Drug intelligence / Profile preview

KFRX-03

Development stage
Preclinical
Lead developer
KeifeRx
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Allosteric Modulators → Classical Binding Small Molecules → Small Molecules, Irreversible Covalent Inhibitors → Covalent Small Molecules → Small Molecules
Administration
Oral
01

Overview

KFRX-03 is a small molecule, oral tyrosine kinase inhibitor developed by KeifeRx for the treatment of neurodegenerative and immune diseases. It selectively inhibits ABL1 (ABL proto-oncogene 1), FYN (tyrosine-protein kinase FYN), and c-Kit (stem cell growth factor receptor) kinases. The drug is designed to cross the blood-brain barrier, restore autophagy, and remove toxic protein buildup in the brain, which may be beneficial in conditions such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease, mastocytosis, Lyme disease, Parkinson's disease with dementia (PDD), frontotemporal dementia, mast cell activation syndromes (MCAS), and urticaria. KFRX-03 aims to provide new therapeutic options with high central nervous system exposure while minimizing off-target side effects[1][3][5].

02

Targets

ABL1 (ABL proto-oncogene 1, non-receptor tyrosine kinase)KIT (c-KIT proto-oncogene receptor tyrosine kinase)FYN (Proto-oncogene tyrosine-protein kinase Fyn)

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