Drug intelligence / Profile preview

KFRX01

Development stage
Unknown
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

KFRX01 is an oral, low-dose, bioequivalent reformulation of the BCR-ABL tyrosine kinase inhibitor nilotinib, developed by KeifeRx to treat neurodegenerative diseases such as early Alzheimer’s disease and Parkinson’s disease by repurposing its kinase inhibition profile for central nervous system activity.[1][3][16] At substantially lower daily doses than the oncology regimen, KFRX01 is formulated to enhance brain penetration and induce autophagy, promoting intracellular clearance of toxic proteins (including amyloid and tau), while also modulating microglial-mediated neuroinflammation and mast cell–driven immune pathways to improve safety and tolerability relative to standard nilotinib.[1][3][16] The program is Phase 2/3–ready and is positioned as a potentially complementary approach to existing amyloid-targeting therapies through intracellular amyloid reduction and inflammation control in neurodegenerative disorders.[1]

Other names
nilotinib bioequivalent
02

Targets

KIT (c-KIT proto-oncogene receptor tyrosine kinase)ABL1 (ABL proto-oncogene 1, non-receptor tyrosine kinase)

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