BrainStorm Cell Therapeutics announced new survival data from 10 participants in its Expanded Access Program (EAP) for NurOwn, its expanded autologous stem cell therapy for patients with amyotrophic lateral sclerosis (ALS).
The EAP included participants who had previously completed the company’s Phase 3 clinical trial, which failed to meet its primary endpoints (and is now back for a Phase 3b).
The new analysis examined survival times from the onset of ALS symptoms through participation in the Phase 3 trial, followed by EAP Periods 1 and 2, and subsequent survival data obtained from publicly available records.
EAP participants entered the Phase 3 trial relatively early in their ALS progression, with a mean ALSFRS-R (ALS Functional Rating Scale-Revised; a measure of ALS progression where higher scores indicate better function) score of 35.8 at baseline (range: 32–40). At the start of the EAP, the group’s mean ALSFRS-R score was 31.4 (range: 27–38; with one participant scoring notably lower at 13).
Key observations:
- 90% of participants (9 out of 10) survived more than five years from symptom onset. According to published research, approximately 10% of ALS patients typically survive beyond five years. It was noted that the single death recorded in this cohort occurred following elective euthanasia.
- The median survival in the EAP group was 6.8 years (range: 6 to 7 years) from symptom onset.
- The company notes that given the expected survival rate of approximately 10% at five years for ALS, the likelihood of observing these outcomes purely by chance is very low.
“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 provided compassionate access to eligible ALS patients who previously participated in the Phase 3 trial. According to the company, data from this cohort emphasize the importance of continuing development work, specifically through the upcoming registrational Phase 3b controlled clinical trial.
