Applied Therapeutics Q3 2024 Financial Results Released

Applied Therapeutics, Inc. reported its financial outcomes for the third quarter ending September 30, 2024. The clinical-stage biopharmaceutical firm is advancing a pipeline of innovative drug candidates targeting crucial molecular markers in conditions with significant unmet medical needs.

In the latest quarter, significant strides have been made towards transitioning from a clinical-stage company to a commercial enterprise. Regulatory submissions for govorestat, targeting two rare diseases, Classic Galactosemia and SORD Deficiency, are in progress. These steps are pivotal as the company prepares for transformative events expected in the upcoming year. Shoshana Shendelman, PhD, Founder and CEO, expressed confidence in govorestat’s potential to address the underlying mechanisms of these diseases, with plans to bring the therapy to patients in 2025.

Regarding Classic Galactosemia, the New Drug Application (NDA) for govorestat is currently under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date set for November 28, 2024. The Marketing Authorization Application (MAA) has also been submitted to the European Medicines Agency (EMA) and is under review by the Committee for Medicinal Products for Human Use (CHMP), with a decision anticipated in the first quarter of 2025. The submission packages for both NDA and MAA are supported by data showcasing significant reductions in galactitol levels, positive clinical outcomes in pediatric patients, and a favorable safety profile. Data from the Phase 3 ACTION-Galactosemia Kids study and the Phase 1/2 study in adults solidify the evidence base for govorestat’s efficacy. If approved, govorestat would be the first drug indicated for Galactosemia, marking a milestone for Applied Therapeutics as it launches its first commercial product.

In relation to SORD Deficiency, following discussions with the FDA’s Neurology I Division, the company is planning an NDA submission for govorestat in early Q1 2025 under Accelerated Approval. The review process for SORD Deficiency is independent of the ongoing Classic Galactosemia review. Data from the Phase 3 INSPIRE study have transitioned patients to open-label treatment for ongoing safety assessment.

Clinical data and the developmental characterization of govorestat for Classic Galactosemia were presented at key medical conferences, including the 2024 Annual Symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM) and the American Society of Human Genetics (ASHG) Annual Meeting 2024. These presentations delved into the disease’s pathogenesis, clinical study designs, and the results of pivotal trials.

Financially, Applied Therapeutics reported cash and short-term investments totaling $98.9 million as of September 30, 2024, up from $49.9 million at the end of 2023. Research and development expenses for the quarter were $14.8 million, an increase from $10.8 million in the same quarter the previous year, driven by higher clinical and regulatory expenses related to govorestat. General and administrative expenses rose to $15.0 million from $4.7 million, attributed to increased legal and professional fees, commercialization costs for govorestat, personnel expenses, and data storage costs. The net loss for the third quarter was $68.6 million, or $0.48 per share, compared to a net loss of $42.4 million, or $0.47 per share, in the third quarter of 2023.

Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel drug candidates targeting central nervous system penetrants for rare metabolic diseases, including Classic Galactosemia, SORD Deficiency, and PMM2-CDG. Additionally, the company is working on AT-001 for Diabetic Cardiomyopathy and AT-003 for Diabetic Retinopathy, expanding its pipeline to address significant medical needs.