Drug intelligence / Profile preview

dorsomorphin

Development stage
Preclinical
Lead developer
Massachusetts General Hospital
Modality
Small Molecules
Administration
Intraperitoneal, Intravenous
01

Overview

Dorsomorphin is a potent, reversible, and ATP-competitive small molecule inhibitor. It was initially identified as an inhibitor of AMP-activated protein kinase (AMPK), but it is also widely recognized for its potent inhibition of Bone Morphogenetic Protein (BMP) type I receptors, including ALK2 (ACVR1), ALK3 (BMPR1A), and ALK6 (BMPR1B). By blocking BMP signaling, dorsomorphin inhibits the phosphorylation of Smad1/5/8. It has been used extensively in research to study metabolic regulation, vascular biology, and embryonic development. While it has shown potential in preclinical models for treating conditions like anemia of chronic disease by suppressing hepcidin expression, it is primarily utilized as a pharmacological tool in vitro and in vivo rather than as a clinical therapeutic.

Other names
6-[4-(2-Piperidin-1-ylethoxy)phenyl]-3-pyridin-4-ylpyrazolo[1,5-a]pyrimidine
02

Targets

AMPK (Adenosine monophosphate–activated protein kinase)VEGFR2 (Vascular endothelial growth factor receptor 2)TAK1 (Transforming growth factor beta–activated kinase 1)AKT (RAC-alpha serine/threonine-protein kinase)MAPK3 (Mitogen-activated protein kinase 1)RK (Ribokinase)BMPR1BBMPR1AACVR1 (Activin receptor type I)

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