false000175942500017594252025-01-132025-01-13

UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
__________________________________________
FORM 8-K
__________________________________________
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): January 13, 2025
__________________________________________
Mirum Pharmaceuticals, Inc.
(Exact name of Registrant as Specified in Its Charter)
__________________________________________
Delaware001-3898183-1281555
(State or Other Jurisdiction
of Incorporation)
(Commission File Number)
(IRS Employer
Identification No.)
989 East Hillsdale Boulevard
Suite 300
Foster City, California
94404
(Address of Principal Executive Offices)(Zip Code)
Registrant’s Telephone Number, Including Area Code:650 667-4085
N/A
(Former Name or Former Address, if Changed Since Last Report)
__________________________________________
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
o   Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
o   Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
o   Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
o   Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
Title of each class
Trading
Symbol(s)
Name of each exchange on which registered
Common stock, par value $0.0001 per share
MIRM

Nasdaq Global Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐


Item 2.02 Results of Operations and Financial Condition.
On January 13, 2025, Mirum Pharmaceuticals, Inc. (the “Company”) issued a press release announcing, among other things, the Company's preliminary unaudited net product sales for the fiscal year ended December 31, 2024, preliminary unaudited net product sales of LIVMARLI (maralixibat) oral solution and CHOLBAM and CHENODAL, and preliminary unaudited cash, cash equivalents and investments. The full text of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.
Item 7.01 Regulation FD Disclosure.
On January 13, 2025, in connection with its participation in the J.P. Morgan Healthcare Conference, the Company posted a corporate slide presentation in the “Investors” portion of its website at www.mirumpharma.com. A copy of the presentation is furnished as Exhibit 99.2 to this Current Report on Form 8-K. The Company undertakes no obligation to update, supplement or amend the materials attached hereto as Exhibit 99.2.
The information in this Current Report on Form 8-K, including Exhibit 99.1 and 99.2, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that Section, nor shall it be deemed to be incorporated by reference into any filing of the Company under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d)Exhibits.
Exhibit
No.
Description
104Cover Page Interactive Data File (embedded within the Inline XBRL document)


SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
Mirum Pharmaceuticals, Inc.
Date: January 13, 2025By:/s/ Christopher Peetz
Christopher Peetz
Chief Executive Officer


Exhibit 99.1
Mirum Pharmaceuticals Announces Preliminary Unaudited 2024 Net Product Sales and Cash Balance and Provides Corporate Updates
–2024 net product sales of approximately $336 million exceeds upper end of guidance range; preliminary and unaudited estimate
–2025 expected global net product sales of $420 million to $435 million
–VISTAS study of volixibat in primary sclerosing cholangitis expected to complete enrollment in second half 2025; topline data expected 2026
FOSTER CITY, Calif. – January 13, 2025 - Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided its preliminary and unaudited estimates for full-year 2024 net product sales, year-end cash balance, corporate updates, and full-year 2025 outlook.
“2024 marked a significant year for Mirum as we accelerated our commercial business and achieved significant development milestones,” said Chris Peetz, chief executive officer of Mirum. “We continued our leadership in cholestatic disease with a label expansion for LIVMARLI and positive interim analyses for volixibat in PSC and PBC. Additionally, we advanced our rare genetic neurology efforts with the NDA submission for chenodiol and the in-licensing of MRM-3379 for Fragile X syndrome. With proven commercial execution in ultra-rare disease and a compelling pipeline in larger indications, we believe we are well-positioned for sustained growth in the years ahead.”
Future Expectations and Milestones
•2025 Guidance: expect continued revenue growth with global net product sales of approximately $420 million to $435 million and positive cash flow from operations
•Volixibat VISTAS study in primary sclerosing cholangitis (PSC) expected to complete enrollment in second half 2025; topline data expected in 2026
•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
•FDA Prescription Drug User Fee Act (PDUFA) date for chenodiol in cerebrotendinous xanthomatosis (CTX) is March 28, 2025
•Expect to initiate Phase 2 study for MRM-3379 in Fragile X Syndrome (FXS) in 2025
2024 Highlights
Commercial: Continued leadership in rare disease with a franchise in hepatology and genetic neurology
•2024 estimated LIVMARLI net product sales of approximately $213 million and CHOLBAM and CHENODAL net product sales of approximately $123 million
•Total estimated net product sales of approximately $99 million in Q4 2024 including approximately $64 million in LIVMARLI net sales and approximately $35 million in net sales from CHOLBAM and CHENODAL
•Expanded global footprint; 30 countries with commercial access, including successful reimbursement negotiation and launch in the four major European markets
Regulatory and Pipeline: Expanding Mirum's leadership across multiple rare diseases and larger orphan settings
•Positive interim results for volixibat in VISTAS PSC and VANTAGE PBC studies
•Volixibat granted breakthrough therapy designation for treatment of cholestatic pruritus in PBC by the U.S. Food and Drug Administration (FDA)
•Volixibat granted orphan drug designation for the treatment of cholestatic pruritus in PBC by the FDA
•LIVMARLI approved by the FDA for cholestatic pruritus in progressive familial intrahepatic cholestasis (PFIC) patients 12 months and older
•LIVMARLI approved in Europe for treatment of PFIC in patients three months and older
•Initiated the LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions
•New Drug Application (NDA) submitted for chenodiol in CTX
•In-licensed global rights to PDE4D inhibitor MRM-3379 for FXS


