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- Study failed to meet primary and secondary efficacy endpoints -
WARREN, N.J. , Nov. 26, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT ) today announced that 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 favorable clinical effect with lowering of plasma neurofilament light chain (NfL), a biomarker of neuronal damage, statistical significance was not achieved (p= 0.52). In addition, significance was not achieved on the secondary efficacy endpoints.
"We wish to thank all of the patients, their families and physicians who participated in the CardinALS trial," said Matthew B. Klein , M.D., Chief Executive Officer, PTC Therapeutics. "We are of course disappointed that we were not able to demonstrate treatment efficacy and provide a potential therapy that could address the significant unmet medical need of ALS patients."
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.
About Amyotrophic Lateral Sclerosis (ALS) Amyotrophic lateral sclerosis, sometimes referred to as ALS, motor neuron disease, or Lou Gehrig's disease, is a rare, progressive, and fatal neurodegenerative disease that affects motor neurons in the brain and spinal cord. 1,2 ALS becomes life-threatening as people with this disease ultimately lose the ability to move, speak, eat, and breathe. 1
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As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful.
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.
[1] Kiernan MC, et al. Amyotrophic lateral sclerosis. The Lancet, 2011; 377(9769):942-955.
[2] Dhasmana S, et al. The panoramic view of amyotrophic lateral sclerosis: A fatal intricate neurological disorder, Life Sciences 2022;288:120156. https://doi.org/10.1016/j.lfs.2021.120156 .
SOURCE PTC Therapeutics, Inc.