Applied Therapeutics Q2 2024 Financial Results

Applied Therapeutics, Inc., a clinical-stage biopharmaceutical company, reported its financial results for the second quarter ending June 30, 2024. The company has been making significant regulatory progress with its lead drug candidate, govorestat, for the treatment of Classic Galactosemia and SORD Deficiency.

Govorestat for Classic Galactosemia and SORD Deficiency
Govorestat, also known as AT-007, is under review by the U.S. FDA and the European Medicines Agency (EMA) for the treatment of Classic Galactosemia. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date for November 28, 2024. The EMA is also reviewing a Marketing Authorization Application (MAA) with a decision expected in early Q1 2025.

In preparation for the FDA's review, a discrepancy in the NIH Toolbox data was found. The vendor initially used an adult formula for calculating the cognition and motor skills scores of pediatric patients. Correcting this to the pediatric formula resulted in significantly improved cognition data for those treated with govorestat, which was statistically significant compared to placebo. This adjustment did not substantially change the motor skills data. These updates have been communicated to both the FDA and EMA.

Govorestat has been granted Pediatric Rare Disease designation, which qualifies it for a Priority Review Voucher (PRV) upon approval. Clinical data supporting the NDA and MAA submissions include Phase 3 registrational data from the ACTION-Galactosemia Kids study and Phase 1/2 data from adult patients with Galactosemia.

Additionally, the FDA's Genetic Metabolic Diseases Advisory Committee (GeMDAC) is tentatively scheduled to meet on October 9, 2024, to discuss the NDA for govorestat for Classic Galactosemia.

Regarding SORD Deficiency, the company has aligned with the Neurology I Division of the FDA to submit an NDA under Accelerated Approval. This submission is expected in early Q1 2025. If approved, govorestat would be Applied Therapeutics' first commercial product.

Financial Highlights
As of June 30, 2024, the company reported cash and cash equivalents totaling $122.2 million, a significant increase from $49.9 million as of December 31, 2023. This increase is attributed to various financing activities and is expected to fund the company's operations into 2026.

Research and development expenses for the quarter decreased to $10.0 million from $11.9 million in the same period of the previous year. This reduction was primarily due to decreased expenses associated with the near completion of govorestat and AT-001, offset by increased regulatory and personnel expenses.

General and administrative expenses rose to $10.6 million from $5.3 million, driven by higher legal and professional fees, commercial expenses in anticipation of govorestat's potential commercialization, and increased personnel costs.

The company reported a net income of $2.9 million for the second quarter of 2024, compared to a net loss of $29.6 million for the same period in 2023. This improvement was partly due to a change in the fair value of warrant liabilities.

Recent Developments
Applied Therapeutics was added to the Russell 3000 Index in June 2024, which tracks the performance of the largest 3,000 U.S. stocks. This inclusion is expected to increase the company's visibility among investors.

The company also deepened its engagement with the patient community by participating in several medical and patient advocacy group conferences, including events organized by the Hereditary Neuropathy Foundation and the Galactosemia Foundation.

Looking Ahead
Applied Therapeutics continues to focus on advancing its drug candidates through regulatory pathways. The potential approval of govorestat for Classic Galactosemia and SORD Deficiency represents significant milestones. The company remains committed to improving the lives of patients with these rare neurological diseases by bringing transformative treatments to market.