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: *
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:
Recent & Upcoming Milestones
Highest Development Phases
Phase 3: GNE Myopathy|Distal Myopathies
Phase 2: Thrombocytopenia|GNE Myopathy|Distal Myopathies
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
ACTRN12618000188235p |
SCI/D | N/A |
Not yet recruiting |
Unknown |
2019-01-31 |
|||
NCT02731690 |
UX001-CL203 | P2 |
Terminated |
Distal Myopathies|GNE Myopathy |
2018-01-10 |
2019-03-20 |
Treatments |
|
2015-004553-41 |
2015-004553-41 | P2 |
Terminated |
GNE Myopathy |
2018-01-10 |
2022-03-13 |
Treatments |
|
NCT02845609 |
SA-thrombo | P2 |
Completed |
Thrombocytopenia |
2017-09-01 |
2019-03-20 |
Treatments |
|
NCT04671472 |
NPC-09-1 | P3 |
Completed |
Distal Myopathies|GNE Myopathy |
2022-03-29 |
13% |
2022-05-17 |
Patient Enrollment|Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments|Trial Status |
NCT02736188 |
UX001-CL302 | P3 |
Terminated |
GNE Myopathy|Distal Myopathies |
2018-01-10 |
2019-03-20 |
||
2016-000360-42 |
2016-000360-42 | P3 |
Terminated |
GNE Myopathy |
2018-01-10 |
2025-07-09 |
Treatments |
|
NCT02377921 |
GNEM | P3 |
Completed |
Distal Myopathies|GNE Myopathy |
2017-06-09 |
2019-03-20 |
Treatments |
|
2014-005432-33 |
2014-005432-33 | P3 |
Completed |
GNE Myopathy |
2017-06-09 |
2025-07-04 |
Treatments |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
10/01/2023 |
PubMed |
[Efficacy of Aceneuramic Acid for Distal Myopathy with Rimmed Vacuoles]. |
|
08/11/2023 |
PubMed |
Efficacy confirmation study of aceneuramic acid administration for GNE myopathy in Japan. |
|
01/01/2023 |
PubMed |
Phase II/III Study of Aceneuramic Acid Administration for GNE Myopathy in Japan. |