Data Interpretability RiskPhase I findings are difficult to interpret because disease heterogeneity can skew outcomes, reducing confidence that early improvements will generalize to broader patient populations.
Early-stage Clinical UncertaintyResults from early trials carry inherent uncertainty about long-term safety, durability, and regulatory acceptance, which can constrain valuation until larger trials provide confirmatory evidence.
Small Sample Size LimitationsSmall cohort sizes in early-stage trials limit statistical confidence and make it uncertain whether observed benefits will persist in larger, more diverse studies.