- Transaction reduces royalty and milestone obligations for LIVMARLI® and volixibat

- Cash flow positive transaction

Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that it has acquired Satiogen Pharmaceuticals, Inc., a San Diego-based company. Satiogen, now a wholly-owned Mirum subsidiary, was an existing licensing partner for LIVMARLI® (maralixibat) oral solution and volixibat. Through the transaction, Mirum obtained all Satiogen licensing payments and Satiogen-owned intellectual property relating to LIVMARLI and volixibat.

"The acquisition of Satiogen is a strategic step that consolidates the economics of our commercial and pipeline programs," said Chris Peetz, president and chief executive officer of Mirum. "The Satiogen team laid some of the foundational groundwork for the potential for IBAT inhibitors in liver disease that is now translating to substantial benefits for patients and a successful launch of LIVMARLI in Alagille syndrome. We thank the Satiogen team as we build on their work to continue to advance important therapies for rare disease."

The transaction will result in a reduction of total licensing royalty obligations for LIVMARLI and volixibat to high single digit to low teens. The total potential consideration for the acquisition consisted of a combination of 841,792 shares of common stock, 199,993 of which is subject to achievement of a milestone, and approximately $2.8 million in cash in respect of an equivalent amount of cash on the books of Satiogen acquired by Mirum at the closing. Mirum will wholly own the Satiogen subsidiary, which will receive a 2% royalty.

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 (ALGS) one year of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis (PFIC) and biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's website.

IMPORTANT SAFETY INFORMATION

LIVMARLI can cause serious side effects, including:

Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get 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 on the right side of the stomach (abdomen) or loss of appetite.

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

A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin 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 starting and during treatment.

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

Prescribing information

About Volixibat

Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Phase 1 and Phase 2 studies of volixibat demonstrated on-target fecal bile acid excretion, a pharmacodynamic marker of IBAT inhibition, in addition to decreases in LDL cholesterol and increases in 7αC4 which are markers of bile acid synthesis. Volixibat has been evaluated in more than 400 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.

Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS Phase 2b clinical trial), intrahepatic cholestasis of pregnancy (OHANA Phase 2b clinical trial), and primary biliary cholangitis (VANTAGE Phase 2b clinical trial).

About Mirum Pharmaceuticals

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum's approved medication is LIVMARLI® (maralixibat) oral solution which is approved in the United States 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 diseases affecting children and adults. LIVMARLI, an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the MARCH Phase 3 clinical trial for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b clinical trial for patients with biliary atresia. In addition, Mirum has an expanded access program open across multiple countries for eligible patients with ALGS and PFIC.

Mirum has submitted a Marketing Authorization Application to the European Medicines Agency for LIVMARLI for the treatment of cholestatic liver disease in patients with Alagille syndrome.

Mirum's second investigational treatment, volixibat, an oral IBAT inhibitor, is being evaluated in three potentially registrational studies including the VISTAS Phase 2b clinical trial for adults with primary sclerosing cholangitis, the OHANA Phase 2b clinical trial for pregnant women with intrahepatic cholestasis of pregnancy, and the VANTAGE Phase 2b clinical trial for adults with primary biliary cholangitis.

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About Satiogen Pharmaceuticals

Satiogen was founded in late 2007 and has been operating as a virtual company by outsourcing all activities required to validate the bile acid brake concept. Satiogen's focus has been on preclinical experiments for proof-of-concept, on collaborating in clinical trials that confirm effects in humans, and on assembling an intellectual property portfolio. For more information, visit www.satiogen.com.

Forward-Looking Statements

Statements contained in this press release regarding 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 expected reduction to Mirum's total licensing royalty obligations, the expected benefits of the acquisition transaction on Mirum's business and operations, including consolidation of the economics of Mirum's commercial and pipeline programs, the anticipated impact for patients with liver disease and the launch of LIVMARLI, Mirum's continued advancement of therapies for rare disease and the potential success and impacts of Mirum's IBAT programs. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as "will," "anticipate," "expect," "potential," "intend" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of the COVID-19 pandemic, and the other risks described in Mirum's filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.

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