STIFEL 2026 HEALTHCARE CONFERENCE - MIRUM PHARMACEUTICALS INC

Financial Performance:

• Raised full-year revenue guidance to $715–$720 million, citing strong momentum across all four approved medicines.
• Projected to achieve cash flow positivity in 2027; currently cash flow positive in the first half of the year, with expected negative cash flow in the second half due to heavy R&D and CMC investments for Berlovitug and Velixibat filings.
• Raised 2027 commercial peak revenue guidance for Berlovitug (HDV) to >$1 billion (up from $750 million), primarily driven by higher assumed pricing relative to competitor Hepcludex.
• Commercial business (Livmarly) driving rapid growth; AllerGEAL is ~50% penetrated and growing steadily, while PFIC (adult/pediatric) remains a significant growth engine.
• FOP guidance stands at >$200 million, leveraging a highly engaged, diagnosed patient base of ~900 worldwide.

Guidance and Future Outlook:

• Revenue growth expected to continue in 2027, with significant contributions anticipated from the recently approved Berlovitug and FOP.
• Berlovitug (HDV) filing targeted for the first half of next year, following Phase 3 studies (Azure 1 & 4) meeting primary endpoints at 24 weeks.
• Velixibat (PBC) data expected in Q1 2027, with a pivotal study design confirmed via FDA Breakthrough Therapy designation interactions.
• Long-term commercial strategy involves expanding sales force by ~40 reps by year-end to target undiagnosed adult PFIC and new indications.
• Management expects to dominate the HDV market alongside competitor Verve, anticipating significant market expansion via reflex testing as more drugs enter the space.

Business Segments and Product Lines:

• Livmarly (MIREP & PFIC): Two approved indications (AllerGEAL, PFIC). EXPAND Phase 3 data (biliary atresia) showed clear benefit, potentially adding ~1,000 addressable patients in the US.
• Fibromodulin (FOP): Recently approved; early launch data is encouraging in a fixed, diagnosed population.
• Berlovitug (HDV): Phase 3 data (up to 108 weeks) shows 100% ALT normalization, 100% virologic response, and >70% TND rates; filing planned for H1 next year.
• Velixibat (PBC/PSC): Interim data for PBC showed >2-point placebo-adjusted itch improvement; PSC filing delayed to allow further FDA interaction before NDA submission.
• Sales Force: Expanding to cover adult PFIC, Berlovitug, and Velixibat; currently covering only top decile of adult hepatologists in PFIC space.

Market and Competitive Landscape:

• HDV: Addressable market estimated at 15,000 diagnosed patients in the US (growing to potentially millions via reflex testing); 2 million Hep B patients exist in the US, with a small fraction currently tested for HDV.
• PSC: ~50,000 US patients, ~60% suffering from significant itch; ~70% of PSC patients also have IBD (Velixibat data shows no exacerbation risk).
• PFIC: Adult PFIC opportunity estimated at 2,000 patients in the US; currently low diagnosis rates with ~50/50 market share split between Livmarly and competitor Bylvay (Ipsen).
• Competition: HDV market to be shared with Verve; PBC market includes PPAR agonists which do not universally address itch.
• Pricing: Berlovitug pricing assumed higher than Gilead's Hepcludex (currently ~$200M run rate ex-US); specific Velixibat pricing strategy TBD based on data and dynamics.

Risks and Challenges:

• Regulatory Uncertainty: PSC filing timeline dependent on FDA interaction outcomes; Velixibat PBC labeling implications for biliary atresia still under FDA discussion.
• Diagnosis Rates: Significant upside for PFIC and HDV relies on increased genetic and reflex testing adoption, which is currently in "early days."
• Clinical Endpoints: HDV TND is not a cure; viral rebound possible upon withdrawal, necessitating long-term maintenance (96 weeks) before considering therapy cessation.
• Patent Litigation: Paragraph IV patent litigation expected, with Markman hearing anticipated next summer, potentially delaying generic entry.

Management Commentary and Tone:

• Tone is highly confident regarding commercial momentum and pipeline depth, emphasizing "rapid revenue growth" and "strong momentum."
• Management views the HDV space as a "patient finding" opportunity where multiple companies benefit from increasing awareness and testing infrastructure.
• Expressed strong conviction in the clinical profile of Berlovitug (long-term data) and Velixibat (safety in IBD patients).
• Acknowledged heavy R&D investment in the second half of the year as a necessary step to set up future growth and filings.

Analyst Questions and Answers:

• FDA Interaction (PSC): FDA recommended Phase 3 at pre-NDA, but Miriam chose to delay filing to align the review team with their data package; no specific data issues were cited.
• Virologic Endpoints: Clarified that while assays vary, the key clinical goal is virus suppression and ALT normalization; TND is the current objective before attempting withdrawal.
• Market Size (HDV): Baseline 15,000 patients; potential expansion to millions as testing practices shift from risk-based to reflex testing post-approval of multiple therapies.
• Competitive Pricing: Berlovitug guidance increase driven by Hepcludex pricing; competition with Verve expected to be complementary in driving market education.
• Livmarly IP: Dosing patents secured through 2040, based on the unique plateau mechanism of iBAT inhibition; solid position against future generics.

Other Key Points:

• Safety Profile: Velixibat showed no IBD exacerbation in PSC patients with comorbid IBD, addressing a key clinical concern.
• Biliary Atresia: EXPAND data supports a new indication, filling a gap left by Ipsen's failed biliary atresia study; opportunity size comparable to AllerGEAL.
• Commercial Synergy: Expansion of the adult sales force will create leverage for the entire portfolio (Berlovitug, Velixibat, FOP) by targeting undiagnosed hepatology patients.
• Global Reach: FOP has a highly engaged global population, while HDV and PSC opportunities are significant both in the US and internationally.