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PBX-001

Development stage
Preclinical
Lead developer
PinotBio
Modality
DNA Intercalators/Alkylators → Nucleic Acid-Directed Small Molecules → Small Molecules, Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics, Monoclonal Antibodies → Antibody-Based Therapeutics
Administration
Intravenous
01

Overview

PBX-001 is an antibody-drug conjugate (ADC) developed by Pinotbio that targets the Trop-2 antigen, which is overexpressed in various cancers. The drug consists of a monoclonal antibody directed against Trop-2, linked via a pH-sensitive cleavable linker to a novel camptothecin payload (FL-118). Upon binding to Trop-2 on cancer cells, PBX-001 is internalized and releases its cytotoxic payload either within the acidic tumor microenvironment or after lysosomal degradation inside the cell. This dual-release mechanism enhances tumor-specific killing and leverages the bystander effect for improved efficacy. The camptothecin-based payload inhibits topoisomerase I and resists efflux by drug-resistance pumps such as ABCG2, thereby overcoming common mechanisms of resistance in cancer therapy. Preclinical studies indicate activity against lung cancer, bladder cancer, and triple-negative breast cancer[1][2][3][4].

Other names
PINOT-ADC PBX-001
02

Targets

TOP1 (DNA Topoisomerase I)TACSTD2 (Tumor-associated calcium signal transducer 2)

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