PRO051: Additional Extension study data

Additional data from an extension phase of a 5-week, open-label Phase I/IIa trial in 12 patients ages 5-16 showed that weekly subcutaneous PRO051 improved mean 6-minute walking distance (6MWD) by 35.2 meters from baseline at the end of the 12-week extension, with 3 patients improving by >=65 meters. In the Phase I/IIa trial, new dystrophin expression was observed in 56-100% of muscle fibers in 10 patients, as measured by post-treatment biopsies, and increased in a dose-dependent manner to up to 15.6% of the expression observed in healthy muscle. The most common adverse events were irritation at the injection site, mild and variable proteinuria, and increased urinary alpha-1 microglobulin (A1M) levels. Data were published in the New England Journal of Medicine. ...