Modest differences in outcomes for doses of Audentes myopathy gene therapy

Audentes presented updated data from the Phase I/II ASPIRO trial of AT132 to treat X-linked myotubular myopathy (XLMTM) showing that the trial's low and high doses had comparable safety and efficacy profiles.

Chairman and CEO Matthew Patterson told BioCentury that Audentes Therapeutics Inc. (NASDAQ:BOLD) will rely on additional analyses in the coming months to determine which of the two doses it will take forward, and make a decision in 3Q19 on whether to submit a BLA and MAA or if additional trials will be needed to support the submissions...