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PC-002L is a next-generation small molecule formulation of PC-002 (sepantronium bromide) being developed by Cothera Bioscience. It functions as an inhibitor of the deubiquitinase (DUB) enzyme USP7 (Ubiquitin-specific protease 7). By inhibiting USP7, the drug prevents the removal of ubiquitin chains from oncogenic proteins, thereby promoting their proteasomal degradation. This mechanism is particularly relevant in cancers driven by MYC family proteins, such as Small Cell Lung Cancer (SCLC) with N-MYC amplification, where the stabilization of these proteins is critical for tumor cell survival. PC-002L is designed to improve upon the pharmacological and therapeutic properties of the original PC-002 formulation and is currently in preclinical development.
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