Product Description
PXL770 is a novel drug candidate that directly activates adenosine monophosphate-activated protein kinase (AMPK); Based on its central metabolic role, we believe that targeting AMPK offers the opportunity to pursue a wide range of indications to treat chronic metabolic diseases, including diseases that affect the liver, such as NASH and rare metabolic disorders (e.g. adrenoleukodystrophy). (Sourced from: https://www.poxelpharma.com/en_us/pipeline/nash)
Mechanisms of Action: AMPK Activator
Novel Mechanism: Yes
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Fast Track - AdrenoleukodystrophyOrphan Drug - AdrenoleukodystrophyOrphan Drug - Kidney Diseases|Polycystic Kidney, Autosomal Dominant *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Poxel
Company Location:
Company CEO:
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Recent & Upcoming Milestones
Highest Development Phases
Phase 2: Adrenoleukodystrophy|Adreno-myeloneuropathy|Non-alcoholic Fatty Liver Disease|Fatty Liver, Alcoholic
Phase 1: Type 2 Diabetes|Hepatitis, Alcoholic|Fatty Liver, Alcoholic|Non-alcoholic Steatohepatitis|Non-alcoholic Fatty Liver Disease|Healthy Volunteers
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT05441904 |
PXL770-009 | P1 |
Completed |
Hepatitis, Alcoholic|Non-alcoholic Steatohepatitis|Fatty Liver, Alcoholic |
2021-03-29 |
2022-07-02 |
Primary Endpoints|Treatments |
|
NCT03950882 |
PXL770-003 | P1 |
Completed |
Non-alcoholic Fatty Liver Disease|Fatty Liver, Alcoholic |
2020-03-29 |
2% |
2021-06-12 |
|
NCT03886103 |
PXL770-005 | P1 |
Completed |
Healthy Volunteers |
2019-03-15 |
|||
NCT03395470 |
PXL770-002 | P1 |
Completed |
Type 2 Diabetes |
2018-03-16 |
55% |
2019-03-22 |
Treatments |
NCT05146284 |
X-ALD or ALD | P2 |
Withdrawn |
Adrenoleukodystrophy|Adreno-myeloneuropathy |
2024-09-01 |
76% |
2024-12-17 |
Patient Enrollment|Primary Endpoints|Treatments|Trial Status |
2021-006223-18 |
START770 | P2 |
Terminated |
Adreno-myeloneuropathy |
2022-07-29 |
2025-05-06 |
Treatments|Trial Status |
|
NCT03763877 |
PXL770-004 | P2 |
Completed |
Fatty Liver, Alcoholic|Non-alcoholic Fatty Liver Disease |
2020-08-03 |
19% |
2021-10-29 |
Primary Endpoints |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
12/10/2025 |
News Article |
Poxel Publishes Its Financial Results for the First Half of 2025 |
|
11/13/2025 |
News Article |
Poxel Reports Revenue for the Third Quarter 2025 |
|
10/31/2025 |
News Article |
Appointment of Yves Decadt as Member of Poxel's Board of Directors |
|
10/16/2025 |
News Article |
Poxel Reports Financial Results for Full Year 2024 and Provides Operational and Financial Update |
|
05/01/2023 |
PubMed |
A novel direct adenosine monophosphate kinase activator ameliorates disease progression in preclinical models of Autosomal Dominant Polycystic Kidney Disease. |
|
08/01/2022 |
PubMed |
Beneficial Effects of the Direct AMP-Kinase Activator PXL770 in In Vitro and In Vivo Models of X-Linked Adrenoleukodystrophy. |
|
07/01/2022 |
PubMed |
Defective AMPK regulation of cholesterol metabolism accelerates atherosclerosis by promoting HSPC mobilization and myelopoiesis. |