Fostrox + Lenvima® holds promise of greatly improved outcomes for advanced liver cancer patients, reveals new data from Medivir at ESMO GI

• New results from Medivir’s Phase 1b / 2a open label trial of fostrox + Lenvima® show great promise treating second-line hepatocellular carcinoma (HCC) patients, an extremely hard to treat population
• Fostrox + Lenvima® achieved a 24% overall response rate (ORR) and estimated median time to progression (TTP) of 10.8 months¹ (4.1 – NE) – with one patient still on treatment after 22 months
• Results come despite very poor prognosis for most second-line HCC patients today, with just 5 – 10% responding to current standard of care treatment, and a typical TTP of only 3 - 4 months
• Medivir’s fostrox (fostroxacitabine bralpamide) is an orally-administered, liver-targeted inhibitor of DNA replication. The mechanism is different to existing first-line HCC treatments, delivering its cell-killing compound selectively to tumor cells while minimizing harm to healthy cells

Stockholm, Sweden, June 27, 2024 — Medivir AB (Nasdaq Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, today presented positive new data from its ongoing phase Ib / IIa study of fostroxacitabine bralpamide (fostrox) + Lenvima® in advanced hepatocellular carcinoma (HCC) at the ESMO GI (European Society of Medical Oncology, Gastrointestinal Cancers) Cancers Congress in Munich, Germany.

Today’s ESMO GI update, poster number 176P, is being presented by Dr Hong Jae Chon on Thursday June 27, shows:

• An overall response rate (ORR) of 24%, with a disease control rate (DCR) of 81%, while the median TTP is now 10.8 months, with 25% of patients still on treatment
• One patient remains on treatment after 22 months, benefiting from a sustained partial response
• Biopsies confirm selective DNA damage to tumor cells, while there is no impact on normal liver function as measured by ALT/ AST levels and stable ALBI score over time

The lack of impact on normal liver function supports the previously reported encouraging safety and tolerability profile, where only 5% of patients had to discontinue due to adverse events and the need for dose modification was lower than expected.

Jens Lindberg, CEO at Medivir, said: “With new data, including clear evidence of tumor selectivity, the clinical benefit of adding fostrox to Lenvima has greatly improved. We are particularly encouraged by the duration of benefit, with patients staying on treatment much longer than anticipated. Second-line hepatocellular carcinoma remains an indication with substantial need for improved outcomes for patients, with no approved treatment options after current standard of care. The data presented at ESMO GI makes us even more convinced of fostrox's future potential in the treatment of HCC. We are now initiating study feasibility and finalizing the study protocol and synopsis which will lead to the opening of IND in the US, which is expected in H2 2024.”

Dr Hong Jae Chon, Professor at CHA Bundang Hospital in Korea, and investigator in the fostrox + Lenvima® study, commented: “Advanced hepatocellular cancer is a challenging disease to treat, especially in second-line after patients have stopped responding to an immunotherapy (IO) combination. There is a need for new treatments that do not worsen liver function and that have different mechanisms of action compared with first line. These data for fostrox + Lenvima show highly encouraging clinical benefits for patients. They indicate that when adding fostrox to Lenvima, efficacy is better than expected from Lenvima alone. I look forward to further explore the efficacy of fostrox plus Lenvima in a randomized, controlled trial.”

The data are from Medivir’s ongoing phase 1b/2a open-label, multi-center, dose-escalation and dose-expansion study, evaluating the safety and efficacy of fostrox in combination with Lenvima in patients for whom current first- or second-line treatment has proven ineffective or is not tolerable.

HCC is the most common type of liver cancer, accounting for more than 80% of cases worldwide.² There are approximately 660,000 patients diagnosed with primary liver cancer per year globally and current five-year survival is less than 20 percent³.