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aceneuramic acid · 1 trial · 4 indications
Muscle strength based on the maximum voluntary isometric contraction (MVIC) against a dynamometer was measured bilaterally in the following upper extremity muscle groups: gross grip, shoulder abductors, elbow flexors, and elbow extensors. The UEC is derived from the sum of the average of the right and left total force values (measured in kg).
| Arm | Type | Description |
|---|---|---|
| Aceneuramic Acid Extended-Release (Ace-ER) | EXPERIMENTAL | Ace-ER 6 g/day, divided 3 times per day (TID) for 48 weeks. |
| Placebo | PLACEBO_COMPARATOR | Matching placebo TID for 48 weeks. |
| Name | Type | Description |
|---|---|---|
| aceneuramic acid extended-release (Ace-ER) | DRUG | tablets for oral use |
| Placebo | DRUG | tablets for oral use |
Inclusion Criteria: * Male or female, aged 18 to 55 years, inclusive * Willing and able to provide written, signed informed consent after the nature of the study has been explained, and before any research-related procedures are conducted * Have a documented diagnosis of GNEM, HIBM, distal myopathy...
Aceneuramic acid is an investigational small molecule being studied for the treatment of Hereditary Inclusion Body Myopathy, also known as GNE Myopathy. It is being developed by Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) for this rare disease indication.
Aceneuramic acid is a form of sialic acid, a sugar molecule that is deficient in patients with Hereditary Inclusion Body Myopathy. The drug is designed to replace this missing sialic acid, addressing the underlying metabolic defect in the condition.
Aceneuramic acid is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company traded on NASDAQ under the ticker RARE. The company focuses on developing therapies for rare and ultra-rare diseases.
Aceneuramic acid is in Phase 3 clinical development. It has completed one Phase 3 trial, but it is still considered investigational and has not been approved by regulatory authorities. The drug is not yet available for commercial use.
Aceneuramic acid has been studied in one completed Phase 3 clinical trial, registered as NCT02377921. This was a randomized, double-blind, placebo-controlled study that enrolled 89 patients with Hereditary Inclusion Body Myopathy across multiple countries, including the United States, Canada, and European nations.
Aceneuramic acid is a form of sialic acid, specifically the free acid form of N-acetylneuraminic acid. In the context of Hereditary Inclusion Body Myopathy, it is used to supplement deficient sialic acid levels in patients.