Drug intelligence / Profile preview

caficrestat

Development stage: Phase 3
Lead developer: Applied Therapeutics
Modality: Small Molecules
Administration: Oral, Subcutaneous

Overview

Caficrestat (AT-001) is a novel, potent, and selective small molecule inhibitor of aldose reductase (AKR1B1), developed by Applied Therapeutics. It is being investigated primarily for the treatment of diabetic cardiomyopathy and diabetic peripheral neuropathy, as well as other diabetic complications such as type 2 diabetes and acute myocardial infarction. Caficrestat works by inhibiting the enzyme aldose reductase, thereby disrupting the polyol pathway that leads to sorbitol accumulation—a process implicated in tissue damage associated with diabetes-related complications. The drug has shown a favorable safety profile in clinical trials and is administered orally or subcutaneously[1][2][3][4][5][6].

Other names: AT 001AT001AT-001

Targets

AR (Adrenergic receptors)

Beyond the preview

Go deeper on caficrestat.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Follow clinical development from study design and recruitment through results.

Trial phase
Status
Readouts

Indications & development

Explore development by indication, patient population, and geography.

Indications
Development status
Countries

Licensing & deals

Trace asset ownership, licensing agreements, and commercial partnerships.

Partners
Deal terms
Milestones

Patents & exclusivity

Explore the patent landscape and regulatory exclusivity around an asset.

Patents
Expiration dates
Exclusivity

Competitive landscape

Compare development programs by target, modality, and indication.

Competing assets
Targets
Development stage

Research & analysis

Connect source evidence and development news to your research questions.

Publications
News
Analysis

Bring the full picture into focus.

See how Gosset can support your research on caficrestat.

Explore the full profile