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Structure-Activity-Relationship Exploration and Animal Validation of Novel Assembly Modulator Small Molecule Chemical Series with Pan-Cancer Selective Cytotoxicity

Lingappa, A. F.; Mallesh, S.; Michon, M.; Shivarudraswamy, D.; Lin, J.; Prasad, M. D.; Dey, D.; Solas, D.; Lingappa, V. R.

2025-03-04 biochemistry
10.1101/2025.02.28.640839 bioRxiv
Show abstract

We recently described a novel class of chemical compounds initially identified as modulators of host-catalyzed HIV capsid assembly, that have been advanced to display broad anti-tumor cytotoxicity. Here we explore in more detail the structure-activity-relationship (SAR) of the chemical series. PAV-0805, an advanced analog, was found to be nontoxic at 10mg/kg in mice and nontoxic to healthy human peripheral blood mononuclear cells (PBMCs). PAV-0805 has broad anti-cancer activity in the NCI-60 cancer cell line screen, exhibiting cytotoxicity to a diversity of brain, breast, colon, lung, ovarian prostate, renal, and skin and blood cancers. In the aggressive 4T1 mouse allograft breast cancer model, PAV-0805 reduced tumor growth and metastasis when treatment was initiated early (primary tumor volume of 50mm3) or late (primary tumor volume of 500mm3). In both cases, treatment with PAV-0805 inhibited both tumor growth and metastasis to a degree comparable to early treatment with paclitaxel. PAV-0805 targets Protein Disulfide Isomerase in a dynamic multi-protein complex comprised of gene products involved in the cancer hallmarks of resisting cell death and reprogramming energy metabolism.

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