Core Insights - Esperion has nominated ESP-2001, a highly-specific allosteric ATP citrate lyase (ACLY) inhibitor, for preclinical development targeting primary sclerosing cholangitis (PSC), a disease with no approved treatments [1][2] - The company aims to file an Investigational New Drug (IND) application with the FDA to initiate first-in-human clinical studies in 2026 [1][2] Company Developments - The nomination of ESP-2001 signifies a strategic expansion of Esperion's therapeutic focus beyond cardiovascular diseases [2] - Esperion continues its partnership with Evotec to utilize their INDiGO platform, which is designed to accelerate the development of small molecule drug candidates [3] Disease Overview - PSC is a rare autoimmune liver disease characterized by chronic inflammation and scarring of the bile ducts, leading to severe complications such as cirrhosis and liver failure [4][6] - There are approximately 76,000 diagnosed PSC patients in the U.S. and Europe, with no approved treatment options available [5] Market Opportunity - ESP-2001 represents a potential market opportunity exceeding $1 billion annually, given its exclusive global development and commercialization rights held by Esperion [5] - The drug may qualify for Orphan Drug and Fast Track designations from the FDA, as well as PRIME designation from the European Medicines Agency [5]