Mirum Pharmaceuticals Highlights Data Demonstrating Impact of LIVMARLI (maralixibat) on Patients with Alagille Syndrome at European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) Annual Meeting - Seite 2

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H-O-012: Predictors of 6-year event-free survival in patients with ALGS treated with maralixibat, an IBAT inhibitor

Dr. Ron Sokol (Oral Presentation)

**Nominee for Alex Mowat Prize for Best Oral Presentation in Hepatology**

Data from the pooled maralixibat clinical studies with up to six years of follow up were analyzed to determine predictors of event-free and transplant-free survival in patients with ALGS. 76 patients met the criteria for inclusion in this analysis and 43 variables were tested as predictors. The laboratory markers that were predictive of event-free and transplant-free survival included specific thresholds of week 48 total bilirubin and week 48 serum bile acids. In addition, improvement in pruritus from baseline to week 48 was also predictive of event-free and transplant-free survival. 76% and 79% of maralixibat-treated ALGS patients remained event-free and transplant-free, respectively, at six years after treatment initiation. These data highlighted potential prognostic markers that could help to inform medical management for patients receiving maralixibat.

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H-P-047: Significant improvement in cholestatic pruritus in patients with Alagille syndrome treated with maralixibat, an ileal bile acid transporter inhibitor: Real-world experience

Dr. Regino Gonzalez-Peralta, Dr. Douglas Mogul (Poster presentation)

The goal of this assessment was to evaluate the real-world application of maralixibat in children with ALGS and cholestatic pruritus. Three patients with ALGS were enrolled in the maralixibat Expanded Access Program with a variety of clinical manifestations. During the course of treatment with maralixibat, pruritus was assessed using the Clinician Scratch Scale (0-4 where 0=none and 4=cutaneous mutilation, hemorrhage and scarring was evident), and liver enzymes were monitored at varying time intervals in the months prior to and after starting maralixibat.

The data showed that treatment with maralixibat led to significant and durable amelioration of pruritus in these individual children with ALGS.

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H-P-058: Maralixibat improves growth in patients with Alagille syndrome: A 4-year analysis

Dr. Binita Kamath (Poster presentation)

The analysis evaluated the impact of long-term maralixibat treatment on the growth and nutritional status of patients with ALGS, a disease for which patients typically present with significant growth deficits. Height and weight z-scores were evaluated in patients who participated in the maralixibat ICONIC study (along with the long-term, open label extension studies). The analysis included patients with ALGS who had both baseline and 204-week assessments for all eight parameters, and this group was divided into four subgroups based on height and weight z-score quartiles. No changes beyond standard of care in supplementation occurred during the study.