Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Investor Presentation (January 2025)
51 slides from Mirum Pharmaceuticals's investor presentation, Jan 13, 2025
Slide 1: Mirum Pharmaceuticals: Transforming Lives in Rare Disease
J a n u a r y 2 0 2 5 Mirum Pharmaceuticals: Transforming Lives in 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: Mirum Pharmaceuticals: High Growth Global Rare Disease Company
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
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 (ALGS) three months of age and older. European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment
Slide 5: Strong Execution Positions Mirum as a High-Growth, Global Biotech
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 require
Slide 6: LIVMARLI: Alagille Syndrome 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 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
Slide 9: Well-Characterized Safety Profile of LIVMARLI
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, respect
Slide 10: CHOLBAM & chenodiol Bile Acid Portfolio
CHOLBAM & chenodiol 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 Strong Commercial Synergies
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 2
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 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/E
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 refract
Slide 17: Volixibat: IBAT Inhibitor for Cholestasis in Adults
IBAT Inhibitor for Cholestasis in Adults Volixibat
Slide 18: PSC & PBC: Immuno-inflammatory Rare Liver Disease
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: • S
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 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
Slide 21: IBAT Inhibition Reduces Pruritus and Serum Bile Acids in PSC
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 Pri
Slide 22: Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus
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 Enrol
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 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
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 FXS
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 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
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: Dedicated to Transforming the Treatment of Rare Diseases
Dedicated to Transforming the Treatment of Rare Diseases Mirum Pharmaceuticals
Slide 33: Well-Positioned to Execute on Our Strategy
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 an
Slide 34: 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 symbols, but
Slide 35: Supplemental Materials
Supplemental Materials
Slide 36: LIVMARLI Important Safety Information
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 live
Slide 37: 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 38: ALGS: A Debilitating Disease with Severe Cholestasis
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 studie
Slide 39: 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 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 experience
Slide 40: Significant Improvement in Transplant-Free Survival in 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 41: PFIC: Progressive Diseases of Bile-Related Transporters
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
Slide 42: 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 43: 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 44: 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 45: RESTORE Phase 3: Potentially NDA enabling for CTX
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 fro
Slide 46: RESTORE Phase 3 study: All Primary and Secondary Endpoints Met
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 Adv
Slide 47: Intellectual Property Overview
Intellectual Property Overview
Slide 48: LIVMARLI IP Coverage in the United States to 2040+
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
Slide 49: 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 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 N
Slide 50: LIVMARLI IP Coverage in Europe to 2040+
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 – P
Slide 51: IP Coverage for Pipeline Indications in Europe
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
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