Brainstorm Cell Therapeutics Inc. (BCLI) Corporate Mid-Year Update on NurOwn Program (Transcript)

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.