US biopharmaceutical company PTC Therapeutics Inc (NASDAQ: PTCT) on Tuesday reported topline results for its CardinALS trial of utreloxastat in Amyotrophic Lateral Sclerosis (ALS) patients.

The global Phase 2 placebo-controlled CardinALS study did not meet its primary endpoint of slowing disease progression on the composite ALSFRS-R and mortality analysis.

While there was modest numerical benefit recorded on the primary endpoint and correlation of favourable clinical effect with lowering of plasma neurofilament light chain (NfL), a biomarker of neuronal damage, statistical significance was not achieved. Nor did the trial achieve its secondary endpoints.

Utreloxastat was demonstrated to be safe and well tolerated in the CardinALS trial. However, due to the lack of efficacy and biomarker signal, further development is not planned at this time.

ALS is a rare, progressive and fatal neurodegenerative disease.