Discovery of SARS-CoV-2 papain-like protease (PL<sup>pro</sup>) inhibitors with efficacy in a murine infection model.

Garnsey, Michelle R; Robinson, Matthew C; Nguyen, Luong T; Cardin, Rhonda; Tillotson, Joseph; Mashalidis, Ellene; Yu, Aijia; Aschenbrenner, Lisa et al. · Sci Adv · 2024

basic_science · Level V

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Abstract

Vaccines and first-generation antiviral therapeutics have provided important protection against COVID-19 caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). However, there remains a need for additional therapeutic options that provide enhanced efficacy and protection against potential viral resistance. The SARS-CoV-2 papain-like protease (PL<sup>pro</sup>) is one of the two essential cysteine proteases involved in viral replication. While inhibitors of the SARS-CoV-2 main protease have demonstrated clinical efficacy, known PL<sup>pro</sup> inhibitors have, to date, lacked the inhibitory potency and requisite pharmacokinetics to demonstrate that targeting PL<sup>pro</sup> translates to in vivo efficacy in a preclinical setting. Here, we report the machine learning-driven discovery of potent, selective, and orally available SARS-CoV-2 PL<sup>pro</sup> inhibitors, with lead compound PF-07957472 (<b>4</b>) providing robust efficacy in a mouse-adapted model of COVID-19 infection.

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