Mirum Pharmaceuticals, Inc. · NASDAQ:MIRM

Mirum Pharmaceuticals — Corporate Update (October 2026)

13 slides from Mirum Pharmaceuticals's corporate update, Oct 12, 2026

  1. Slide 1: Mirum Pharmaceuticals EXPAND Phase 3 Topline Results

    October 2026 Mirum Pharmaceuticals EXPAND Phase 3 Topline Results MIRUM PHARMACEUTICALS

  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

  3. Slide 3: IBAT Inhibition in Pediatric Cholestatic Liver Disease

    IBAT Inhibition in Pediatric 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 addresses he

  4. Slide 4: LIVMARLI Label Expansion Opportunity in Cholestatic Pruritus

    LIVMARLI Label Expansion Opportunity in Cholestatic Pruritus Broad Unmet Need in Multiple Ultra-Rare Cholestatic Conditions Pruritus in Ultra-Rare Characteristic Estimated Addressable Cholestatic Settings Cholestatic Burden US/EU Biliary Atresia Patients1 Elevated sBA e.g., Biliary Atresia, Secondar

  5. Slide 5: EXPAND Phase 3 Study

    EXPAND Phase 3 Study Key Inclusion Criteria 20wk +20wks • Diagnosis of cholestatic liver disease excluding ALGS, PFIC, Maralixibat 285 ug/kg BID1 PSC, PBC, and ICP Maralixibat n=52 Open Label Extension • Moderate to severe cholestatic Placebo pruritus • Total serum bile acids >2×ULN Primary Endpoint

  6. Slide 6: EXPAND Phase 3 Baseline Demographics – All Pediatric Population

    EXPAND Phase 3 Baseline Demographics – All Pediatric Population MRX Placebo (N=22) (N=24) Age at enrollment in years (SD) 6.6 (4.4) 6.0 (4.0) Sex (Female %) 10 (45.5) 11 (45.8) ItchRO(Obs) Score (SD) 2.65 (0.96) 2.65 (0.70) sBA, mol/L (SD) 149.7 (92.9) 155.4 (98.6) ALT, U/L (SD) 95.4 (59.4) 134.2 (

  7. Slide 7: CFB in ItchRO(Obs) Improvement: Primary Endpoint Met

    EXPAND Phase 3 Primary Endpoint Met Rapid, Sustained, Clinically Meaningful Reductions in Pruritus; sNDA Submission Planned 1H 2027 Average Pruritus Score (ItchRO[Obs]) Over 20 Weeks (All Pediatric Population) Biliary Atresia Subgroup 0 -1.15 CFB in ItchRO(Obs)2 PBO-adjusted reduction; p=0.0002 -0.5

  8. Slide 8: EXPAND Phase 3 Safety Summary (Safety Population)

    EXPAND Phase 3 Safety Summary (Safety Population) MRX Placebo n (%) (n=25) (n=27) Participants with any treatment-emergent adverse event (TEAE) 24 (96.0) 26 (96.3) Grade 3 or higher TEAEs 6 (24.0) 6 (22.2) Related to study drug 1 (4.0) 0 Serious TEAEs 5 (20.0) 5 (18.5) TEAE that led to death 0 0 TEA

  9. Slide 9: Expanding Impact in Rare Cholestatic Pruritus

    Expanding Impact in Rare Cholestatic Pruritus Path to Sustained Long-Term EXPAND Growth PFIC Phase 3 sNDA submission targeted H1 ‘27 ✓ Continued growth in new Rx Biliary Atresia: ~2,000 US/EU addressable ✓ Strong adherence & persistence ✓ Weight-based dosing ALGS Approved ✓ Int’l market expansion 4,

  10. Slide 10: Delivering High Impact Medicines for Rare Disease

    MIRUM PHARMACEUTICALS Delivering High Impact Medicines for Rare Disease 10

  11. 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

  12. Slide 12: Thank You

    THANK YOU mirumpharma.com ©2026 Mirum Pharmaceuticals, Inc. All rights reserved. All service marks, trademarks and tradenames appearing in this present ation are the property of their respective owners. Solely for convenience, the trademarks and tradenames referred to in this presentation appear wit

  13. Slide 13: LIVMARLI Important Safety Information

    LIVMARLI Important Safety Information IMPORTANT SAFETY INFORMATION LIVMARLI can cause serious side effects, including: Liver injury: Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In

Mirum Pharmaceuticals — Corporate Update (October 2026) on 6ix