Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Q4 2025 Earnings Presentation
60 slides from Mirum Pharmaceuticals's earnings presentation, Feb 25, 2026
Slide 1: Mirum Pharmaceuticals: Delivering High Impact Medicines for Rare Disease
Mirum Pharmaceuticals: Delivering High Impact Medicines for Rare Disease F e b r u a r y 2 0 2 6
Slide 2: Forward-Looking Statements
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
Slide 3: Strong Commercial Performance
Strong Commercial Performance 521 4 CAGR 91% 75 179 336 Potentially Registrational $4B+ 2022 2023 2024 2025 Topline Readouts Net Product Sales ($M)1 $630-$650M 2026 Net Product Sales Guidance Expected in Next 18 Months Peak Revenue Potential of Mirum Portfolio3 4 FDA Breakthrough Designations2 AZURE
Slide 4: Commercial Portfolio with Pipeline of Growth Opportunities
Commercial Portfolio with Pipeline of Growth Opportunities 3 APPROVED RARE DISEASE MEDICINES, 5 ADDITIONAL INDICATIONS IN DEVELOPMENT IN HIGH-NEED ORPHAN INDICATIONS Indication Preclinical Phase 1 Phase 2 and Phase 3 Approved Alagille Syndrome (ALGS)1 FDA and EMA approved Progressive Familial Intrah
Slide 5: Strong Execution Positions Mirum as a Global Rare Disease Leader
Strong Execution Positions Mirum as a Global Rare Disease Leader Global Commercial Reach Annual Net Product Sales $630-$650M $521M Hepatology & GI $336M Rare Genetic Neurology & Metabolics $179M $75M 2022 2023 2024 2025 2026E 1 Portfolio with Multi-Billion Dollar Revenue Potential2 LIVMARLI Volixiba
Slide 6: LIVMARLI
LIVMARLI® Alagille Syndrome (ALGS) Progressive Familial Intrahepatic Cholestasis (PFIC)
Slide 7: IBAT Inhibition In Cholestatic Liver Disease
IBAT Inhibition In Cholestatic Liver Disease Targeting IBAT Removes Circulating Bile Acids Addressing Toxic Bile Acid Accumulation Cholestatic Liver Disease Targeting IBAT Lowers Bile Acids IBAT Inhibition Clinical Benefits1-5 Defined by impaired bile flow & Mechanism directly addresses hepatotoxic
Slide 8: LIVMARLI: A Leading Medicine for Ultra-Rare Cholestatic Pruritus
LIVMARLI: A Leading Medicine for Ultra-Rare Cholestatic Pruritus $360M 69% YoY Growth $213M $142M $75M Available in Both Oral Solution and Tablet Formulation 2022 2023 2024 2025 Annual Net Product Sales 8
Slide 9: Expanding Impact in Ultra-Rare Cholestatic Pruritus
Expanding Impact in Ultra-Rare Cholestatic Pruritus Path to Sustained Long-Term EXPAND Growth PFIC Phase 3 Ultra-Rare Cholestasis ✓ Continued growth in new Rx 1,000+ potential US/EU ✓ Strong adherence & persistence prevalence ✓ Weight-based dosing ALGS Approved 1,000+ US/EU prevalence ✓ Int’l market
Slide 10: Bile Acid Portfolio
Bile Acid Portfolio CHOLBAM® & CTEXLI®
Slide 11: Bile Acid Replacement Therapies for Rare Genetic Diseases
Bile Acid Replacement Therapies for Rare Genetic Diseases Bile Acid Replacement Therapies Provide Bile Acids the Liver Cannot Produce Impaired Bile Acid Synthesis Bile Acid Replacement Therapies Driven by single-enzyme defects and peroxisomal (PEX) disorders 7-DHC Smith-Lemli-Optiz Syndrome (SLOS) ✓
Slide 12: Growing Business with Significant Commercial Synergies
Growing Business with Significant Commercial Synergies Bile Acid Portfolio Addresses Multiple Increased Awareness & High Need Settings Diagnosis Driving Growth $161 ® CTX Approved $123 $109 $103 BASD PBD-ZSD Approved 2022 2023 1 2024 2025 Annual Net Product Sales ($M) 1 Travere Therapeutics, Inc. an
