PTC axes utreloxastat program after phase 2 ALS failure
But utreloxastat had little effect on ALS in the phase 2 trial. PTC powered the study to show whether twice-daily oral dosing of utreloxastat could achieve a two-point difference on ALSFRS, an instrument for evaluating the functional status of ALS patients. The drug candidate fell short of that bar.
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Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect ...
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