Mirum Pharmaceuticals Showcases Five New LIVMARLI (maralixibat) Presentations at the EASL Congress - Seite 2

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LBP-35: Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): Data from the MARCH-ON study
Late-breaker poster presentation

The analysis evaluated the long-term maintenance response to maralixibat in patients who were randomized to receive maralixibat (MRX-MRX) or placebo (PBO-MRX) in MARCH and continued treatment with maralixibat in MARCH-ON. Eighty-five patients from MARCH enrolled in MARCH-ON (47 MRX and 38 placebo). Baseline was defined as the start of maralixibat treatment for each group. The results showed:

• Significant improvements observed in the first 26 weeks of the MARCH study were sustained from Baseline to Week 52 in MARCH-ON for pruritus severity, sBA levels, total bilirubin, height -score and weight Z-score in the MRX-MRX group.
• Newly gained statistically significant reductions in pruritus severity and sBA levels were observed in the key efficacy endpoints from Baseline to Week 26 in the MRX-PBO group, in line with observations from the initial MARCH maralixibat group.
• No new safety signals were identified. The most frequent treatment emergent adverse events were gastrointestinal-related, in line with the mechanism of action of IBAT inhibition, mostly mild and transient. Patients who previously received maralixibat in MARCH were less likely to have events in MARCH-ON compared to MARCH.
• The data suggest overall improved liver health following maralixibat treatment in patients with PFIC, which are maintained over time.

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WED-257: Impact of maralixibat on cholestatic pruritus in adults aged 16 years and older with Alagille syndrome

Previous data in ALGS has been largely focused on pediatric populations; however, 24-40.3% of patients with ALGS reach 18 years of age with their native liver and may require treatment for cholestasis and pruritus. This analysis reported, for the first time, the efficacy and safety of maralixibat in adult patients with ALGS; ≥16 years transitioning to adult care and participants aged >16 years who initiated treatment in the ALGS clinical program. Results demonstrated that: