Pipeline
Applied Therapeutics is developing a pipeline of novel products that target molecules and pathways with proven roles in disease biology.
Aldose Reductase Inhibitors (ARI)
Aldose Reductase is an enzyme involved in the pathophysiology of numerous diseases with high unmet medical need. Applied Therapeutics has developed a new generation of ARIs that have been developed for conditions such as Galactosemia and Diabetic Cardiomyopathy.
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
PRECLINICAL
PHASE 1
PHASE 2
PHASE 3
DOSING
ROUTE
MILESTONES
Aldose Reductase Franchise
QD Oral
Pediatric Phase 3 outcomes trial completed; MAA validated Q4 2023 & NDA accepted Q1 2024; PDUFA date Aug 2024
QD Oral
Positive pilot study data; Phase 3 12-month interim data positive; 24 months ongoing
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.
