Mirum Pharmaceuticals Presents Long-Term LIVMARLI® (maralixibat) Data and New Alagille Syndrome Caregiver Burden Analysis at NASPGHAN 2022 Annual Meeting

- Six-year natural history comparison with maralixibat demonstrates event-free and transplant-free survival in patients with Alagille syndrome (ALGS)

- Characterization of four-year growth improvement in Alagille syndrome with maralixibat

- New analysis evaluating health-related quality of life impact assessed on caregivers of children with Alagille syndrome

- Mirum to host a scientific symposium on the clinical benefits of treating cholestasis in children

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) shared data from three posters presented during the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition Annual Meeting (NASPGHAN) taking place October 12-16, 2022, in Orlando, Florida.

“Our presence at NASPGHAN provides an exciting opportunity for Mirum to showcase maralixibat six-year event-free survival benefit over the natural history standard of care, as well as long-term improvements in growth in patients with Alagille syndrome,” said Pam Vig, PhD, head of research and development at Mirum. “Further, while the burden of pruritus on patients with Alagille syndrome has been well-characterized through prior analyses, the impact to caregivers has been underappreciated, from mental health to financial distress.”

Scientific Symposium

The company will also host a scientific symposium: “Beyond Itch: Exploring the Clinical Benefits of Treating Cholestasis in Children,” featuring Drs. William F. Balistreri (chair), Regino P. Gonzalez-Peralta, Binita M. Kamath, and Saeed Mohammad.

The event will take place on Thursday, October 13, 2022, from 7:15-8:45pm ET in Sebastian L 1/2.

Presentations

The posters presented will be available within the Publications and Presentations section on Mirum’s website after they are presented at NASPGHAN.

Poster #290: Maralixibat Improves Growth in Patients with Alagille Syndrome: A 4-Year Analysis Presented on Friday, October 14, 2022 – 12:00-2:30pm ET, Poster Session II 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 clinical studies and the long-term open-label extensions. 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.

These 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 a serum bile acid threshold