Drug intelligence / Profile preview

DMUC4064A

Development stage
Phase 1
Lead developer
Genentech
Modality
Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics, Small Molecules, Monoclonal Antibodies → Antibody-Based Therapeutics
Administration
Intravenous
01

Overview

DMUC4064A is an antibody-drug conjugate (ADC) composed of a humanized IgG1 monoclonal antibody targeting MUC16 (also known as cancer antigen 125 or CA125), conjugated to the cytotoxic agent monomethyl auristatin E (MMAE) via a protease-cleavable linker. The drug uses THIOMAB™ technology for site-directed conjugation, resulting in a consistent drug-to-antibody ratio and improved homogeneity. Its mechanism of action involves binding to MUC16-expressing tumor cells, internalization of the ADC, and intracellular release of MMAE, which disrupts microtubules leading to cell cycle arrest and apoptosis. DMUC4064A was developed primarily for platinum-resistant ovarian cancer and unresectable pancreatic cancer. In phase I clinical trials, it demonstrated manageable safety with common adverse events including fatigue, nausea, abdominal pain, blurred vision, diarrhea, anemia, and ocular toxicities such as dry eye and keratitis. Despite promising early results in platinum-resistant ovarian cancer patients with high MUC16 expression (ORR 46%), further development appears discontinued[1][2][3][5][6].

Other names
anti-MUC16/MMAE antibody-drug conjugateMMUC3333AMMUC-3333AMMUC 3333A
02

Targets

TUBB (Tubulin (alpha and beta subunits))MUC16 (Mucin-16)

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