Applied Therapeutics is on track for a potentially transformative year as it awaits a decision from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for govorestat in the treatment of Classic Galactosemia. The FDA's decision is expected by the Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024. Simultaneously, the company is preparing an NDA submission for govorestat to treat SORD Deficiency, anticipated in early Q1 2025.
Regulatory Milestones for Govorestat
The NDA for govorestat in Classic Galactosemia is currently under review within the FDA's Division of Rare Diseases and Medical Genetics. Govorestat has been granted Pediatric Rare Disease designation, which would qualify Applied Therapeutics for a Priority Review Voucher (PRV) upon approval. In parallel, the Marketing Authorization Application (MAA) for govorestat in Classic Galactosemia is under review by the European Medicines Agency (EMA), with a decision expected in early Q1 2025. The NDA and MAA submissions are supported by data demonstrating rapid and sustained reduction in galactitol, leading to meaningful clinical benefits in pediatric patients, along with a favorable safety profile. These submissions include clinical outcomes data from the Phase 3 ACTION-Galactosemia Kids study in children aged 2-17, the Phase 1/2 ACTION-Galactosemia study in adult patients, and preclinical data.
Following a Type C meeting with the FDA, Applied Therapeutics plans to submit an NDA under Accelerated Approval for govorestat in SORD Deficiency in early Q1 2025. The review and potential approval of govorestat for SORD Deficiency are independent of the ongoing review for Classic Galactosemia. If govorestat is approved for Classic Galactosemia, the SORD Deficiency submission will be a supplementary New Drug Application (sNDA). Patients from the Phase 3 INSPIRE study have transitioned to open-label govorestat treatment for continued safety data collection.
Clinical Data Presentations
Applied Therapeutics presented clinical data and development characterization of govorestat for Classic Galactosemia at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium and the American Society of Human Genetics (ASHG) Annual Meeting 2024. These presentations highlighted the mechanism of disease pathogenesis in Classic Galactosemia, the design of the first clinical outcomes study in Classic Galactosemia, and the results of the ACTION-Galactosemia Kids study.
Financial Position
As of September 30, 2024, Applied Therapeutics reported $98.9 million in cash and cash equivalents and short-term investments, compared to $49.9 million at December 31, 2023. Research and development expenses for the three months ended September 30, 2024, were $14.8 million, compared to $10.8 million for the same period in 2023. General and administrative expenses were $15.0 million for the three months ended September 30, 2024, compared to $4.7 million for the same period in 2023. The net loss for the third quarter of 2024 was $68.6 million, or $0.48 per basic and diluted common share, compared to a net loss of $42.4 million, or $0.47 per basic and diluted common share, for the third quarter 2023.
Applied Therapeutics is focused on developing novel drug candidates against validated molecular targets in indications of high unmet medical need. Govorestat, a central nervous system penetrant Aldose Reductase Inhibitor (ARI), is being developed for CNS rare metabolic diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. The company is also developing AT-001, a potent ARI, for Diabetic Cardiomyopathy (DbCM), and AT-003, an ARI for Diabetic retinopathy.
