Medivir (MVIR) Carnegie Healthcare Day 2025 summary

Market opportunity and disease landscape

• Second-line hepatocellular carcinoma (HCC) market projected to grow from $2.8bn in 2024 to $4.0bn by 2030, driven by rising fatty liver disease and improved first-line treatments.

• Fatty liver disease prevalence is increasing, raising liver cancer risk 17-fold and affecting over 25% of US adults.

• No approved second-line treatments exist after immunotherapy combinations, highlighting a significant unmet need.

Fostrox mechanism and clinical results

• Fostrox is the first oral, liver-targeted prodrug designed to selectively kill tumor cells while sparing healthy liver tissue.

• Combination of fostrox + Lenvima demonstrated a median time to progression (TTP) of 10.9 months and median overall survival (OS) of 13.7 months, outperforming Lenvima monotherapy.

• Tumor reduction was observed regardless of fostrox dose reduction or baseline AFP levels.

• Stable liver function was maintained during treatment, as shown by ALT, AST, and ALBI score monitoring.

Clinical development and regulatory progress

• Positive phase 1b/2a data presented at EASL Liver Cancer Summit; IND approval obtained for global phase 2b FOCUS-2 study.

• FOCUS-2 is a randomized, multi-country trial comparing fostrox + Lenvima to Lenvima alone, with ORR as the primary endpoint and readout expected in 2027.

• Study design includes dose optimization, interim analysis, and is powered for accelerated approval and potential breakthrough therapy designation.

• Preparations include site selection, regulatory submissions, and drug supply readiness across 40 sites in 8-9 countries.