Corporate and Financial: Sustained financial strength
•Achieved positive cash flow from operations in Q3 2024
•Cash, cash equivalents and investments of approximately $287 million as of December 31, 2024 compared to $286.3 million as of December 31, 2023
•Announced the appointment of Joanne Quan, MD as Chief Medical Officer
The foregoing amounts relating to 2024 financial data are unaudited and preliminary and are subject to completion of financial closing procedures. Additional information and disclosure would be required for a more complete understanding of the company’s financial position and results of operations as of December 31, 2024.
Mirum will present at the 43rd annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 15, 2025, at 9:45 a.m. PT. The presentation and question and answer session will be webcast live and can be accessed by visiting the Investors and Media section of Mirum’s corporate website. The replay of the webcast will be available for 30 days.
About LIVMARLI® (maralixibat) oral solution
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 Breakthrough Therapy designation for ALGS and PFIC type 2 and 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 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.
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 (VISTAS study), and primary biliary cholangitis (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.
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 Cerebrotendinous Xanthomatosis
Cerebrotendinous xanthomatosis (CTX) is a rare, progressive and underdiagnosed disorder of cholesterol metabolism affecting many parts of the body. In people with CTX, the body is unable to break down cholesterol properly causing toxins (e.g., cholestanol and bile alcohols) to build up throughout the body over time. The disorder is inherited in an autosomal recessive genetic manner. Signs and symptoms of CTX include neonatal cholestasis (jaundice or bile flow interruption), chronic diarrhea, the development of bilateral cataracts before the age of 18, development of tendon xanthomas (fatty deposits in the tendons) during teenage years or later, and neurologic deterioration. The types, combinations and severity of symptoms can be different from person to person making diagnosis challenging and often delayed.
About chenodiol tablets
Chenodiol tablets 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. More recently, the US Food and Drug Administration (FDA) granted chenodiol orphan drug designation for cerebrotendinous xanthomatosis (CTX). CTX is a rare progressive disorder that can affect the brain, spinal cord, tendons, eyes and arteries. Chenodiol is not yet indicated for the treatment of CTX but has received a medical necessity determination in the U.S. by the FDA.


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, CHOLBAM® (cholic acid) capsules, and CHENODAL® (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 is also initiating 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 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 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. Chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the U.S. Lastly, Mirum is 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, commercial results for our approved products, including continued growth in year over year net product sales, being on track to achieve our revised financial guidance, our expected financial results as of December 31, 2024, including our net product sales and cash, cash equivalents and investments, delivering life changing medicines for patients suffering from rare diseases, the results, enrollment, conduct and progress of Mirum’s ongoing and planned studies for its product candidates, including newly in-licensed product candidates, the timing and results of interim analyses of our ongoing studies and the regulatory approval path for its product candidates globally, including the anticipated PDUFA date for chenodiol for CTX and additional international launches expected in 2025. 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” 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 and subsequent 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.
Contacts
Investor Contacts:
Andrew McKibben
ir@mirumpharma.com
Media Contact:
Erin Murphy
media@mirumpharma.com

J a n u a r y 2 0 2 5 Mirum Pharmaceuticals: Transforming Lives in Rare Disease Exhibit 99.2

 
Forward-Looking Statements This presentation contains "forward-looking" statements that are based on our management’s beliefs and assumptions and on information currently available to management. Forward-looking statements include all statements other than statements of historical fact contained in this presentation, including information concerning our business strategy, objectives and opportunities, including the future opportunities for LIVMARLI, CHOLBAM, chenodiol, and Mirum's clinical candidates. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements, including, but not limited to: the results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, including newly in-licensed product candidates, and our plans and expectations for commercializing LIVMARLI, CHOLBAM and chenodiol in the United States and rest of world; the costs of our business strategy, commercialization plans and development programs, the financial impact or revenues from any commercialization we undertake; estimates of the number of patients impacted by the diseases or related diseases that we seek to treat and who are appropriate for treatment with our commercial products; the potential clinical benefits of LIVMARLI, CHOLBAM and chenodiol and any of our product candidates, including volixibat and MRM-3379; our expected growth; our ability to obtain necessary regulatory approvals for our product candidates or predictions of the outcome of any regulatory consideration and, if and when approved, market acceptance of our products; our dependence on third- party clinical research organizations, manufacturers, suppliers and distributors; the design, implementation, timelines and outcomes of our clinical trials; the impact of competitive products and therapies; our ability to obtain necessary additional capital; our ability to attract and retain key employees; our ability to manage the growth and complexity of our organization; our ability to maintain, protect and enhance our intellectual property; and our ability to continue to stay in compliance with applicable laws and regulations. You should refer to the section entitled “Risk Factors” set forth in our Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and other filings we make with the Securities and Exchange Commission (SEC) from time to time (available at http://www.sec.gov) for a discussion of important factors that may cause our actual results to differ materially from those expressed or implied by our forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Neither we nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements. We undertake no obligation to update any forward-looking statements after the date of this presentation except as may be required by law. This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and growth and other data about our industry. These data involve a number of assumptions and limitations, and Mirum makes no representation to the accuracy of such estimates. Projections, assumptions and estimates of the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk. The trademarks included herein are the property of the owners thereof and are used for reference purposes only. We use our website (www.mirumpharma.com), LinkedIn page (www.linkedin.com/company/mirum-pharmaceuticals), and social media accounts (i.e.(https://twitter.com/mirumpharma) as channels of distribution of information about our company, product candidates, planned announcements, attendance at upcoming conferences and other matters. Such information may be deemed material information and we may use these channels to comply with our disclosure obligations under Regulation FD. Therefore, investors should monitor our website, LinkedIn page, and social media accounts in addition to following our SEC filings, press releases, public conference calls and webcasts. 2

