Sarepta Therapeutics Inc's (NASDAQ: SRPT) EMBARK Phase 3 study of Elevidys in ambulatory boys (those who can walk) with Duchenne muscular dystrophy aged 4-7 years failed to meet the primary endpoint.
Elevidys-treated patients improved by 2.6 points on their North Star Ambulatory Assessment (NSAA), a measure of motor function 52 weeks after treatment, compared to 1.9 points in placebo-treated patients.
Needham writes that Sarepta will face an uphill battle to expand the label with existing data, and as a result, it lowered the price target from $185 to $82.
Management attributed missing on NSAA to a relatively short follow-up and a milder patient population compared to previous ...