Slide 13: Pipeline
Pipeline Significant Expansion Opportunities within Pipeline Indications
Slide 14: LIVMARLI (maralixibat)
LIVMARLI (maralixibat)
Slide 15: LIVMARLI Label Expansion Opportunity in Cholestatic Pruritus
LIVMARLI Label Expansion Opportunity in Cholestatic Pruritus Broad Unmet Need in Multiple Ultra-Rare Cholestatic Conditions Pruritus in Ultra-Rare Characteristic Estimated Addressable Cholestatic Settings Cholestatic Burden US/EU Patients e.g., Biliary Atresia, Secondary Elevated sBA Sclerosing Chol
Slide 16: EXPAND Phase 3 Study Enrolling
EXPAND Phase 3 Study Enrolling Primary Endpoint 20wk +20wks Change in pruritus from Patients with LIVMARLI 285ug/kg BID1 Baseline to 20wk Cholestatic Pruritus LIVMARLI n~45 PBO Open Label Extension Primary Endpoint Secondary Endpoints Safety & tolerability Key Inclusion Criteria Markers of disease a
Slide 17: Volixibat
Volixibat IBAT Inhibitor for Cholestasis in Adults
Slide 18: PSC and PBC: Immuno-inflammatory Rare Liver Disease
PSC and PBC: Immuno-inflammatory Rare Liver Disease Elevated Bile Acid Levels Drive Severe Symptom Burden (Pruritus, Fatigue) and Progressive Liver Disease Bile Acid Overload Obstruction of bile flow via impairment of intrahepatic and extrahepatic bile ducts Bile Acid Accumulation PSC: fibrotic stri
Slide 19: Primary Sclerosing Cholangitis
Primary Sclerosing Cholangitis
Slide 20: PSC: Pruritus is Common and Often Moderate to Severe
PSC: Pruritus is Common and Often Moderate to Severe Pruritus is a Registrational Endpoint Pruritus Is a Significant Burden No Approved Therapies; Significant Opportunity in PSC 8 PSC US Patients with Pruritus Often Moderate to Severe Median worst itch score (0-10 NRS) from last itching episode1 ~30
Slide 21: IBAT Inhibition Reduces Pruritus and Serum Bile Acids in PSC
IBAT Inhibition Reduces Pruritus and Serum Bile Acids in PSC 70 Mean (SE) ItchRO Weekly Sum Score (Max = 70) 60 Pruritus 50 40 CAMEO Study -70% 30 20 Δ -28.3 (-42.2, -14.3) P = 0.0078 IBAT inhibitor Proof of Concept in PSC 10 Data from patients with ItchRO >3 at Baseline, n=8 0 Baseline Week 14/ET S
Slide 22: Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus
Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus Positive Interim Analysis Exceeded prespecified efficacy and safety thresholds for continuation 28wk 2+ years PSC patients with moderate-to-severe pruritus VLX 20mg BID1 20 mg BID dose selected VLX Open Label PBO Extension VISTAS
Slide 23: Primary Biliary Cholangitis
Primary Biliary Cholangitis
Slide 24: PBC: Most Prevalent Cholestatic Liver Disease
PBC: Most Prevalent Cholestatic Liver Disease Significant Opportunity Across 1st & 2nd Line Settings 60% No approved therapies for pruritus controlled on UDCA 31k ~50k (1st Line) with active pruritus I found myself itching my arms so ~85k much that I had bruises on my arms... - Rose, patient with PB
Slide 25: Phase 2b Study of Volixibat in PBC Patients with Cholestatic Pruritus
Phase 2b Study of Volixibat in PBC Patients with Cholestatic Pruritus Positive Interim Analysis Rapid and statistically significant improvement in pruritus PBC patients with moderate-to-severe pruritus 28wk 2+ years Reductions in sBA and VLX 20mg BID1 VLX Open Label improvements in fatigue PBO Exten
