Applied Therapeutics Reveals Pivotal Study Data In Hereditary Neuropathy

  • Applied Therapeutics Inc APLT announced sorbitol reduction data from the ongoing global Phase 3 INSPIRE trial of oral AT-007 in approximately 50 patients with SORD Deficiency in the U.S. and Europe. 
  • SORD Deficiency is a hereditary axonal neuropathy caused by mutations in the Sorbitol Dehydrogenase gene, resulting in an inability to metabolize the sugar sorbitol, and accumulation of high levels of toxic sorbitol, causing motor neuron degeneration and loss of mobility and motility. 
  • AT-007 (govorestat) is a central nervous system penetrant Aldose Reductase Inhibitor, which blocks the conversion of glucose to sorbitol. 
  • Related: Applied Therapeutics' Lead Candidate Pediatric Study Failed To Reach Statistical Significance.
  • In a pre-specified interim analysis of the ongoing Phase 3 INSPIRE trial, AT-007 reduced sorbitol levels by a mean of approximately 52% (or approximately 16,000ng/ml) over 90 days of treatment vs. placebo.
  • In the INSPIRE trial, a baseline cross-sectional analysis of the relationship between sorbitol level, age (or duration of disease), and clinical outcome measures demonstrated a statistically significant correlation between sorbitol level and key clinical outcome measures. 
  • The company is working with the FDA to determine the appropriate regulatory path forward, as well as the data required for an NDA submission, 
  • The INSPIRE study will continue in a blinded format to the 12-month interim clinical outcomes assessment. If the primary clinical outcome measure (10-meter-walk/run) reaches statistical significance at 12 months, the study will be completed and unblinded. 
  • If not, the study will continue in a blinded format for 24 months, where clinical outcomes will be assessed in a final statistical analysis. AT-007 continues to be safe and well tolerated to date.
  • Price Action: APLT shares are up 3% at $1.03 on the last check Thursday.
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