Chenodiol

- First quarter 2025 total revenue of $111.6 million; 2025 guidance increased to $435 to $450 million - VISTAS study in PSC expected to complete enrollment in third quarter of 2025; topline data expected in second quarter of 2026 - LIVMARLI oral tablet formulation FDA approved - Conference call to provide business updates today, May 7 at 1:30 p.m. PT/4:30 p.m. ET FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the first quarter 2025 and provided a business update. “It’s been a strong start to the year with commercial growth and multiple milestones achieved across our pipeline,” said Chris Peetz, chief executive officer of Mirum. “We’re pleased with the FDA’s approval of LIMARLI’s tablet formulation, which provides more options for the ALGS and PFIC community in the U.S. in the form of a convenient single tablet dose. Our VISTAS study of volixibat in PSC is enrolling well and we expect to complete enrollment in the third quarter of this year. We’re excited to continue our strong execution across our commercial medicines and pipeline throughout the year.” Commercial: Raising full year revenue guidance to $435 to $450 million First quarter 2025 global net product sales of $111.6 million. First quarter 2025 LIVMARLI net product sales were $73.2 million representing 71% growth over first quarter 2024 net product sales. Bile Acid Medicines net product sales were $38.4 million representing 47% growth over first quarter 2024 net product sales. LIVMARLI oral tablet formulation approved. LIVMARLI approved in Japan for ALGS and PFIC. CTEXLI (chenodiol) approved in the US for cerebrotendinous xanthomatosis. Regulatory and Pipeline: Clinical milestones on track Volixibat VISTAS study in primary sclerosing cholangitis (PSC) expected to complete enrollment in third quarter of 2025; topline data expected in the second quarter of 2026. Oral presentation of 28-week interim data from volixibat in PBC (VANTAGE study) at EASL on Friday May 9th at 9:30am CEST. Volixibat VANTAGE study in primary biliary cholangitis (PBC) expected to complete enrollment in 2026. LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions expected to complete enrollment in 2026. Expect to initiate Phase 2 study for MRM-3379 in Fragile X Syndrome (FXS) in 2025. Corporate and Financial: Strong balance sheet and financial independence Total revenue for the quarter ended March 31, 2025, was $111.6 million compared to $69.2 million for the quarter ended March 31, 2024. Total operating expenses were $126.8 million for the quarter ended March 31, 2025 compared to $95.7 million for the quarter ended March 31, 2024. Total operating expenses for the quarter ended March 31, 2025 included $21.9 million of non-cash stock-based compensation, intangible amortization, and other non-cash expenses compared to $17.1 million for the quarter ended March 31, 2024. As of March 31, 2025, Mirum had unrestricted cash, cash equivalents and investments of $298.6 million compared to $292.8 million as of December 31, 2024. Business Update Conference Call Mirum will host a conference call today, May 7th at 1:30 p.m. PT/4:30 p.m. ET, to provide business updates. Join the call using the following details: Conference Call Details: U.S./Toll-Free: +1 833 470 1428 International: +1 404 975 4839 Passcode: 549600 You may also access the call via webcast by visiting the Events & Presentations section on Mirum’s website. A replay of this webcast will be available for 30 days. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI® (maralixibat) oral solution is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com. LIVMARLI has received orphan designation for ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams 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), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. 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 is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. US Prescribing Information EU SmPC Canadian Product Monograph 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. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About CHOLBAM® (cholic acid) capsules The FDA approved CHOLBAM (cholic acid) capsules in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of CHOLBAM has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy. CHOLBAM® (cholic acid) Indication CHOLBAM is a bile acid indicated for Treatment of bile acid synthesis disorders due to single enzyme defects. Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption. LIMITATIONS OF USE The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment. Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose. Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis. ADVERSE REACTIONS The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy. Please see full Prescribing Information for additional Important Safety Information. About CTEXLI™ (chenodiol) tablets CTEXLI™ (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries. IMPORTANT SAFETY INFORMATION CTEXLI can cause side effects, including: Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching. Most Common Side Effects: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection. Tell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines. US Prescribing Information About Mirum Pharmaceuticals 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 and LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It 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 other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. 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 of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). 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, significant increase or continued commercial results for our approved medicines, including continued financial growth, the potential achievement of our yearly financial guidance, continued strong execution across our approved medicines and pipeline, the results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, the timing and results of interim analyses of our ongoing studies and the regulatory approval path for our product candidates in any indication or any specific territory. 