Slide 26: VANTAGE Interim Analysis: Baseline Characteristics Well Balanced
VANTAGE Interim Analysis: Baseline Characteristics Well Balanced Volixibat BID 20mg Volixibat BID 80 mg Placebo Characteristic (n=10) (n=10) (n=11) 53.9 52.3 62.1 Age (years), mean (SD) (15.8) (5.9) (9.7) 8 9 10 Female, n (%) (80) (90) (91) 6.8 6.3 6.2 Adult ItchRO Score, mean (SD) (1.6) (1.8) (1.5)
Slide 27: VANTAGE Interim Analysis: Reduction in Pruritus from Baseline
VANTAGE Interim Analysis: Reduction in Pruritus from Baseline RAPID AND STATISTICALLY SIGNIFICANT REDUCTIONS IN PRURITUS 1 Average Pruritus Score (Adult ItchRO) Over 28 Weeks 0 PBO Adjusted CFB in Adult ItchRO1 -1 Response2: Improvement -2 -3 VLX 20mg -2.4 (P=0.0039) -4 VLX 80mg -2.6 -5 (P=0.0010) -
Slide 28: VANTAGE Interim Analysis: Other Observations
VANTAGE Interim Analysis: Other Observations • 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
Slide 29: Brelovitug
Brelovitug Chronic Hepatitis Delta Virus (HDV)
Slide 30: HDV Is the Most Severe Form of Viral Hepatitis
HDV Is the Most Severe Form of Viral Hepatitis No Approved Therapies in the US Hepatitis Delta Virus >50% Liver-Related Death in 10 Years1 ~15,000~ US pts diagnosed, insured, under care4 5yrs Avg. Progression to Cirrhosis and Liver Failure2 ~40,000 Est. US Prevalence Requires Hepatitis B coinfection
Slide 31: Brelovitug: Preliminary Efficacy and Favorable Safety Profile in HDV
Brelovitug: Preliminary Efficacy and Favorable Safety Profile in HDV Brelovitug Phase 2 Study Results High rate of virologic response and ALT normalization at 48wks1 Virologic Response Virologic response + ALT normalization (HDV RNA ≥2 log reduction or TND) Fully human anti-HBsAg monoclonal antibody
Slide 32: Ongoing Brelovitug Phase 3 Trials Supporting FDA and EMA Submissions
Ongoing Brelovitug Phase 3 Trials Supporting FDA and EMA Submissions All Studies Enrolling; No ALT Limitation for Study Participation 24wk FDA Registration AZURE 1 n=200 2:2:1 300mg 1x Weekly vs 900mg 1x Monthly vs Delayed Tx Start Extension (96wks) Delayed Tx→ 300mg 1x Weekly Enabling Studies Inter
Slide 33: MRM-3379
MRM-3379 PDE4D Inhibitor for Fragile X Syndrome (FXS)
Slide 34: Fragile X Syndrome (FXS): Rare X-Linked Genetic Disorder
Fragile X Syndrome (FXS): Rare X-Linked Genetic Disorder Mutation in the X-linked FMR1 Gene Decreases cAMP • Leading inherited form of intellectual disability and autism spectrum disorder FMRP cAMP cAMP cAMP cAMP • Symptoms more pronounced in males • Diagnosed by genetic testing ~50,000 Males in the
Slide 35: MRM-3379: Selective PDE4D inhibitor for FXS
MRM-3379: Selective PDE4D inhibitor for FXS PDE4D MRM-3379 Regulates cellular signaling by breaking down cAMP Highly expressed in brain regions critical for learning, memory, • Oral, selective PDE4D inhibitor emotional regulation • 5:1 brain/plasma ratio, potentially increasing therapeutic window •
Slide 36: MRM-3379 Was Evaluated in a Mouse Model of FXS
MRM-3379 Was Evaluated in a Mouse Model of FXS MRM-3379 improved multiple behavioral domains relevant to the FXS phenotype in a mouse model FMR1 KO2 Mouse Model of FXS Hyperactivity Phenotype Was Reversed Nesting Phenotype Was Reversed Marble Burying Phenotype Was Reversed Results support the potent
Slide 37: Phase 2 Study of MRM-3379 in Fragile X Syndrome
