Clinical Trial ChallengesTroriluzole has a checkered history as the drug previously failed in a Ph2/3 SCA study in 2017, failed in a Ph3 trial in generalized anxiety disorder (GAD), failed in a Ph2/3 study in obsessive compulsive disorder (OCD), and failed in a Phase 2/3 study in Alzheimer's disease.
Financial PerformanceThe company has high cash burn and there is some risk in near-term read-out, such as the SMA trial potentially missing its targets.
Regulatory IssuesThis opportunity carries a significant regulatory risk given the lack of robust placebo-controlled evidence, as the product failed in a Phase 2/3 trial and a Phase 3 trial, and the company received a refuse to file letter previously for its NDA filing based on subgroup analysis.