Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Investor Presentation (January 2024)
42 slides from Mirum Pharmaceuticals's investor presentation, Jan 8, 2024
Slide 1: Mirum Pharmaceuticals: Transforming Lives in Rare Disease
January 2024 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: Established leader in life changing rare disease medicines
Established leader in the development and commercialization of life changing rare disease medicines Standard of Care Medicines Late-Stage Indications Countries with Reimbursed Access 3 4 18
Slide 4: Strong Execution Positions Mirum as a High-Growth, Global Biotech
Strong Execution Positions Mirum as a High-Growth, Global Biotech ALGS $500M US market opportunity Continued US penetration International expansion in target markets Expanding indications (PFIC Submissions) Rare Disease Commercial Excellence Net Product Sales Growing penetration across range of bile
Slide 5: Commercial Portfolio with Pipeline of Growth Opportunities
Commercial Portfolio with Pipeline of Growth Opportunities *Received 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 6: 2024 Strategic Goals to Build Value
5 Clinical Programs 2024 Strategic Goals to Build Value INDUSTRY LEADING TEAM DELIVERING REMARKABLE TREATMENTS TO PATIENTS WORLDWIDE Drive continued growth across all three commercial medicines Expand labels for LIVMARLI (PFIC) & CHENODAL (CTX) Leverage IBAT Inhibition Expertise in Adult Cholestatic
Slide 7: Commercial Overview
Commercial Overview
Slide 8: Efficient Global Rare Disease Commercial Execution
Efficient Global Rare Disease Commercial Execution 1Estimate as of December 31, 2023 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 positio
Slide 9: LIVMARLI in ALGS
Long-term data set showing significant effects on pruritus and event-free survival LIVMARLI® (maralixibat) Oral Solution in ALGS
Slide 10: IBAT Inhibition In Cholestatic Liver Disease
IBAT Inhibition In Cholestatic Liver Disease Targeting IBAT Removes Circulating Bile Acids Addressing Toxic Bile Acid Accumulation Pruritus Bilirubin (PFIC) Xanthomas (ALGS) Event-free survival Quality of Life Growth 1. Gonzales E et al. Lancet. 2021;398:1581-1592. 2. Loomes KM et al. Hepatol Commun
Slide 11: ALGS: A Debilitating Disease with Severe Cholestasis
ALGS: A Debilitating Disease with Severe Cholestasis Kamath 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
Slide 12: LIVMARLI: Positioned for Long Term Growth in ALGS
LIVMARLI: Positioned for Long Term Growth in ALGS 1Estimate as of December 31, 2023 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
Slide 13: LIVMARLI Well-Characterized Safety Profile
LIVMARLI Well-Characterized Safety Profile Safety Data of LIVMARLI in ALGS Includes 5 Years of Follow-up from 3 Randomized Studies Most common adverse events were diarrhea, abdominal pain (41.6 and 38.6 events per 100 person-years respectively) GI adverse reactions were generally mild or moderate se
Slide 14: CHENODAL & CHOLBAM Bile Acid Portfolio
CHENODAL & CHOLBAM Bile Acid Portfolio
Slide 15: Bile Acid Replacement Therapies for Rare Genetic Diseases
Bile Acid Replacement Therapies for Rare Genetic Diseases 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) Smit
Slide 16: High Performing, Profitable Business with Strong Commercial Synergies
High Performing, Profitable Business with Strong Commercial Synergies CTX Genetic disorder with toxic cholestanol & bile alcohol build-up FDA medical necessity status ~1-2k US Prevalence BASD Group of genetic disorders with abnormal bile acid production ~200-300 US Prevalence PBD-ZSD Genetic, multis
Slide 17: Pipeline
Significant expansion opportunities within pipeline indications Pipeline
Slide 18: MARCH-PFIC Study: LIVMARLI in PFIC
Phase 3 MARCH-PFIC Study: Highly statistically significant effects in All-PFIC LIVMARLI (maralixibat) in PFIC
Slide 19: PFIC: Progressive Diseases of Bile-Related Transporters
PFIC: Progressive Diseases of Bile-Related Transporters Kamath BM et al. J Pediatr Gastroenterol Nutri. 2018, Kamath BM et al, Liver Transpl 2012, Vandriel SM, et al. EASL 2020 (oral presentation), Mirum market research and company estimates Genetic disease leading to bile acid build-up and progress
Slide 20: Positive Phase 3 Data Across Broad Spectrum of PFIC Types
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 De
Slide 21: Phase 3: Summary of Treatment Emergent Adverse Events
