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.

PTC Therapeutics (NASDAQ:PTCT) said a Phase 2 placebo-controlled study of its ALS drug candidate utreloxastat failed to meet its primary and secondary efficacy endpoints. The biotech company said the drug failed to demonstrate statistical significance in the slowing of disease progression,

Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect PTC, instead postulating that the upcoming FDA decision on its phenylketonuria candidate sepiapterin will be a stronger driver of the biotech’s stock.