Product Description
AT-007 is an investigational, novel Aldose Reductase Inhibitor (ARI) being developed for the treatment of several rare diseases.
Mechanisms of Action: AKR Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Fast Track - GalactosemiasOrphan Drug - Congenital Disorders of GlycosylationOrphan Drug - GalactosemiasOrphan Drug - SORD DeficiencyPriority Review - *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Applied Therapeutics
Company Location: NEW YORK NY 10017
Company CEO: Shoshana Shendelman
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Czech Republic, France, Germany, Italy, Spain, United Kingdom, United States, Unknown Location
Active Clinical Trial Count: 5
Recent & Upcoming Milestones
- Clinical Outcomes Reported - Applied Therapeutics presented P3 Charcot-Marie-Tooth Disease|Deficiency Diseases|SORD Deficiency results on 2025-05-18 for Govorestat
- PDUFA date set for Govorestat to treat classic galactosemia; EMA decision expected Q1 2025, accelerated approval for SORD deficiency.
- FDA accepted NDA for Govorestat for Galactosemia with pdufa target action date of August 28, 2024, granting priority review.
Highest Development Phases
Phase 3: Charcot-Marie-Tooth Disease|Galactosemias|Other|SORD Deficiency
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT07191912 |
CMT-SORD | P3 |
Not yet recruiting |
SORD Deficiency|Charcot-Marie-Tooth Disease |
2028-10-01 |
26% |
2025-09-26 |
|
2023-001104-33 |
2023-001104-33 | P3 |
Active, not recruiting |
Other |
2025-04-12 |
2025-07-09 |
Primary Completion Date|Start Date|Study Completion Date|Treatments|Trial Status |
|
NCT04902781 |
AT-007-1002 | P3 |
Completed |
Galactosemias |
2023-03-03 |
8% |
2025-10-30 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments|Trial Status |
2024-518250-16-00 |
AT-007-1005 | P3 |
Not yet recruiting |
SORD Deficiency |
2026-11-30 |
2025-05-02 |
Treatments |
|
NCT05397665 |
INSPIRE | P3 |
Active, not recruiting |
SORD Deficiency |
2024-10-12 |
17% |
2025-09-16 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
12/11/2025 |
News Article |
Cycle Pharmaceuticals to Acquire Applied Therapeutics |
|
11/20/2025 |
News Article |
Applied Therapeutics Announces Corporate Updates |
|
07/02/2025 |
News Article |
Inventus Signs Milling Agreement With McEwen Mining |
|
06/17/2025 |
News Article |
Inventus Reports Additional High-Grade Confirmation Drilling and Commences Bulk Sample Extraction at the Pardo Gold Project |
|
09/04/2023 |
PubMed |
Effects of Thermal-Strain-Induced Atomic Intermixing on the Interfacial and Photoluminescence Properties of InGaAs/AlGaAs Multiple Quantum Wells. |
|
05/22/2023 |
PubMed |
Sorbitol reduction via govorestat ameliorates synaptic dysfunction and neurodegeneration in sorbitol dehydrogenase deficiency. |
|
03/15/2023 |
PubMed |
Design and Outcomes of a Novel Multidisciplinary Ophthalmic Genetics Clinic. |