PTC Therapeutics ALS drug fails to meet endpoints as trials shut down
The announcement means the company will be pulling the plug on the CardinALS trial after its amyotrophic lateral sclerosis drug failed to halt disease progression.
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.
PTC Scraps ALS Asset Utreloxastat After Mid-Stage Fail, Analysts Look to PKU Opportunity
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.
PTC Therapeutics: Phase 2 CardinALS study did not meet its primary endpoint
PTC Therapeutics (PTCT) announced that the global Phase 2 placebo-controlled CardinALS study did not meet its primary endpoint of slowing
PTC's utreloxastat fails Phase 2 clinical study for ALS
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,
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Macquarie Spinout Lands $16.75M for ALS Gene Therapy
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