First-In-Human Effects of PPT1 Inhibition Using the Oral Treatment with GNS561/Ezurpimtrostat in Patients with Primary and Secondary Liver Cancers

First-In-Human Effects of PPT1 Inhibition Using the Oral Treatment with GNS561/Ezurpimtrostat in Patients with Primary and Secondary Liver Cancers

Introduction: GNS561/Ezurpimtrostat is a first-in-class, orally bioavailable, small molecule that blocks cancer cell proliferation by inhibiting late-stage autophagy and dose-dependent build-up of enlarged lysosomes by interacting with the palmitoyl-protein thioesterase 1 (PPT1). Methods: This phase...

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Main Authors: Abou-Alfa, G.K (Author), Ansaldi, C. (Author), Awada, A. (Author), Bestion, E.N (Author), Decaens, T. (Author), Dreyer, C. (Author), Halfon, P. (Author), Harding, J.J (Author), Kotecki, N. (Author), Menut, A. (Author), Merle, P. (Author), Mezouar, S. (Author), Paradis, V. (Author), Rachid, M. (Author), Raymond, E. (Author), Roth, G. (Author)
Format: Article
Language:English
Published: S. Karger AG 2022
Subjects:
Autophagy
Cholangiocarcinoma
GNS561/ ezurpimtrostat
Hepatocellular carcinoma
Lysosome
Palmitoyl-protein thioesterase 1
Phase 1 trial
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