Applied Therapeutics Announces Positive Data Trend in AT-007 ACTION-Galactosemia Kids Pediatric ...
Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced that the ACTION-Galactosemia Kids Phase 3 trial has demonstrated a trend in clinical benefit favoring AT-007 vs. placebo
• The study did not yet reach statistical significance at 12 months of treatment; however, AT-007 (gavorostat) demonstrated a trend in clinical benefit vs. placebo
• Applied Therapeutics plans to meet with the EMA to discuss potential submission of the current data package for conditional approval in the EU
• Trial will continue to 18 months in blinded format
NEW YORK, Oct. 06, 2022 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced that the 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 6 months by a firewalled Data Monitoring Committee (DMC) until the study reaches statistical significance. Review of the data at 12 months of treatment by the DMC indicated that while the study primary endpoint has not yet reached statistical significance, a trend exists favoring AT-007 vs. placebo. The clinical benefit at this early time point was most pronounced in patients with significant deficits in clinical performance at baseline. Safety data demonstrated that AT-007 continues to be safe and well tolerated. The study will proceed in blinded format to the next review at 18 months of treatment. In the meantime, the Company will meet with the EMA to discuss potential submission of an MAA based on existing data for conditional approval.
“Galactosemia is a debilitating disease that greatly impacts patients and families, and there are currently no approved treatments available,” said Riccardo Perfetti, MD, PhD, Chief Medical Officer of Applied Therapeutics. “The data to date in the ACTION-Galactosemia Kids study provides hope for patients and families living with this disease. We know that entering a child in a long-term placebo-controlled study is a difficult decision, and we are truly thankful to the families who are participating in the ACTION-Galactosemia clinical program.”
About AT-007
