Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Investor Presentation (January 2026)
60 slides from Mirum Pharmaceuticals's investor presentation, Jan 12, 2026
Slide 1: Mirum Pharmaceuticals: Delivering High Impact Medicines for Rare Disease
J a n u a r y 2 0 2 6 Mirum Pharmaceuticals: Delivering High Impact Medicines for Rare Disease Exhibit 99.2
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: Delivering Significant Value Through a Purpose-Built Rare Disease Model
3 1 Assuming closing of proposed acquisition of Bluejay Therapeutics, expected in January 2026, subject to customary closing conditions 2 Annual net product sales 2022-2024 and preliminary unaudited 2025 net product sales subject to completion of financial closing procedures 3 Includes designations
Slide 4: Commercial Portfolio with Pipeline of Growth Opportunities
Commercial Portfolio with Pipeline of Growth Opportunities 1Received U.S. FDA approval for cholestatic pruritus in patients with Alagille syndrome 3 months of age and older. European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of cholest
Slide 5: Strong Execution Positions Mirum as a Global Rare Disease Leader
Strong Execution Positions Mirum as a Global Rare Disease Leader 5 Hepatology & GI Rare Genetic Neurology & Metabolics 1 Estimate as of December 31, 2025 is preliminary and unaudited and is subject to completion of financial closing procedures. Additional information and disclosure would be required
Slide 6: LIVMARLI: ALGS and PFIC
Alagille Syndrome (ALGS) Progressive Familial Intrahepatic Cholestasis (PFIC) LIVMARLI®
Slide 7: IBAT Inhibition in Cholestatic Liver Disease
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
Slide 8: LIVMARLI: A Leading Medicine for Ultra-Rare Cholestatic Pruritus
LIVMARLI: A Leading Medicine for Ultra-Rare Cholestatic Pruritus 8 1 Estimate as of December 31, 2025 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
Slide 9: Expanding Impact in Ultra-Rare Cholestatic Pruritus
Expanding Impact in Ultra-Rare Cholestatic Pruritus 9 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 Ultra-Rare Cholestasis 1,000+ potential US/EU prevalence Path to Sustained Long-Term Growth ✓ Continu
Slide 10: CHOLBAM & CTEXLI Bile Acid Portfolio
CHOLBAM® & CTEXLI® Bile Acid Portfolio
Slide 11: Bile Acid Replacement Therapies for Rare Genetic Diseases
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)
Slide 12: Growing Business with Significant Commercial Synergies
Growing Business with Significant Commercial Synergies 12 Bile Acid Portfolio Addresses Multiple High Need Settings 1 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 potent
Slide 13: Pipeline
Significant Expansion Opportunities within Pipeline Indications Pipeline
Slide 14: LIVMARLI
LIVMARLI
Slide 15: LIVMARLI Label Expansion Opportunity in Cholestatic Pruritus
LIVMARLI Label Expansion Opportunity in Cholestatic Pruritus 15 Broad Unmet Need in Multiple Ultra-Rare Cholestatic Conditions 1 Mirum Market Research Pruritus in Ultra-Rare Cholestatic Settings Elevated sBA Severe pruritus Stunted growth Impaired QoL Characteristic Cholestatic Burden Estimated Addr
Slide 16: EXPAND Phase 3 Study Enrolling
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 cholest
Slide 17: Volixibat: IBAT Inhibitor for Cholestasis in Adults
IBAT Inhibitor for Cholestasis in Adults Volixibat
Slide 18: PSC and PBC: Immuno-inflammatory Rare Liver Disease
PSC and 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: •
Slide 19: Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus
Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus 191 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 Confi
Slide 20: Primary Sclerosing Cholangitis
Primary Sclerosing Cholangitis
Slide 21: PSC: Pruritus is Common and Often Moderate to Severe
PSC: Pruritus is Common and Often Moderate to Severe 21 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
Slide 22: IBAT Inhibition Reduces Pruritus and Serum Bile Acids in PSC
IBAT Inhibition Reduces Pruritus and Serum Bile Acids in PSC 22 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 Pri
Slide 23: Primary Biliary Cholangitis
Primary Biliary Cholangitis
Slide 24: PBC: Most Prevalent Cholestatic Liver Disease
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
