Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM
Mirum Pharmaceuticals — Corporate Update (September 2026)
11 slides from Mirum Pharmaceuticals's corporate update, Sep 28, 2026
Slide 1: AZURE-1 Phase 3 Topline Results
MIRUM PHARMACEUTICALS AZURE-1 Phase 3 Topline Results AZURE-1 Phase 2b 48-Week Topline Results September 2026
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: Brelovitug: Chronic Hepatitis Delta Virus (HDV)
BRELOVITUG CHRONIC HEPATITIS DELTA VIRUS (HDV) AZURE-1 Phase 3 Topline Results AZURE-1 Phase 2b 48-Week Topline Results 3
Slide 4: HDV Is the Most Severe Form of Viral Hepatitis
HDV Is the Most Severe Form of Viral Hepatitis A Rapidly Progressive Disease with Significant Unmet Need Hepatitis Delta Virus >50% Liver-Related Death in 10 Years1 ~15,000 US pts diagnosed, insured, under care4 5yrs Avg. Progression to Cirrhosis and Liver Failure2 ~40,000 Est. US Prevalence Require
Slide 5: Brelovitug AZURE-1 Phase 2b/3 Study Design
Brelovitug: Preliminary Efficacy and Favorable Safety Profile in HDV Brelovitug AZURE-1 Phase 2b/3 Study Design 24wk Fully human anti-HBsAg monoclonal antibody SC injection 1x Weekly or 1x Monthly 300mg 1x Weekly or 900mg 1x Monthly1 Extension (96wks) Delayed Tx→ 300mg 1x Delayed Tx Start Weekly Pri
Slide 6: AZURE-1 Phase 3 Key Baseline Disease Characteristics
AZURE-1 Phase 3 Key Baseline Disease Characteristics 300 mg QW 900 mg Q4W Delayed Treatment N=59 N=65 N=29 Cirrhosis, n (%)1 24 (41) 27 (42) 12 (41) Liver stiffness, kPa, mean (SD) 14.3 (10.78) 13.7 (8.01) 14.6 (11.49) ALT, U/L, mean (SD) 130.4 (106.88) 121.3 (95.07) 121.0 (84.29) ALT ≥5x ULN, n (%)
Slide 7: AZURE-1 Phase 3 Week 24 Met Primary Endpoint
AZURE-1 Phase 3 Week 24 Met Primary Endpoint 300 mg QW* 900 mg Q4W Delayed Treatment N=59 N=65 N=29 Primary Endpoint 33/59 (56%) 29/65 (45%) 0/29 (0%) (Virologic Response + ALT normalization) p <0.0001 p <0.0001 Virologic response 51/59 (86%) 55/65 (85%) 0/29 (0%) HDV RNA ≥2 log10 reduction or TND H
Slide 8: AZURE-1 Phase 3 Safety Through Week 24
AZURE-1 Phase 3 Safety Through Week 24 300 mg QW 900 mg Q4W Delayed Treatment Participants who experienced, n (%) N=59 N=65 N=29 AEs Any 27 (45.8) 40 (61.5) 8 (27.6) Related to treatment 14 (23.7) 22 (33.8) 0 Grade 3+ 0 0 1 (3.4)* Serious 0 0 1 (3.4)* AE leading to discontinuation of study drug, n (
Slide 9: AZURE-1 Phase 2b: Responses Continue to Deepen at Week 48
AZURE-1 Phase 2b: Responses Continue to Deepen at Week 48 No New Safety Signals Observed Through 48 Weeks of Treatment 300 mg QW 900 mg Q4W N=20a N=20 24 wks 48wks 24 wks 48 wks Primary Endpoint 45% 35% 55% 55% (Virologic Response + ALT normalization) p = 0.003 p = 0.024 Virologic response 100% 95%b
Slide 10: Ongoing Brelovitug Phase 3 Trials Supporting FDA and EMA Submissions
Ongoing Brelovitug Phase 3 Trials Supporting FDA and EMA Submissions No ALT Limitation for Study Participation 24wk FDA Registration AZURE-1 n~200 300mg 1x Weekly Extension (96wks) Enabling Studies 2:2:1 vs 900mg 1x Monthly vs Delayed Tx Start Delayed Tx→ 300mg 1x Weekly Primary Endpoint Topline Dat
Slide 11: Well-Positioned to Execute on Our Planned Strategy
Well-Positioned to Execute on Our Planned Strategy 5 potential approval and label expansion opportunities expected 2026 2027 2026 FY Guidance VISTAS (PSC) positive topline results VANTAGE (PBC) topline results in Q1 AZURE-1 (HDV) positive interim analysis Brelovitug HDV BLA submission in H1
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