BCLI: Initiation - Potential 10x Return on Successful Phase 3b. Promising Post Hoc and Biomarker Analysis Underpins Our NurOwn® Clinical Effectiveness Expectation
14 Jun 2024
Phase 3Clinical ResultCell Therapy
FROM ACF EQUITY RESEARCH HEALTHCARE TEAM
NASDAQ: BCLI
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LONDON, June 14, 2024 /PRNewswire/ --
INITIATION NOTE: BrainStorm Cell Therapeutics Inc. (Nasdaq: BCLI) is focused on developing autologous mesenchymal stem cell (MSC) therapies for the treatment of neurodegenerative diseases (NDDs) – BCLI's primary target is the fatal amyotrophic lateral sclerosis (ALS/MND/Lou Gehrig's). Post hoc analysis of BCLI's PIII trial data shows BCLI's NurOwn® (debamestrocel, MSC-NTF), has statistically significant clinical effects on early-stage ALS sufferers and that placebo trialists deteriorate faster. Peer reviewed research (Mar 2024) indicated that BCLI's NurOwn® has a positive impact on NfL biomarkers for ALS. These and other factors have persuaded the FDA to sign up to the SPA binding commitment for a NurOwn® PIIIb ALS trial. BCLI has regained regulatory minimum value NASDAQ compliance. Our valuation captures only the smaller by patients US market and excludes the larger EU/UK market.
Post hoc analysis of BCLI's PIII early stage (mild-moderate) ALS sufferers (baseline ALSFRS-R scores >=35) treated with BCLI's NurOwn® (debamestrocel, MSC-NTF) revealed positive clinical responses with respect to slowing of ALS disease progression (primary endpoint). However the PIII trial cohort consisted (unexpectedly) of 23% advanced ALS sufferers, clouding the primary and secondary end point statistical analysis (possible floor effects). The new PIIIb trial is designed to recruit a cohort of participants with ALSFRS-R scores >=35. Additionally, more recent peer reviewed research found that certain biomarkers involved in ALS pathology, specifically NfL, LAP and Galectin-1 were found to be predictive of positive clinical outcomes in NurOwn® (debamestrocel, MSC-NTF),-treated participants. If the new trial design is successfully executed, as we forecast, we expect a strongly positive valuation inflection point for BCLI.
ALS: High Unmet Medical Need Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder (NDD) that affects motor nerve cells in the brain and the spinal cord. There are an estimated ~450k ALS patients worldwide (30k US and 51k European). Median survival post diagnosis is 2 to 5 years. Current treatment options have very limited efficacy. Tofersen addresses just 2% of ALS sufferers. There is a high unmet need to slow/reverse ALS progression.
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