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CPT-004 is a lead compound in the "capton" class of therapeutics developed by Enabled Therapeutics. Captons are structurally modified small-molecule drugs designed to mitigate excitotoxicity, inflammation, and other mechanisms of central nervous system (CNS) pathology. CPT-004 is administered orally in an inactive form that crosses the blood-brain barrier more efficiently due to reduced charge and polar surface area. Once in the brain, it becomes irreversibly activated only under neuropathological conditions—specifically where reactive oxygen species (ROS) are elevated due to tissue damage or dysfunction. This activation is strictly confined to distressed brain tissue, ensuring targeted therapeutic action while minimizing effects on healthy tissue. Preclinical studies have demonstrated drug-like pharmacokinetics, widespread distribution throughout the brain at therapeutic levels, and nearly quantitative activation in models of CNS injury such as seizures[1].
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