Mirum Hits All-Time High - Is There More Upside Ahead?

(RTTNews) - Mirum Pharmaceuticals Inc. (MIRM) has kicked off the year with commercial growth and successful pipeline milestones, propelling its shares to an all-time high of $53.

The company has three approved ultra-rare medicines and 4 clinical programs. Its marketed drugs include Livmarli oral solution and tablets, Cholbam capsules, and Ctexli tablets.

-- Livmarli is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 3 months of age and older, and progressive familial intrahepatic cholestasis (PFIC) five years of age and older in the U.S. The drug is also approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. Livmarli is also approved in Japan for ALGS and PFIC.

-- Cholbam is indicated for the treatment of bile acid synthesis disorders due to single enzyme defects (SEDs) and as an adjunctive treatment of peroxisomal disorders (PDs), including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption.

-- Ctexli is a synthetic form of the bile acid chenodeoxycholic acid that is indicated for the treatment of adults with cerebrotendinous xanthomatosis.

For the first quarter ended March 31, 2025, total revenue jumped to $111.6 million from $69.2 million in the year-ago quarter. Sales of Livmarli contributed $73.2 million to the total, reflecting a 71% year-over-year growth compared to the first quarter of 2024.

Net loss for the first quarter of 2025 narrowed to $14.67 million or $0.30 per share from $25.27 million or $0.54 per share in Q1 2024.

Based on its strong first-quarter results, the company expects total revenue for 2025 to range between $435 million and $450 million, representing an increase of approximately 29% to 34% compared to the $336.4 million reported in 2024.

Pipeline & Near-term Catalysts

Mirum's lead drug candidate, Volixibat, is being evaluated in the following trials:

-- A phase IIb study in Primary Sclerosing Cholangitis patients with cholestatic pruritus, dubbed VISTAS. -- A phase IIb study in Primary Biliary Cholangitis patients with cholestatic pruritus, dubbed VANTAGE.

Enrollment in the VISTAS study is anticipated to be completed this quarter, while the VANTAGE study is on track to complete enrollment in 2026.

Up next is MRM-3379, an investigational oral drug for the treatment of fragile X syndrome. A phase II study for MRM-3379 in Fragile X Syndrome is due to be initiated this year.

The company is also evaluating its flagship drug Livmarli in additional settings of cholestatic pruritus in a phase III trial, dubbed EXPAND. Enrollment in this study is expected to be completed in 2026.

Mirum Pharma shares began trading on the Nasdaq Global Market on July 18, 2019, under the symbol "MIRM", priced at $15 per share.

When we alerted readers to MIRM on May 22, 2024, it was trading at $25.81. The stock hit an all-time high of $54.78 during intraday trading yesterday, before closing at $53.28.