Fresh safe­ty, tol­er­a­bil­i­ty da­ta snap­shot from ear­ly-stage study of Wave's DMD drug spooks in­vestors

Wave Life Sci­ences irked in­vestors with the lat­est snap­shot of safe­ty da­ta on its ex­per­i­men­tal Duchenne mus­cu­lar dy­s­tro­phy (DMD) drug on Tues­day, caus­ing Wall Street to ques­tion whether the re­sults from the ear­ly-stage study fore­shad­owed trou­ble ahead.

The ex­per­i­men­tal drug, su­vodirsen, is be­ing test­ed in the same DMD pop­u­la­tion that Sarep­ta’s $SRPT pi­o­neer­ing Ex­ondys 51 is ap­proved to treat — boys who are amenable to ex­on 51 skip­ping. In the Phase I study, 36 pa­tients re­ceived a dose of 0.5 mg/kg, 1 mg/kg, 2 mg/kg, 5 mg/kg, 7 mg/kg or 10 mg/kg of su­vodirsen (n=26) or place­bo (n=10) in five as­cend­ing dose co­horts and were fol­lowed for 85 days.

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