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.