Mirum Pharmaceuticals (MIRM) H.C. Wainwright 28th Annual Global Investment Conference summary

Commercial performance and strategy

• Achieved $176.2 million in Q2 revenue, raising full-year guidance to $680–$700 million.

• LIVMARLI drives growth, with strong performance in Alagille syndrome and PFIC, and a 13% sequential revenue increase in Q2.

• Commercial expansion targets broader adult PFIC market, aiming to reach 2,000 potential adult patients in the U.S.

• International growth expected, with focus on Europe, Latin America, and the Middle East through distributors.

• Commercial team expansion underway, with full deployment expected by end of year or early next.

Pipeline and clinical milestones

• Near-term catalysts include PDUFA date for zilurgisertib (FOP), AZURE-1 and AZURE-4 readouts for hepatitis delta, and EXPAND basket study data.

• LIVMARLI label expansion and genetic testing initiatives are increasing adult PFIC diagnoses and prescriptions.

• EXPAND study targets rare cholestatic pruritus indications, with half of enrollment in biliary atresia; unblinding expected next quarter.

• Volixibat pivotal studies for PSC and PBC are adaptive in design, with NDA submission for PSC planned in first half of next year.

• VANTAGE study for PBC showed strong interim results, supporting breakthrough designation and pivotal status.

Competitive positioning and differentiation

• LIVMARLI differentiates through pronounced itch reduction, strong patient persistence, and favorable tolerability.

• Volixibat targets both biochemically controlled and second-line PBC patients, with consistent pruritus response.

• Zilurgisertib offers once-daily oral dosing for FOP, with pivotal data in patients as young as 12 years old.

• Ongoing efforts to build awareness of genetic testing in adult cholestatic disease, supported by academic research.