 
Mirum Pharmaceuticals: High Growth Global Rare Disease Company 3 Proven Track Record of Execution and Efficiency 1 Estimate as of December 31, 2024 is preliminary and unaudited and is subject to complet ion of financial closing procedures. Additional information and disclosure would be required for a more complete understanding of the company's financial position and results of operations as of December 31, 2024 2 Mirum Pharmaceuticals Inc. 10-Q for period ending September 30, 2024 $420-435M 2025 Net Product Sales Guidance 3 Approved Ultra-Rare Medicines Advanced Pipeline In Larger Orphan Settings 5 Clinical Programs Strong Financial Position ~$287M Cash Balance1 Achieved positive cash flow from operations in Q3 20242 Founded November 2018

 
Commercial Portfolio with Pipeline of Growth Opportunities 1Received U.S. FDA approval for cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older. European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older 2 Received U.S. FDA approval for cholestatic pruritus in patients with Progressive Familial Intrahepatic Cholestasis (PFIC) 12 months of age and older. European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of progre ssive familial intrahepatic cholestas is (PFIC) in patients 3 months of age and older 3 Chenodal® is currently commercially available under medical necessity for CTX as determined by the FDA 4 Bile acid synthesis disorders include Peroxisome biogenesis disorder-Zellweger Spectrum Disorder (PBD-ZSD) and Smith Lemli Opitz Syndrome (SLOS) 4 Alagille Syndrome (ALGS)1 Progressive Familial Intrahepatic Cholestasis (PFIC)2 Cholestatic Pruritus (Additional Settings) Cerebrotendinous Xanthomatosis (CTX)3 Bile Acid Synthesis Disorders (BASD)4 volixibat Primary Sclerosing Cholangitis (PSC) Primary Biliary Cholangitis (PBC) MRM-3379 Fragile X Syndrome (FXS) VISTAS positive interim analysis, expect enrollment completion H2 2025 VANTAGE positive interim analysis, expect enrollment completion 2026 RESTORE Phase 3 positive, PDUFA date March 28, 2025 Phase 1 ApprovedPreclinical Phase 2b/Phase 3 FDA and EMA approved 3 APPROVED RARE DISEASE PRODUCTS, 5 ADDITIONAL INDICATIONS IN DEVELOPMENT IN HIGH-NEED ORPHAN INDICATIONS FDA and EMA approved FDA approved Indication Approved for radiolucent gallstones EXPAND Phase 3, expect enrollment completion 2026 Phase 2, initiating 2025 Granted FDA Breakthrough Therapy Designation

 
Strong Execution Positions Mirum as a High-Growth, Global Biotech 5 Hepatology & GI Rare Genetic Neurology & Metabolics 1 Estimate as of December 31, 2024 is preliminary and unaudited and is subject to completion of financial closing procedures. Additional information and disclosure would be required for a more complete understanding of the company's financial position and results of operations as of December 31, 2024 2 2025 Net Product Sales Guidance 3 Mirum Estimates Volixibat (PSC and PBC) $1Bn+ MRM-3379 (FXS) $1Bn+ $75M $179M ~$336M 2022 2023 2024E 2025E $420-435M 21 Annual Net Product SalesGlobal Commercial Reach Pipeline with Multi-Billion Dollar Revenue Potential3

 
Alagille Syndrome (ALGS) Progressive Familial Intrahepatic Cholestasis (PFIC) LIVMARLI®

 
IBAT Inhibition In Cholestatic Liver Disease 7 Targeting IBAT Removes Circulating Bile Acids Addressing Toxic Bile Acid Accumulation Pruritus Bilirubin (PFIC) Xanthomas (ALGS) Transplant-free survival Quality of life Growth 1. Gonzales E et al. Lancet. 2021;398:1581-1592. 2. Loomes KM et al. Hepatol Commun. 2022;6(9):2379-2390. 3. Thompson R. Serum bile acid control in long-term maralixibat-treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency. Presented at: EASL 2020; August 2020. Accessed Apr il 29, 2021. https://linkinghub.elsevier.com/retrieve/pii/S0168827820307571 4. van Wessel DBE et al. J Hepatol. 2021;73(1):84-93. 5. Sokol J, Gonzales E, Kamath BM, et al. Predictors of 6-year event-free survival in patients with Alagille syndrome treated with maralixibat, an IBAT inhibitor. Paper presented at: European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN): Annual Meeting; June 22-25, 2022; Copenhagen, Denmark. Inhibits reuptake by IBAT & prevents recirculation Increases fecal bile acid excretion Cholestatic Liver Disease Defined by impaired bile flow & hepatotoxic build-up of bile acids Targeting IBAT Lowers Bile Acids Mechanism directly addresses bile acid accumulation IBAT Inhibition Clinical Benefits1-5 Severe pruritus Cellular damage Poor outcomes

 
LIVMARLI: A Leading Medicine for Ultra Rare Cholestatic Pruritus 8 ALGS PFIC EXPAND Approved 4,000-5,500 US/EU prevalence ~200/yr annual incidence Approved 1,000+ US/EU prevalence ~70/yr annual incidence Phase 3 Pruritus in Rare Cholestatic Settings 1,000+ US/EU prevalence Sustained Long Term Growth ✓ Continued growth in new Rx ✓ Strong adherence & persistence ✓ Weight-based dosing ✓ Int’l market expansion ✓ IP Protection to 2040+

 
Well-Characterized Safety Profile of LIVMARLI 9 Safety Data of LIVMARLI includes 5 Years of follow-up from 3 randomized studies in ALGS, and 93-patient randomized MARCH study in PFIC Most common adverse events were diarrhea and abdominal pain (ALGS: 41.6 and 38.6 events per 100 person-years, respectively; PFIC: 57.4% vs 19.6% pbo, 27.7% vs 15.2% pbo, respectively) GI adverse reactions were generally mild or moderate severity and self-limiting 6% of patients experienced dose reductions or interruptions due to diarrhea, abdominal pain (ALGS, PFIC) LIVMARLI can cause serious side effects, including liver injury. Changes in certain liver tests are common in patients but may worsen during treatment and should therefore be monitored prior to and during treatment. These changes may be a sign of liver injury and, in PFIC, can be serious or may lead to transplant or death.

