PTC Therapeutics (PTCT) said late Tuesday that a global phase 2 trial of Utreloxastat in patients with amyotrophic lateral sclerosis failed to meet its primary and secondary efficacy endpoints.
The study didn't meet its primary endpoint of slowing disease progression on the composite ALSFRS-R and mortality analysis, while significance wasn't achieved on the secondary efficacy endpoints, the company said.
Although Utreloxastat was safe and well tolerated in the trial, further development is currently not planned due to the lack of efficacy and biomarker signal, PTC said.
Amyotrophic lateral sclerosis is a rare and fatal neurodegenerative disease that affects motor neurons in the brain and spinal cord.
The company's shares were down 5.5% in after-hours activity.
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