Telomir Pharmaceuticals Inc. (NASDAQ:TELO) has announced promising preclinical data for its lead small molecule candidate, Telomir-1, in the treatment of Wilson's disease. The study demonstrated that Telomir-1 significantly improved neurological, behavioral, liver, and kidney functions in a genetically relevant animal model of the disease. Results showed a dose-dependent reversal of neurological dysfunction, organ damage, copper accumulation, and mortality. Additionally, Telomir-1 normalized critical liver health markers, indicating potential protection of liver function. These findings justify further development towards human clinical trials, which are planned for the first half of 2026. The company aims to submit an Investigational New Drug $(IND.AU)$ application by the end of the year, advancing Telomir-1 as a potential disease-modifying treatment for rare disorders involving copper dysregulation. Results of the preclinical study were presented on June 11, 2025.
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