Phase 3: Summary of Treatment Emergent Adverse Events Treatment Emergent Adverse Event (TEAE) LIVMARLI (n=47) Placebo (n=46) Any TEAE, n (%) 47 (100%) 43 (93.5%) Severe TEAE, n (%) 3 (6.4%) 3 (6.5%) Serious TEAE, n (%) 5 (10.6%) 3 (6.5%) TEAE leading to discontinuation, n (%) 1 (2.1%) 0 TEAE leading
Slide 22: Volixibat: IBAT Inhibitor for Cholestasis in Adults
IBAT Inhibitor for Cholestasis in Adults Volixibat
Slide 23: PSC & PBC: Immuno-inflammatory Rare Liver Disease
PSC & PBC: Immuno-inflammatory Rare Liver Disease Bile Acid Overload Obstruction of bile flow via impairment intrahepatic and extrahepatic bile ducts Elevated Bile Acid Levels Drive Severe Symptom Burden and Progressive Liver Disease Bile Acid Accumulation Associated with: Severe symptomatic burden
Slide 24: Primary Sclerosing Cholangitis
Primary Sclerosing Cholangitis
Slide 25: PSC: Pruritus is Common and Often Moderate to Severe
PSC: Pruritus is Common and Often Moderate to Severe 54,000 patients across US & EU ~65% with active pruritus1 1 Mirum market research 2 “How would you rate the worst itch you experienced from your last episode of itching?” (n = 257); Kowdley et al, EASL 2022 No Approved Therapies; Pruritus Is A Sig
Slide 26: IBAT Inhibition Reduces Pruritus and Serum Bile Acids in PSC
IBAT Inhibition Reduces Pruritus and Serum Bile Acids in PSC CAMEO Study: Open label Maralixibat 10mg QD for 14wks, N=27 ItchRO: 0-10 Numerical Rating Scale (0=No Itch, 10=Worst Itch Imaginable) Volixibat: Highly active on bile acid pathway; 48-week safety data in prior studies CAMEO Study IBAT inhi
Slide 27: Phase 2b Study of Volixibat in PSC
Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus Primary Endpoint Change in pruritus from Baseline to 28wk Secondary Endpoints Safety & tolerability Markers of disease and QoL Long Term Assessments Changes in pruritus and QoL Markers of disease progression Safety Up to 2yrs Blin
Slide 28: Primary Biliary Cholangitis
Primary Biliary Cholangitis
Slide 29: PBC: Most Prevalent Cholestatic Liver Disease
PBC: Most Prevalent Cholestatic Liver Disease 230,000 patients across US & EU *Mirum market research Significant Global Opportunity Across 1st & 2nd Line Settings 1st line therapy patients (ALP <1.67xULN) 2nd line therapy patients (ALP >1.67xULN) ~60% with active pruritus* Primary Biliary Cholangiti
Slide 30: Phase 2b Study of Volixibat in PBC
Phase 2b Study of Volixibat in PBC Patients with Cholestatic Pruritus Primary Endpoint Change in pruritus from Baseline to 28wk Secondary Endpoints Safety & tolerability Markers of disease and QoL Long Term Assessments Changes in pruritus and QoL Markers of disease progression Safety Up to 2yrs Topl
Slide 31: Dedicated to transforming the treatment of rare diseases
Dedicated to transforming the treatment of rare diseases Mirum
Slide 32: Mirum is a Leading High-Growth, Global Rare Disease Biotech
Mirum is a Leading High-Growth, Global Rare Disease Biotech 1 Estimate as of December 31, 2023 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 financi
Slide 33: Thank You
Thank You
Slide 34: Supplemental Materials
Supplemental Materials
Slide 35: LIVMARLI Important Safety Information
LIVMARLI (maralixibat) oral solution Important Safety Information IMPORTANT SAFETY INFORMATION LIVMARLI can cause serious side effects, including: Changes in liver tests: Changes in certain liver tests are common in patients with Alagille syndrome but may worsen during treatment with LIVMARLI. These
Slide 36: 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 37: 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. 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 gr
Slide 38: 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 39: MARCH PFIC: Significant Improvements in Markers of Liver Disease
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
Slide 40: 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 41: RESTORE Phase 3: Potentially NDA enabling for CTX
RESTORE Phase 3: Potentially NDA enabling for CTX, Orphan Exclusivity 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 baselin
Slide 42: RESTORE Phase 3: All Primary and Secondary Endpoints Met
RESTORE Phase 3 study: All Primary and Secondary Endpoints Met Data Mean Difference with 95% CI P-values from a paired t-test comparing CHENODAL 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
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