RegeNXBio shares fall, despite positive new data on RGX-202

6 June 2025

US gene therapy company RegeNXBio (Nasdaq: RGNX) yesterday announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates include positive functional, safety and biomarker data for RGX-202, RegeNXBio’s potential best-in-class, investigational gene therapy for Duchenne muscular dystrophy.

The functional data demonstrate consistent benefit among dose level 2 participants at 9 and 12 months following treatment with RGX-202, said RegeNXBio, whose shares slumped almost 13% to $8.78.

"Today's findings support the potential of RGX-202 to positively change the disease course for Duchenne and meaningfully benefit patients living with this degenerative disease. At the same dose being used in the pivotal trial, RGX-202 participants exceeded natural history across all key measures, including the North Star Ambulatory Assessment, which is striking," said Dr Steve Pakola, chief medical officer of RegeNXBio.

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