Applied Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative drug candidates targeting validated molecular pathways for diseases with significant unmet medical needs. Their approach utilizes advancements in technology to identify and target specific molecules and pathways involved in disease biology. This includes leveraging new FDA guidance on biomarkers and established clinical endpoints, which can expedite the process from clinical development to regulatory approval, ultimately facilitating quicker access to treatments for patients in need.

One of the key drug candidates in their pipeline is Govorestat (AT-007), an investigational Aldose Reductase Inhibitor (ARI) designed to treat rare diseases such as Galactosemia, SORD Deficiency, and PMM2-CDG. Govorestat has demonstrated the ability to cross the blood-brain barrier and has received various designations from regulatory agencies, including Orphan Drug and Pediatric Rare Disease designations from the FDA. Clinical studies have shown that Govorestat effectively reduces toxic metabolite levels associated with these conditions, indicating its potential as a transformative treatment option. The ongoing research and clinical trials aim to establish its efficacy and safety across different patient populations, reinforcing the company's commitment to addressing critical health challenges.

United States

Applied Therapeutics is a clinical-stage biopharmaceutical company that uses our novel drug-development approach to create transformative, life-changing treatments for the patients who desperately need them.

Age-related diseases

Cardiovascular Diseases

Eye Diseases

Genetic Diseases

Metabolic Diseases

Rare diseases

Applied Therapeutics

Sorbitol dehydrogenase deficiency

AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in clinical development for treatment of Galactosemia. AT-007 has been studied in an animal model of Galactosemia, which demonstrated that AT-007 reduces toxic galactitol levels and prevents disease complications.&nbsp;Applied Therapeutics&nbsp;is conducting a biomarker based development program in patients with Galactosemia, based on the recently released industry guidance on drug development for low prevalence, slowly progressing rare metabolic diseases. The company received Orphan Designation for AT-007 for Galactosemia in&nbsp;May 2019 and Pediatric Rare Disease Voucher (PRV) designation in 2020.

Govorestat (AT-007) 

Galactosemia

Rare genetic disease

Govorestat  (AT-007)

Diabetic cardiomyopathy

Genetic disorder

Hemophilia

AT-001 is an investigational oral, novel, potent Aldose Reductase inhibitor in Phase 3 clinical development for the treatment of Diabetic Cardiomyopathy. AT-001 has been previously studied in a Phase 1/2 study in approximately 120 patients with type 2 diabetes, a subset of which had DbCM.

Caficrestat (AT-001)

Congenital disorder of glycosylation

Phosphomannomutase 2 deficiency

AT-007, a novel CNS penetrant Aldose Reductase inhibitor (recently given the INN scientific name gavorestat) has demonstrated biological activity in a validated model of PMM2-CDG.&nbsp; Applied Therapeutics&nbsp;plans to initiate a clinical study in 2021 and is currently working with CDG experts and the FDA to design a robust clinical program.
<a href="https://ir.appliedtherapeutics.com/news-releases/news-release-details/fda-grants-007-pediatric-rare-disease-designation-and-orphan">Source&nbsp;</a>

Applied Therapeutics

Company Details