PTC halts development of ALS drug after trial miss
PTC Therapeutics (Nasdaq: PTCT) has announced that its Phase II trial for utreloxastat, aimed at treating amyotrophic lateral sclerosis (ALS), failed to meet its primary endpoint.
While a modest numerical benefit was observed in the primary endpoint, as well as in a biomarker of neuronal damage, the CardinALS study showed no statistically significant improvement in slowing deterioration, following a commonly used measure of disease progression.
Secondary efficacy endpoints also did not yield significant results. Despite demonstrating safety and tolerability, PTC stated that further development of utreloxastat is not planned.
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