Slide 25: Phase 2b Study of Volixibat in PBC Patients with Cholestatic Pruritus
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
Slide 26: VANTAGE Interim Analysis: Baseline Characteristics Well Balanced
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
Slide 27: VANTAGE Interim Analysis: Reduction in Pruritus from Baseline
VANTAGE Interim Analysis: Reduction in Pruritus from Baseline 27 Heneghan et al, EASL 2025 1Adult ItchRO is a 0-10 worst itch numerical rating scale where 0 = no itch and 10 = worst possible itch 2LS mean (95% CI) change from Baseline to the average of the last 12 weeks of treatment. LS means and P
Slide 28: VANTAGE Interim Analysis: Other Observations
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
Slide 29: MRM-3379 PDE4D Inhibitor for Fragile X Syndrome (FXS)
MRM-3379 PDE4D Inhibitor for Fragile X Syndrome (FXS)
Slide 30: Fragile X Syndrome (FXS): Rare X-Linked Genetic Disorder
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 cA
Slide 31: MRM-3379: Selective PDE4D inhibitor for FXS
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 • O
Slide 32: MRM-3379 Was Evaluated in a Mouse Model of FXS
MRM-3379 Was Evaluated in a Mouse Model of FXS 32Carter, et al. FXS-NDD 2025 MRM-3379 improved multiple behavioral domains relevant to the FXS phenotype in a mouse model Hyperactivity Phenotype Was Reversed Nesting Phenotype Was Reversed Marble Burying Phenotype Was Reversed FMR1 KO2 Mouse Model of
Slide 33: Phase 2 Study of MRM-3379 in Fragile X Syndrome
Phase 2 Study of MRM-3379 in Fragile X Syndrome 33 Primary Endpoint Safety & tolerability Key Secondary Endpoint NIH-TCB Crystallized Cognition Composite (CCC) Composed from the Picture Vocabulary Test (PVT) and Oral Reading Recognition Test (ORRT) Key Inclusion Criteria • Males, 13-45 years of age
Slide 34: Chronic Hepatitis Delta Virus (HDV): Brelovitug
Chronic Hepatitis Delta Virus (HDV) Mirum's acquisition of brelovitug from Bluejay Therapeutics, Inc. remains subject to regulatory approval. The information in this section is derived from public information and Mirum's internal research. Brelovitug
Slide 35: HDV Is the Most Severe Form of Viral Hepatitis
HDV Is the Most Severe Form of Viral Hepatitis 35 No Approved Therapies in the US ~15,000~ US pts diagnosed, insured, under care4 1Negro, F. & Lok, A. S JAMA 2023 2Miao et al, The Journal of Infectious Diseases 2019 3Sagnelli, C. et al. HBV/HDV Co-Infection: Epidemiological and Clinical Changes, Rec
Slide 36: Virologic Response Demonstrated in P2 Clinical Trial
Virologic Response Demonstrated in P2 Clinical Trial Fully human anti-HBsAg monoclonal antibody SC injection 1x Weekly or 1x Monthly Brelovitug: Preliminary Efficacy and Favorable Safety Profile in HDV Safety: Parallel ALT reductions; Low rates of flulike symptoms, No >grade 2 AEs, no SAEs, no disco
Slide 37: Ongoing Brelovitug Phase 3 Trials Supporting FDA and EMA Filings
Ongoing Brelovitug Phase 3 Trials Supporting FDA and EMA Filings 37 1 Patients in delayed Tx start arm switch to 300mg 1x Weekly at week 12 2 Virologic Response = HDV RNA ≥2 log reduction or TND 300mg 1x Weekly vs 900mg 1x Monthly vs Delayed Tx Start Primary Endpoint Week 24 Virologic Response2 + AL
Slide 38: Delivering High Impact Medicines for Rare Disease
Delivering High Impact Medicines for Rare Disease Mirum Pharmaceuticals
Slide 39: Well-Positioned to Execute on Our Planned Strategy
Well-Positioned to Execute on Our Planned Strategy 39 2027 2026 FY Guidance $630-650M 2026 Net Product Sales Guidance Cash Flow Positive in 2027 $392M Cash Balance3 4 potentially registrational topline readouts expected in the next 18 months1 2026 ❑ VANTAGE (PBC) complete enrollment in H2 ❑ VISTAS (
Slide 40: Thank You
©2026 Mirum Pharmaceuticals, Inc. All rights reserved. All service marks, trademarks and tradenames appearing in this presentation are the property of their respective owners. Solely for convenience, the trademarks and tradenames referred to in this presentation appear without the ® and symbols, but
Slide 41: Supplemental Materials
Supplemental Materials
Slide 42: LIVMARLI Important Safety Information
LIVMARLI Important Safety Information 42 IMPORTANT SAFETY INFORMATION LIVMARLI can cause serious side effects, including: Liver injury: Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury.
