Drug intelligence / Profile preview

KFRX-04

Development stage
Preclinical
Lead developer
KeifeRx
Modality
Small Molecules
Administration
Oral
01

Overview

**KFRX-04** is a novel small molecule tyrosine kinase inhibitor (TKI) developed by KeifeRx, optimized for low-dose oral delivery with blood-brain barrier penetration. It acts as a dual inhibitor of **PIKFYVE** (phosphoinositide kinase, FYVE-type zinc finger containing) and **c-Kit** (stem cell growth factor receptor), promoting autophagy to clear toxic proteins and modulating mast cell activity to reduce inflammation. Primarily targeted for mast cell-related disorders, it shows promise in neurodegenerative conditions due to its central nervous system activity.[1][3][5][11]

02

Targets

PIKFYVE (Phosphoinositide kinase, FYVE-type zinc finger containing enzyme)KIT (c-KIT proto-oncogene receptor tyrosine kinase)

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