Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Corporate Update (June 2024)
15 slides from Mirum Pharmaceuticals's corporate update, Jun 17, 2024
Slide 1: Interim Analysis Results of the VISTAS and VANTAGE Phase 2b Studies
Mirum Pharmaceuticals: Interim Analysis Results of the VISTAS (PSC) Phase 2b and VANTAGE (PBC) Phase 2b Studies J u n e 2 0 2 4
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 development and commercialization of rare disease medicines
Established leader in the development and commercialization of life changing rare disease medicines Standard of Approved Late Stage 3 Care Medicines 4 Indications 3 Indications
Slide 4: Strong Execution Positions Mirum as a High-Growth, Global Biotech
Strong Execution Positions Mirum as a High-Growth, Global Biotech 2018 2021 2023 2024+ 2 $310-320M 2024 Net Product Sales Guidance Founded November 2018 Approved Sept, 20211 3 Commercial Medicines Positive Interims for Volixibat (Alagille Syndrome) Global Commercial Footprint LIVMARLI Approved for P
Slide 5: Commercial Portfolio with Pipeline of Growth Opportunities
Commercial Portfolio with Pipeline of Growth Opportunities 3 APPROVED RARE DISEASE PRODUCTS, 3 ADDITIONAL INDICATIONS IN DEVELOPMENT IN HIGH-NEED ORPHAN INDICATIONS Indication Patients (US+EU) Preclinical Phase 1 Phase 2b/Phase 3 Approved Alagille Syndrome1 4,000 – 5,500 FDA and EMA Approved Progres
Slide 6: Volixibat
Volixibat Interim Analysis Results of the VISTAS (PSC) Phase 2b and VANTAGE (PBC) Phase 2b Studies 6
Slide 7: IBAT Inhibition In Cholestatic Liver Disease
IBAT Inhibition In Cholestatic Liver Disease Targeting IBAT Removes Circulating Bile Acids Addressing Toxic Bile Acid Accumulation Cholestatic Liver Disease Targeting IBAT Lowers Bile Acids IBAT Inhibition Clinical Benefits1-5 Defined by impaired bile flow & Mechanism directly addressing bile Transp
Slide 8: PSC & PBC: Immuno-inflammatory Rare Liver Disease
PSC & PBC: Immuno-inflammatory Rare Liver Disease Elevated Bile Acid Levels Drive Severe Symptom Burden (Pruritus, Fatigue) and Progressive Liver Disease Bile Acid Overload Obstruction of bile flow via impairment of intrahepatic and extrahepatic bile ducts Bile Acid Accumulation PSC: fibrotic strict
Slide 9: Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus
Phase 2b Study of Volixibat in PSC Patients with Cholestatic Pruritus Positive Interim Analysis Exceeded prespecified efficacy and safety thresholds for continuation PSC patients with moderate-to-severe pruritus 28wk Up to 2yrs VLX 20mg BID1 VLX Open Label 20 mg BID dose selected PBO Extension Confi
Slide 10: Phase 2b Study of Volixibat in PBC Patients with Cholestatic Pruritus
Phase 2b Study of Volixibat in PBC Patients with Cholestatic Pruritus Positive Interim Analysis Rapid and statistically significant improvement in pruritus 28wk Up to 2yrs PBC patients with moderate-to-severe pruritus VLX 20mg BID1 Reductions in sBA and VLX Open Label Extension PBO improvements in f
Slide 11: VANTAGE Interim Analysis: Baseline Characteristics Well Balanced
VANTAGE Interim Analysis: Baseline Characteristics Well Balanced Volixibat BID 20mg Volixibat BID 80 mg Placebo Characteristic (n=10) (n=10) (n=10) 53.9 52.3 62.1 Age (years), mean (SD) (15.83) (5.91) (10.26) 8 9 9 Female, n (%) (80) (90) (90) 6.8 6.3 6.5 Adult ItchRO Score, mean (SD) (1.58) (1.75)
Slide 12: VANTAGE Interim Analysis: Reduction in Pruritus from Baseline
VANTAGE Interim Analysis: Reduction in Pruritus from Baseline RAPID AND STATISTICALLY SIGNIFICANT REDUCTIONS IN PRURITUS Placebo volixibat 20 mg volixibat 80 mg volixibat 20+80 mg (n=10) (n=10) (n=10) (n=20) 0 LS Mean change (SE) in the Adult ItchRO** -1 score from Baseline -2 -3 -1.50 (0.585)* p=0.
Slide 13: VANTAGE Interim Analysis: Other Observations
VANTAGE Interim Analysis: Other Observations • Significant reduction in pruritus as early as Week 1 • Significant improvements in fatigue at Week 16 • 75% of patients on volixibat achieved >50% reduction in sBA • No new safety signals: – No clinically meaningful changes in liver laboratory tests for
Slide 14: Mirum is a Leading High-Growth, Global Rare Disease Biotech
Mirum is a Leading High-Growth, Global Rare Disease Biotech 2024 Value Drivers Growing commercial business Continued strong revenue growth $179M 2023 net product sales1 LIVMARLI: Continued US/Intl demand growth $310-320M expected 2024 net product sales Bile Acid portfolio: Growth in line with histor
Slide 15: Thank You
Thank You ©2024 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 ™