 
CHOLBAM & chenodiol Bile Acid Portfolio

 
Bile Acid Replacement Therapies for Rare Genetic Diseases 11 Impaired Bile Acid Synthesis Driven by single-enzyme defects and peroxisomal (PEX) disorders Bile Acid Replacement Therapies Provide Bile Acids the Liver Cannot Produce Peroxisome biogenesis disorder- Zellweger Spectrum Disorder (PBD-ZSD) Smith-Lemli-Optiz Syndrome (SLOS) Deficiency of bile acids CDCA, CA Accumulation of toxic intermediates • Progressive liver disease • Impairment of organ function • Irreparable neurological damage • Significant morbidity 7-DHC 5β-RD CYP27A1 Cholesterol Defects in genes and enzymes involved in production of essential bile acids PEX Bile Acid Replacement Therapies ✓ Restoration of bile acid homeostasis ✓ Reduction of toxic intermediates ✓ Improvement and prevention of adverse clinical manifestations Bile Acid Synthesis Disorders (BASD) Cerebrotendinous Xanthomatosis (CTX) CDCA: chenodeoxycholic acid, CA: Cholic Acid

 
Growing Business with Strong Commercial Synergies 12 Bile Acid Portfolio Addresses Multiple High Need Settings1 1 Appadurai et al. Molecular Genetics and Metabolism 2015; Cross JL et al. Clin Genet. 2015; PBD-ZSD: Klouwer, et al. Orphanet J Rare Dis 2015.; Orpha.net Disorder of bile acid synthesis 2011 2 Travere Therapeutics, Inc. and Mirum Pharmaceuticals, Inc. 10-K filings; 2023 net product sales excludes an approximate ~$5M reserve recorded by Travere Therapeutics, Inc. for potential repayment obligations attributed to 2015-2020 net product sales in France 3Estimate as of December 31, 2024 is preliminary and unaudited and is subject to completion of financial closing procedures. Additional information and disclosure would be required for a more complete understanding of the company's financial position and results of operations as of December 31, 2024 *Chenodal® is currently commercially available under medical necessity for CTX as determined by the FDA $89 $96 $103 $109 ~$123 2020 2021 2022 2023 2024 Net Product Sales ($M) Consistent Year-over-Year Growth 1,000-2,000 US prevalence Est. only ~10% diagnosed ~200-300 US prevalence 1,000-2,000 US prevalence CTX PDUFA Date Mar 28, 2025 FDA Medical Necessity Status BASD (incl. PBD-ZSD) Approved SLOS Approved 2 3 *

 
Significant Expansion Opportunities within Pipeline Indications Pipeline

 
LIVMARLI

 
LIVMARLI Label Expansion Opportunity in Cholestatic Pruritus 15 Broad Unmet Need in Multiple Rare Cholestatic Conditions 1 Mirum Market Research Pruritus in Rare Cholestatic Settings Elevated sBA Severe pruritus Stunted growth Impaired QoL Characteristic Cholestatic Burden Estimated Addressable US/EU Patients 1,000+ with cholestatic pruritus1 LIVMARLI Is Uniquely Positioned to Address the Burden of Cholestatic Pruritus e.g. Biliary Atresia, Secondary Sclerosing Cholangitis, Others

 
EXPAND Phase 3 Study Enrolling 16 Primary Endpoint Change in pruritus from Baseline to 20wk Secondary Endpoints Safety & tolerability Markers of disease and QoL +20wks Key Inclusion Criteria • Diagnosis of cholestatic liver disease excluding ALGS, PFIC, PSC, PBC, and ICP • Moderate to severe refractory cholestatic pruritus • Total sBA >2× ULN Primary Endpoint LIVMARLI 285ug/kg BID1 LIVMARLI Open Label Extension 20wkDay 1 1 LIVMARLI 285ug/kg is equivalent to 300 ug/kg maralixibat chloride. BID, twice daily PBO Patients with Cholestatic Pruritus n~45 Enrollment Completion Expected 2026

 
IBAT Inhibitor for Cholestasis in Adults Volixibat

 
PSC & PBC: Immuno-inflammatory Rare Liver Disease Bile Acid Overload Obstruction of bile flow via impairment of intrahepatic and extrahepatic bile ducts Elevated Bile Acid Levels Drive Severe Symptom Burden (Pruritus, Fatigue) and Progressive Liver Disease Bile Acid Accumulation Associated with: • Severe symptomatic burden • Reduced bile acid synthesis • Inflammation and fibrosis of bile ducts and liver • Progressive liver damage PSC: fibrotic strictures of bile ducts 54,000 patients US/EU 65% of patients with active pruritus PBC: inflammatory driven cholestasis 230,000 patients US/EU 60% of patients with active pruritus IBAT inhibition Reduces Pruritus and sBA in PSC & PBC 18

