Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Q2 2025 Earnings Presentation
53 slides from Mirum Pharmaceuticals's earnings presentation, Aug 6, 2025
Slide 1: Mirum Pharmaceuticals: Transforming Lives in Rare Disease
Mirum Pharmaceuticals: Transforming Lives in Rare Disease A u g u s t 2 0 2 5
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: Mirum Pharmaceuticals: High Growth Global Rare Disease Company
Mirum Pharmaceuticals: High Growth Global Rare Disease Company 3 Approved Advancing a Pipeline In Strong Ultra-Rare Medicines Larger Orphan Settings Financial Position $490-510M 4 $322M 2025 Net Product Sales Guidance Clinical Programs Cash Balance1 Founded November 2018 Expect Positive Cash Flow in
Slide 4: Commercial Portfolio with Pipeline of Growth Opportunities
Commercial Portfolio with Pipeline of Growth Opportunities 3 APPROVED RARE DISEASE PRODUCTS, 4 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 FDA and
Slide 5: Strong Execution Positions Mirum as a High-Growth, Global Biotech
Strong Execution Positions Mirum as a High-Growth, Global Biotech Global Commercial Reach Annual Net Product Sales $490-510M $336M Hepatology & GI Rare Genetic $179M Neurology & Metabolics $75M 2022 2023 2024 2025E 1 Portfolio with Multi-Billion Dollar Revenue Potential2 LIVMARLI Volixibat MRM-3379
Slide 6: LIVMARLI
LIVMARLI® Alagille Syndrome (ALGS) Progressive Familial Intrahepatic Cholestasis (PFIC)
Slide 7: IBAT Inhibition In Cholestatic Liver Disease
IBAT Inhibition In Cholestatic Liver Disease Targeting IBAT Removes Circulating Bile Acids Addressing Toxic Bile Acid Accumulation Cholestatic Liver Disease Targeting IBAT Lowers Bile Acids IBAT Inhibition Clinical Benefits1-5 Defined by impaired bile flow & Mechanism directly addresses hepatotoxic
Slide 8: LIVMARLI: A Leading Medicine for Ultra Rare Cholestatic Pruritus
LIVMARLI: A Leading Medicine for Ultra Rare Cholestatic Pruritus Path to Sustained Long Term EXPAND Growth PFIC Phase 3 Ultra Rare Cholestasis ✓ Continued growth in new Rx 1,000+ potential US/EU ✓ Strong adherence & persistence prevalence ✓ Weight-based dosing ALGS Approved 1,000+ US/EU prevalence ✓
Slide 9: Well-Characterized Safety Profile of LIVMARLI
Well-Characterized Safety Profile of LIVMARLI Safety Data Most common adverse GI adverse reactions 6% of patients of LIVMARLI includes 5 events were diarrhea were generally mild or experienced dose reductions Years of follow-up from 3 and abdominal pain moderate severity or interruptions due to rand
Slide 10: Bile Acid Portfolio
Bile Acid Portfolio CHOLBAM & CTEXLI
Slide 11: Bile Acid Replacement Therapies for Rare Genetic Diseases
Bile Acid Replacement Therapies for Rare Genetic Diseases Bile Acid Replacement Therapies Provide Bile Acids the Liver Cannot Produce Impaired Bile Acid Synthesis Bile Acid Replacement Therapies Driven by single-enzyme defects and peroxisomal (PEX) disorders 7-DHC Smith-Lemli-Optiz Syndrome (SLOS) ✓
Slide 12: Growing Business with Significant Commercial Synergies
Growing Business with Significant Commercial Synergies Bile Acid Portfolio Addresses Multiple Consistent Year-over-Year Growth High Need Settings Net Product Sales ($M) $123 CTX $109 Approved $103 BASD PBD-ZSD Approved 2022 2023 1 2024 1 Travere Therapeutics, Inc. and Mirum Pharmaceuticals, Inc. 10-
Slide 13: Pipeline
Pipeline Significant Expansion Opportunities within Pipeline Indications
Slide 14: LIVMARLI
LIVMARLI
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 Chola
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 & PBC: Immuno-inflammatory Rare Liver Disease
PSC & 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 strict
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: MRM-3379
MRM-3379 PDE4D Inhibitor for FXS
Slide 30: 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 31: 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 32: Phase 2 Study of MRM-3379 in Fragile X Syndrome
Phase 2 Study of MRM-3379 in Fragile X Syndrome Phase 2 Study Design 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 Composit
Slide 33: Mirum Pharmaceuticals
Mirum Pharmaceuticals Dedicated to Transforming the Treatment of Rare Diseases
Slide 34: Well-Positioned to Execute on Our Strategy
Well-Positioned to Execute on Our Strategy Multiple Value-Creating Anticipated Milestones 2025 2026 2027 2025 FY Guidance: ❑ CTEXLI FDA approved ❑ VISTAS (PSC) topline results in Q2 ❑ EXPAND topline results in H1 • $490-510M ❑ LIVMARLI tablet approved ❑ EXPAND complete enrollment ❑ VANTAGE (PBC) top
Slide 35: Thank You
Thank You ©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 sy
Slide 36: Supplemental Materials
Supplemental Materials
Slide 37: 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 38: 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 39: 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 40: LIVMARLI Available in Both Oral Solution and Tablet Formulation
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 40
Slide 41: ALGS: A Debilitating Disease with Severe Cholestasis
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 experienced ≥1 point
Slide 42: 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 43: 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 44: PFIC: Progressive Diseases of Bile-Related Transporters
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 Subtypes Impaired QoL (P
Slide 45: 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 46: 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 47: 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 48: 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 49: Intellectual Property Overview
Intellectual Property Overview
Slide 50: 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 51: 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 52: 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) 1 Orange Book Liste
Slide 53: 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 Orange No.Book 2,084,172 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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