Long-Term LIVMARLI Data in ALGS and PFIC Highlighted at EASL Congress

Data Highlights Long-Term Efficacy of LIVMARLI in Alagille Syndrome
New findings presented at the European Association for the Study of the Liver (EASL) Annual Congress underscore the sustained benefits of long-term LIVMARLI treatment in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). Mirum Pharmaceuticals, Inc. shared data from studies highlighting the significant clinical improvements observed over extended treatment periods with LIVMARLI® (maralixibat) oral solution.

Key Study Findings:

1. Alagille Syndrome (ALGS):
Presented by Dr. Doug Mogul, the analysis from the MERGE study revealed that patients with ALGS treated with LIVMARLI for up to seven years showed sustained clinical benefits. Nearly all patients who continued treatment experienced meaningful reductions in pruritus and serum bile acids. Additionally, improvements in height were noted, signifying overall health benefits.

2. Progressive Familial Intrahepatic Cholestasis (PFIC):
Another significant analysis presented by Professor Richard J. Thompson focused on growth improvements in PFIC patients. Data from the MARCH/MARCH-ON trials involving 60 patients demonstrated statistically significant improvements in height and weight z-scores over 82 weeks of treatment with LIVMARLI.

3. Liver Health Improvement in PFIC:
Dr. Lorenzo D’Antiga presented findings on the relationship between reductions in serum bile acid (sBA) levels and key liver health markers in PFIC patients. The analysis indicated that decreases in sBA levels were associated with reductions in bilirubin levels, suggesting potential disease-modifying effects of LIVMARLI and its positive impact on liver health.

About LIVMARLI® (maralixibat) Oral Solution:
LIVMARLI is an orally administered, once-daily ileal bile acid transporter (IBAT) inhibitor. It is approved for the treatment of cholestatic pruritus in ALGS patients three months and older and in PFIC patients five years and older in the United States. Additionally, it holds approval from the European Commission and Health Canada for the treatment of cholestatic pruritus in ALGS patients as young as two months. LIVMARLI has received a positive opinion from the CHMP in Europe for the treatment of PFIC in patients three months and older, with a final decision expected by the third quarter of 2024.

Safety Information:
LIVMARLI has known side effects, including potential liver injury, gastrointestinal issues, and fat-soluble vitamin deficiency. Liver function monitoring through blood tests and physical exams is recommended before and during treatment. Patients may experience liver-related symptoms such as nausea, vomiting, jaundice, dark urine, abdominal pain, bloating, appetite loss, and unusual bruising or bleeding. Gastrointestinal symptoms like diarrhea and stomach pain are also common. Monitoring for vitamin deficiencies, bone fractures, and bleeding is advised due to the risk of fat-soluble vitamin deficiency.

About Mirum Pharmaceuticals:
Mirum Pharmaceuticals, Inc. is dedicated to transforming the treatment of rare diseases affecting children and adults. Its product portfolio includes three approved medications: LIVMARLI, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets. LIVMARLI is approved for treating rare liver conditions such as ALGS and PFIC across various regions including the U.S. and Europe. Mirum is also pursuing further clinical research with investigational treatments like volixibat and CHENODAL for other debilitating liver diseases.

The continuous research and positive findings presented at the EASL Annual Congress reinforce the potential of LIVMARLI in providing long-term clinical benefits for patients with ALGS and PFIC, improving their overall quality of life and health outcomes.