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 “expected,” “will,” “could,” “would,” “guidance,” “potential,” “continue” 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 geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 26, 2025, and subsequent filings with the Securities and Exchange Commission, which are available at www.sec.gov. 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. Mirum Pharmaceuticals, Inc. Condensed Consolidated Statement of Operations Data (in thousands, except share and per share amounts) (Unaudited) Three Months Ended March 31, 2025 2024 Revenue: Product sales, net $ 111,585 $ 68,917 License and other revenue — 305 Total revenue 111,585 69,222 Operating expenses: Cost of sales (1) 23,018 17,830 Research and development 46,044 32,222 Selling, general and administrative 57,706 45,638 Total operating expenses (2) 126,768 95,690 Loss from operations (15,183 ) (26,468 ) Other income (expense): Interest income 3,023 3,633 Interest expense (3,596 ) (3,577 ) Other income, net 2,108 1,757 Net loss before provision for income taxes (13,648 ) (24,655 ) Provision for income taxes 1,029 624 Net loss (14,677 ) (25,279 ) Net loss per share, basic and diluted $ (0.30 ) $ (0.54 ) Weighted-average shares of common stock outstanding, basic and diluted 48,889,058 46,927,550 (1) Amounts include intangible amortization expense as follows: Intangible amortization $ 5,893 $ 5,402 (2) Amounts include stock-based compensation expense as follows: Cost of sales $ 331 $ — Research and development 5,229 3,861 Selling, general and administrative 10,243 7,589 Total stock-based compensation $ 15,803 $ 11,450 Mirum Pharmaceuticals, Inc. Condensed Consolidated Balance Sheet Data (in thousands) (Unaudited) March 31, December 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 211,822 $ 222,503 Short-term investments 65,852 57,812 Accounts receivable 95,852 78,286 Inventory 22,418 22,403 Prepaid expenses and other current assets 14,874 11,784 Total current assets 410,818 392,788 Restricted cash 425 425 Long-term investments 20,912 12,526 Intangible assets, net 243,845 249,819 Other noncurrent assets 14,245 15,196 Total assets $ 690,245 $ 670,754 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 11,639 $ 14,618 Accrued expenses and other current liabilities 115,938 111,933 Total current liabilities 127,577 126,551 Operating lease liabilities, noncurrent 7,003 7,972 Convertible notes payable, net, noncurrent 308,509 308,082 Other liabilities 13,900 2,509 Total liabilities 456,989 445,114 Commitments and contingencies Stockholders’ equity: Preferred stock — — Common stock 5 5 Additional paid-in capital 892,367 870,189 Accumulated deficit (658,858 ) (644,181 ) Accumulated other comprehensive loss (258 ) (373 ) Total stockholders’ equity 233,256 225,640 Total liabilities and stockholders’ equity $ 690,245 $ 670,754 Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Erin Murphy media@mirumpharma.com

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will present data at three upcoming medical congresses. Digestive Disease Week (DDW) will be held in San Diego, May 3-6, 2025. The European Association for the Study of the Liver (EASL) will take place May 7-10 in Amsterdam, the Netherlands. The 57th European Society for Paediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting will follow May 14-17 in Helsinki, Finland. Data from Mirum’s LIVMARLI and volixibat studies will be highlighted in oral and poster presentations during the congresses. “We are excited to see the continued evidence generated by Mirum medicines and product candidates across a number of settings,” said Joanne Quan, MD, chief medical officer at Mirum. “In particular, we look forward to sharing updated data from the interim analysis of the VANTAGE study of volixibat in patients with pruritus due to PBC. We are encouraged by the positive results which show a meaningful decrease in pruritus that lasts for the duration of the placebo-controlled period.” EASL – May 7-10, 2025 Abstract #OS-059 (Oral): Volixibat for the treatment of cholestatic pruritus in primary biliary cholangitis: an adaptive, randomized, placebo-controlled phase 2B trial (VANTAGE): 28-week interim analysis Friday, May 9 from 8:30-8:45am CEST Immune-mediated and Cholestatic Diseases Session, McIntyre Room Presented by: Michael Heneghan, MD, MMedSc, FRCPI, King’s College, London, UK New 28-week data were reported from the Phase 2 VANTAGE study evaluating volixibat in patients with cholestatic pruritus related to primary biliary cholangitis (PBC). Thirty-one patients with moderate-to-severe pruritus were randomized (20mg VLX=10, 80mg VLX=10, placebo=11). Data showed that volixibat led to early and significant reductions in PBC-associated cholestatic pruritus and were maintained throughout the duration of the study. Further, 70% of volixibat-treated patients had a ≥50% reduction in serum bile acid levels from baseline. Additionally, patients treated with volixibat experienced improvements in fatigue and sleep. No new safety signals were observed through 28 weeks of treatment. The most common treatment-emergent adverse event was diarrhea which was mild to moderate in severity and transient in nature. Additional data will be shared following the formal presentation on May 9. EASL Symposium Wednesday, May 7 from 1:30-2:30pm CEST Adult Cholestatic Liver Disease: Focusing on Clinical Outcomes and the Future of IBAT Inhibitors An archive of the symposium will be available following the event. DDW Oral Presentations Presentation 784: Volixibat for the Treatment of Cholestatic Pruritus in Primary Biliary Cholangitis: An Adaptive, Randomized, Placebo-controlled Phase 2B Trial (VANTAGE): Interim