First-in-human phase I/II study of NEOD001 in patients with light chain amyloidosis and persistent organ dysfunction
Antibodies, Monoclonal, Humanized
Immunoglobulin Light Chains
Multiple Organ Failure
Monthly infusions of NEOD001 were safe and well tolerated. Recommended future dosing was 24 mg/kg. Organ response rates compared favorably with those reported previously for chemotherapy. A phase II expansion is ongoing. A global phase III study (NCT02312206) has been initiated. Antibody therapy targeting misfolded proteins is a potential new therapy for the management of AL amyloidosis.