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Entrada Therapeutics reported Phase 1/2 data for ENTR-601-44, an exon-44 skipping oligonucleotide for Duchenne muscular dystrophy (DMD), that fell short of analyst expectations, sending the Boston-based company's shares down more than 50% on May 7, 2026.
In the first dosing cohort of the ELEVATE-44-201 study in ambulatory patients aged 6–17, treated patients showed a 2.36 percentage-point increase in dystrophin from a baseline of 4%, well below William Blair and Oppenheimer base-case forecasts of roughly 10–11% and far lower than Avidity/Novartis’ del-zota readout (~25%). Entrada said the therapy showed a favorable safety profile and a statistically significant improvement in time-to-rise, a functional measure.
The company has begun dosing a second cohort at double the original dose (12 mg/kg) with results expected by the end of 2026.
The trial included sites in the U.K. and EU; Entrada previously faced an FDA hold that was lifted in February 2025.


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