Drug intelligence / Profile preview

PXS64

Development stage
Preclinical
Lead developer
Syntara
Modality
Other Chimeric Degraders → Targeted Protein Degraders (TPDs) → Small Molecules
01

Overview

PXS64 is a small molecule analogue of mannose-6-phosphate (M6P) developed as an anti-fibrotic agent. It acts as an inhibitor of the cation-independent mannose 6-phosphate receptor (CI-M6PR), thereby blocking the activation of latent transforming growth factor-beta 1 (TGF-β1). By inhibiting TGF-β1 signaling, PXS64 reduces the production of extracellular matrix proteins such as collagen and fibronectin and suppresses myofibroblast differentiation. Preclinical studies have demonstrated that PXS64 ameliorates fibrosis in models of idiopathic pulmonary fibrosis and kidney fibrosis by decreasing fibrotic and inflammatory markers in human lung fibroblasts, IPF patient fibroblasts, and human kidney tubular cells[1][2][3][4][8]. The drug is considered a potential candidate for further preclinical development in fibrotic diseases.

Other names
mannose-6-phosphate analoguemannose6-phosphate analoguemannose 6-phosphate analogueM6P analogueM-6P analogueM 6P analogueCI-M6PR inhibitorCI-M-6PR inhibitorCI-M 6PR inhibitor
02

Targets

IGF2R (Cation-independent mannose-6-phosphate receptor)

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