BrainStorm presents NurOwn survival data at NEALS Meeting

BrainStorm (BCLI) announced the presentation of two posters featuring NurOwn at the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis Consortium, or NEALS, Meeting, which took place virtually October 21 – 24. The posters ‘Debamestrocel Long-Term Benefits on Survival and Neurodegeneration in ALS Expanded Access Program’ and ‘An Overview of The Phase 3b Clinical Trial of Debamestrocel in ALS’ highlight the results achieved with ALS patients who participated in the Expanded Access Program, or EAP, for NurOwn and summarize the details of BrainStorm’s upcoming Phase 3b trial in ALS. Ten participants from Brainstorm’s prior Phase 3 clinical trial were enrolled in an open label Expanded Access Program, or EAP. The EAP spanned two 28-week periods, with a break in time between the periods. Participants received an intrathecal dose of Debamestrocel every 8 weeks, for a maximum of 6 doses over the two periods. Baseline characteristics from 10 EAP participants, captured at the time they entered the Phase 3 trial, were matched against a comparable cohort from the PRO-ACT historical database using propensity score matching, or PSM. Matching covariates for PSM included time since disease onset, pre-baseline ALSFRS-R slope, age, Slow/Forced Vital Capacity, and site of onset, with a 10:1 matching ratio. A Kaplan-Meier plot was generated, and a log rank test was performed to compare survival between the two groups. A longitudinal plot of neurofilament light was generated to assess long-term effects on neurodegeneration. At the last available visit in the EAP, 9/10 participants were alive. The survival curves revealed a statistically significant difference in favor of debamestrocel with a median survival time of 46.6 months for the debamestrocel group compared to 41.1 months for the matched control. Among the six ‘early-start’ participants, a continual reduction in NfL was observed. In contrast, for those who received placebo in the Phase 3, the group median NfL change was 37% by the end of phase 3, indicating worsening neurodegeneration. However, after these participants received debamestrocel in the EAP, the majority showed a stabilization in NfL levels. Up to approximately 200 participants with ALS are expected to enroll in the two-part trial, to receive 3 doses of either debamestrocel or placebo for 24 weeks, followed by an open label period of receiving 3 doses debamestrocel for another 24 weeks. Participants in both treatment arms will be able to receive standard of care while on study. The key entry criteria will include: age 18 to 75 years old, ALS diagnosis defined by the revised El Escorial criteria as laboratory-supported probable, clinically probable, or definite, symptom onset within 24 months of screening, 2 points on each item of the ALSFRS-R, ALSFRS-R total score 45, and upright Slow Vital Capacity 65% of predicted. The primary efficacy endpoint will be a comparison of change in ALSFRS-R from baseline to week-24 for debamestrocel vs. placebo. Other outcome assessments will include CAFS, SVC, HHD ALSAQ-40 questionnaire, the ZBI, and survival. CSF and blood samples will be collected for analysis of biomarkers of neuroinflammation, neurodegeneration, and neuroprotection. A sample using an oral swab can be collected for DNA evaluation of ALS-related genes.