Mirum Banks on Livmarli, Overdependence a Concern

Mirum Pharmaceuticals, Inc.'s MIRM lead product, Livmarli (maralixibat), an orally administered ileal bile acid transporter ("IBAT") inhibitor, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) worldwide. Sales of Livmarli have been rising steadily since its approval and launch in 2021.

The FDA approved a label expansion for Livmarli oral solution to include the treatment of cholestatic pruritus in patients aged five years and older with progressive familial intrahepatic cholestasis ("PFIC") in March.

Also, during the second quarter of 2024, Livmarli was approved for treating cholestatic pruritus in PFIC patients aged 12 months and above in the United States.

The European Commission approved Livmarli for the treatment of PFIC in patients aged three months and older in July.

In the first half of 2024, Livmarli generated sales worth $90.1 million, up 46.3% on a year-over-year basis. The label expansion and the continuous demand for the drug should drive sales further in the days ahead.

Shares of Mirum have rallied 37.8% year to date against the industry's decrease of 1.7%.

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In June 2024, Mirum submitted a new drug application for chenodiol for treating cerebrotendinous xanthomatosis patients in the United States.

Chenodiol is not currently approved but has received a medical necessity medicine status from the FDA for the treatment of CTX, a rare autosomal genetic disorder.

We remind investors that in August 2023, Mirum acquired all assets of Travere Therapeutics' bile acid products, which added two other products to its commercial portfolio — Cholbam (cholic acid) capsules and Chenodal (chenodiol) tablets. Cholbam is approved for treating bile acid synthesis disorders and Zellweger spectrum disorders.

The addition of these two commercialized products helps Mirum to diversify its revenue stream.

This apart, MIRM is developing its pipeline candidate, volixibat, an oral IBAT inhibitor, in two separate phase IIb studies for the treatment of primary biliary cholangitis and primary sclerosing cholangitis.

If successfully developed and upon potential approval, volixibat may provide a novel approach to the treatment of adult cholestatic diseases.

The continued demand for Livmarli and the addition of Cholbam and Chenodal should help Mirum establish a leading pediatric hepatology franchise. However, the company remains heavily dependent on Livmarli for revenues, which is a concern. Any regulatory setback for the drug will hurt the company's growth prospects.

Also, any developmental setback related to the pipeline will have an adverse effect on the company's growth prospects. Also, it remains to be seen whether Cholbam and Chenodal are able to reduce the heavy dependence on Livmarli for revenues in the days ahead.

Zacks Rank & Stocks to Consider

Mirum currently carries a Zacks Rank #3 (Hold).

Some top-ranked stocks in the biotech sector are Illumina, Inc. ILMN and Fulcrum Therapeutics, Inc. FULC, both sporting a Zacks Rank #1 (Strong Buy) at present.

In the past 60 days, estimates for Illumina's 2024 earnings per share have moved up from $1.07 to $3.16. Earnings per share estimates for 2025 have improved from $2.93 to $4.49. Year to date, shares of ILMN have lost 6.4%.

ILMN's earnings beat estimates in each of the trailing four quarters, the average surprise being 463.46%.

In the past 60 days, estimates for Fulcrum Therapeutics' 2024 loss per share have narrowed from $1.24 to 48 cents. Loss per share estimates for 2025 have narrowed from $1.71 to $1.51. Year to date, shares of FULC have surged 36.9%.

FULC's earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.

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