Mirum Pharmaceuticals Sells Rare Pediatric Disease Priority Review Voucher

FOSTER CITY, Calif.–(BUSINESS WIRE)–Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare liver disease, announced today that it has entered into a definitive agreement to sell its Priority Review Voucher for rare pediatric diseases (“PRV”) for $110 million.

The PRV was granted by the US Food and Drug Administration in September 2021 with the approval of LIVMARLI™ (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

Under the agreement, Mirum will receive a payment of $110 million upon closing of the transaction, which is subject to customary closing conditions and is expected to occur upon the expiration of applicable U.S. antitrust clearance requirements.

Jefferies LLC acted as the exclusive financial advisor to Mirum for this transaction.

About LIVMARLI™ (maralixibat) oral solution

LIVMARLI™ (maralixibat) oral solution is an orally administered, once-daily ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older and is the only FDA-approved drug for the treatment of cholestatic pruritus associated with Alagille syndrome. For more information, visit LIVMARLI.com.

LIVMARLI is currently being evaluated in late stage clinical trials of other rare cholestatic liver diseases, including progressive familial intrahepatic cholestasis and biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan drug designation for ALGS, PFIC and biliary atresia. For more information about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section of the company’s website.

About Alagille Syndrome

Alagille syndrome (ALGS) is a rare genetic disorder in which bile ducts are abnormally narrow, malformed, and reduced in number, leading to bile buildup in the liver and eventually progressive liver disease. The estimated incidence of ALGS is one in every 30,000 people.1 In patients with ALGS, multiple organ systems can be affected by the mutation, including the liver, heart, kidneys, and central nervous system.2 The buildup of bile acids prevents the liver from working good at removing waste products from the bloodstream and according to recent reports, 60% to 75% of patients with ALGS undergo liver transplantation before they reach adulthood.3 Signs and symptoms resulting from liver damage in ALGS may include jaundice (yellowing of the skin) ), xanthomas (disfiguring cholesterol deposits under the skin) and pruritus (itching)2. The pruritus experienced by patients with ALGS is one of the most severe of all chronic liver disease and is present in most affected children by the third year of life.4


LIVMARLI can cause serious side effects, including:

Changes in liver testing. Changes in certain liver tests are common in patients with Alagille syndrome and may worsen during treatment with LIVMARLI. These changes can be a sign of liver damage and can be serious. Your healthcare provider should do blood tests to check your liver function before and during treatment. Tell your healthcare provider right away if you develop any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark, or brown, urine, pain in the right side of the stomach (abdomen), or loss of appetite .

Stomach and intestinal (gastrointestinal) problems. LIVMARLI may cause stomach and intestinal problems during treatment, including diarrhoea, stomach pain and vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severe than usual for you.

A condition called fat-soluble vitamin (FSV) deficiency, caused by low levels of certain vitamins (vitamins A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome, but may worsen during treatment. Your healthcare provider should do blood tests before and during treatment.

Other common side effects reported during treatment were bone fractures and gastrointestinal bleeding.

Prescribing Information

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum’s approved drug is LIVMARLI™ (maralixibat) oral solution approved in the US for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver disease in children and adults. Maralixibat (LIVMARLI), an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver disease and includes the MARCH Phase 3 trial for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b trial for patients with biliary atresia. In addition, Mirum has opened an extensive access program in Canada, Australia, the UK and several countries in Europe for eligible patients with Alagille syndrome.

Mirum has applied to the European Medicines Agency for a marketing authorization for maralixibat for the treatment of cholestatic liver disease in patients with Alagille syndrome.

Mirum’s second investigational treatment, volixibat, also an oral IBAT inhibitor, is being evaluated in two pivotal trials, including the OHANA Phase 2b trial for pregnant women with intrahepatic cholestasis of pregnancy and the VISTAS Phase 2b trial for adults with primary sclerosing cholangitis . Mirum plans to begin a Phase 2b trial in primary biliary cholangitis later this year.

To expand its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize VTX-803 and VTX-802 gene therapy programs for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and research to enable new drug studies.

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Forward-Looking Statements

Statements in this press release about matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the closing of the PRV sale and Mirum’s receipt of the proceeds of the proposed sale. Because such statements are subject to risks and uncertainties, actual results could differ materially from those expressed or implied by such forward-looking statements. Words such as “will”, “would”, “would”, “potentially” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based on Mirum’s current expectations and contain assumptions that may never materialize or prove incorrect. Actual results could differ materially from those anticipated in such forward-looking statements due to various risks and uncertainties, including, but not limited to, risks and uncertainties associated with Mirum’s business in general, the impact of the COVID-19 19 pandemic and the other risks described in Mirum’s filings with the Securities and Exchange Commission. All forward-looking statements in this press release speak only as of the date they are made and are based on management’s assumptions and estimates as of that date. Mirum assumes no obligation to update such statements to reflect events that occur or circumstances that exist after the date they are made, except as required by law.


1Dank, et al. Archives of Childhood Illness 1977

2Johns Hopkins drug. hopkinsmedicine.org/health/conditions-and-diseases/Alagille-syndrome

3Vandriel, et al. GALA EASL 2020; Kamath, et al. Hepatology Communication 2020

4Elisofon, et al. Journal of Pediatric Gastroenterology and Nutrition 2010

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