BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced new survival data from 10 participants in its Expanded Access Program (EAP) for NurOwn® (autologous MSC-NTF cells) in amyotrophic lateral sclerosis (ALS). The EAP enrolled 10 participants who had previously completed the Phase 3 clinical trial.
The analysis reviewed survival from the time of first symptom onset through participation in the Phase 3 trial, EAP Periods 1 and 2, followed by additional survival data collected through publicly available records.
EAP participants presented at the start of the Phase 3 trial relatively early in their disease course, with Mean ALSFRS-R at Baseline of 35.8 (Range: 32 – 40), followed by Mean ALSFRS-R at the start of the EAP of 31.4 (Range: 27 – 38; with one outlier score of 13).
Key findings include:
- 90% of participants (9/10) survived more than five years from the onset of ALS symptoms, compared to published estimates indicating that approximately 10% of individuals with ALS survive beyond five years. Notably, the single death in the cohort occurred following elective euthanasia.
- The median survival observed in the EAP cohort was 6.8 years (range: 6 to 7 years) from symptom onset.
- Given that approximately 10% of individuals with ALS survive beyond five years[1], the probability of observing such extended survival in 9 out of 10 participants purely by chance is extremely low. This strongly suggests that the survival outcomes in this cohort are unlikely to be the result of random variation alone.
“These survival data provide encouraging real-world insights into the long-term experience of ALS patients treated with NurOwn under expanded access,” said Chaim Lebovits, President and CEO of BrainStorm Cell Therapeutics. “We believe these findings add further evidence to support the upcoming Phase 3b clinical trial of NurOwn, to be conducted under an FDA Special Protocol Assessment (SPA). Our goal remains to generate high quality and conclusive data to demonstrate the benefit of NurOwn in people living with ALS.”
Bob Dagher, MD, Chief Medical Officer of BrainStorm Cell Therapeutics, stated: “The fact that 9 out of 10 EAP participants surpassed five years of survival from first symptom – in contrast with the very modest expected survival pattern typically seen in ALS – represents a clinically meaningful observation. Furthermore, 6 out of 10 patients are still alive, even post seven years from symptom onset. These data further strengthen the scientific rationale to pursue our pivotal Phase 3b trial of NurOwn in ALS, under full alignment with the FDA guidance.”
The NurOwn EAP was designed to provide compassionate access to NurOwn for eligible ALS participants who completed the Phase 3 trial. The observed durability of survival in this cohort, as reflected in the publicly sourced survival data, underscores the importance of advancing the upcoming registrational Phase 3b controlled clinical trial.