 
Primary Sclerosing Cholangitis

 
PSC: Pruritus is Common and Often Moderate to Severe 20 Pruritus Is a Significant Burden Median worst itch score (0-10 NRS) from last itching episode1 8 1 Kowdley KV, et al. Presented at EASL 2022. Survey conducted in 482 patients with PSC; not all patients responded to all questions 2 Mirum Market Research No Approved Therapies; Significant Opportunity in PSC ~30k PSC US Patients with Pruritus Often Moderate to Severe ~20k PSC Patients in the US2 65% with active pruritus Pruritus is a Registrational Endpoint It is…like your blood is itchy. The bile is in your blood…you can't reach the itch. - Nicola, patient with PSC1 This debilitating itch is merciless, all consuming, and overwhelming. - Kristina, patient with PSC1

 
IBAT Inhibition Reduces Pruritus and Serum Bile Acids in PSC 21 CAMEO Study: Open label maralixibat 10mg QD for 14wks, N=27 ItchRO: 0-10 Numerical Rating Scale (0=No Itch, 10=Worst Itch Imaginable) All values are mean (95% CI) Volixibat: Highly Active on Bile Acid Pathway; 48-Week Safety Data in Prior Studies 0 10 20 30 40 50 60 70 CAMEO Study IBAT inhibitor Proof of Concept in PSC Data from patients with ItchRO >3 at Baseline, n=8 Baseline Week 14/ET Pruritus -70% Mean (SE) ItchRo Weekly Sum Score (Max = 70) 0 20 40 60 80 100 Bile Acids -40% Mean (SE) sBA levels (umol/L) Baseline Week 14/ET Δ -28.3 (-42.2, -14.3) P = 0.0078 Δ -32.06 (−75.0, 10.9) P = 0.078 Significant Reductions in Pruritus and Bile Acids

 
Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus 221 Participants are randomized 1:1 between Volixibat 20mg and Placebo. BID, twice daily 2+ years Confirmatory Analysis (n = ~120) VLX 20mg BID1 VLX Open Label Extension 28wk PBO PSC patients with moderate-to-severe pruritus Enrollment Completion Expected H2 2025 Primary Endpoint Change in pruritus from baseline to 28wk Exceeded prespecified efficacy and safety thresholds for continuation 20 mg BID dose selected VISTAS continues with no changes Positive Interim Analysis

 
Primary Biliary Cholangitis

 
PBC: Most Prevalent Cholestatic Liver Disease 24 ~85k 20k 31k 60% controlled on UDCA (1st Line) 40% uncontrolled on UDCA (2nd Line) PBC Patients with Pruritus in the US Often Moderate to Severe No approved therapies for pruritus with active pruritus with active pruritus Diagnosed PBC Patients in the US2 Significant Opportunity Across 1st & 2nd Line Settings 1PBCers Organization. PBCers Stories. Retrieved from website https://pbcers.org/stories/. Accessed October 23, 2024. 2Mirum Market Research ~50kI found myself itching my arms so much that I had bruises on my arms... - Rose, patient with PBC1 I began itching all over…It was unlike any itch I ever experienced. I scratched so much, my skin was raw. - Donna, patient with PBC1 Similar rate of pruritus in 1st & 2nd line (~60%)

 
Phase 2b Study of Volixibat in PBC Patients with Cholestatic Pruritus 251 Participants are randomized 1:1 between Volixibat 20mg and Placebo. BID, twice daily 2+ years Confirmatory Analysis (n = up to 200) VLX 20mg BID1 VLX Open Label Extension 28wk PBO PBC patients with moderate-to-severe pruritus Enrollment Completion Expected 2026 Primary Endpoint Change in pruritus from baseline to 28wk Positive Interim Analysis Rapid and statistically significant improvement in pruritus Reductions in sBA and improvements in fatigue 20 mg BID dose selected Granted FDA Breakthrough Therapy Designation

 
VANTAGE Interim Analysis: Baseline Characteristics Well Balanced 26 Characteristic Volixibat BID 20mg (n=10) Volixibat BID 80 mg (n=10) Placebo (n=11) Age (years), mean (SD) 53.9 (15.8) 52.3 (5.9) 62.1 (9.7) Female, n (%) 8 (80) 9 (90) 10 (91) Adult ItchRO Score, mean (SD) 6.8 (1.6) 6.3 (1.8) 6.2 (1.5) sBA in umol/L, mean (SD) 53 (53) 44 (73) 31 (52) ALP (U/L), mean (SD) 238 (134) 232 (107) 167 (114) Kowdley et al, AASLD 2024 Adult ItchRO is a 0-10 worst-itch numerical rating scale completed once daily

 
VANTAGE Interim Analysis: Reduction in Pruritus from Baseline 27Kowdley et al, AASLD 2024 1LS mean (95% CI) change from Baseline to the average of the last 12 weeks of treatment. LS means and P values were calculated using an MMRM model. Placebo (n=11)VLX 80 mg BID (n=10)VLX 20 mg BID (n=10) Week -5 -4 -3 -2 -1 0 C h an ge f ro m B as el in e BL 16 RAPID AND STATISTICALLY SIGNIFICANT REDUCTIONS IN PRURITUS -2.4 (P=0.0041) -2.6 (P=0.0011) PBO Adjusted Response1: VLX 20mg VLX 80mg Average Pruritus Score (Adult ItchRO) Over 16 Weeks

