and Webcast Information](index=3&type=section&id=Conference%20Call%20and%20Webcast%20Information) Details for BrainStorm's conference call and webcast to discuss Q2 2024 results and recent progress were provided [Conference Call and Webcast Details](index=3&type=section&id=Conference%20Call%20and%20Webcast%20Details) BrainStorm provided conference call and webcast details for Q2 2024 results, including dial-in numbers and webcast URL - A conference call and webcast for the investment community was scheduled for 8:30 AM ET on August 14, 2024, to discuss second quarter results and recent progress[1](index=1&type=chunk)[9](index=9&type=chunk) - Access details include Toll Free: **877-545-0523**, International: **973-528-0016**, Participant Access Code: **308245**, and Webcast URL: **https://www.webcaster4.com/Webcast/Page/2354/51009**[8](index=8&type=chunk) - A replay of the conference call is available for **14 days** via Toll Free: **877-481-4010**, International: **919-882-2331**, and Replay Passcode: **51009**[9](index=9&type=chunk) [Company Overview and Disclosures](index=3&type=section&id=Company%20Overview%20and%20Disclosures) BrainStorm Cell Therapeutics develops autologous adult stem cell therapeutics for neurodegenerative diseases, with forward-looking statement disclosures [About BrainStorm Cell Therapeutics Inc.](index=3&type=section&id=About%20BrainStorm%20Cell%20Therapeutics%20Inc.) BrainStorm Cell Therapeutics is a leading developer of autologous adult stem cell therapeutics for neurodegenerative diseases, holding NurOwn® rights - BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases[10](index=10&type=chunk) - The company holds exclusive, worldwide licensing agreement for the NurOwn® technology platform, which produces autologous MSC-NTF cells[10](index=10&type=chunk) - Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. FDA and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS)[10](index=10&type=chunk) [Notice Regarding Forward-Looking Statements](index=4&type=section&id=Notice%20Regarding%20Forward-Looking%20Statements) The press release contains forward-looking statements subject to substantial risks and uncertainties, including regulatory approvals and clinical development - This press release contains "forward-looking statements" regarding future events such as FDA meetings, SPA, ADCOM, PDUFA action dates, clinical development of NurOwn, and the future success of BrainStorm[11](index=11&type=chunk) - These statements are subject to substantial risks and uncertainties, including management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, and prospects for future regulatory approval of NurOwn[11](index=11&type=chunk) - Readers should not place undue reliance on BrainStorm's forward-looking statements, which are based on the beliefs, expectations, and opinions of management as of the date of this press release[11](index=11&type=chunk) [Contacts](index=4&type=section&id=Contacts) Contact information for media and investor relations inquiries is provided - Media Contact: Lisa Guiterman, Phone: **+1 202-330-3431**, Email: **lisa.guiterman@gmail.com**[12](index=12&type=chunk) - IR Contact: Michael Wood, Phone: **+1 646-597-6983**, Email: **mwood@lifesciadvisors.com**[12](index=12&type=chunk)

- Company reached alignment with the U.S. FDA on the Chemistry, Manufacturing, and Controls (CMC) aspects of its planned Phase 3b clinical trial for NurOwn® in ALS- SPA in place for Phase 3b NurOwn® trial in ALS- Enhanced leadership team with appointment of  Hartoun Hartounian Ph.D. as EVP and Chief Operating Officer, and promotion of Dr. Bob Dagher to EVP and Chief Medical Officer- Conference call and webcast at 8.30am ET todayNEW YORK, Aug. 14, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDA ...

Core Viewpoint - BrainStorm Cell Therapeutics Inc. will hold a conference call on August 14, 2024, to update shareholders on Q2 financial results and provide a corporate update [1] Group 1: Conference Call Details - The conference call is scheduled for 8:30 a.m. Eastern Time on August 14, 2024 [1] - Participants can submit questions in advance until 5:00 p.m. Eastern Time on August 12, 2024 [1] - The call can be accessed via toll-free and international numbers, with a specific access code provided [2] Group 2: Company Overview - BrainStorm Cell Therapeutics is focused on developing autologous adult stem cell therapeutics for neurodegenerative diseases [3] - The company holds rights to the NurOwn® technology platform for producing autologous MSC-NTF cells through an exclusive licensing agreement [3] Group 3: Clinical Development - Autologous MSC-NTF cells have received Orphan Drug designation from the FDA and EMA for treating amyotrophic lateral sclerosis (ALS) [4] - A Phase 3 trial for ALS has been completed, investigating the safety and efficacy of repeat-administration of autologous MSC-NTF cells [4] - The company has also completed a Phase 2 open-label multicenter trial for progressive multiple sclerosis (MS) [4]

ACF Equity Research FROM ACF EQUITY RESEARCH HEALTHCARE TEAMLONDON, July 24, 2024 /PRNewswire/ -- NASDAQ: BCLIREAD ACF EQUITY RESEARCH'S UPDATE NOTE HERE READ ACF EQUITY RESEARCH'S RELATED THEMATIC RESEARCH NOTE ON REGENERATIVE MEDICINE – NEURODEGENERATIVE DISEASE STEM CELLS & ALS/MND/LOU GEHRIG'S HEREUPDATE NOTE: BrainStorm Cell Therapeutics Inc. (Nasdaq: BCLI) develops NurOwn® stem cell therapy for Neurodegenerative diseases (NDDs) – BCLI's first target is ALS (MND/Lou Gehrig's). Since initiation – PIIIb ...

