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ALS ‘Urgency’, US FDA Regulatory Environment Justify Filing NurOwn, BrainStorm Says

 
• By Sue Sutter

BrainStorm discovered a statistical error while preparing the stem cell therapy's BLA, and corrected data show a statistically significant treatment difference for a key secondary endpoint in less advanced ALS. However, the Phase III trial still failed its primary clinical efficacy endpoint.

Steep climb
BrainStorm's ALS drug may face a steep climb to US regulatory approval. • Source: Shutterstock

BrainStorm Cell Therapeutics Inc. is citing the regulatory environment, the US Food and Drug Administration’s “urgency” on drugs for amyotrophic lateral sclerosis, as well as a newly corrected subgroup analysis for a secondary endpoint in its Phase III trial, in explaining why it plans to seek approval of the stem cell therapy NurOwn.

The success of a biologics license application submission would seem to face long odds given the Phase III trial failed its primary endpoint and secondary endpoints. In addition, the FDA...

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