PTC Therapeutics Discontinues Utreloxastat Program Following Phase 2 ALS Trial Failure

Trial Outcome:
The global Phase 2 placebo-controlled CardinALS study of utreloxastat in ALS patients did not meet its primary endpoint of slowing disease progression12.

Safety and Tolerability:
Utreloxastat was found to be safe and well-tolerated in the trial, but lacked efficacy and biomarker signals necessary for further development1.

Program Discontinuation:
Due to the lack of efficacy, PTC Therapeutics has decided not to pursue further development of utreloxastat for ALS treatment at this time12.

Company Background:
PTC Therapeutics is a global biopharmaceutical company focused on developing and commercializing treatments for rare disorders, including ALS1.

Disease Impact:
ALS is a life-threatening neurological disorder that progressively affects the ability to move, speak, eat, and breathe, with no cure currently available12.

Sources:

1. https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-topline-results-cardinals-trial

2. https://www.fiercebiotech.com/biotech/ptc-fails-phase-2-als-trial-axes-utreloxastat-program