Summary of Brainstorm Cell Therapeutics Inc. Mid-Year Update on NurOwn Program Company Overview - **Company**: Brainstorm Cell Therapeutics Inc. (NASDAQ: BCLI) - **Focus**: Development of NurOwn, a cell therapy for neurodegenerative disorders, particularly ALS (Amyotrophic Lateral Sclerosis) Key Developments 1. **Phase 3b Trial Initiation**: - Priority is to initiate the Phase 3b trial for ALS, with a constructive Type C meeting held with the FDA on June 24, 2024, to discuss chemistry, manufacturing, and controls (CMC) aspects [6][8] - The FDA has shown continued support, and the company is aligned with the agency on resolving outstanding CMC questions [6][7] 2. **Clinical Research Organization (CRO) Engagement**: - A contract has been signed with a leading CRO recognized for expertise in ALS and cell therapy to support the Phase 3b trial [9] - The CRO is preparing clinical sites and project plans to begin dosing the first patient before the end of 2024 [9] 3. **Funding and Financial Strategy**: - The company raised $4 million through the sale of common stock and warrants, which will support near-term milestones but is not sufficient for the entire Phase 2b study [10] - Additional financing options, including non-dilutive grants, are being explored [10][19] 4. **Leadership Changes**: - Dr. Haro Hartounian has been appointed as Chief Operating Officer, bringing over 32 years of experience in biopharmaceuticals, particularly in cell and gene therapy [11] 5. **Regulatory Strategy**: - The company is committed to conducting the Phase 3b trial to gather robust data for a future Biologics License Application (BLA) submission [28] - The decision to conduct an additional Phase 3b trial was made to align with FDA expectations and to provide comprehensive data for ALS treatment approval [28][29] Clinical Trial Details 1. **Trial Design and Objectives**: - The Phase 3b trial will have a shorter screening period of about eight weeks, focusing on patients diagnosed within two years and with preserved respiratory function [42][37] - Entry criteria will ensure patients are in the early stages of ALS, aiming for a higher baseline ALSFRS score than in previous trials [42][37] 2. **Manufacturing and BLA Readiness**: - Securing a commercial manufacturing partner early is crucial for BLA filing readiness and reducing time to market [24] - The manufacturing process is well-defined, and the company is prepared for the FDA inspection as part of the BLA approval process [24][33] Market and Competitive Landscape - The company believes that positive results from the Phase 3b trial will lead to a reevaluation of its market value, allowing for future financing on more favorable terms [20][22] - The focus on early intervention in ALS treatment aligns with emerging trends in neurodegenerative disease therapies [52] Additional Insights - The complexity of cell therapy manufacturing was emphasized, with a focus on the unique aspects of NurOwn's production process [49] - The company is committed to transparency regarding challenges and is focused on developing NurOwn as a potential treatment for ALS [29] Conclusion - Brainstorm Cell Therapeutics is poised to advance its NurOwn program with a clear strategy for trial initiation, regulatory engagement, and financial planning, while addressing the urgent need for effective ALS treatments.

Core Viewpoint - BrainStorm Cell Therapeutics Inc. is set to hold a mid-year corporate update on July 8, 2024, to discuss positive developments in the NurOwn® program for neurodegenerative diseases [1] Company Overview - BrainStorm Cell Therapeutics is a leading developer of autologous adult stem cell therapeutics targeting neurodegenerative diseases [3] - The company holds exclusive rights to the NurOwn® technology platform, which produces autologous MSC-NTF cells, and has received Orphan Drug designation from the FDA and EMA for ALS treatment [3] - BrainStorm has completed a Phase 3 trial for ALS (NCT03280056) and a Phase 2 trial for progressive MS (NCT03799718), both supported by grants from various organizations [3] Conference Call Details - The conference call will feature presentations from the President and CEO, Chaim Lebovits, and the newly appointed COO, Hartoun Hartounian, along with a Q&A session [1] - Participants can submit questions in advance until July 5, 2024 [1] - The call can be accessed via toll-free and international numbers, with a replay available until July 22, 2024 [2]

NEW YORK, June 27, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) (the "Company"), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that it has entered into a securities purchase agreement with a single institutional investor (the "Holder") for the purchase and sale of 11,111,111 shares of the Company's common stock (or prefunded warrants in lieu thereof) at a purchase price of $0.36 per share in a registered direct offering.In a conc ...

Core Viewpoint - BrainStorm Cell Therapeutics Inc. has appointed Dr. Hartoun Hartounian as the new EVP and COO, which is expected to enhance the company's operational efficiency and support the commercial readiness of its ALS treatment, NurOwn® [1][2] Company Overview - BrainStorm Cell Therapeutics is a developer of autologous adult stem cell therapeutics aimed at treating neurodegenerative diseases, particularly ALS [4] - The company holds exclusive rights to the NurOwn® technology platform, which has received Orphan Drug designation from the FDA and EMA for ALS treatment [4] Leadership Appointment - Dr. Hartounian has over 32 years of experience in the biopharmaceutical industry, particularly in cell and gene therapy [1][2] - His previous achievements include founding BioCentriq, which was acquired for $73 million in 2022, and leading multiple biotechnology companies [1][2] Responsibilities and Expectations - In his new role, Dr. Hartounian will oversee all operational aspects, including CMC and commercialization, to streamline logistics and navigate complexities [2] - The company anticipates that Dr. Hartounian's expertise will be crucial in advancing NurOwn and achieving strategic objectives [2] Clinical Development - BrainStorm has completed a Phase 3 trial for NurOwn in ALS, which investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells [4][5] - The company has also conducted a Phase 2 trial for progressive MS, supported by grants from relevant organizations [5]