Details of the Drug

General Information of Drug (ID: DMR9XMZ)

Drug Name
BBP-671
Indication
Disease Entry ICD 11 Status REF
Methylmalonic acidemia 5C50.E0 Phase 1 [1]
Propionic acidemia 5C50.E0 Phase 1 [1]
Drug Type
Small molecule
Cross-matching ID
TTD ID
DXB8M6

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Pantothenate kinase 3 (PANK3) TTFBUWA PANK3_HUMAN Activator [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

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.
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.