About Applied Therapeutics

Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need.

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Our Approach

Applied Therapeutics leverages technological advances to target molecules and pathways with proven roles in disease biology.

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  • Applied Therapeutics' first development target is an enzyme called Aldose Reductase. Utilizing advanced crystallography, the company has developed a unique understanding of structural changes within the enzyme active site, which has enabled functional design of new compounds to improve binding affinity and specificity. These new compounds have higher Aldose Reductase inhibitory activity and less off-target activity.

Applied Therapeutics is also leveraging new FDA guidance for the use of biomarkers as well as established clinical endpoints to bring products to market.

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  • In rare diseases such as Galactosemia, the company is following FDA guidance on slowly progressive, low-prevalence rare diseases that are associated with substrate deposition and are caused by single enzyme defects. This guidance allows for the use of biomarker-based trials as a basis for approval.

  • In more common diseases such as Diabetic Cardiomyopathy, the company is utilizing established and validated clinical endpoints, such as functional capacity (Peak VO2) and biomarkers such as NT-proBNP (a cardiac stress biomarker) to demonstrate clinical effectiveness and safety.

  • By applying the right science and the right development strategies, the company believes it can accelerate time from clinical development to approval, and into the hands of patients who urgently need treatment. When someone is fighting a fatal or debilitating disease for which no drugs are available, every day counts.

By applying the right science and the right development strategies, Applied Therapeutics aims to rapidly advance new treatments for patients with high unmet medical need.

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Our PIPELINE

Applied Therapeutics is building a pipeline of novel drugs to treat diseases with no treatment options. Our Aldose Reductase Inhibitor (ARI) franchise is in development across multiple disease areas, including Galactosemia, Diabetic CardiomyopathyDiabetic Peripheral Neuropathy and other diseases. The company’s preclinical pipeline also includes a PI3 kinase inhibitor in development for orphan oncology indications.

Our Mission

Our mission is to create transformative treatments for patients with high unmet need conditions. 

By applying the right science and the right development strategies, Applied Therapeutics aims to rapidly advance new treatments for patients with high unmet medical need.

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