Company: MIRM
Filing Date: 2025-08-12
Form Type: S-3ASR
Source: 0001193125-25-178937
Chunk: 45

Company: Mirum Pharmaceuticals, Inc.
Filing Date: 2025-08-12
Form: S-3ASR
Chunk 45
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bam (also known as
Kolbam) pursuant to an asset purchase agreement dated July 16, 2023.

The U.S. Food and Drug Administration (“FDA”)
approved Cholbam in March 2015, as the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients
with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder. Chenodiol is standard of care for the treatment of cerebrotendinous xanthomatosis (“CTX”) in the United States with a medical necessity
recognition by the FDA and was commercialized under the brand name Chenodal. We submitted a new drug application for chenodiol for the

S-2

treatment of CTX in 2024 and received FDA approval for the treatment of adults with CTX in February 2025, which is commercialized under the brand name Ctexli. We currently commercialize Chenodal,
Cholbam and Ctexli in the U.S. through our specialized and focused commercial team. We have also assumed license and distribution agreements with several rare disease companies for the commercialization of Cholbam and chenodiol in additional
countries.

We are developing Livmarli for certain rare cholestatic conditions through the Phase 3 EXPAND study, which we initiated in the
fourth quarter of 2024. In addition, we are advancing our product candidate, volixibat, a novel, oral, minimally-absorbed agent designed to inhibit IBAT, for the treatment of adult patients with cholestatic liver diseases. We are developing
volixibat in the setting of primary sclerosing cholangitis (“PSC”) and primary biliary cholangitis (“PBC”), and in October 2024, we announced that the FDA granted Breakthrough Therapy Designation for volixibat as a potential
treatment for cholestatic pruritus in patients with PBC. We conducted an interim analysis of our VISTAS Phase 2b clinical trial in PSC and reported interim data from our VANTAGE Phase 2b clinical trial in PBC in June 2024. We expect the VISTAS Phase
2b clinical trial in PSC to complete enrollment in the third quarter of 2025 with topline data expected in the second quarter of 2026, and we expect the VANTAGE Phase 2