 
VANTAGE Interim Analysis: Other Observations 28 • Significant reduction in pruritus as early as Week 1 • Significant improvements in fatigue at Week 16 • 70% of patients on volixibat achieved >50% reduction in sBA • No new safety signals: – No clinically meaningful changes in liver laboratory tests for patients on volixibat – 77% of patients on volixibat experienced diarrhea which was mild in severity and led to 1 discontinuation – 3 patients experienced serious TEAEs, including one in the placebo arm; none related to study drug Kowdley et al, AASLD 2024

 
MRM-3379 PDE4D Inhibitor for FXS

 
Fragile X Syndrome(FXS): Rare X-Linked Genetic Disorder 30 Mutation in the X-linked FMR1 Gene Decreases cAMP • Leading inherited form of intellectual disability and autism spectrum disorder • Symptoms more pronounced in males • Diagnosed by genetic testing No Approved Therapies for FXS FMRP cAMP cAM P cAM P cAM P Impaired Cognition, Learning and Behavior ~50,000 Males in the US/EU with FXS ~2/3 with full mutation of FMR1 gene1 1Hunter et al.– American Journal of Medical Genetics 2014

 
MRM-3379: Selective PDE4D inhibitor for FXS 31 In FXS patients, PDE4D inhibition improves cognition and daily function1 PDE4D Regulates cellular signaling by breaking down cAMP cAMP levels Highly expressed in brain regions critical for learning, memory, emotional regulation PDE4-D Inhibition FXS • Oral, selective PDE4D inhibitor • 5:1 brain/plasma ratio, potentially increasing therapeutic window • Efficacy in preclinical models of memory • Well tolerated in SAD and MAD clinical trials MRM-3379 Initiating Phase 2 Dose Ranging Study in 2025 1 Kravis et al., Nature Medicine 2021

 
Dedicated to Transforming the Treatment of Rare Diseases Mirum Pharmaceuticals

 
Well-Positioned to Execute on Our Strategy 33 1 Estimate as of December 31, 2024 is preliminary and unaudited and is subject to completion of financial closing procedures. Additional information and disclosure would be required for a more complete understanding of the company's financial position and results of operations as of December 31, 2024 ❑ CTX PDUFA Mar 28, 2025 ❑ VISTAS (PSC) complete enrollment in H2 ❑ Initiating MRM-3379 P2 study in FXS ❑ VANTAGE (PBC) complete enrollment ❑ EXPAND complete enrollment ❑ VISTAS (PSC) topline 2025 2026 2025 FY Guidance: • $420-435M Net Product Sales Guidance • Positive Cash Flow from Operations ~$287M Cash, Cash Equivalents and Investments1 Multiple Value-Creating Anticipated Milestones

 
©2025 Mirum Pharmaceuticals, Inc. All rights reserved. All service marks, trademarks and tradenames appearing in this presentation are the property of their respective owners. Solely for convenience, the trademarks and tradenames referred to in this presentation appear without the ® and symbols, but those references are not in tended to ind icate, in any way, that we will not asser t, to the fu llest extent under applicable law, our r ights, or the right of the applicable licensor to these trademarks and tradenames. The information here in is for in formational purposes only and represents the current view of Mirum Pharmaceuticals, Inc. as of the date of th is presentation (or as of an ear lier date if specifically noted). Thank You

 
Supplemental Materials

 
LIVMARLI (maralixibat) Oral Solution Important Safety Information 36 IMPORTANT SAFETY INFORMATION LIVMARLI can cause serious side effects, including: Liver injury: Changes in certain liver tests are common in patients but may 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 and physical exams before starting and during treatment to check your liver function. Stomach and intestinal (gastrointestinal) problems: LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain during treatment. Fat Soluble Vitamin Deficiency: This vitamin deficiency is common in patients with Alagille syndrome but may worsen during treatment.

 
CHOLBAM Important Safety Information 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. 37

 
ALGS: A Debilitating Disease with Severe Cholestasis 38Kamath BM et al. J Pediatr Gastroenterol Nutri. 2018, Kamath BM et al, Liver Transpl 2012, Vandriel SM, et al. EASL 2020 (oral presentation), Gonzales E et al. Lancet. 2021;398:1581-1592. *Post Hoc Analysis, included data from 3 long-term studies (N=76); Transplant-free survival was defined as time to liver transplant or death; Sokol J, Gonzales E, Kamath BM, et al. ESPGHAN: Annual Meeting 2022 Genetic disease leading to severe cholestasis, unbearable pruritus and multi- system effects 88% Affected by cholestatic pruritus 6 in 10 Progress to transplant or death by adulthood Pruritus Reduction Leads to Improved Transplant-Free Survival Lowers Serum Bile Acids Alagille Syndrome Significantly Reduces Pruritus 84% 83% of patients with a >1-point reduction in ItchRO[Obs] remained transplant-free 6 years after starting LIVMARLI* of patients experienced ≥20% reduction in sBA levels of participants experienced ≥1 point reduction in ItchRO[Obs] in cholestatic pruritus 93%

 
ICONIC: ALGS Pivotal Study Shows Significant Long-term Benefit 1 Gonzales E et al. Lancet. 2021;398:1581-1592. -100 -80 -60 -40 -20 0 Week 18 Week 48 Se ru m b ile a ci d ( μ m ol /L ) Reduced sBA -2.5 -2 -1.5 -1 -0.5 0 Week 18 Week 48 It ch RO (O bs ) Reduced Pruritus 84% of participants experienced clinically meaningful improvements (≥1 point reduction in ItchRO[Obs]) in cholestatic pruritus LIVMARLI also improved other key symptoms of ALGS including growth, quality of life, and fatigue. Clinically Meaningful and Sustained Improvements in Pruritus, sBA, Growth, and QoL from Baseline.1 83% of patients experienced ≥20% reduction in sBA levels View data published in The Lancet Supplemental Material 39

