Sarepta Therapeutics Inc (NASDAQ: SRPT) shares were advancing Wednesday following the release of the results of an early stage trial evaluating its gene therapy candidate MYO-101 in limb-girdle muscular dystrophy type 2E.
Sarepta said the three clinical trial participants who received MYO-101 at a dose of 5*13vg/kg showed robust expression of transduced beta-SG at about two months.
MYO-101 transduces skeletal and cardiac muscle with a gene that codes for the full-length, native beta-SG protein, the lack of which causes LGMD2E.
As opposed to the 20-percent pre-defined measure of success for expression, actual mean protein expression was 51 percent, the company said.
Mean fiber intensity, as measured by IHC, was 47 percent compared to normal control.
Sarepta also noted that all patients showed a notable decrease in serum creatine kinase, a biomarker associated with muscle damage.
Separately, Sarepta said it has exercised its option to acquire Myonexus Therapeutics, the company behind gene therapies for five LGMDs. Subject to the closing of the transaction, Sarepta said it will pay Myonexus shareholders $165 million.
Sarepta is being touted as a M&A candidate due to its rich gene therapy pipeline.
Why It's Important
LGMD2E is an autosomal recessive muscular dystrophy, with affected people showing neuromuscular symptoms before the age of 10. Progression of the disease may lead to a loss of ambulation in teen years and often death by the age of 30.
There is no approved therapy for the indication.
Following the positive results, the company said it will continue the pivotal trial focused on developing MYO-101 for LGMD2E.
Sarepta shares were up 9.86 percent at $153.85 at the time of publication Wednesday.
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