Brainstorm Cell Therapeutics(US:BCLI)2025-11-14 22:01Clinical 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[89]. - The FDA granted Fast Track designation and Orphan Drug status for NurOwn® for ALS, providing potential for extended exclusivity[91]. - 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[89]. - The BLA for NurOwn® was withdrawn on November 3, 2023, in coordination with the FDA, viewed as a withdrawal without prejudice[89]. - A Special Protocol Assessment (SPA) request for a planned Phase 3b clinical trial was submitted to the FDA on February 23, 2024, and written agreement was received on April 9, 2024[104]. - 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[89]. - The company has reached alignment with the FDA on the Chemistry, Manufacturing, and Controls (CMC) aspects of the Phase 3b clinical trial for NurOwn®[137]. - The company announced the completion of a productive meeting with the FDA on December 7, 2023, to discuss plans for a SPA for a Phase 3b registrational trial[137]. - The FDA issued a Refusal to File (RTF) letter on November 10, 2022, citing insufficient completeness for substantive review[121]. - Following discussions with the FDA, the company requested to file the BLA over Protest on February 6, 2023, which was confirmed on February 7, 2023[123]. Financial Performance and Funding - General and administrative expenses for Q3 2025 decreased by $855,000 to $1,148,000 compared to $2,003,000 in Q3 2024, primarily due to reductions in payroll and other costs[180]. - Net loss for Q3 2025 was $2,106,000, an improvement from a net loss of $2,708,000 in Q3 2024, resulting in a net loss per share of $0.19 compared to $0.51[183]. - For the nine months ended September 30, 2025, net loss was $7,873,000, down from $8,650,000 in the same period of 2024, with a net loss per share of $0.91 versus $1.80[184]. - Cash used in operating activities for the nine months ended September 30, 2025 was $6,235,000, primarily due to clinical trial costs and payroll[191]. - Cash and cash equivalents as of September 30, 2025 amounted to $236,000[190]. - The company raised $6,100,000 from financing activities during the nine months ended September 30, 2025, through sales of common stock and short-term loans[192]. - The company anticipates needing substantial additional financing to fund operations and clinical trials, with potential dilution to existing shareholders[205]. - The company is considering multiple alternatives to meet capital needs, including public and private sales of common stock and convertible promissory notes[186]. - A recent securities purchase agreement was entered into, issuing a promissory note of $182,400, with a 12% interest rate that increases to 22% if not timely paid[202]. - The company has filed for delisting from Nasdaq and is now quoted on the OTCQB Venture Market under the symbol "BCLI"[206]. - The company faces additional compliance requirements due to state-level regulations affecting trading liquidity[206]. - The company is assessing its ability to obtain funding from third parties and the costs associated with clinical trials and R&D programs[207]. - The company emphasizes the importance of regulatory approvals and the associated time and costs[207]. Research and Development - The company is actively engaged in research for the clinical development of NurOwn® and MSC-NTF derived exosomes in various neurodegenerative disorders[161]. - The company has received $400,000 out of $500,000 awarded for ALS biomarker research from The ALS Association and I AM ALS[155]. - The company is considering advancing NurOwn® as an innovative treatment option in Alzheimer's Disease based on emerging scientific insights[151]. - Research and development expenditures for the three months ended September 30, 2025, were $899,000, a decrease of $146,000 compared to the same period in 2024[176]. - For the nine months ended September 30, 2025, research and development expenditures increased to $3,323,000, up by $395,000 from $2,928,000 in the same period in 2024[178]. Clinical Trial Results - Completed Phase 1/2 and Phase 2 open-label clinical trials of NurOwn® in ALS patients, demonstrating tolerability and preliminary signs of activity[105]. - Phase 2 trial involved 48 patients randomized 3:1 to receive NurOwn® or placebo, with no discontinuations due to adverse events[108]. - NurOwn® treatment showed higher response rates in the ALS functional rating scale compared to placebo, particularly in rapid progressors[111]. - Statistically significant increases in neurotrophic factors and reductions in inflammatory markers were observed post-treatment in NurOwn® participants[114]. - Phase 3 trial enrolled a population of rapid progressors, but did not achieve statistically significant results despite showing numerical improvements[117]. - New analyses indicated a statistically significant treatment difference in a secondary endpoint for participants with a baseline ALSFRS-R score of at least 35[119]. - The Phase 2 PMS clinical trial enrolled 20 patients, with 80% (16 patients) completing the study, and no deaths or serious adverse events related to multiple sclerosis were reported[147]. - 38% of NurOwn® treated patients showed at least a 10-point improvement in the MS Walking Scale (MSWS-12) from baseline to week 28[148]. - NurOwn® treated patients exhibited a mean improvement of 10% in the timed 25-foot walk (T25FW) and a 4.8% improvement in the 9-hole peg test (9-HPT) compared to matched historical controls[157]. Company Operations and Intellectual Property - The company employs 23 individuals across the United States and Israel, with a focus on recruiting and retaining talent to support its objectives[93]. - The proprietary NurOwn® technology involves a multi-step manufacturing process that includes harvesting and isolating stem cells from the patient's bone marrow[96]. - The company has a strong intellectual property portfolio and holds exclusive rights to commercialize NurOwn® technology through a licensing agreement with Ramot, Tel Aviv University[91]. - The company has developed a validated cryopreservation process for MSC, allowing multiple doses of NurOwn® from a single bone marrow harvest[125]. - As of November 1, 2023, the company optimized its manufacturing capabilities for NurOwn® by leveraging partnerships and operational resources[129]. - The company contracted with City of Hope and Dana Farber Cancer Institute for the manufacturing of NurOwn® for its Phase 3 clinical study[126][127]. - The company holds approximately 30 granted patents and has pending applications across key global markets, including the U.S., Europe, and South America[156]. - The company qualifies as a smaller reporting company, omitting certain market risk disclosures[212]. - The company has no off-balance sheet arrangements that could materially affect its financial condition[211]. - The company’s accounting policies include stock-based compensation, which is estimated using the Black-Scholes option valuation model[210]. - Changes in assumptions for stock-based compensation could significantly impact net loss and net loss per share[210].