Pipeline

Applied Therapeutics is developing a pipeline of novel products that target molecules and pathways with proven roles in disease biology. By leveraging new FDA guidance for the use of biomarkers as well as established clinical endpoints, this approach can accelerate the time from clinical development to approval and to patients who urgently need treatment.

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

Adult study completed; pediatric study ongoing
NDA expected Q4 2021

Oral

Pilot study underway; Registrational study Q4 2021

Oral

Phase 2 ready; clinical study start 2021;
Expanded Access open

BID Oral

Ph 3 trial initiated in Q3 2019; data 1H ‘23

Oral

Sub-study embedded in DbCM Ph 3 trial

Oral

Ph 1 expected 2021

PI3 Kinase Franchise

SC/Oral

Proof of concept preclinical 2021

Peripheral T-cell lymphoma, cutaneous T-cell lymphoma and T-cell acute lymphoblastic leukemia

COMPOUND

MILESTONEs

Aldose Reductase Franchise

AT-007

Adult study completed; pediatric study ongoing
NDA expected Q4 2021

AT-007

Pilot study underway; Registrational study Q4 2021

AT-007

Phase 2 ready; clinical study start 2021;
Expanded Access open

AT-007

Ph 3 trial initiated in Q3 2019; data 1H ‘23

AT-001

Sub-study embedded in DbCM Ph 3 trial

AT-003

Ph 1 expected 2021

PI3 Kinase Franchise

AT-104

Proof of concept preclinical 2021

1 Full results of pivotal Phase 2 adult ACTION-Galactosemia released in 1H 2020.
2 T-cell acute lymphoblastic leukemia, peripheral T-cell lymphoma and cutaneous T-cell lymphoma.

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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