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
Company: Applied Therapeutics, Inc.
Company Location:
Company Founding Year: None
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Czech Republic, Italy, United Kingdom, United States
Active Clinical Trial Count: 3
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
- FDA review of Govorestat for classic galactosemia progressing smoothly, with PDUFA target action date set for Nov 28, 2024.
- Govorestat NDA accepted for classic galactosemia, granted priority review by FDA with PDUFA target action date of August 28, 2024.
Highest Development Phases
Phase 3: Other|SORD Deficiency
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
2024-518250-16-00 |
AT-007-1005 | P3 |
Not yet recruiting |
SORD Deficiency |
2026-11-30 |
2025-05-02 |
Treatments |
|
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 |
|
NCT05397665 |
INSPIRE | P3 |
Completed |
SORD Deficiency |
2024-10-12 |
9% |
2026-06-10 |
Primary Endpoints |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
02/03/2026 |
News Article |
Cycle Pharmaceuticals Completes Acquisition of Applied Therapeutics |
|
01/29/2026 |
News Article |
Applied Therapeutics Shares Letter to Stockholders Recommending Tender of Shares Following Offer Expiration Extension |
|
12/11/2025 |
News Article |
Cycle Pharmaceuticals to Acquire Applied Therapeutics |
|
11/20/2025 |
News Article |
Applied Therapeutics Announces Corporate Updates |