Jan. 7 at 3:07 AM
$RGNX $SRPT RGX-202's Phase I/II trial endpoints focus on secondary functional measures like NSAA and timed tests (TTStand, 10MWR, TTClimb), where dose level 2 patients showed mean improvements exceeding natural history controls (e.g., +4.5 points on NSAA vs. expected decline; all improved on timed tests with velocity changes past MCID).��Comparison to SRPT's ELEVIDYSSarepta (SRPT) ELEVIDYS Phase III (EMBARK) missed its primary endpoint of NSAA change at week 52 (+2.6 vs. +1.9 placebo, not statistically significant), though post-hoc and other analyses suggested signals.�
RGX-202 differentiates by consistently beating matched external natural history on all measures in a smaller cohort, with robust microdystrophin expression (46-118% of control) tied to functional gains, unlike ELEVIDYS where biomarker success did not fully translate to primary endpoint success.��
