FOSTER CITY, Calif. - Mirum Pharmaceuticals, Inc. (NASDAQ: NASDAQ:MIRM) announced that new data from its long-term treatment studies with LIVMARLI (maralixibat) oral solution will be presented at the upcoming European Association for the Study of the Liver (EASL) Annual Congress in Milan, Italy. The data highlight sustained clinical benefits and improvements in liver health markers and growth in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC).
The MERGE study results, to be presented on Thursday, reveal that patients with ALGS maintained clinical benefits through seven years of LIVMARLI treatment. Moreover, data from the MARCH/MARCH-ON trials indicate that maralixibat is associated with improved growth in PFIC patients and that serum bile acid level improvements correlate with better liver health markers in children with PFIC.
LIVMARLI, an ileal bile acid transporter (IBAT) inhibitor, is currently approved by the U.S. Food and Drug Administration for treating cholestatic pruritus in ALGS patients three months and older and PFIC patients five years and older. The European Commission and Health Canada have also approved LIVMARLI for similar indications. Moreover, Mirum has submitted LIVMARLI for approval in Europe for PFIC patients two months and older.
The medication has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for these conditions. Notably, LIVMARLI is not recommended for PFIC type 2 patients with a severe defect in the bile salt export pump (BSEP) protein due to potential liver injury risks.
Mirum Pharmaceuticals is focused on developing therapies for rare diseases affecting both children and adults. Its portfolio includes three approved medications: LIVMARLI, CHOLBAM (cholic acid) capsules, and CHENODAL (chenodiol) tablets.
The company's research pipeline features investigational treatments for liver diseases, with volixibat being evaluated for primary sclerosing cholangitis and primary biliary cholangitis, and CHENODAL showing positive results in a Phase 3 study for cerebrotendinous xanthomatosis (CTX) in 2023.
The upcoming symposium sponsored by Mirum on June 5 will delve into the pediatric experience with IBAT inhibitors and their potential application in adult hepatology, specifically for primary sclerosing cholangitis (PSC).
This article is based on a press release statement from Mirum Pharmaceuticals, Inc.
InvestingPro Insights
As Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) continues to make strides in the hepatology field with its promising treatment studies and regulatory achievements, a closer look at the company's financial health through InvestingPro data reveals a mixed picture.
With a market capitalization of approximately $1.17 billion, Mirum's financial scale is substantial in the biopharmaceutical industry, particularly for a company focusing on rare liver diseases. The company's revenue has shown impressive growth over the last twelve months as of Q1 2024, surging by 133.9%, indicating strong market uptake of its products and potential for future expansion.
Despite the positive revenue trajectory, Mirum operates at a loss, with an operating income margin of -48.43% and a negative return on assets of -32.18% for the same period. This is consistent with the InvestingPro Tips, which suggest that analysts do not expect the company to be profitable this year. Still, the same tips also highlight that Mirum’s liquid assets surpass its short-term obligations, which may provide some financial stability as it continues to invest in research and development.
The company's stock is currently trading near its 52-week low, which, coupled with the lack of dividend payouts to shareholders, may raise concerns for potential investors looking for immediate returns. Nevertheless, for those with a longer-term outlook, the substantial revenue growth and the company's commitment to addressing unmet needs in rare liver diseases could present an opportunity, especially considering the fair value estimates. According to InvestingPro, the fair value of Mirum's stock stands at $27.15, which suggests a potential upside from the previous close price of $24.76.
For investors seeking more comprehensive analysis, InvestingPro offers additional tips on Mirum Pharmaceuticals, which can be accessed through the platform. With the use of the coupon code PRONEWS24, users can get an additional 10% off a yearly or biyearly Pro and Pro+ subscription, unlocking further insights that could inform investment decisions.
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