Glycoprotein Non-Metastatic Melanoma Protein B (GPNMB): A Translational Pharmacodynamic Biomarker for PIKfyve Inhibition With VRG50635.

Geraldine Gontier¹, Grace Kim¹, Chao Wang¹, Kevin Zhu¹, Raymond Gau¹, Ningzhe Zhang¹, Swati Naphade¹, Alistair Stewart¹, Martin J Schmidt¹, Robert Galemmo¹, Brian Shook¹, Anil Tarachandani¹, Irene Choi¹, Shane Raines², Robert H Scannevin¹, Henrieke W Grievink³, Lars M G Smits^3,4, Philip H C Kremer^3,4, Diego Cadavid¹

¹Verge Genomics, South San Francisco, California, USA.

²2b Analytics, Wallingford, Pennsylvania, USA.

³Centre for Human Drug Research, Leiden, the Netherlands.

⁴Leiden University Medical Center, LUMC, Leiden, the Netherlands.

PubMed PMC DOI PDF

Abstract

Glycoprotein non-metastatic melanoma protein B (GPNMB) was investigated as a pharmacodynamic (PD) biomarker for PIKfyve inhibition across translational studies ex vivo, in vitro, in animals, and in the clinic, demonstrating significant response to VRG50468 in cells, in vivo in the central nervous system (CNS) and in peripheral fluids and tissues. VRG50468 is the active metabolite of VRG50635, a sm…

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