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.
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
PI3 Kinase Franchise
† Peripheral T-cell lymphoma, cutaneous T-cell lymphoma and T-cell acute lymphoblastic leukemia
COMPOUND
MILESTONEs
Aldose Reductase Franchise
AT-007
Positive adult and pediatric biomarker data; pediatric Phase 3 outcomes trial ongoing
AT-007
Positive pilot study data; Phase 3 registrational trial ongoing
AT-007
Phase 2 ready; Expanded Access open
AT-007
Ph 3 registrational trial initiated in Q3 2019; data 1H ‘23
AT-001
Sub-study embedded in DbCM Ph 3 trial
AT-003
Ph 1 expected 2022
PI3 Kinase Franchise
AT-104
Proof of concept preclinical
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.
