Sarepta up 5% premarket on positive data from SRP-9003 trial

Sarepta Therapeutics (NASDAQ:SRPT) is up 5% premarket on announcing nine-month functional results from three Limb-girdle muscular dystrophy Type 2E (LGMD2E) clinical trial participants who received SRP-9003, an investigational gene therapy intended to transduce skeletal and cardiac muscle with a gene that codes for the full-length, native beta-sarcoglycan protein.

In Cohort 1 of the study, three participants ages 4-13 were treated with an infusion of SRP-9003 at a dose of 5×10¹³vg/kg. Improvements in functional outcomes were observed at day 270 (nine months) for all three participants.

At Day 270, mean creatine kinase was significantly reduced compared to baseline. All three participants showed improvements from baseline across all functional measures.

No new safety signals were observed and the safety profile seen to date supports the ability to dose escalate in the next cohort of the study.

https://seekingalpha.com/news/3503968-sarepta-5-percent-premarket-positive-data-srpminus-9003-trial