Phase 2 Study of MRM-3379 in Fragile X Syndrome 12wk Primary Endpoint 16-45 years, N=52 MRM-3379: 3 Dose Arms Primary and Safety & tolerability 1:1:1:1 Secondary Endpoints PBO 13 to <16 years, N=8 MRM-3379 Open Label Key Secondary Endpoint NIH-TCB Crystallized Cognition Composite (CCC) Key Inclusion
Slide 38: Mirum Pharmaceuticals
Mirum Pharmaceuticals Delivering High Impact Medicines for Rare Disease
Slide 39: Well-Positioned to Execute on Our Planned Strategy
Well-Positioned to Execute on Our Planned Strategy 4 potentially registrational topline readouts expected in the next 18 months 2026 2027 2026 FY Guidance ❑ VISTAS (PSC) topline results in Q2 ❑ VANTAGE (PBC) topline results in H1 ❑ AZURE-1 (HDV) Interim Analysis in Q2 ❑ Volixibat PSC Approval/Launch
Slide 40: Thank You
Thank You ©2026 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 sy
Slide 41: Supplemental Materials
Supplemental Materials
Slide 42: LIVMARLI Important Safety Information
LIVMARLI Important Safety Information IMPORTANT SAFETY INFORMATION LIVMARLI can cause serious 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
Slide 43: CHOLBAM Important Safety Information
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 I
Slide 44: CTEXLI Important Safety Information
CTEXLI Important Safety Information 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
Slide 45: Mirum Quarterly Net Product Sales
Mirum Quarterly Net Product Sales Net Product Sales ($M) Q1 2024 Q2 2024 Q3 2024 Q4 2024 FY 2024 Q1 2025 Q2 2025 Q3 2025 Q4 2025 FY 2025 LIVMARLI US 30.8 35.5 43.5 44.7 154.5 49.5 56.9 64.2 74.0 244.7 LIVMARLI International 12.1 11.7 15.6 19.4 58.8 23.7 * 31.2* 28.1* 32.3 115.3 LIVMARLI Total 42.8 4
Slide 46: LIVMARLI Available in Both Oral Solution and Tablet Formulation
Supplemental Material LIVMARLI Available in Both Oral Solution and Tablet Formulation Flexible Dosing Options for Patients 10mg 15mg 20mg 30mg Size for comparison One LIVMARLI Tablet Per Dose 46
Slide 47: ALGS: A Debilitating Disease with Severe Cholestasis
Supplemental Material ALGS: A Debilitating Disease with Severe Cholestasis Alagille Syndrome Genetic disease leading to severe cholestasis, unbearable pruritus and multi- system effects Lowers Serum Bile Acids 83% of patients experienced ≥20% reduction in sBA levels 88% Significantly of participants
Slide 48: ICONIC: ALGS Pivotal Study Shows Significant Long-term Benefit
Supplemental Material ICONIC: ALGS Pivotal Study Shows Significant Long-term Benefit Clinically Meaningful and Sustained Improvements in Pruritus, sBA, Growth, and QoL from Baseline.1 Reduced Pruritus 0 -0.5 84% ItchRO(Obs) -1 of participants experienced -1.5 clinically meaningful improvements -2 (≥
Slide 49: Significant Improvement in Transplant-Free Survival in Patients with ALGS Treated with LIVMARLI
Supplemental Material Significant Improvement in Transplant-Free Survival in Patients with ALGS Treated with LIVMARLI Post-hoc Analysis of Long-Term Impact: >1-point Reduction in ItchRO[Obs] Was a Predictor of Transplant-Free Survival* Transplant-Free Survival Over 6 Years of Treatment with LIVMARLI
Slide 50: PFIC: Progressive Diseases of Bile-Related Transporters
Supplemental Material PFIC: Progressive Diseases of Bile-Related Transporters PFIC (Progressive Familial Intrahepatic Cholestasis) Multiple genetic subtypes Significant Improvements in Pruritus, Serum Bile Acids, and Bilirubin Severe pruritus Stunted growth Improvements Consistent Across Multiple Su
