Mirum Pharmaceuticals to Showcase LIVMARLI® (maralixibat) Data at EASL and ESPGHAN Annual Congresses

- Eight abstracts accepted, including three oral presentations

- Maralixibat data highlighting predictors of event-free survival in patients with Alagille syndrome nominated for prestigious Alex Mowat Prize for best oral presentation in hepatology

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) will present data at the upcoming International Liver Congress™ of the European Association for the Study of the Liver (EASL) and the 54^th Annual European Society for Paediatric Gastroenterology, Hepatology, and Nutrition Annual Meeting (ESPGHAN), taking place in London and Copenhagen, respectively. The Company will present data from its LIVMARLI® (maralixibat) clinical studies and will unveil a multi-national survey of patient-reported outcomes data assessing pruritus and overall disease burden in adults with primary sclerosing cholangitis.

Mirum will also host a scientific symposium during each meeting:

• EASL: “Patient, caregiver, and physician: Understanding perspectives in adult and pediatric cholestasis”
  • Thursday, June 23, 2022 from 6:30-8:00 p.m. BST
• ESPGHAN: “Cholestatic Disease in Children: From Diagnosis to Patient Management”
  • Thursday, June 23, 2022 from 6:15-7:15 p.m. CEST

**ESPGHAN: June 22-25, 2022, Copenhagen, Denmark**

All abstracts are available via the Journal of Paediatric Gastroenterology and Nutrition and the page number for each abstract is enclosed with the title below.

Oral presentations

H-O-008: Maralixibat treatment response is associated with improved health-related quality of life in patients with bile salt export pump (BSEP) deficiency (pg. 650)

Oral presentation: June 23, 2022 at 5:00 p.m. CEST

Presenter: Dr. Douglas Mogul (on behalf of Dr. Kathy Loomes)

H-O-012: Predictors of 6-year event-free survival in patients with Alagille syndrome treated with maralixibat, an IBAT inhibitor (pg. 658)

Oral presentation: June 24, 2022 at 8:45 a.m. CEST

**Nominated for the Alex Mowat Prize for Best Oral Presentation in Hepatology**

Presenter: Dr. Ronald Sokol

H-O-018: Maralixibat-treated patients with Alagille syndrome (ALGS) demonstrate improved event-free survival in a natural history comparison with patients from the GALA database: application of real-world evidence analytics (pg. 670)

Oral presentation: June 24, 2022 at 9:45 a.m. CEST

Presenter: Dr. Bettina Hansen

Poster presentations

H-P-058: Maralixibat improves growth in patients with Alagille syndrome: a 4-year analysis (pg. 778)

Poster: June 24, 2022 from 2:00-2:45 p.m. CEST

Authors: Dr. Binita Kamath, et al

H-P-057: Response to treatment with maralixibat in Alagille syndrome is associated with improved health-related quality of life (pg. 776)

Poster Walk: June 24, 2022 at 2:00-2:45 p.m. CEST

Author: Dr. Binita Kamath, et al

H-P-047: Significant improvement in cholestatic pruritus in patients with Alagille syndrome treated with maralixibat, an ileal bile acid transporter inhibitor: Real-world experience (pg. 763)

Poster Walk: June 24, 2022 from 2:00-2:45 p.m. CEST

Authors: Dr. Regino Gonzalez-Peralta and Dr. Doug Mogul

**International Liver Congress – EASL: June 22-26, 2022, London, United Kingdom**

All abstracts are available via the EASL website and the page number for each abstract is enclosed with the title below.

Poster Presentations

Poster 634: Dosing ileal bile acid transporter inhibitors in the fasted state minimizes gastrointestinal adverse effects while maintaining pharmacodynamic effect (pg. S599)

Author: Dr. Cory Kostrub, et al.

Poster 756: Impact of pruritus in primary sclerosing cholangitis (PSC): a multinational survey (pg. S312)

Author: Dr. Kris Kowdley, et al.

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. LIVMARLI is not approved for use in PFIC or 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 U.S. 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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Forward-Looking Statements

This press release includes forward-looking statements pertaining to the Company’s planned participation at a scientific conference, which may include discussion of the Company’s clinical and research data, including the discovery, development and commercialization of our product candidates and technologies, and the therapeutic potential thereof, the continuation of our clinical trials, and the success of our collaborations with partners and any potential future collaborations. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those relating to our preclinical research and clinical programs and other risks identified under the heading “Risk Factors” included in our most recent Form 10-Q and Form 10-K filings and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Mirum's current views with respect to future events, and Mirum does not undertake and specifically disclaims any obligation to update any forward-looking statements, except as required by law.

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