Product Description
Aceneuramic acid is an N-acetylneuraminic acid that is keto-neuraminic acid in which one of the hydrogens of the amino group is replaced by an acetyl group. It is a member of N-acetylneuraminic acids, a member of acetamides and a 2-oxo monocarboxylic acid. It derives from a keto-neuraminic acid. It is a conjugate acid of an aceneuramate.
Mechanisms of Action: No Mechanism
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: Saudi Arabia
Approved Indications: None
Known Adverse Events: None
Company: Ultragenyx
Company Location: NOVATO CA 94949
Company CEO: Emil D. Kakkis
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Highest Development Phases
Phase 3: Distal Myopathies|GNE Myopathy
Phase 2: Distal Myopathies|GNE Myopathy|Thrombocytopenia
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| 2014-005432-33 | P3 |
Completed |
GNE Myopathy |
2017-06-09 |
|
| 2015-004553-41 | P2 |
Terminated |
GNE Myopathy |
2018-01-10 |
|
| 2016-000360-42 | P3 |
Terminated |
GNE Myopathy |
2018-01-10 |
|
| SCI/D | N/A |
Not yet recruiting |
Unknown |
2019-01-31 |
|
| GNEM | P3 |
Completed |
Distal Myopathies|GNE Myopathy |
2017-06-09 |
|
| UX001-CL203 | P2 |
Terminated |
GNE Myopathy|Distal Myopathies |
2018-01-10 |
|
| UX001-CL302 | P3 |
Terminated |
GNE Myopathy|Distal Myopathies |
2018-01-10 |
|
| SA-thrombo | P2 |
Completed |
Thrombocytopenia |
2017-09-01 |
|
| NPC-09-1 | P3 |
Completed |
Distal Myopathies|GNE Myopathy |
2022-03-29 |
13% |