caplacizumab

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Ligands
caplacizumab

GtoPdb Ligand ID: 9123

Synonyms: ALX-0081 | anti-vWF Nanobody | Cablivi® | caplacizumab-yhdp

caplacizumab is an approved drug (EMA (2018), FDA (2019))

Comment: Caplacizumab (ALX-0081) is a nanobody targeting von Willebrand factor (vWF) [5], with anti-thrombotic action [1]. Nanobody^TM technology was commercially developed by Ablynx. Nanobodies are antibody derivatives comprising only the single variable domains (variable heavy, V_H) whilst retaining full antigen binding capacity. Their relatively small size (compared to complete antibodies) provides the potential for alternative modes of delivery and the possibility of brain penetrance.
Annotated peptide sequences for this therapeutuc are available from its IMGT/mAb-DB record. BLAST protein sequence analysis of the peptide sequence submitted with the INN document for caplacizumab reveals a 100% match with SEQ ID NO: 90 claimed in patent US7807162 [4], which is preferred humanized variant 12A2H1 in the patent document.

References
1. Bartunek J, Barbato E, Heyndrickx G, Vanderheyden M, Wijns W, Holz JB. (2013) Novel antiplatelet agents: ALX-0081, a Nanobody directed towards von Willebrand factor. J Cardiovasc Transl Res, 6 (3): 355-63. [PMID:23307200]
2. Peyvandi F, Scully M, Kremer Hovinga JA, Cataland S, Knöbl P, Wu H, Artoni A, Westwood JP, Mansouri Taleghani M, Jilma B et al.. (2016) Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med, 374 (6): 511-22. [PMID:26863353]
3. Scully M, Cataland SR, Peyvandi F, Coppo P, Knöbl P, Kremer Hovinga JA, Metjian A, de la Rubia J, Pavenski K, Callewaert F et al.. (2019) Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura. N Engl J Med, 380 (4): 335-346. [PMID:30625070]
4. Silence K. (2010) Single domain VHH antibodies against von Willebrand factor. Patent number: US7807162 B2. Assignee: Ablynx N.V.. Priority date: 20/05/2006. Publication date: 05/10/2010.
5. Ulrichts H, Silence K, Schoolmeester A, de Jaegere P, Rossenu S, Roodt J, Priem S, Lauwereys M, Casteels P, Van Bockstaele F et al.. (2011) Antithrombotic drug candidate ALX-0081 shows superior preclinical efficacy and safety compared with currently marketed antiplatelet drugs. Blood, 118 (3): 757-65. [PMID:21576702]

References

1. Bartunek J, Barbato E, Heyndrickx G, Vanderheyden M, Wijns W, Holz JB. (2013)
Novel antiplatelet agents: ALX-0081, a Nanobody directed towards von Willebrand factor.
J Cardiovasc Transl Res, 6 (3): 355-63. [PMID:23307200]

2. Peyvandi F, Scully M, Kremer Hovinga JA, Cataland S, Knöbl P, Wu H, Artoni A, Westwood JP, Mansouri Taleghani M, Jilma B et al.. (2016)
Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura.
N Engl J Med, 374 (6): 511-22. [PMID:26863353]

3. Scully M, Cataland SR, Peyvandi F, Coppo P, Knöbl P, Kremer Hovinga JA, Metjian A, de la Rubia J, Pavenski K, Callewaert F et al.. (2019)
Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura.
N Engl J Med, 380 (4): 335-346. [PMID:30625070]

4. Silence K. (2010)
Single domain VHH antibodies against von Willebrand factor.
Patent number: US7807162 B2. Assignee: Ablynx N.V.. Priority date: 20/05/2006. Publication date: 05/10/2010.

5. Ulrichts H, Silence K, Schoolmeester A, de Jaegere P, Rossenu S, Roodt J, Priem S, Lauwereys M, Casteels P, Van Bockstaele F et al.. (2011)
Antithrombotic drug candidate ALX-0081 shows superior preclinical efficacy and safety compared with currently marketed antiplatelet drugs.
Blood, 118 (3): 757-65. [PMID:21576702]