Results Monday, May 5 from 2:15-2:30pm PT Cholestatic Liver Disease session, Room 6C Presented by Mitchell Schiffman, MD, Bon Secours Liver Institute, Virginia, USA Presentation 1086: Comprehensive Analysis of Cholestasis Genetic Panel Results From 2016–2022 in Children and Young Adults: Insights into Diagnostic Yield Tuesday, May 6 from 10:00-10:15am PT Human and Experimental Cholestatic and Autoimmune Liver Diseases, Room 6F Presented by Brett Hoskins, DO, Indiana University School of Medicine, Indiana, USA Poster Presentations Poster Su1756: Genetic Insights Into PFIC-associated Genes in Unexplained Chronic Cholestasis and Liver Disease: Frequency and Implications of Variant Combinations Sunday, May 4 from 12:30-1:30pm PT Pediatric Hepatology Session, DDW Poster Hall, C-E Presented by Brett Hoskins, DO, Indiana University School of Medicine, Indiana, USA Poster Mo1627: Improvements in Pruritus, Serum Bile Acids, and Total Bilirubin Following Treatment with Maralixibat in Patients with Primary Sclerosing Cholangitis Monday, May 5 from 12:30-1:30pm PT Human and Experimental Cholestatic and Autoimmune Liver Diseases, DDW Poster Hall C-E Presented by Jessica Hochberg, MD, University of Miami Miller School of Medicine, Florida, USA ESPGHAN – May 14-17, 2025 Oral presentations Abstract 1066: Improvements in pruritus are associated with growth in patients with progressive familial intrahepatic cholestasis: Data from the MARCH-ON trial Saturday, May 17 from 9:15-11:15am EEST Nutrition and Hepatology Session, Hall 3G Presented by Professor Richard Thompson, King’s College, London, UK Abstract 1089: The relationship between serum bile acids and event-free survival following the use of maralixibat for progressive familial intrahepatic cholestasis (PFIC): Data from the MARCH/MARCH-ON trials Saturday, May 17 from 1:10-2:40pm EEST Hepatology Abstracts, Hall 3G Presented by Professor Richard Thompson, King’s College, London, UK Abstract 1110: Improvements in pruritus after maralixibat treatment are associated with improved health-related quality of life for patients with cholestatic liver disease Saturday, May 17 from 1:10-2:40pm EEST Hepatology Abstracts, Hall 3G Presented by Dr. Emmanuel Gonzales, Hépatologie Pédiatrique, Hôpital Bicêtre, AP-HP. Université Paris-Saclay, Le Kremlin-Bicêtre, France Abstract 1098: Bile acid subspecies are correlated with pruritus and bilirubin improvement in PFIC patients treated with maralixibat: Data from MARCH and MARCH-ON Saturday, May 17 from 1:10-2:40pm EEST Hepatology Abstracts, Hall 3G Presented by Dr. Henkjan Verkade, University of Groningen, Beatrix Children’s Hospital and University Medical Center Groningen, Netherlands Poster presentations Abstract 1102: Greater improvements in bilirubin were observed in pruritus responders after maralixibat treatment in patients with PFIC: data from the MARCH/MARCH-ON trials Friday, May 16 from 3:30-4:20pm EST General Hepatology 02 Session, e-Poster Station 08 Presented by Professor Richard Thompson, King’s College, London, UK Abstract 1094: Impact of long-term maralixibat treatment on concomitant medication use for the treatment of cholestatic pruritus in Alagille syndrome: Real-world experience in the United States Friday, May 16 from 3:30-4:20pm EEST General Hepatology 03 & Transplantation Session, e-Poster Station 09 Presented by Dr. Tony Tokman, Mirum Pharmaceuticals, Inc., Foster City, California USA Abstract 1107: Clinical benefits of maralixibat for patients with Alagille syndrome are durable through 7 years of treatment: Data from the MERGE study Friday, May 16 from 3:30-4:20pm EEST General Hepatology Session 02, e-Poster Station 08 Presented by Dr. Emmanuel Gonzales, Hépatologie Pédiatrique, Hôpital Bicêtre, AP-HP. Université Paris-Saclay, Le Kremlin-Bicêtre, France ESPGHAN Symposium Thursday, May 15 from 12:45-1:45pm EEST Hall 3C Growing Knowledge and Evidence: Long-term impact of IBAT inhibition Full presentations will be available within the Publications and Presentations section on Mirum’s website. 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. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases, in both liquid and tablet formulations. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. U.S. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including: Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams 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), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. 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 is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. LIVMARLI is available in oral solution or tablet formulations and is taken by mouth 30 minutes before a meal. For Alagille syndrome, LIVMARLI is taken one time each day in the morning. For PFIC, LIVMARLI is taken two times each day. If you take the oral solution, use the oral dosing dispenser to measure your dose. 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/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It 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 other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. 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 of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). 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 anticipated presentations of data from Mirum’s LIVMARLI and volixibat studies, including the VANTAGE study of volixibat in patients with pruritus due to PBC. 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,” “could,” “can,” “would,” “potential,” “hope,” “opportunity,” 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 macroeconomic and geopolitical developments, and the other risks described in Mirum’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission on February 26, 2025, and subsequent filings with the U.S. Securities and Exchange Commission, which are available at www.sec.gov. 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.