Drug intelligence / Profile preview

AT-20494

Development stage
Preclinical
Lead developer
Janssen Pharmaceutical
Modality
Small Molecules
Administration
Oral
01

Overview

**AT-20494** is an orally bioavailable small molecule and a novel rapamycin analog (rapalog) that selectively inhibits mTORC1, the mechanistic target of rapamycin complex 1, a master regulator of cellular metabolism overactive in chronic diseases. Discovered by Navitor Pharmaceuticals scientists, it represents the first fully selective mTORC1 inhibitor advancing to human studies. Preclinical data demonstrate efficacy in reducing cyst burden, kidney volume, fibrosis, and inflammation in PKD1-mutant mouse models of autosomal dominant polycystic kidney disease (ADPKD), with potential broader applications in aging-related conditions driven by mTORC1 dysregulation.[1][3][5]

Other names
NV-20494NV20494NV 20494AT 20494AT20494AT-20494
02

Targets

Mechanistic target of rapamycin complex 1

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