AKTX-101

AKTX-101 is a next-generation antibody-drug conjugate (ADC) developed for cancer therapy. It targets the TROP2 receptor (tumor-associated calcium signal transducer 2) on cancer cells and delivers a proprietary novel payload called PH1 via a tumor-selective linker. Unlike traditional ADCs that use tubulin inhibitors or DNA-damaging agents as cytotoxic payloads, the PH1 payload in AKTX-101 is a spliceosome inhibitor designed to disrupt RNA splicing within cancer cells. This disruption leads to tumor-specific cell death through mechanisms such as unfolded protein response and endoplasmic reticulum stress, while also generating immunostimulatory effects by accumulating mis-spliced proteins that act as neoantigens to stimulate anti-tumor immunity. The design aims to minimize off-target toxicity and overcome resistance mechanisms common with other ADCs by being less susceptible to MDR transporter-mediated drug resistance. Preclinical studies have shown superior activity, prolonged survival, reduced resistance, better tolerability and safety compared to current TROP2-targeting ADCs; it also demonstrates potential synergy with checkpoint inhibitors[1][5][6][7][9][10].

Brand names

Torpedo

Other names

Anti TROP2 antibody drug conjugateAnti TROP2 ADC - Akari Therapeutics