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CP-235 (also known as CPT-2008 or CPT-235) is a first-in-class, orally bioavailable small molecule inhibitor of mitochondrial reverse electron transport (RET) being developed by Cerepeut. The compound was originally discovered through collaborative research at Stanford University and the University of California, San Francisco (UCSF). CP-235 specifically targets mitochondrial complex I by binding to its NDUFS3 and NDUFV1 subunits. This binding prevents the pathological interaction of phosphorylated tau (pTau) with complex I, thereby breaking a self-perpetuating feedback loop where pTau drives RET, and RET-induced oxidative stress promotes further tau hyperphosphorylation. By blocking this cycle, CP-235 reduces reactive oxygen species (ROS) production, restores the NAD+/NADH redox balance, and mitigates neuroinflammation and neurodegeneration. Cerepeut is advancing CP-235 through preclinical and IND-enabling studies for the treatment of tauopathies and age-related neurodegenerative diseases, with progressive supranuclear palsy (PSP) planned as the lead clinical indication, followed by Alzheimer's disease, Parkinson's disease, and frontotemporal dementia.
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