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FOSTER CITY, Calif. - Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM), a biopharmaceutical company with a market capitalization of $1.94 billion and impressive revenue growth of 81% in the last twelve months, has received approval from the U.S. Food and Drug Administration (FDA) for a tablet formulation of LIVMARLI® (maralixibat), aimed at treating cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). According to InvestingPro data, the company maintains a strong gross profit margin of 76%, reflecting efficient operations in its specialty pharmaceutical business. This new tablet option is expected to enhance the convenience for patients and healthcare providers by offering a solid form in addition to the existing liquid formulation.
Peter Radovich, president and chief operating officer at Mirum, highlighted the success of LIVMARLI since its launch and expressed optimism that the new tablet formulation will positively impact patients’ lives by providing an additional treatment option. The availability of LIVMARLI tablets is scheduled for June through Mirum Access Plus. The company’s strong financial position is evidenced by its healthy current ratio of 3.1, indicating robust liquidity to support its commercialization efforts. For deeper insights into Mirum’s financial health and growth potential, investors can access comprehensive analysis through InvestingPro’s detailed research reports.
LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is currently approved in the U.S. for ALGS patients three months and older and for PFIC patients 12 months and older. It is also approved in Europe for ALGS patients two months and older and PFIC patients three months and older. Mirum is conducting the Phase 3 EXPAND study to explore the use of LIVMARLI in further settings of cholestatic pruritus.
The FDA’s approval comes with a limitation of use for PFIC type 2 patients with a severe defect in the bile salt export pump (BSEP) protein. Side effects of LIVMARLI can include liver injury and gastrointestinal problems, among others. Healthcare providers are advised to conduct regular blood tests and physical exams to monitor liver function and check for fat-soluble vitamin (FSV) deficiency, which is common in patients with ALGS and PFIC but can worsen during treatment.
Mirum Pharmaceuticals focuses on developing treatments for rare diseases affecting both children and adults. Alongside LIVMARLI, the company has two other approved medications: CHOLBAM® (cholic acid) capsules and CTEXLI™ (chenodiol) tablets. Mirum is also advancing its late-stage pipeline, including volixibat and MRM-3379 for other rare diseases. The company’s stock has demonstrated strong performance, with a 61% return over the past year, though InvestingPro analysis suggests the stock is currently trading near its Fair Value. Investors seeking detailed valuation metrics and additional ProTips can explore InvestingPro’s comprehensive research platform.
The information in this article is based on a press release statement from Mirum Pharmaceuticals, Inc.
In other recent news, Mirum Pharmaceuticals reported fourth-quarter revenue of $99.41 million, exceeding analyst expectations of $95.4 million and marking a 55.6% increase compared to the previous year. However, the company posted a fourth-quarter adjusted loss of $1.85 per share, significantly missing the anticipated $0.30 loss per share. For the full year 2024, Mirum’s total net product sales reached $336.4 million, a substantial increase from $178.9 million in 2023. Mirum’s LIVMARLI drug, which generated $213.3 million in 2024 sales, recently received approval in Japan for treating rare liver diseases, marking a significant milestone for the company.
Furthermore, H.C. Wainwright raised Mirum’s stock target to $72, maintaining a Buy rating, following the company’s announcement that it is on track to meet its 2025 sales guidance. The firm projects global net product sales between $420 million to $435 million for 2025, suggesting continued growth. Cantor Fitzgerald also provided insights into the biotech sector’s struggles, noting a challenging climate for biotech investors in 2025. Despite these challenges, Mirum remains focused on expanding its product reach and anticipates several clinical milestones, including the completion of its VISTAS study of volixibat in 2025.
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