pabinafusp alfa   Click here for help

GtoPdb Ligand ID: 11772

Synonyms: IZCARGO® | JR-141 | JR141
Approved drug
pabinafusp alfa is an approved drug (Japan (2021))
Compound class: Antibody
Comment: Pabinafusp (JR-141) is a fusion protein of the Fab domains of an anti-transferrin receptor monoclonal and the enzyme iduronate 2-sulfatase (IDS; P22304) [5], an enzyme that is deficient in patients with the lysosomal storage disease mucopolysaccharidosis type II (MPS II; Hunter syndrome) [1-2]. It is an intravenously administered enzyme replacement therapy. The anti-transferrin receptor component facilitates receptor-mediated movement of the fused enzyme across the blood-brain barrier [4].
1. Bonuccelli G, Di Natale P, Corsolini F, Villani G, Regis S, Filocamo M. (2001)
The effect of four mutations on the expression of iduronate-2-sulfatase in mucopolysaccharidosis type II.
Biochim Biophys Acta, 1537 (3): 233-8. [PMID:11731225]
2. Demydchuk M, Hill CH, Zhou A, Bunkóczi G, Stein PE, Marchesan D, Deane JE, Read RJ. (2017)
Insights into Hunter syndrome from the structure of iduronate-2-sulfatase.
Nat Commun, 8: 15786. [PMID:28593992]
3. Okuyama T, Eto Y, Sakai N, Nakamura K, Yamamoto T, Yamaoka M, Ikeda T, So S, Tanizawa K, Sonoda H et al.. (2021)
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II.
Mol Ther, 29 (2): 671-679. [PMID:33038326]
4. Sonoda H, Morimoto H, Koshimura Y, Kinoshita M, Takagi H, Yui Y. (2015)
Novel anti-transferrin receptor antibody that passes through blood-brain barrier.
Patent number: WO2015098989A1. Assignee: JCR Pharmaceuticals. Priority date: 25/12/2013. Publication date: 02/07/2015.
5. Sonoda H, Morimoto H, Yoden E, Koshimura Y, Kinoshita M, Golovina G, Takagi H, Yamamoto R, Minami K, Mizoguchi A et al.. (2018)
A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II.
Mol Ther, 26 (5): 1366-1374. [PMID:29606503]