Drug intelligence / Profile preview

NEOD001 + bortezomib

Development stage
Discontinued
Lead developer
Prothena
Modality
Monoclonal Antibodies → Antibody-Based Therapeutics, Small Molecules
Administration
Intravenous, Subcutaneous
01

Overview

**NEOD001 + bortezomib** is an investigational combination therapy for amyloid light-chain (AL) amyloidosis. NEOD001 (also known as birtamimab) is a humanized IgG1 monoclonal antibody targeting a conserved epitope in misfolded κ and λ light chains, designed to neutralize toxic soluble light chain aggregates and promote macrophage-induced phagocytosis of organ-deposited insoluble amyloid. Bortezomib is a small molecule proteasome inhibitor that disrupts plasma cell function and reduces amyloidogenic free light chains. The combination seeks to both suppress production of amyloidogenic light chains (via bortezomib) and directly clear existing amyloid deposits (via NEOD001)[1][4]. NEOD001 was developed specifically for AL amyloidosis; bortezomib is widely used in myeloma and amyloidosis and is a standard backbone in current treatment regimens.

Other names
birtamimab + bortezomib
02

Targets

PSMB9 (Immunoproteasome subunit beta type-1i)AL (Amyloid light-chain protein)PSMB1 (26S Proteasome (β1-Subunit))PSMB5 (Proteasome subunit beta Type-5)

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