Pipeline

Applied Therapeutics is developing a pipeline of novel products that target molecules and pathways with proven roles in disease biology.

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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

QD Oral

Phase 2 ready;
Expanded Access open

BID Oral

Phase 3 trial topline data reported

BID Oral

Sub-study embedded in DbCM Phase 3 trial

Oral

Phase 1 ready

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.  

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