Applied Therapeutics' Lead Candidate Pediatric Study Failed To Reach Statistical Significance

Applied Therapeutics Inc's APLT ACTION-Galactosemia Kids Phase 3 trial has demonstrated a trend in clinical benefit favoring AT-007 vs. placebo.
The study is designed to evaluate the impact of AT-007 vs. placebo on clinical outcomes in children with Classic Galactosemia, with a review of safety and efficacy every six months.
Classic galactosemia is an inherited condition in which the body cannot properly digest galactose, a sugar found in all foods containing milk. The sugar buildup can cause seizures, blood infections, liver damage, or even death if left untreated.
A data review at 12 months by the monitoring committee indicated that while the study's primary endpoint has not yet reached statistical significance, a trend exists favoring AT-007 vs. placebo.
Safety data demonstrated that AT-007 continues to be safe and well tolerated.
The study will proceed in a blinded format to the next review at 18 months of treatment.
In the meantime, the company will meet with the European Medicines Agency to discuss the potential application submission based on existing data for conditional approval.
Price Action: APLT shares are down 30.53% at $0.66 on the last check Thursday.