REGENXBIO (NASDAQ:RGNX) shared positive interim data from its Phase I/II AFFINITY DUCHENNE trial on Wednesday, bolstering the case for its gene therapy candidate, RGX-202.
The data, reflecting a cutoff of Jan. 5, 2026, focused on participants receiving the pivotal dose of 2x10^14 GC/kg, a cohort that has demonstrated significant functional improvements compared to external natural history controls.
Clinical outcomes measured by the North Star Ambulatory Assessment (NSAA) showed a mean improvement of +4.9 points at one year compared to the cTAP external control group.
Notably, the effect was even more pronounced in older participants (aged 8 and above), who saw a +5.2 point improvement.
Cardiac MRI measures remained stable across the cohort, a critical metric for Duchenne patients who often face progressive heart muscle weakening.
The safety profile for RGX-202 appears increasingly robust.
The company reported no serious adverse events (SAEs) or adverse events of special interest (AESIs).
Liver markers—frequently a point of concern in high-dose AAV gene therapies—remained below thresholds that would necessitate clinical intervention.