Slide 51: MARCH PFIC: Positive Phase 3 Data Across Broad Spectrum of PFIC Types
Supplemental Material MARCH PFIC: Positive Phase 3 Data Across Broad Spectrum of PFIC Types Significant Pruritus Improvements in All-PFIC Patients LIVMARLI Placebo (n=33) (n=31) 0.0 Change from baseline in pruritus morning score Proportion of pruritus score -0.5 -0.6 assessments ≤ 1 point: (ItchRO[O
Slide 52: MARCH PFIC: Significant Improvements in Markers of Liver Disease
Supplemental Material MARCH PFIC: Significant Improvements in Markers of Liver Disease Significant Improvements in All-PFIC Patients (PFIC1, PFIC2, PFIC3, PFIC4, PFIC6) Serum Bile Acid Bilirubin1 50 4 LIVMARLI (n=33) Placebo (n=31) LIVMARLI Placebo Change from baseline in serum BA, µmol/L 3 3 Change
Slide 53: PFIC: sBA Response Associated with Transplant-Free Survival
Supplemental Material PFIC: sBA Response Associated with Transplant-Free Survival INDIGO Phase 2: 100% 5-yr Transplant Free Survival in sBA Responders* 100 sBA Responders 90 80 70 60 TFS (%) 50 40 30 20 10 Log-Rank p=0.0006 0 52 104 156 208 260 312 Time from enrolment (weeks) Loomes K et al. Hepatol
Slide 54: Well-Characterized Safety Profile of LIVMARLI
Well-Characterized Safety Profile of LIVMARLI Safety Data Most common adverse GI adverse reactions 6% of patients of LIVMARLI includes 5 events were diarrhea were generally mild or experienced dose reductions Years of follow-up from 3 and abdominal pain moderate severity or interruptions due to rand
Slide 55: Real World Evidence: IBAT Inhibition Reduces Pruritus and sBA in PSC
Supplemental Material Real World Evidence: IBAT Inhibition Reduces Pruritus and sBA in PSC LIVMARLI Compassionate Use in PSC Patients with Pruritus n=7 Pruritus Bile Acids 6 of 7 subjects with complete or near complete resolution Most patients showed reductions in sBA levels Baseline 4 Baseline 500
Slide 56: Intellectual Property Overview
Intellectual Property Overview
Slide 57: LIVMARLI IP Coverage in the United States to 2040+
LIVMARLI IP Coverage in the United States to 2040+ 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, 2037) Orange Book Listed – Patent No. 11,376,251 ALGS Orphan Designation (2030) [Pending
Slide 58: IP Coverage for Pipeline Indications in the United States
IP Coverage for Pipeline Indications in the United States Indication Composition of Matter (2027) Patent No. 7,956,085 PBC Granted Orphan Designation, 7 years from approval PBC Volixibat PSC Eligible for Orphan Designation, 7 years from approval PSC [Pending] Method of Treatment: Dosing (2032, 2040)
Slide 59: LIVMARLI IP Coverage in Europe to 2040+
LIVMARLI IP Coverage in Europe to 2040+ Indication Method of Treatment: [Approved for Grant]Dosing Method(2031, of 2040) Treatment: Dosing (2040) Orange Book Listed – Patent No. 11,229,647 / 11,497,745 / 11,918,578 / 11,260,053 Method of Orphan Treatment (2032) Designation (2034) Orange Book Listed
Slide 60: IP Coverage for Pipeline Indications in Europe
IP Coverage for Pipeline Indications in Europe Indication Composition of Matter Method of Treatment: (2027) Dosing (2031, 2040) Patent OrangeNo. 2,084,172 Book Listed – Patent No. 11,229,647 / 11,497,745 / 11,918,578 / 11,260,053 Method of Treatment (2032) PBC Granted Orphan Designation, 10 years fr
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