Sarepta resumes dosing at U.K. sites after DMD trial halt

Sarepta Therapeutics Inc. (NASDAQ:SRPT) President and CEO Doug Ingram said the company has resumed dosing at U.K. sites in the Phase III ESSENCE trial evaluating golodirsen (SRP-4053) and casimersen (SRP-4045) to treat Duchenne muscular dystrophy amenable to exon 53 or 45 skipping, respectively. During a presentation Tuesday at the Barclays Global Healthcare Conference in Miami, Ingram said the U.K.'s Medicine and Healthcare products Regulatory Agency (MHRA) reviewed the study and allowed dosing to resume.

Sarepta had halted dosing at U.K. study sites last month as a result of a serious adverse event (SAE) that triggered an MHRA stopping rule, Ingram said. The SAE was rhabdomyolysis and elevated creatine kinase levels, both of which are common in DMD patients, he noted (see BioCentury Extra, Feb. 9).

Last month, Sarepta said an IDMC had concluded that dosing could continue based on a review of safety data from all ESSENCE patients.

Sarepta said Monday it plans to complete the submission of a rolling NDA to FDA by year end seeking accelerated approval of golodirsen to treat DMD amenable to exon 53 skipping. The company believes ESSENCE may serve as a postmarketing confirmatory study for golodirsen, if it receives accelerated approval (see BioCentury Extra, March 12).

Golodirsen and casimersen are phosphorodiamidate morpholino oligomers (PMOs) targeting exons 53 and 45, respectively.

Ingram's presentation took place during market hours Tuesday. Sarepta gained $1.80 to $80.90 on Tuesday, and climbed another $1.37 to $82.27 on Wednesday.