PTC Therapeutics Announces Topline Results of CardinALS Trial of Utreloxastat in ALS Patients

- 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

About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-class treatments for patients who have little to no treatment options. PTC's strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients. PTC believes this allows it to maximize value for all its stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, Instagram, LinkedIn and X.

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References:

[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.