Mirum Pharmaceuticals ((MIRM)) announced an update on their ongoing clinical study.
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Study Overview: The LEAP-US study, officially titled Long-Term SafEty and Clinical Outcomes of LivmArli in Patients in the United States, aims to evaluate the long-term safety and clinical outcomes of Livmarli in patients with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC). This 5-year observational cohort study is significant as it seeks to provide crucial insights into the prolonged effects of Livmarli, a drug designed to manage these rare liver conditions.
Intervention/Treatment: The study focuses on the drug Livmarli, administered to patients diagnosed with ALGS or PFIC. Livmarli is intended to improve liver function and quality of life in these patients, with dosages of 380 mcg/kg once daily or 570 mcg/kg twice daily.
Study Design: This is a prospective, observational cohort study, meaning it will follow a group of patients over time without manipulating the treatment they receive. The primary purpose is to observe the long-term effects of Livmarli in real-world settings.
Study Timeline: The study began on December 11, 2023, with an estimated primary completion date yet to be announced. The latest update was submitted on May 21, 2025, indicating ongoing recruitment and data collection efforts.
Market Implications: The continuation of this study could influence Mirum Pharmaceuticals’ stock performance positively, as successful long-term outcomes may enhance investor confidence and market positioning. Competitors in the rare liver disease treatment space will likely monitor these developments closely, as positive results could strengthen Mirum’s market share.
The LEAP-US study is currently ongoing, with further details available on the ClinicalTrials portal.