PTC Therapeutics, Inc., an international biopharmaceutical company dedicated to the innovation, development, and marketing of clinically unique medicines, announced that the phase 2 CardinALS study, a global and placebo-controlled trial, failed to meet its main goal of slowing ALS disease progression as assessed by the composite ALSFRS-R and mortality metrics. Although a slight numerical advantage was noted in primary outcomes and a link was found between a beneficial clinical impact and reduced levels of plasma neurofilament light chain (NfL)—an indicator of neuronal injury—statistical significance was not attained (p=0.52). Furthermore, secondary efficacy outcomes did not reach statistical significance.
“We extend our gratitude to all patients, their families, and healthcare professionals involved in the CardinALS study,” stated Matthew B. Klein, M.D., CEO of PTC Therapeutics. “While we are disheartened by the inability to demonstrate treatment effectiveness and offer a promising therapy to address the critical needs of ALS patients, we are grateful for the insights gained.”
Despite utreloxastat proving to be safe and well-tolerated in the CardinALS trial, no further development is planned at this stage due to the absence of efficacy and definitive biomarker signals.
Amyotrophic lateral sclerosis, also known as ALS, motor neuron disease, or Lou Gehrig’s disease, is a rare, progressively fatal neurodegenerative condition that impacts motor neurons in both the brain and spinal cord. ALS becomes life-threatening as individuals with this illness eventually lose the abilities to move, speak, eat, and breathe.
PTC is recognized as a global biopharmaceutical company focused on discovering, developing, and commercializing clinically unique medications that benefit both children and adults with rare disorders.