Applied Therapeutics, Inc. provided a regulatory update on the AT-007 Galactosemia program. Following discussions with the FDA at the end of the year, the Company has decided to hold on submitting an NDA for AT-007 for treatment of Galactosemia pending additional discussions with the agency. Although the Galactosemia program had previously been discussed in the context of an NDA for Accelerated Approval based on reduction in galactitol, the FDA has now indicated that clinical outcomes data will likely be required for approval.

The ongoing ACTION-Galactosemia Kids Phase 3 study is evaluating the impact of AT-007 treatment vs. placebo on clinical outcomes over time, including cognition, speech, behavior and motor skills. Clinical outcomes are assessed every 6 months by a firewalled committee.

The first assessment will be completed in the first quarter of 2022, and then every 6 months thereafter until the study reaches statistical significance. AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency and PMM2-CDG. In an animal model of Galactosemia, AT-007 reduced toxic galactitol levels and prevented disease complications.

In clinical trials, AT-007 significantly reduced plasma galactitol levels vs. placebo in adults and children with Galactosemia. AT-007 is currently being studied in a Phase 3 clinical outcomes trial (ACTION-Galactosemia Kids) in children ages 2-17 with Galactosemia, as well as a long-term open label study in adults with Galactosemia.

AT-007 has received both Orphan Drug and Pediatric Rare Disease designations from the U.S. Food and Drug Administration (FDA) for the treatment of Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia.