PTC Therapeutics announced on November 26, 2024, that its global Phase 2 placebo-controlled CardinALS study of utreloxastat for Amyotrophic Lateral Sclerosis (ALS) did not meet its primary endpoint. The study aimed to slow disease progression based on a composite ALSFRS-R and mortality analysis.
Statistical significance was not achieved on the primary endpoint (p=0.52), nor on any of the secondary efficacy endpoints. While a modest numerical benefit was recorded and a correlation with lowering of plasma neurofilament light chain (NfL) was observed, these were not statistically significant.
Due to the lack of demonstrated efficacy and biomarker signal, PTC Therapeutics has decided to discontinue further development of utreloxastat. The company expressed disappointment in not being able to provide a potential therapy for ALS patients.
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