- 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.