For Duchenne muscular dystrophy patients taking a 50 milligram dose of Sarepta Therapeutics' (NASDAQ: SRPT) eteplirsen for 48 weeks, that's how much longer the average patient walked during a six-minute walk test than a placebo group, which began taking the drug after 24 weeks. Not only did the 50mg group outperform placebo, but it showed an average 27.1 meter increase in distance walked over the treatment period -- an astonishing improvement over the 36-week data, which showed a 5.2 meter decline for the 50mg group.
Not only did the walk test show a statistically significant benefit, but measures of dystrophin, a vital protein that protects muscle fibers, showed statistically significant increases in both the initial treatment and delayed treatment/placebo group.
This is the kind of data that could merit an accelerated approval from the Food and Drug Administration. We'll have to wait to find out for sure, but shareholders are already piling in, bidding up shares by more than 110% in early morning trading.
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