REGENXBIO Announces Positive Topline Results from Pivotal Phase III AFFINITY DUCHENNE® Study of RGX-202
REGENXBIO announced positive topline results from the pivotal Phase III AFFINITY DUCHENNE study of RGX-202, a gene therapy for Duchenne Muscular Dystrophy. The trial met its primary endpoint with 93% of participants achieving microdystrophin expression above 10% at Week 12 (p<0.0001). Additionally, 80% of participants achieved microdystrophin expression above 40%. RGX-202 demonstrated a statistically significant correlation between microdystrophin expression and functional improvement, as measured by the North Star Ambulatory Assessment (NSAA) and timed function tests.
Key figures
- Drug Name
- RGX-202
- Phase Of Trial
- Phase III
- Safety profile
- Well-tolerated with a favorable safety profile; mean GGT and total bilirubin did not exceed the upper limit of normal up to one year post-treatment (n=9)
- Functional improvement
- Statistically significant correlation with functional improvement (NSAA change from baseline, correlation= .094, p = 0.0002)
- Microdystrophin expression
- 93% of participants achieved >10% microdystrophin expression at Week 12
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