Applied Therapeutics (APLT)

Executive Summary

Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing transformative treatments for rare diseases with high unmet need, particularly CNS metabolic disorders. Its lead asset, govorestat, is a novel, CNS-penetrant aldose reductase inhibitor (ARI) being investigated for galactosemia, a rare genetic disease with no approved therapies. The company has completed a pivotal Phase 3 study (INSPIRE) evaluating govorestat in children with galactosemia, demonstrating significant improvements in clinical outcomes and biomarker reduction. Additionally, govorestat is being explored in other CNS indications driven by aldose reductase pathology, such as sorbitol dehydrogenase deficiency. The company also has a preclinical pipeline targeting additional rare diseases. Financially, Applied Therapeutics maintains a strong balance sheet to fund operations through key milestones. With a clear regulatory pathway and high unmet need, the company is positioned for potential near-term value creation, pending regulatory interactions and commercial launch preparations.

Upcoming Catalysts (preview)

• Q1 2027FDA Approval Decision for Govorestat in Galactosemia75% success
• H2 2026Phase 3 Data Readout for Govorestat in Sorbitol Dehydrogenase Deficiency60% success
• Q2 2027Regulatory Submission in EU for Govorestat in Galactosemia70% success