Possible factors behind Sarepta’s DMD miss

Sarepta believes the functional endpoint miss in a Phase II trial of its Duchenne muscular dystrophy gene therapy candidate is due to a randomization issue, but investors may be reading the negative result through to its broader gene therapy platform. 

Sarepta Therapeutics Inc. (NASDAQ:SRPT) fell 50% to $84.45 in after-hours trading on Thursday after reporting SRP-9001 met the biological endpoint in the SRP-9001-102 trial, although with lower expression than expected, and missed the primary functional endpoint. ...