 
Significant Improvement in Transplant-Free Survival in Patients with ALGS Treated with LIVMARLI *Transplant-free survival was defined as time to liver transplant or death; post-hoc analysis included data from 3 long-term studies (N=76) Sokol J, Gonzales E, Kamath BM, et al. Predictors of 6-year event-free survival in patients with Alagille syndrome treated with maralixibat, an IBAT inhibitor. Paper presented at: European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN): Annual Meeting; June 22-25, 2022; Copenhagen, Denmark. 40 Transplant-Free Survival Over 6 Years of Treatment with LIVMARLI* Post-hoc Analysis of Long-Term Impact: >1-point Reduction in ItchRO[Obs] Was a Predictor of Transplant-Free Survival* of patients remained transplant-free 6 years after starting LIVMARLI of patients who had ≤1-point reduction in ItchRO(Obs) (n=30) remained transplant-free 6 years after starting LIVMARLI Supplemental Material

 
PFIC: Progressive Diseases of Bile-Related Transporters 41 ~80% Require liver transplant by 18yrs of age PFIC (Progressive Familial Intrahepatic Cholestasis) Significant Improvements in Pruritus, Serum Bile Acids, and Bilirubin 62% With Minimal to No Itch (Proportion of pruritus score assessments ≤1 after 26wks of treatment)** Severe pruritus Stunted growth Impaired QoL Improvements Consistent Across Multiple Subtypes (PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status)* Multiple genetic subtypes Karpen et al, JPGN 2021; Englert et al, Transplantation 2007;84: 1361–1363; Thompson, et al. Oral Presentation, AASLD 2022 *LIVMARLI is not recommended in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in non-functional or complete absence of bile salt export pump (e.g. BSEP-3 variant which accounts for approximately 21% of PFIC type 2 patients) ** Proportion of pruritus score assessments recorded as a 0 or 1 on the 0-4 ItchRo[Obs]

 
-1.8 -0.6 -2.0 -1.5 -1.0 -0.5 0.0 MARCH PFIC: Positive Phase 3 Data Across Broad Spectrum of PFIC Types Data are LS Mean with standard error bars. Effect size compared the difference between LIVMARLI and placebo, averaged over the last 3 time periods using a repeated measures mixed effect model. * LIVMARLI LS Mean = Placebo LS Mean; #LS Mean Delta with 95% CI Thompson, et al. Oral Presentation, AASLD 2022 42 LIVMARLI (n=33) Placebo (n=31) C h a n ge f ro m b a se lin e in p ru ri tu s m o rn in g sc o re (I tc h R O [O bs ]) Δ: -1.17 (-1.705, -0.642)# p < 0.0001* Proportion of pruritus score assessments ≤ 1 point: 62% LIVMARLI vs 28% placebo (p<0.0001) Significant Pruritus Improvements in All-PFIC Patients Supplemental Material

 
-157 -200 -150 -100 -50 0 50 MARCH PFIC: Significant Improvements in Markers of Liver Disease Data are LS Mean with standard error bars. Effect size compared the difference between LIVMARLI and placebo, averaged over the last 3 time periods using a repeated measures mixed effect model. * LIVMARLI LS Mean = Placebo LS Mean; #LS Mean Delta with 95% CI; ## Data are mean with standard error bars Thompson, et al. Oral Presentation, AASLD 2022 1 Bilirubin was not a prespecified primary or secondary endpoint that was in hierarchical order 43 3 C h a n ge f ro m b as el in e in s er u m B A , µ m o l/ L LIVMARLI (n=33) Placebo (n=31) -4 -3 -2 -1 0 1 2 3 4 0 2 4 6 8 10 12 14 16 18 20 22 24 26 Study Week LIVMARLI Placebo C h a n ge f ro m B as e li n e , (m g/ d L) # # Δ: -160 µmol/L (-220.84, -99.97)# p < 0.0001* Bilirubin1Serum Bile Acid Significant Improvements in All-PFIC Patients (PFIC1, PFIC2, PFIC3, PFIC4, PFIC6) Δ: -2.0 (-3.98, -0.03)# p=0.0471* Supplemental Material

 
PFIC: sBA Response Associated with Transplant-Free Survival Loomes K et al. Hepatol Commun. 2022;6:2379-2390; n=19 (7 sBA responders, 12 sBA non-responders) *NAPPED criteria (van Wessel et al, 2021): sBA responders defined as having an average sBA of <102 μmol/L (if baseline sBA ≥102 μmol/L), OR a ≤-75% average percent change from baseline 44 Time from enrolment (weeks) T FS (% ) 100 60 40 20 80 10 30 50 70 90 0 52 208 260 312104 156 Log-Rank p=0.0006 sBA Responders INDIGO Phase 2: 100% 5-yr Transplant Free Survival in sBA Responders* Supplemental Material

