BrainStorm Cell Therapeutics Shares Fall After Updated Biomarker Data From Lead Cell Therapy Candidate

BrainStorm Cell Therapeutics Inc BCLI presented new biomarker data from the Phase 3 trial of its late-stage investigational amyotrophic lateral sclerosis (ALS) treatment, NurOwn, at the 2023 ALS and Related Motor Neuron Diseases Gordon Research Conference. 

These data show that treatment with NurOwn significantly elevated markers of neuroprotection and lowered markers of neuroinflammation and neurodegeneration, including neurofilament light (NfL), over time compared to placebo in all trial participants.

Statistical modeling identifies three biomarkers that predict clinical outcomes observed with NurOwn-treatment in the Phase 3 trial (BCT-002), including change in Galectin-1 and baseline biomarkers NfL and LAP/TGFβ1.

NurOwn-treated participants had reduced NfL values from baseline to week 20 compared to placebo (p<0.05).

NfL baseline levels were prognostic of ALS disease progression, confirming results from other ALS trials. Participants with a greater decline from baseline at week 28, as measured by ALSFRS-R total score, had higher baseline NfL values, r=-0.33, p=0.0064.

Causal Inference using a natural disease progression model showed a relationship between reductions due to NurOwn in NfL changes from baseline and ALSFRS-R changes from baseline.

After adjusting for the predicted changes due to natural disease progression, the correlation between NurOwn-driven changes at the final measure in NfL (week 20) and ALSFRS-R (week 28) was r=-0.365, p=0.087. This analysis was conducted on participants with all ALSFRS-R items >1 at baseline.

Last year, BrainStorm received a refusal to file letter from the FDA regarding its marketing application seeking approval for NurOwn for ALS.

Last month, the company announced the FDA would convene a meeting of the Peripheral and Central Nervous System Drugs Advisory Committee to NurOwn application, scheduled for September 27. PDUFA action date is targeted to occur by December 8.

Price Action: BCLI shares are down 4.50% at $1.91 during the premarket session on the last check Friday.