Fostrox + Lenvima continues to show promising tumor control in HCC, all patients have now dosed at least two treatment cycles

• Early and confirmed anti-tumor activity, 16 of 20 patients with disease control at first scan and sustained tumor control for 13 of 18 patients with >3 months follow-up
• Durable clinical benefit, median time to progression (to date) ~4.5 months with >50% of patients still on treatment
• Consistently good safety and tolerability profile

STOCKHOLM, Oct. 5, 2023 /PRNewswire/ -- Medivir AB (Nasdaq:MVIR) (Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, announced today that all patients in the ongoing phase 2a study in advanced hepatocellular carcinoma (HCC) has now dosed at least two treatment cycles and the treatment continues to show very promising clinical benefit. The study evaluates safety and efficacy with the candidate drug fostrox in combination with Lenvima® in patients for whom current first- or second-line treatment has proven ineffective or is not tolerable.

The study, which is an open-label, multi-center, dose-escalation and dose-expansion study, continues to show early and durable clinical benefit across the cohort, as measured by local review. Of the 13 patients with sustained tumor control at 3 months, 3 patients achieved Partial Response and 10 patients Stable Disease, providing 17% Overall Response Rate (ORR) and 72% Disease Control Rate (DCR). The patient who has benefited the longest remains on treatment after 13.5 months, with sustained partial response.

The combination remains tolerable with no unexpected new safety events and adverse events are transient and manageable. Continued lower need for dose reductions or interruptions than expected. The observed tolerability is of outmost importance as patients with HCC often have an increased sensitivity to drugs in general due to impaired liver function.

"Overall, the consistent early and durable signals of clinical benefit and the favorable safety profile are very encouraging. Patients with advanced HCC, who have progressed on at least one line of treatment, are a very difficult-to-treat population with few effective treatment options. The overall response rate of 17% already at 2^nd scan is very encouraging and indicates a higher share of responders than historically seen in second line treatment of HCC. We can also see that for many patients, their target lesions continue to shrink with longer follow-up," says Dr. Pia Baumann, CMO at Medivir.

Medivir plans to provide an update in connection with the company's upcoming quarterly results presentation on Friday October 27. This will include a more comprehensive update of local review data as most of the patients will have had the opportunity to have at least three scans, with each scan scheduled to take place every 6 weeks. In addition to the data update, the company will also provide further context what these more mature data could mean for future clinical practice in second line HCC, a patient population for whom there are no approved medical treatments today.

For additional information, please contact;

Magnus Christensen, CFO, Medivir AB
Telephone: +46 8 5468 3100
E-mail: [email protected]

About fostrox

Fostrox is an oral pro-drug, designed to selectively treat liver cancers and to minimize side effects. It has the potential to become the first liver-targeted and orally administered drug for patients with HCC. Fostrox has completed a phase 1b monotherapy study, and a combination study in HCC currently ongoing.

About primary liver cancer

Primary liver cancer is the third leading cause of cancer-related deaths worldwide and hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver. Although existing therapies for advanced HCC can extend the lives of patients, treatment benefits are insufficient and death rates remain high. There are approximately 660,000 patients diagnosed with primary liver cancer per year globally and current five-year survival is less than 20 percent¹⁾. HCC is a heterogeneous disease with diverse etiologies, and lacks defining mutations observed in many other cancers. This has contributed to the lack of success of molecularly targeted agents in HCC. The limited overall benefit, taken together with the poor overall prognosis for patients with intermediate and advanced HCC, results in a large unmet medical need.

About Medivir

Medivir develops innovative drugs with a focus on cancer where the unmet medical needs are high. The drug candidates are directed toward indication areas where available therapies are limited or missing and there are great opportunities to offer significant improvements to patients. Medivir is focusing on the development of fostroxacitabine bralpamide (fostrox), a pro-drug designed to selectively treat liver cancer and to minimize side effects. Collaborations and partnerships are important parts of Medivir's business model, and the drug development is conducted either by Medivir or in partnership. Birinapant, a SMAC mimetic, is exclusively outlicensed to IGM Biosciences (Nasdaq: IGMS) to be developed in combination with IGM-antibodies for the treatment of solid tumors. Medivir's share (ticker: MVIR) is listed on Nasdaq Stockholm's Small Cap list. www.medivir.com.

1) Rumgay et al.,European Journal of Cancer 2022 vol.161, 108-118. 

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SOURCE Medivir