Regenxbio (RGNX) said Wednesday new interim results of its gene therapy RGX-202 study in Duchenne muscular dystrophy showed evidence of "positively" changing disease trajectory.
The company said favorable safety profile continued with no serious adverse events or adverse events of special interest observed in the phase I/II study.
The firm also said it expects pivotal topline data in Q2 and plans to seek a pre-Biologics License Application meeting with the US Food and Drug Administration in mid-2026.
Shares were down more than 6% in midday trading.
Price: 9.22, Change: -0.68, Percent Change: -6.87
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