Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Investor Presentation (January 2023)
33 slides from Mirum Pharmaceuticals's investor presentation, Jan 9, 2023
Slide 1: January 2023 Corporate Presentation
January 2023 Corporate Presentation 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: Leading the Way in Rare Diseases
LIVMARLI approved for cholestatic pruritus In Alagille Syndrome Robust Pipeline Strong Team and Balance Sheet Leading the Way in Rare Diseases Delivering remarkable treatments to patients in need worldwide
Slide 4: What Sets Mirum Apart
What Sets Mirum Apart 1 Cash, cash equivalents, restricted cash equivalents and investments; Estimate as of December 31, 2022, preliminary and unaudited and are subject to completion of financial closing procedures. Additional information and disclosure would be required for a more complete understa
Slide 5: IBAT Inhibition Targets Bile Acids in Cholestasis
IBAT Inhibition Targets Bile Acids in Cholestasis Cholestatic Liver Disease Abbreviation: IBAT - Ileal Bile Acid Transporter IBATi Reduces Bile Acids IBATi Clinical Impacts Inhibits reuptake by IBAT Interrupts recirculation of bile acids Increases fecal bile acid excretion Serum bile acid reductions
Slide 6: Commercial-Stage with Pipeline of Growth Opportunities
Commercial-Stage with Pipeline of Growth Opportunities *Received U.S. FDA approval for cholestatic pruritus in patients with ALGS one year of age and older. European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in
Slide 7: LIVMARLI for ALGS: Long-term data on pruritus and event-free survival
Long-term data set showing significant effects on pruritus and event-free survival LIVMARLI® (maralixibat) oral solution for ALGS
Slide 8: 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), Mirum market research and company estimates Genetic disease leading to severe cholestasis, unbearable pr
Slide 9: LIVMARLI Worldwide ALGS Commercialization
LIVMARLI Worldwide ALGS Commercialization Approved in the United States and Europe 1 Estimate as of December 31, 2022, preliminary and unaudited Launch Q4 2021 2022 Estimate 2023 Continued US & International growth Multiple Drivers of Commercial Growth Total Net Product Sales US business positioned
Slide 10: LIVMARLI US Commercialization Strategy
LIVMARLI US Commercialization Strategy Awareness & Use Disease education, unmet need and LIVMARLI clinical data Mirum Access Plus Prescription to fill, reimbursement, exclusive specialty pharmacy Long-term Support Dedicated case management to support adherence, outcomes Comprehensive support from fi
Slide 11: ICONIC: ALGS Pivotal Study Showing Significant Long-term Benefit
ICONIC: ALGS Pivotal Study Showing Significant Long-term Benefit Gonzales et al., Lancet 2021; 398: 1581–92 Highly significant, clinically meaningful and sustained improvements in pruritus from baseline *Denotes statistical significance, 95% CI excludes zero (compared with baseline, overall populati
Slide 12: Significant Improvement in Event-Free Survival in Patients with ALGS
Significant Improvement in Event-Free Survival in Patients with ALGS Treated with LIVMARLI Compared to Untreated Cohort Hansen BE, et al. Presentation. AASLD 2021. CI: confidence interval; EFS: event-free survival; HR: hazard ratio; MRX: maralixibat; SAP: statistical analysis plan. *Cox regression m
Slide 13: LIVMARLI Well-Characterized Safety Profile
LIVMARLI Well-Characterized Safety Profile LIVMARLI Prescribing Information Safety Data of LIVMARLI in ALGS Includes 5 Years of Follow-up from 3 Randomized Studies Events observed over 5% of patients Number of events per 100 person-years Diarrhea 41.6 Abdominal pain 38.6 Vomiting 19.8 Nausea 2.9 Fat
Slide 14: MARCH-PFIC Study: Highly statistically significant effects in All-PFIC
Phase 3 MARCH-PFIC Study: Highly statistically significant effects in All-PFIC LIVMARLI (maralixibat) for PFIC
Slide 15: PFIC: Progressive Diseases of Bile-Related Transporters
PFIC: Progressive Diseases of Bile-Related Transporters Progressive Familial Intrahepatic Cholestasis (PFIC) Genetic disease of bile transporters, resulting in cholestasis Severe pruritus, common indication for surgery or liver transplant ~20% transplant-free survival at 18 years of age Incidence of
Slide 16: Positive Phase 3 Data Across Broad Spectrum of PFIC Subtypes
Positive Phase 3 Data Across Broad Spectrum of PFIC Subtypes 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
