Drugs / caficrestat
last change Dec 2025 re-read 3 minutes ago

caficrestat

also known as AT001 · Aldose reductase-in-1

Small molecule targets AKR1B1 via inhibition

Developed for
cardiomyopathy · COVID-19 · disorder of defective peroxisome oxidative status
Investigated by
Alltech Life Sciences Inc. · Applied Therapeutics, Inc. · NYU Langone Health

Trials 3

2012201320142015201620172018201920202021202220232024202520262027
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial
Phase 1 NCT01731093 Mar 2012 → Oct 2013 disorder of defective peroxisome oxidative status Alltech Life Sciences Inc. Completed No outcome recorded
Phase 21 trial
Phase 2 NCT04365699 Apr 2020 → Jan 2021 COVID-19 NYU Langone Health Completed No outcome recorded
Phase 31 trial · 1 missed
Phase 3 NCT04083339 Sep 2019 → Dec 2023 overdue cardiomyopathy Applied Therapeutics, Inc. Active not recruiting Missed primary

News releases announcing trial results or a regulatory action · 1

DateIssuerRelease
2024-03-12 Nacuity Pharmaceuticals, Inc. Regulatory Arctic Therapeutics and Nacuity Pharmaceuticals Announce European Medicines Agency Approval to Initiate First Clinical Trial of AT-001 (NPI-001) for the Treatment of HCCAA nacuity.com ↗
EMA approved clinical trial of AT-001 (NPI-001) in Iceland

All press releases naming this drug 1 release

DateIssuerRelease

Evidence & citations 7 cited values

Every value below carries the sentence it was read from. 5 sources stand behind the page.

FieldValueCited text
Known as caficrestat “The study team hypothesizes that aldose reductase inhibition with AT-001 (caficrestat) might represent a novel therapeutic approach to reduce inflammation and risk of adverse...” NCT04365699 ↗
Known as Aldose reductase-in-1 ChEMBL registry synonym — accepted as the source's own label CHEMBL4297455 ↗
Known as AT001 “At present AT001 is the sole name for the active substance.” NCT04083339 ↗
3

NCT04365699 ↗

NCT04083339 ↗

NCT01731093 ↗

Action Inhibit “a highly selective aldose reductase inhibitor” PMID 38597864 ↗ Apr 2024
1

“Aldose reductase inhibitor” CHEMBL4297455 ↗

Modality Small molecule — CHEMBL4297455 ↗
Route Oral “Mode of Administration: Oral” NCT04365699 ↗
Target AKR1B1 “Aldose reductase inhibitor” CHEMBL4297455 ↗