Financial PerformanceThere is some risk in near-term read-out, such as the SMA trial potentially missing its targets, and the company has high cash burn.
Market ConcernsInvestor concerns over plateauing effect are driving a selloff.
Regulatory RiskThis 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.