BrainStorm Cell Therapeutics Shared Positive NurOwn® Data at 2024 NEALS Meeting

BrainStorm Cell Therapeutics Inc. recently showcased two significant posters on NurOwn® (MSC-NTF0 or debamestrocel) during the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis Consortium (NEALS) Meeting, held virtually from October 21 to 24. The posters, titled '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,' outlined the findings from the Expanded Access Program (EAP) for ALS patients treated with NurOwn, as well as the planned Phase 3b trial design.

In the study titled 'Debamestrocel Long-Term Benefits on Survival and Neurodegeneration in ALS Expanded Access Program,' ten participants from BrainStorm's previous Phase 3 trial were enrolled in an open-label EAP. Six of these participants received Debamestrocel, while four received a placebo. The participants, over two 28-week periods with a break in between, were administered Debamestrocel intrathecally every eight weeks, up to six doses. Their baseline characteristics were matched with a comparable cohort from the PRO-ACT historical database using propensity score matching (PSM).

The PSM included factors such as time since disease onset, ALSFRS-R slope before the baseline, age, Slow/Forced Vital Capacity (SVC/FVC), and site of onset, with a 10:1 matching ratio. A Kaplan-Meier plot and a log rank test were used to compare survival rates between the two groups. A longitudinal plot of neurofilament light (NfL) was generated to evaluate long-term effects on neurodegeneration.

The results were promising, showing that at the last available visit in the EAP, nine out of ten participants were alive. The survival analysis showed a statistically significant improvement in the debamestrocel group, with a median survival time of 46.6 months compared to 41.1 months for the control group. Additionally, early-start participants who received Debamestrocel during the Phase 3 study demonstrated a continual reduction in NfL levels, indicating reduced neurodegeneration. Participants who initially received a placebo showed significant NfL increase by the end of Phase 3, but after receiving Debamestrocel in the EAP, most experienced stabilization in NfL levels.

Dr. Bob Dagher, Executive VP and Chief Medical Officer at BrainStorm, expressed satisfaction with the findings, noting that the long-term survival benefit and reductions in NfL were encouraging. This aligns with their understanding of NurOwn's mechanism of action, providing extended benefits to patients.

The 'An Overview of The Phase 3b Clinical Trial of Debamestrocel in ALS' poster detailed the upcoming trial, which is anticipated to enroll approximately 200 ALS participants. The trial will be conducted in two parts: Part-A, where participants will receive three doses of either Debamestrocel or placebo over 24 weeks, and Part-B, an open-label period of 24 weeks with three doses of Debamestrocel.

Key entry criteria for participants include being aged 18 to 75 years, having an ALS diagnosis as per the revised El Escorial criteria, symptom onset within 24 months of screening, and various other clinical measures. The primary efficacy endpoint will be the change in ALSFRS-R score from baseline to week 24, comparing Debamestrocel with placebo. Secondary assessments will include clinical function and survival assessments, SVC, HHD, ALSAQ-40 questionnaire, ZBI, and survival. Biomarkers for neuroinflammation, neurodegeneration, and neuroprotection will be analyzed from CSF and blood samples.

Dr. Dagher emphasized the importance of confirming NurOwn's efficacy in early-stage ALS patients through the Phase 3b trial. He highlighted that BrainStorm has aligned with the US FDA on the trial's Special Protocol Assessment and Chemistry, Manufacturing, and Controls, thereby reducing regulatory risks. BrainStorm looks forward to advancing the trial and providing further updates.

NurOwn® technology represents a promising therapeutic approach for neurodegenerative diseases, producing autologous MSC-NTF cells that secrete neurotrophic factors targeting specific disease pathways. With a strong commitment to ALS, BrainStorm continues to pioneer innovations in this field through extensive clinical research and trials.