Applied Therapeutics' Investigational Drug Shows Clinical Benefit In Rare Metabolic Disorder, Chalks Out FDA Submission Plans

  • Applied Therapeutics Inc APLT announced results from the ACTION-Galactosemia Kids study of govorestat (AT-007) for galactosemia, a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. 
  • When not appropriately metabolized, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications.
  • Treatment with govorestat demonstrated consistent and sustained clinical benefits in activities of daily living, behavioral symptoms, cognition, adaptive behavior, and tremors.
  • While statistical significance defined as a p-value of <0.05 was not met on the primary endpoint, a systematic improvement over time was demonstrated for the overall primary endpoint (p=0.1030) and pre-specified sensitivity analyses, including cognition (p=0.0698).
  • Individual speech and language components of the primary endpoint were not impacted, which is suspected to be due to a lack of progression in the placebo group and concomitant speech therapy received by almost all children in the trial. 
  • A post-hoc analysis of the global statistical test, including behavior and activities of daily living but excluding speech & language components, demonstrated a statistically significant benefit of active treatment vs. placebo (p=0.0205), which strengthened over time.
  • Govorestat provided a statistically significant benefit on tremors at 18 months (p=0.0428).
  • Govorestat continued to be safe and well-tolerated in all age groups; no treatment-related serious adverse events (SAEs) were reported.
  • The company plans to request a pre-NDA meeting with the FDA as soon as possible to discuss a potential NDA submission in 2H of 2023; it plans to submit a European Marketing Authorization Application in mid-2023.
  • Concurrently, Applied Therapeutics announced a private placement of 9.7 million shares at $0.946 per share and 22 million pre-funded warrants at $0.945 for a gross proceed of $30 million.
  • Price Action: APLT shares are up 9.94% at $1.04 on the last check Monday.
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