Slide 17: Significant Improvements in Markers of Liver Disease
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 Mean Delta w
Slide 18: Summary of Treatment Emergent Adverse Events
Summary of Treatment Emergent Adverse Events Diarrhea was predominantly mild with no severe events, and transient with a median duration of 5.5 days One patient had a treatment emergent adverse event of mild diarrhea that led to discontinuation Treatment Emergent Adverse Event (TEAE) LIVMARLI (n=47)
Slide 19: sBA Response Associated with Transplant-Free Survival
sBA Response Associated with Transplant-Free Survival Loomes K et al, Hepatology Communications 2022 *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 ≤-75% average percent change from baseline INDIGO Phase 2
Slide 20: Phase 2b in post-Kasai Biliary Atresia: Data 2H 2023
Phase 2b in post-Kasai (HPE) Biliary Atresia: Data 2H 2023 LIVMARLI (maralixibat) for Biliary Atresia
Slide 21: Biliary Atresia: Severe Cholestasis Leading to Transplantation
Biliary Atresia: Severe Cholestasis Leading to Transplantation 1 0.5 to 0.8 per 10,000 live births, Sanchez-Valle et al , Advances in Pediatrics 2017; 2Shneider et al, J Pediatr. 2006; 3Harpavat et al, AASLD 2020 Leading cause of pediatric liver transplant, rapidly progressing cholestatic disease El
Slide 22: Enrolling EMBARK Study in Biliary Atresia
Enrolling EMBARK Study in Biliary Atresia Primary endpoint data expected in 2H 2023 Biliary Atresia patients ≤3 months age at HPE N~72 Open Label Long Term Extension 6mo Day 1 +18mo Primary Endpoint Change in total bilirubin Secondary Endpoints Change in serum bile acid Bilirubin normalization Bioma
Slide 23: IBATi for Cholestasis in Adults: Volixibat
IBATi for Cholestasis in Adults Volixibat
Slide 24: Volixibat: Targeting Adult Cholestasis
VOLIXIBAT: Targeting Adult Cholestasis Highly active on bile pathway; 48-week safety data in prior studies ~29,000 cases in US No approved therapies Pruritus: Potential Registration Endpoint 130,000 Cases in US No approved therapies for pruritus Pruritus: Potential Registration Endpoint PSC Primary
Slide 25: PSC & PBC: Immuno-inflammatory Rare Liver Disease
PSC & PBC: Immuno-inflammatory Rare Liver Disease Elevated Bile Acid Levels Drive Severe Symptom Burden and Progressive Liver Disease Bile Acid accumulation associated with: Severe symptomatic burden Inflammation and fibrosis of bile ducts and liver Progressive liver damage IBATi Proof of Mechanism:
Slide 26: Volixibat Clinical Studies: PSC and PBC
Volixibat Clinical Studies: PSC and PBC Blinded interim analysis Mid-year 2023: n~45, Dose Selection Confirmatory Analysis n~120 PSC PSC patients with moderate-to-severe pruritus N~120 VLX Open Label Extension PBC PBC patients with moderate-to-severe pruritus N~260 28wk Interim analysis H2 2023: n~6
Slide 27: Leading the way in rare liver disease
Leading the way in rare liver disease Mirum
Slide 28: Opportunity to Significantly Expand in Pipeline Indications
Opportunity to Significantly Expand in Pipeline Indications PATIENT POPULATION ALGS ALGS ALGS +PFIC +BA PFIC BA FDA and EMA approved Pediatric indications Pediatric indications + adult indications >20x increase +VOLIXIBAT (PSC, PBC)
Slide 29: Multiple Near-Term Transformative Milestones in 2023
Mirum: Multiple Near-Term Transformative Milestones in 2023 Potential ALGS partner market approvals* (Q4 ’22+) MARCH-PFIC Phase 3 Met primary and key secondary endpoints ALGS EU approval LIVMARLI VOLIXIBAT EMBARK (BA) Phase 2 data (H2 ’23) EU Commercial Launch (Q1 ‘23) PFIC FDA sNDA Filing (Q1 ‘23)
Slide 30: Recent Company Highlights
Recent Company Highlights Anticipated fourth quarter 2022 net product sales $27M and $76M in total revenue full year 20221 US business positioned to grow 50% year over year in 2023; international markets launching Continued stable operating expenses; Commercial business cash flow positive Cash, cash
Slide 31: Leading the Way in Rare Diseases
LIVMARLI approved for cholestatic pruritus In Alagille Syndrome Robust Pipeline Strong Team and Balance Sheet Leading the Way in Rare Diseases Delivering remarkable treatments to patients in need worldwide
Slide 32: Thank you!
Thank you! ©2023 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 ™
Slide 33: 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
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