Mirum Pharmaceuticals, Inc. presented analyses from its clinical studies evaluating maralixibat in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis type 2 (PFIC2), two rare liver diseases affecting children. An analysis from the Phase 2 INDIGO open-label study evaluating long-term clinical efficacy and transplant-free survival with maralixibat in patients (n=19) with bile salt export pump (BSEP) deficiency, or progressive familial intrahepatic cholestasis type 2 (PFIC2) was presented during the congress. These data demonstrated that patients with long-term maralixibat treatment who achieved serum bile acid (sBA) response had five-year transplant-free survival. In addition, those patients who achieved sBA response also had significant improvements in pruritus, liver parameters, quality of life and growth. The study showed that maralixibat was generally well-tolerated and the most common adverse events were mild to moderate diarrhea and abdominal pain, which were transient in nature; no gastrointestinal events led to discontinuation of maralixibat. Data from more than five years of evaluation across three maralixibat Phase 2 clinical trials and their extension studies were analyzed to assess treatment-emergent adverse events of diarrhea and abdominal pain (GI events) in patients with ALGS. In the 86 patients evaluated, the analysis included incidence, severity, seriousness, time to onset, and duration of events, irrespective of relatedness to treatment with maralixibat, as determined by the investigator.