Pipeline

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

Lead investigational products AT-001 and AT-007, part of our larger Aldose Reductase Inhibitor (ARI) franchise, are currently in clinical trials. Additional clinical trials in other indications are planned.

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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 are in clinical development 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

PRECLINICAL

PHASE 1

PHASE 2

PHASE 3

DOSING
ROUTE

MILESTONES

Aldose Reductase Franchise

QD Oral

Positive adult and pediatric biomarker data; pediatric Phase 3 outcomes trial ongoing

QD Oral

Positive pilot study data; Phase 3 registrational trial ongoing

QD Oral

Phase 2 ready;
Expanded Access open

BID Oral

Ph 3 registrational trial initiated in Q3 2019; data expected YE 2023

Oral

Sub-study embedded in DbCM Ph 3 trial

Oral

Ph 1 expected 2023

COMPOUND

MILESTONES

Aldose Reductase Franchise

AT-007

Galactosemia
Positive adult and pediatric biomarker data; pediatric Phase 3 outcomes trial ongoing

AT-007

SORD Deficiency
Positive pilot study data; Phase 3 registrational trial ongoing

AT-007

PMM2-CDG
Phase 2 ready; Expanded Access open

AT-007

Diabetic Cardiomyopathy
Ph 3 registrational trial initiated in Q3 2019; data expected YE 2023

AT-001

Diabetic Peripheral Neuropathy
Sub-study embedded in DbCM Ph 3 trial

AT-003

Diabetic Retinopathy
Ph 1 expected 2023

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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