Drug intelligence / Profile preview

KTI-2338

Development stage
Preclinical
Lead developer
Keros Therapeutics
Modality
Small Molecules
01

Overview

KTI-2338 is an investigational **small molecule** selective inhibitor of activin receptor-like kinase 2, also known as activin A receptor type 1. It has been described in preclinical work from Keros Therapeutics as a potent, highly selective ALK2 kinase inhibitor with single-digit nanomolar activity, designed to suppress pathological ALK2 mediated SMAD1/5/9 signaling. The program has been studied preclinically in disorders linked to dysregulated ALK2 biology, including **fibrodysplasia ossificans progressiva**, where it reduced osteogenic signaling markers and aberrant Activin A driven responses in human FOP cell models, and iron-restricted anemia settings such as **iron-refractory iron deficiency anemia** and **anemia of inflammation or chronic kidney disease**, where ALK2 inhibition lowered hepcidin and improved iron parameters in animal models. No approved brand name or clinical marketing status was identified.

02

Targets

ACVR1 (Activin receptor type I)

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