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PT-201

Development stage: Preclinical
Lead developer: p53-Therapeutics
Modality: Small Molecules
Administration: Oral

Overview

PT-201 is a preclinical-stage small molecule therapeutic belonging to the xanthine class, being developed by p53-Therapeutics. It is designed to treat cancers harboring mutations in the TP53 tumor suppressor gene. PT-201 functions by bypassing defective p53 signaling to restore a p53-like transcriptome. It acts through a non-canonical pathway involving the activation of the integrated stress response transcription factors ATF3 and ATF4, which subsequently upregulate pro-apoptotic target genes such as Noxa, thereby inducing tumor cell death and targeting an S-phase cell cycle checkpoint.

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