Mirum Pharmaceuticals Presents LIVMARLI Clinical Data and Real-World Evidence at NASPGHAN

• LIVMARLI PFIC primary analysis from MARCH study awarded prestigious Gerard Odell Prize for Excellence in Pediatric Hepatology Research
• 15 abstracts demonstrating robust clinical findings from LIVMARLI studies and real-world experience in ALGS and PFIC

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced the presentation of a series of analyses demonstrating further benefit of LIVMARLI® (maralixibat) oral solution in patients with cholestatic pruritus associated with Alagille syndrome (ALGS) and for patients with progressive familial intrahepatic cholestasis (PFIC), two rare debilitating liver diseases. LIVMARLI is the first medication approved by the U.S. Food and Drug Administration (FDA) to treat cholestatic pruritus in patients with ALGS, and the only to be approved for patients three months of age and older.

Data presented during NASPGHAN demonstrated continued evidence of the impact of LIVMARLI for patients with PFIC as well as both clinical data and real-world use of LIVMARLI in patients with ALGS. Presentations included:

PFIC:

• Data from the MARCH PFIC Phase 3 Study was awarded the prestigious Gerard Odell prize for Excellence in Pediatric Hepatology Research. The MARCH PFIC clinical trial was conducted across the broadest range of PFIC types ever studied. Key data from the study demonstrated significant, rapid, and clinically meaningful improvements in pruritus and serum bile acids (sBA) along with significant improvements in bilirubin and weight z-scores.
• Other key PFIC data included analyses on bilirubin normalization, improvement in sleep, long term maintenance of efficacy from the MARCH-ON extension study, and results from the FIC1 genetic cohort.

ALGS:

• Real-world data in patients with ALGS was presented on adherence to LIVMARLI as well as reduction of other medications and vitamin supplementation while on LIVMARLI. In addition, case studies on delay of liver transplant due to significant reductions in pruritus, and quality of life were shown.
• Other key ALGS data included clinically meaningful reductions in the number and severity of xanthomas, and improvement in pruritus and maintenance of effect in adult patients with ALGS.

“The extensive data being presented at NASPGHAN continues to underscore the impact of LIVMARLI across these pediatric cholestatic indications, and we are excited about what this means for the patient community,” said Pam Vig, PhD, head of research and development at Mirum. “The body of research presented at this conference advances our understanding of LIVMARLI’s clinical effect, which is critical for disease management in the real-world setting.”

The full presentations can be found on Mirum’s website within the Publications & Presentations section, and each is also linked within the titles below.

Alagille Syndrome (ALGS)

• Abstract 69: Maralixibat for the treatment of severe xanthomas in two children with Alagille syndrome (poster)
• Abstract 71: Maralixibat persistency and adherence for the treatment of cholestatic pruritus in Alagille Syndrome: Real-world experience in the United States (poster)
• Abstract 72: Maralixibat impact on concomitant medication use for the treatment of cholestatic pruritus in Alagille syndrome: Real-world experience in the United States (poster)
• Abstract 88: Impact of maralixibat on cholestatic pruritus in young adults aged 16 years and older with Alagille syndrome (poster)
• Abstract 89: Maralixibat, an ileal bile acid transporter inhibitor, delays the need for liver transplant in patients with Alagille syndrome: Real-world experience (poster)
• Abstract 626: Real-world safety experience in patients with Alagille syndrome treated with maralixibat (poster)
• Abstract 632: Maralixibat improves xanthomas and hypercholesterolemia in children with Alagille syndrome: An integrated analysis from two clinical trials (poster)

Progressive Familial Intrahepatic Cholestasis (PFIC)

• Abstract 531: Efficacy and safety of maralixibat in patients with progressive familial intrahepatic cholestasis (MARCH): A randomized placebo-controlled phase 3 study (oral presentation)
  **Gerard Odell Prize for Excellence in Pediatric Hepatology Research Recipient**
• Abstract 65: Maralixibat improves cholestatic pruritus and bile acids in children with FIC1: Data from the MARCH trial (poster)
• Abstract 85: Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): Data from the MARCH-ON study (poster)
• Abstract 86: Maralixibat leads to significant reductions in bilirubin for patients with progressive familial intrahepatic cholestasis: Data from the MARCH trial (poster)
• Abstract 347: Analysis of long-term safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: Data from MARCH-ON (poster)
• Abstract 625: Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: Data from the MARCH trial (poster)
• Abstract 630: Maralixibat leads to significant reductions in pruritus and improvements in sleep for children with progressive familial intrahepatic cholestasis: Data from the MARCH trial (poster)

ALGS and PFIC

• Abstract 363: Safety and tolerability of maralixibat in infants from 2 months of age with Alagille syndrome or progressive familial intrahepatic cholestasis: Results from the RISE study

About LIVMARLI® (maralixibat) oral solution

LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and the only approved medication by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older.

LIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for U.S. residents, please visit LIVMARLI.com.

Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older, and in Europe, in PFIC for patients two months of age and older.

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including 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 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 gastrointestinal bleeding and bone fractures.

US Prescribing Information
EU SmPC
Canadian Product Monograph

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, Cholbam® (cholic acid) capsules, and Chenodal® (chenodiol) tablets.

LIVMARLI, an IBAT inhibitor, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in Canada. Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older and in Europe in PFIC for patients two months of age and older. Cholbam is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. Chenodal has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX).

Mirum’s late-stage pipeline includes three investigational treatments for debilitating liver diseases. The LIVMARLI development program includes the Phase 2b EMBARK study for biliary atresia. Mirum’s second investigational IBAT inhibitor is volixibat, which is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis and Phase 2b VANTAGE study for primary biliary cholangitis. Lastly, Chenodal, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX.

To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter.

Forward-Looking Statements

This press release includes forward-looking statements pertaining to the Company’s planned participation at a scientific conference, including data presentation title and synopsis, which may include discussion of the Company’s clinical and research data, including the therapeutic potential and/or commercial viability of our product candidates and technologies in PFIC and ALGS. 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,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. The accuracy of such statements is subject to a number of risks, uncertainties and assumptions including, but are not limited to, the following factors: the uncertainties inherent in research and development; the uncertainties inherent in business and financial planning, including, without limitation, risks related to Mirum’s business and prospects, adverse developments in our focused markets, or adverse developments in the U.S. or global regulatory environment or economies generally; the continued impact of COVID-19 on our business, operations and financial results; and competitive developments. Other factors that might cause such a difference include those discussed in the Company’s filings with the SEC. 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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