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 3

The data showed that patients with ALGS treated with long-term maralixibat (up to four years) had significantly increased height from baseline to week 204 (p=0.0004). Patients with the lowest height and weight z-scores at baseline had the greatest improvements in height and weight z-scores, and individuals that had the greatest catch-up weight gain also had the greatest catch-up height growth at week 48 (p=0.0013). The analysis also concluded that maralixibat-treated patients who achieved an sBA threshold <200 µmol/L had greater accelerated height, suggesting bile acid homeostasis may facilitate improvement in height deficits. Further analyses, including comparison to natural history cohort of patients with ALGS are needed to fully characterize the impact of maralixibat treatment on growth.

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H-O-008: Maralixibat treatment response is associated with improved health-related quality of life in patients with bile salt export pump (BSEP) deficiency

Dr. Doug Mogul on behalf of Dr. Kathy Loomes (Oral presentation)

The analysis evaluated patients with BSEP deficiency, or PFIC2, following treatment with maralixibat. Patients with PFIC suffer from poor health-related quality of life (HRQoL) compared with healthy children, thus the aim of this analysis was to assess the impact of maralixibat treatment response at week 48 on HRQoL among a subset of children with BSEP deficiency from the INDIGO Phase 2 study.

Overall, patients with BSEP deficiency who achieved sBA treatment response (defined as >75% decrease from baseline or reduction to <102 µmol/L from baseline to week 48) experienced clinically meaningful improvements in PedsQL Generic Core Total Scale Score and Multidimensional Fatigue Total Scale Score, as well as statistically significant improvements in sleep and fatigue, as compared to those who did not achieve an sBA response.

Baseline characteristics were similar for responders and non-responders but baseline HRQoL Multidimensional Fatigue Score was lower for responders. With a minimal clinically important difference (MCID) of 4-5 points, the results demonstrated:

• PedsQL Generic Core Scale: responders improved significantly by >3× MCID (p=0.01)
• Multidimensional Fatigue Scale: responders improved significantly by >4× MCID (p=0.04)
• Family Impact Scale: clinically meaningful improvements did not reach statistical significance

In conclusion, response to maralixibat has the potential to significantly improve HRQoL in patients with PFIC.