Clinical Development RiskClinical outcomes remain uncertain as early signals from small cohorts may not replicate in larger pivotal studies, creating potential downside if efficacy or safety differs.
Financing RiskHigh development costs and cash burn could necessitate additional capital raises, which may dilute shareholders or slow program timelines if funding is not secured on favorable terms.
Regulatory UncertaintyFavorable FDA interactions do not guarantee approval or an expedited pathway, and regulatory determinations could require additional data or delay market authorization.