PTC Therapeutics Reports CardinALS Trial Results for Utreloxastat in ALS Patients

PTC Therapeutics, Inc. recently reported that their global Phase 2 CardinALS study, a placebo-controlled trial, did not meet its primary efficacy objective. The study focused on the progression of Amyotrophic Lateral Sclerosis (ALS) using a composite measure of ALSFRS-R and mortality analysis. Despite a slight numerical benefit observed, statistical significance was not reached, with a p-value of 0.52. Additionally, the trial failed to achieve its secondary efficacy endpoints.

Dr. Matthew B. Klein, CEO of PTC Therapeutics, expressed gratitude to the patients, families, and physicians who participated in the study. He also voiced disappointment over the inability to demonstrate treatment efficacy, which would have addressed a significant unmet medical need for ALS patients.

The experimental drug, Utreloxastat, was shown to be safe and well tolerated among participants. However, due to the lack of efficacy and biomarker signal in the study, the company does not plan to continue its development at this time.

ALS, also known as motor neuron disease or Lou Gehrig's disease, is a rare, progressive, and fatal neurodegenerative condition that impacts motor neurons in the brain and spinal cord. This disorder severely affects patients' ability to move, speak, eat, and breathe, ultimately becoming life-threatening.

PTC Therapeutics is a global biopharmaceutical company dedicated to discovering, developing, and commercializing innovative medicines for rare disorders. The company's strategy involves leveraging its scientific and clinical expertise along with its global commercial infrastructure to bring new therapies to patients with few or no treatment options. PTC's mission is to provide access to best-in-class treatments for these patients, thereby maximizing value for all stakeholders.