STIFEL 2026 HEALTHCARE CONFERENCE - MIRUM PHARMACEUTICALS INC

Explicit Guidance:

• Revenue: FY2024 revenue guidance raised to $715–$720 million, reflecting growth from all approved medicines and strong momentum into FY2025.
• Revenue (New Assets): FY2027+ revenue from Berlovitag (HDV) projected to exceed $1 billion annually (raised from previous $750 million guidance), driven by pricing assumptions exceeding Gilead's Hepcludex ex-U.S. run rate; management indicates a tendency to "lean to the plus" over time.
• Cash Flow: Projected to achieve cash flow positivity in FY2027; cash flow was negative in FY2023, positive in the first half of FY2024, and anticipated to be negative in the second half of FY2024 due to heavy R&D and CMC investments for upcoming filings.
• Share Count/Dilution: No explicit new guidance on share count, but management notes a "heavy R&D lift" and significant investments ahead of FY2027.

Business-Segment Outlook:

• Berlovitag (HDV): Filing planned for H1 of next year (FY2025) following pre-NDA feedback; Phase 3 "Azure One" and "Azure Four" studies met primary endpoints. Data tracked up to 108 weeks show 100% ALT normalization and >70% Treatment-Not-Detectable (TND) rates. Commercial target market currently estimated at 15,000 diagnosed U.S. patients, with significant upside potential as reflex testing increases (similar to the European market post-Hepcludex).
• Felixibat (PSC & PBC):
  • PSC: Filing targeted for H1 of next year (FY2025); FDA feedback suggests a lack of familiarity with the program rather than specific data gaps. Addressable U.S. population estimated at 50,000 patients, with ~60% suffering from significant itch.
  • PBC: Data readout expected in Q1 2027. Interim data showed >2-point pruritus improvement; final data submission to follow.
• Livmarli: Revenue growth expected to continue without plateau. Key drivers include adult PFIC diagnosis (addressable U.S. population ~2,000, currently under-diagnosed) and Biliary Atresia expansion (addressable ~1,000 U.S. patients, no direct competition). Sales force expansion of ~40 reps to be fully operational by year-end.
• FOP (Algeol): Recent launch showing high engagement; first-week performance encouraged. Addressable U.S. population estimated at ~300 patients (900 globally), considered a diagnosed, ready-to-treat market.

Market and Competitive Outlook:

• HDV Market: Anticipated to become a "patient finding" opportunity where multiple approved therapies (Berlovitag, Vird, Gilead) drive market education and testing volumes. Management expects to dominate the market alongside competitors, though specific market share splits are premature to speculate.
• PPAR Competition (PBC): Berlovitag and Felixibat are positioned to capture market share from PPARs, which do not universally address pruritus. Felixibat targets patients well-controlled on UDCA who still have itch (off-label PPAR users) plus those suboptimally controlled.
• Livmarli vs. Bilve: Market share currently estimated at ~50/50 in the adult PFIC space; management aims to increase share by expanding sales force coverage beyond the top decile of hepatologists.
• Pricing Dynamics: Pricing strategy for Felixibat to be determined post-data; potential prioritization of PSC (monotherapy) could lead to "step-throughs" into the PBC market. Berlovitag pricing assumptions already factored into the >$1B guidance based on Hepcludex pricing.

Macro / Industry Outlook:

• Testing Adoption: Anticipated shift from risk-based to reflex testing for Hepatitis D (HDV) following drug approvals, significantly expanding the total addressable market beyond the current 15,000 baseline.
• Regulatory Environment: FDA is engaging closely with Miriam's programs (Breakthrough Designations for PSC and PBC); management expects standard review timelines with some overlap between PSC and PBC reviews.
• Patent Protection: Core dosing patents for Livmarli extend to 2040, with a solid dose patent extending to 2043. A Markman hearing is anticipated next summer.

Strategic Initiatives:

• Sales Force Expansion: Adding ~40 sales representatives by year-end to target adult PFIC, adult HDV, and PBC markets, leveraging existing Livmarli infrastructure.
• R&D & CMC Investments: Heavy investment in CMC (Chemistry, Manufacturing, and Controls) in H2 FY2024 to support filings for Berlovitag and Felixibat, targeting cash flow positivity by FY2027.
• Market Development: Joint initiatives with partner Ipsen to facilitate genetic testing adoption in adult hepatology clinics to accelerate PFIC diagnosis.
• M&A/Partnerships: No specific new M&A discussed; focus remains on internal pipeline execution and strategic commercial expansion.

Risks and Headwinds to Outlook:

• Regulatory Uncertainty: FDA feedback on PSC (Phase 3 recommendation) indicates potential for iterative discussions before submission, though no specific data gaps were identified.
• Competitive Pricing: Berlovitag guidance relies on pricing assumptions higher than Gilead's Hepcludex; actual pricing dynamics and market share splits remain speculative.
• IP Litigation: Paragraph IV patent challenges and Markman hearings could create future uncertainty, though management views the current portfolio as "solid."
• Diagnostic Lag: Growth in adult PFIC is contingent on increased diagnosis rates and genetic testing adoption, which management views as "early days" in the adult market.
• Safety/Tolerability: While flu-like symptoms are low, patient tolerability and real-world adherence remain variables in treatment decisions.

Management Tone on the Future:

• Confidence: High confidence in the robustness of data packages for PSC and HDV; described PSC interim data as "reassuring" regarding IBD exacerbation risks.
• Growth Narrative: Strong optimism regarding "non-plateauing" growth for Livmarli and significant upside in undiagnosed adult populations (PFIC, HDV, PBC).
• Strategic Positioning: Expects the HDV market to be a "win-win" for multiple players, driving overall disease awareness and patient identification. Management expressed confidence in dominating the HDV market alongside competitors.
• Hedges: Frequent qualifiers regarding "patient finding" dynamics and the timeline for widespread testing adoption; noted that pricing and market share splits in HDV are "way too early to speculate."

Miscellaneous:

• Timeline Overlaps: PSC filing expected to precede PBC filing (Q1 2027), but review processes may overlap.
• Clinical Endpoints: Emphasis on "finite therapy" and "cure" as ultimate objectives, though current regulatory path focuses on TND and ALT normalization maintained for 96 weeks.
• Market Dynamics: Management notes that the presence of Gilead in HDV is "not a very high priority" currently but plans to monitor quarterly progress.