 
RESTORE Phase 3: Potentially NDA enabling for CTX, Orphan Exclusivity 45 RESTORE Phase 3 Double-blind placebo controlled cross-over withdrawal study Primary Endpoint • Change from baseline in urine total 23S-pentol (bile alcohol) Secondary Endpoints • Change from baseline in plasma 7αC4 • Change from baseline in plasma cholestanol • Proportion of patients requiring rescue medication All endpoints assessed at the end of double-blind periods Follow-up (30 days) 8wks 4wks* PBO 4wks* Open Label chenodiol 8wks N=13 chenodiol chenodiol PBOOpen Label chenodiol Open Label chenodiol Open Label chenodiol Elevated Levels of Cholestanol Drive Symptomatic Burden and Progression in CTX chenodiol = CDCA *Rescue chenodiol available as needed if symptoms occur or lab values are out of the expected range for patients receiving chenodiol or PBO Double-Blind Baseline defined as the last measurement prior to dosing on Day 1 for Double-Blind period 1 and Day 85 for Double-Blind period 2 THE FIRST AND ONLY PHASE 3 PLACEBO-CONTROLLED STUDY IN CTX Supplemental Material

 
RESTORE Phase 3 study: All Primary and Secondary Endpoints Met 46 Data Mean Difference with 95% CI P-values from a paired t-test comparing chenodiol with placebo is performed on the change from double-blind (DB) baseline (BL) to last non-missing post-BL DB record on natural log-transformed scale Adverse Events • The most commonly reported adverse events while on chenodiol were diarrhea (n=5) and headache (n=3) • The majority of adverse events reported were mild or moderate in severity and not considered to be treatment-related Urine 23S-Pentol (bile alcohol) ng/mL p<0.0001 20-fold increase after withdrawal (CI: 10.3, 43.5) Plasma 7αC4 ng/mL p<0.0001 50-fold increase after withdrawal (CI: 25.0, 66.7) Plasma Cholestanol ug/mL p=0.0083 2.8-fold increase after withdrawal (CI: 1.5, 5.2) % PBO pts requiring rescue chenodiol during double blind withdrawal periods p=0.0006 61.5% (CI: 31.6%, 86.1%) Clinically Meaningful Improvements in Primary and Secondary Endpoints; PDUFA Mar 28th, 2025 Difference at end of DB period (placebo relative to chenodiol) (95% CI) Primary Endpoint Key Secondary Endpoints Supplemental Material

 
Intellectual Property Overview

 
ALGS PFIC LIVMARLI IP Coverage in the United States to 2040+ 48 Method of Treatment: Dosing (2031, 2040) Orange Book Listed – Patent No. 11,229,647 / 11,497,745 / 11,918,578 / 11,260,053 Method of Treatment (2032, 2037) Orange Book Listed – Patent No. 11,376,251 [Pending] Method of Treatment: Dosing (2040, 2043) Method of Treatment (2032) Orange Book Listed – Patent No. 10,512,657 / 11,229,661 Orphan Designation (2030) [Pending] Formulation, Manufacturing, Additional Dosing (2042, 2043) [Pending] Formulation, Manufacturing, Additional Dosing (2042) Orphan Designation (2031) Indication

 
Volixibat PBC PSC CTX MRM-3379 FXS IP Coverage for Pipeline Indications in the United States 491Assumes standard patent term extension [Pending] Additional Dosing (2042) [Pending] Method of Treatment: Dosing (2032, 2040) Orphan Designation, 7 years from approval Composition of Matter (2027) Patent No. 7,956,085 PBC Granted Orphan Designation, 7 years from approval Indication Composition of Matter (2039)1 Patent No. 9,120,770 PSC Eligible for Orphan Designation, 7 years from approval

 
LIVMARLI IP Coverage in the Europe to 2040+ 50 Indication Method of Treatment: Dosing (2031, 2040) Orange Book Listed – Patent No. 11,229,647 / 11,497,745 / 11,918,578 / 11,260,053 Method of Treatment (2032) Orange Book Listed – Patent No. 11,376,251 Method of Treatment (2032) Orange Book Listed – Patent No. 10,512,657 / 11,229,661 Orphan Designation (2030) [Pending] Formulation, Manufacturing, Additional Dosing (2042, 2043) ALGS PFIC [Pending] Formulation, Manufacturing, Additional Dosing (2042) [Approved for Grant] Method of Treatment: Dosing (2040) Method of Treatment (2032, Spain and France 2037) Patent No. 2,771,003 [Approved for Grant] Method of Treatment: Dosing (2040) Method of Treatment (2032) Patent No. 2,771,003 [Pending] Formulation, Manufacturing, Additional Dosing (2042) Orphan Designation (2034) Orphan Designation (2034) 1. Orphan designation out to 2034 for ALGS 2. CHMP positive opinion and COMP favorable opinion for PFIC 1 2

 
IP Coverage for Pipeline Indications in Europe 51 Method of Treatment: Dosing (2031, 2040) Orange Book Listed – Patent No. 11,229,647 / 11,497,745 / 11,918,578 / 11,260,053 Method of Treatment (2032) Orange Book Listed – Patent No. 11,376,251 [Pending] Method of Treatment: Dosing (2040, 2043) Orphan Designation (2030) [Pending] Formulation, Manufacturing, Additional Dosing (2042) Volixibat PBC PSC [Pending] Method of Treatment: Dosing (2040) Composition of Matter (2027) Patent No. 2,084,172 [Pending] Formulation, Manufacturing, Additional Dosing (2042) PSC Eligible for Orphan Designation, 10 years from approval Indication 1Volixibat received a positive opinion on orphan drug designation for the treatment of PBC from EMA Committee for Orphan Medicinal Products (COMP) PBC Eligible for Orphan Designation, 10 years from approval1

 
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Jan. 13, 2025
Cover [Abstract]  
Document Type 8-K
Document Period End Date Jan. 13, 2025
Entity Registrant Name Mirum Pharmaceuticals, Inc.
Entity Incorporation, State or Country Code DE
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