Slide 43: CHOLBAM Important Safety Information
CHOLBAM Important Safety Information LIMITATIONS OF USE The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established. IMPORTANT SAFETY I
Slide 44: CTEXLI Important Safety Information
CTEXLI Important Safety Information IMPORTANT SAFETY INFORMATION CTEXLI can cause side effects, including: Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may
Slide 45: Mirum Quarterly Net Product Sales
Mirum Quarterly Net Product Sales Net Product Sales ($M) Q1 2024 Q2 2024 Q3 2024 Q4 2024 FY 2024 Q1 2025 Q2 2025 Q3 2025 Q4 2025E1 FY 2025E1 LIVMARLI US 30.8 35.5 43.5 44.7 154.5 49.5 56.9 64.2 74 243 LIVMARLI International 12.1 11.7 15.6 19.4 58.8 23.7 31.2 28.1 33 116 LIVMARLI Total 42.8 47.2 59.1
Slide 46: LIVMARLI Available in Both Oral Solution and Tablet Formulation
LIVMARLI Available in Both Oral Solution and Tablet Formulation 46 One LIVMARLI Tablet Per Dose Size for comparison 10mg 15mg 20mg 30mg Flexible Dosing Options for Patients Supplemental Material
Slide 47: ALGS: A Debilitating Disease with Severe Cholestasis
ALGS: A Debilitating Disease with Severe Cholestasis 47Kamath 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 studie
Slide 48: ICONIC: ALGS Pivotal Study Shows Significant Long-term Benefit
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 o l/ L) Reduced sBA -2.5 -2 -1.5 -1 -0.5 0 Week 18 Week 48 It ch R O (O b s) Reduced Pruritus 84% of participants experienc
Slide 49: Significant Improvement in Transplant-Free Survival in Patients with ALGS
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 even
Slide 50: PFIC: Progressive Diseases of Bile-Related Transporters
PFIC: Progressive Diseases of Bile-Related Transporters 50 ~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
Slide 51: MARCH PFIC: Positive Phase 3 Data Across Broad Spectrum of PFIC Types
-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. * L
Slide 52: MARCH PFIC: Significant Improvements in Markers of Liver Disease
-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
Slide 53: PFIC: sBA Response Associated with Transplant-Free Survival
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 ≤
Slide 54: Well-Characterized Safety Profile of LIVMARLI
Well-Characterized Safety Profile of LIVMARLI 54 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, respec
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 55 4 4 3 3 2 4 2 1 2 0 0 0 1 0 0 1 2 3 4 Patient 1 Patient 2 Patient 3 Patient 4 Patient 5 Patient 6 Patient 7 196 62 506 70 148 317 136 38 31 79 14 106 433 35 0 100 200 300 400 500 Patient 1 Patient 2 Patient
Slide 56: Intellectual Property Overview
Intellectual Property Overview
Slide 57: LIVMARLI IP Coverage in the United States to 2040+
ALGS PFIC ALGS & PFIC LIVMARLI IP Coverage in the United States to 2040+ 571Approved for grant 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 [Pendi
Slide 58: IP Coverage for Pipeline Indications in the United States
Volixibat PBC PSC CTX MRM-3379 FXS IP Coverage for Pipeline Indications in the United States 581Assumes standard patent term extension [Pending] Additional Dosing (2042) [Pending] Method of Treatment: Dosing (2032, 2040) Orphan Designation (2032) Composition of Matter (2027) Patent No. 7,956,085 PBC
Slide 59: LIVMARLI IP Coverage in Europe to 2040+
LIVMARLI IP Coverage in Europe to 2040+ 59 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 – Paten
Slide 60: IP Coverage for Pipeline Indications in Europe
IP Coverage for Pipeline Indications in Europe 60 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
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