Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DXB8M6
|
|||
| Drug Name |
BBP-671
|
|||
| Drug Type |
Small molecule
|
|||
| Indication | Methylmalonic acidemia [ICD-11: 5C50.E0] | Phase 1 | [1] | |
| Propionic acidemia [ICD-11: 5C50.E0] | Phase 1 | [1] | ||
| Company |
CoA Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Pantothenate kinase 3 (PANK3) | Target Info | Activator | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04836494) A First-in-human, Randomized, Placebo-controlled, Single and Multiple Ascending Dose Escalation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BBP-671 in Healthy Subjects and In Patients With Propionic Acidemia or Methylmalonic Acidemia. U.S.National Institutes of Health. | |||
| REF 2 | Relief of CoA sequestration and restoration of mitochondrial function in a mouse model of propionic acidemia. J Inherit Metab Dis. 2023 Jan;46(1):28-42. | |||
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