Pipeline
Applied Therapeutics leverages technological advances to target molecules and pathways with proven roles in disease biology. 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.
Applied Therapeutics is developing a pipeline of novel products that target molecules and pathways with proven roles in disease biology to address high unmet need rare diseases.
Aldose Reductase Inhibitors (ARI)
Aldose Reductase is an enzyme implicated in multiple metabolic diseases with high unmet medical need. Applied Therapeutics has developed a new generation of ARIs for the treatment of multiple rare diseases.
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.
COMPOUND
MILESTONES
Aldose Reductase Franchise
govorestat
Galactosemia
Pediatric Phase 3 outcomes trial completed; MAA validated Q4 2023 & NDA accepted
Q1 2024; PDUFA date Aug 2024
govorestat
SORD Deficiency
Positive pilot study data; Phase 3 12-month interim data positive; 24 months
ongoing
govorestat
PMM2-CDG
Phase 2 ready; Expanded Access open
AT-001
Diabetic Cardiomyopathy
Phase 3 trial topline data available
AT-001
Diabetic Peripheral Neuropathy
Sub-study embedded in DbCM Phase 3 trial
AT-003
Diabetic Retinopathy
Phase 1 ready
Compound
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Rights
Status
Phase 3 12-month interim data positive; NDA submission expected Q1 2025
MAA=Marketing Authorization Application; NDA=New Drug Application
Our Approach
Applied Therapeutics leverages technological advances to target molecules and pathways with proven roles in disease biology. 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.
