Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Investor Presentation (January 2022)
38 slides from Mirum Pharmaceuticals's investor presentation, Jan 11, 2022
Slide 1: January 2022 Corporate Presentation
January 2022 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 FDA-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 $500M+ potential in ALGS in US 5 late-stage indications, significant data events upcoming
Slide 4: LIVMARLI is Now Approved in the United States
LIVMARLI is Now Approved in the United States LIVMARLI (maralixibat) oral solution is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older. www.LIVMARLI.com
Slide 5: Commercial-Stage with Pipeline of Growth Opportunities
Commercial-Stage with Pipeline of Growth Opportunities LIVMARLI launched, 5 additional indications under development in high-need orphan indications Alagille syndrome* Progressive Familial Intrahepatic Cholestasis Biliary Atresia LIVMARLI (maralixibat) Oral Solution FDA Approved MAA submitted in Eur
Slide 6: IBAT Inhibitors Interrupt Bile Acid Recirculation
Ileal Bile Acid Transporter (IBAT) Inhibitors Interrupt Bile Acid Recirculation Targeting Bile Acid Excess of Cholestasis Redirects bile acid flow by inhibiting reuptake by IBAT Interrupts recirculation of bile acids to the liver Increases fecal bile acid excretion Abbreviations: sBA: serum bile aci
Slide 7: Long-term data set on pruritus and event-free survival in ALGS
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; 67(2): 148-156, 2Vandriel SM, et al. EASL 2020 (oral presentation) Genetic Disease Leading to Severe Cholestasis, Unbearable Pruritus and Multi-system Effects 2k-2.5k Children in the U.S. 88%
Slide 9: Significant Improvement in Event-Free Survival with LIVMARLI
NEW: Significant Improvement in Event-Free Survival in Patients with ALGS with LIVMARLI Compared to Untreated Cohort 6-year analysis demonstrates a 70% reduction for clinical outcomes vs natural history control from the GALA database CI: confidence interval; EFS: event-free survival; HR: hazard rati
Slide 10: ICONIC: ALGS Pivotal Study Showing 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 11: Safety Data of LIVMARLI in ALGS
Safety Data of LIVMARLI in ALGS Includes 5 Years of Follow-up from 3 Randomized Studies LIVMARLI Well-Characterized Safety Profile 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-soluble vitamin deficiency 11.1
Slide 12: LIVMARLI Commercialization Strategy
LIVMARLI 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 first
Slide 13: ALGS Opportunity and Preliminary Q4 Net Product Revenue
ALGS OPPORTUNITY PRELIMINARY Q4 NET PRODUCT REVENUE* Estimate >90% of covered lives in US have reimbursable pathway as of January 2022 $500M $3.0M LIVMARLI: Approved September 29, 2021 *Unaudited and estimated launch quarter
Slide 14: LIVMARLI Worldwide Commercialization
LIVMARLI Worldwide Commercialization CORE MARKETS US Launched EU Early Access Programs ongoing, Launch anticipated in H2 2022 PARTNER MARKETS DISTRIBUTOR PARTNERS Japan China South Korea Israel Russia Czech Republic (Central & Eastern Europe) UAE (Middle East) In-house Commercial Team
Slide 15: Dramatic and durable response in INDIGO Phase 2 study
Dramatic and durable response in INDIGO Phase 2 study LIVMARLI (maralixibat) for PFIC
Slide 16: 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 17: sBA Response Associated with Improved Pruritus and Growth
sBA Response Associated with Improved Pruritus and Growth Adapted from: Thompson R, et al. NASPGHAN 2020 (poster presentation); Error bars represent standard error of the mean. ItchRO[Obs], Itch Reported outcome [Observer]; sBA, serum bile acid NAPPED response threshold 102 µmol/L 85% decrease 80% d
Slide 18: INDIGO: sBA Response Associated with Transplant-Free Survival
INDIGO: sBA Response Associated with Transplant-Free Survival Supported by Positive Event-Free Survival Analyses with Natural History Cohort Transplant free survival in PFIC2 after maralixibat treatment Thompson et al, AASLD 2020 Time from enrolment (weeks) TFS (%) 100 60 40 20 80 10 30 50 70 90 0 5
Slide 19: PFIC Phase 3 Enrolling, Topline Data Expected H2 2022
PFIC Phase 3 Enrolling – Topline Data Expected H2 2022 Global Study Covering All PFIC Sub-types; Evaluating High Dose Maralixibat 1:1 randomization Baseline 6 Months + Natural history data set comparisons N~ 30 with nt-PFIC2 Up to 60 patients in supplemental cohort of other PFIC subtypes Global clin
Slide 20: Ongoing Phase 2b in post-Kasai Biliary Atresia
Ongoing Phase 2b in post-Kasai Biliary Atresia LIVMARLI (maralixibat) for Biliary Atresia
Slide 21: Biliary Atresia: Severe Cholestasis Leading to Transplantation
Biliary Atresia: A Severe Cholestasis Leading to Transplantation Inflammatory bile duct destruction causing severe cholestasis Bile accumulation in the liver leads to liver damage and transplantation Fatal without Kasai procedure (HPE), standard of care within the first weeks of life Leading cause o
