Applied Therapeutics, Inc. announced presentations at the 2022 Annual Meeting of the Peripheral Nerve Society, May 14 - 17 in Miami, Florida. The data presented at PNS represents a comprehensive understanding of the role of toxic sorbitol in disease pathogenesis in SORD Deficiency, as well as ability to reduce sorbitol levels and prevent disease progression with AT-007 treatment. In a pilot study in patients with SORD Deficiency, sorbitol level correlated with disease severity, and AT-007 treatment substantially reduced sorbitol levels.

In a Drosophila model of disease, elevated sorbitol resulted in neuronal damage and decline in mobility, and AT-007 treatment prevented the disease phenotype by inhibiting sorbitol production. Taken together, this data significantly advances our understanding of the disease and potential for treatment with AT-007.