- Sarepta Therapeutics (NASDAQ:SRPT) released data on SRP-5051 demonstrating the effectiveness of the 30 mg/kg arm for the Duchenne muscular dystrophy therapy.
- Sarepta reported that the 30 mg/kg dose led to a 4x increase in exon skipping and a 2x increase in dystrophin at 24 weeks of dosing.
- In addition, SRP-5051 at this dose was found to have an 18x increase and 8x increase, in, respectively, exon skipping and dystrophin, compared to the company's EXONDYS 51 (eteplirsen).
- SRP-5051 could also have a dosing advantage over EXONDYS 51 as it only requires once monthly dosing as opposed to once weekly.
- However, hypomagnesemia was identified as a safety concern in two patients, but was corrected with magnesium supplements.
- Part A of the MOMENTUM trial is now complete and part B is intended to be the pivotal trial.
- The part B protocol will be amended to address the hypomagnesemia risk.
- Sarepta shares are down 0.1% to $70.90 in premarket trading.