Slide 22: EMBARK: Phase 2b Study Enrolling
EMBARK: Phase 2 Study Enrolling Analyzing 6-month Bilirubin Biomarker Before Long-term Transplant-Free Survival 1:1 randomization Core Study: 6 months Primary Cohort N=72 patients ≤3 months age at HPE Excl. BASM, cystic BA Primary Endpoint Change in total serum bilirubin Secondary Endpoints Change i
Slide 23: IBATi for Cholestasis in Adults: Volixibat
IBATi for Cholestasis in Adults Volixibat
Slide 24: Volixibat Potential in Adult Cholestasis
Volixibat Potential in Adult Cholestasis Intrahepatic Cholestasis of Pregnancy (ICP), Primary Sclerosing Cholangitis (PSC) and Primary Biliary Cholangitis (PBC) High Unmet Need Prevalence PSC: ~29,000 in US Limited Tx Options ICP, PSC - No Approved Therapies PBC – Treatments Sub-optimal Byron D, Min
Slide 25: Volixibat Highly Active on Bile Acid Excretion
Volixibat Highly Active on Bile Acid Excretion Highly active on fecal bile acid excretion Resultant dose-related increase in 7aC4 48-week safety data in prior studies C. Key, AASLD 2019 Broad Data Set Informs Registrational Program Volixibat Markedly Impacts Bile Pathway Selected doses for Phase 2b
Slide 26: ICP: Bile Acid Accumulation Drives Severe Symptoms
ICP: Bile Acid Accumulation Drives Severe Symptoms, Poor Outcomes Monte et al, 1995; Shaw et al, 1982 Normal Pregnancy ICP Pregnancy Bile acids flow from fetus to mother across a natural placental gradient Maternal cholestasis; Accumulation of bile acids reverses natural bile acid gradient, exposing
Slide 27: Accumulation of Maternal BAs Drives Fetal Morbidity/Mortality
Accumulation of Maternal BAs Drives Fetal Morbidity/Mortality Glantz et al, Hepatology 2004; Geenes et al, Hepatology 2014; Ovadia et al, The Lancet 2019; 2019 March of Dimes Report Card 25-57% $50K $2B ICP babies are born preterm Average cost of preterm birth Annual U.S. medical costs Small differe
Slide 28: OHANA: Phase 2b Study in ICP
OHANA: Phase 2b Study Enrolling Evaluating Reductions in sBA, Pruritus and Perinatal Outcomes in ICP 1:1:1 randomization Double-blind Volixibat 20mg Twice Daily Placebo Twice Daily Up to delivery Study Patient Population Primary cohort: N=260 patients with ICP diagnosis Up to 60 patients in suppleme
Slide 29: 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 Obstructed bile flow leads to Severe symptomatic burden Inflammation and fibrosis of the bile ducts and liver Progressive liver damage No approved therapies for PSC N
Slide 30: VISTAS: Volixibat PSC Phase 2b
VISTAS: Volixibat PSC Phase 2b Enrolling Assessing Pruritus Reductions in PSC; Setting with No Approved Therapies 1:1:1 randomization Volixibat 20 mg Twice Daily Placebo (PBO) Twice Daily Day 1 28 weeks Study Patient Population ~120 PSC patients (small duct compensated cirrhosis, concomitant IBD all
Slide 31: VANTAGE: Volixibat PBC Phase 2b Study
VANTAGE: Volixibat PBC Phase 2b Study - Enrolling Evaluating the safety and efficacy of volixibat in patients with itch caused by PBC 1:1:1 randomization Volixibat BID – 20 mg Placebo (PBO) BID 28wk Study Patient Population N = 260 PBC patients Moderate-to-severe pruritus No ALP cutoff Stable doses
Slide 32: Leading the Way in Rare Liver Disease
Leading the Way in Rare Liver Disease Mirum
Slide 33: Opportunity to Significantly Expand in Pipeline Indications
Opportunity to Significantly Expand in Pipeline Indications PATIENT POPULATION ALGS ALGS ALGS +PFIC +BA PFIC BA FDA-approved, EU Marketing submission complete Pediatric indications Pediatric indications + adult indications Pediatric indications (blue) Adult indications (red) >20x increase +VOLIXIBAT
Slide 34: Strong Balance Sheet: Projected 3+ Years Runway
Strong Balance Sheet: Projected 3+ Years Runway SELECTED Balance Sheet Data Sept. 30, 2021 Dec. 31, 2020 Cash, cash equivalents and investments $ 205.0 231.8 Total Assets 235.2 240.9 Total stockholders’ equity (deficit) 56.4 172.1 SELECTED Statements of Operations Data Sept. 30, 2021 Sept. 30, 2020
Slide 35: Transformative Milestones Ahead in 2022
Potential ALGS partner market approvals (Q4) Mirum: Transformative Milestones Ahead in 2022 OHANA (ICP) interim analysis (H2) VISTAS (PSC) interim analysis (H2) MARCH-PFIC Phase 3 data (H2) International early access programs (Q1) Potential ALGS EU approval* (H2) *In Europe, seeking EMA approval for
Slide 36: Leading the Way in Rare Diseases
LIVMARLI FDA-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 $500M+ potential in ALGS in US 5 late-stage indications, significant data events upcoming
Slide 37: Thank you!
Thank you! ©2021 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 38: 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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