CNP-104

CNP-104 is an investigational, biodegradable nanoparticle-based therapy developed for the treatment of primary biliary cholangitis (PBC), a rare autoimmune liver disease. The drug encapsulates the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC-E2), which is a key autoantigen in PBC. Its mechanism of action involves inducing antigen-specific immune tolerance to pathogenic activated PDC-E2 T-cells that drive inflammation in bile ducts, thereby addressing the root cause of PBC and potentially modifying disease progression. In clinical studies, CNP-104 has demonstrated favorable immunological responses—such as reduction in Th17 T cells—and stabilization or improvement in liver health outcomes, including statistically significant reductions in liver stiffness. The drug has received Orphan Drug Designation and Fast Track Designation from the FDA.