12:30 PM EDT, 09/03/2024 (MT Newswires) -- Dyne Therapeutics ( DYN ) said Tuesday new data from a phase 1/2 trial of DYNE-251 in patients with duchenne muscular dystrophy showed "unprecedented dystrophin expression and functional improvement in multiple cohorts."
The company also said the drug candidate demonstrated a "favorable safety profile" with "treatment emergent adverse events" mostly mild or moderate.
There were no related serious adverse events other than in two trial participants at the 40 mg/kg dose level who have both recovered from "events potentially related to study drug," it said.
The company is planning on moving on to next steps in the study and pursuing expedited approvals. Dyne will provide an update on registration by the end of this year.
Dyne shares were down more than 31% in recent trading.
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