Fostroxacitabine bralpamide

Study closed on November 26th, 2024, end-of-treatment data to be presented at an upcoming scientific congress. Patients have stayed on treatment longer than anticipated and the 3 remaining patients have each been on treatment for at least 15 months. The patient with the longest treatment time in the study shows sustained partial response after more than 27 months. Stockholm, Sweden — Medivir AB (Nasdaq Stockholm: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, today announced that the phase 1b/2a study of fostroxacitabine bralpamide (fostrox) + Lenvima® in 2nd or 3rd line advanced liver cancer (HCC), closed on November 26th. The 3 remaining patients in the study have been transferred to compassionate use, allowing continued benefit from the treatment. At the ESMO congress in Barcelona in September, the company presented positive, mature data showing an encouraging tolerability profile and improved outcomes beyond what can be expected from current alternatives in second-line advanced HCC. Patients in the study showed extended duration of benefit, resulting in longer than expected treatment time, as evidenced by a median Time to Progression of 10.9 months. This is substantially longer than previously seen in second-line HCC. With the study now closed, the company plans to present end-of-treatment data at an upcoming scientific congress. - "The objectives of the phase 1b/2a study, evaluating safety and preliminary efficacy of fostrox in combination with Lenvima, were met. Mature data presented at ESMO this year showed that the combination is safe and tolerable with encouraging anti-tumor activity. These results indicate improved outcomes beyond current second-line alternatives in advanced HCC and with a compassionate use program in place, the remaining patients can continue benefitting from the treatment. We can now close the study and focus all our efforts on the planned randomized phase 2b study of fostrox in combination with Lenvima as we strive to make the combination the first, approved treatment option in second-line advanced HCC”, says Dr. Pia Baumann, CMO at Medivir. For additional information, please contact; Magnus Christensen, CFO, Medivir AB Telephone: +46 8 5468 3100 E-mail: magnus.christensen@medivir.com About fostrox Fostrox is a liver-targeted inhibitor of DNA replication that delivers the cell-killing compound selectively to the tumor while minimizing the harmful effect on normal cells. This is achieved by coupling an active chemotherapy (troxacitabine) with a prodrug tail. This design enables fostrox to be administered orally and travel directly to the liver where the active substance is released locally in the liver. With this unique mechanism, fostrox has the potential to become the first liver-targeted, orally administered drug that can help patients with various types of liver cancer. A phase 1b monotherapy study with fostrox has been completed and a phase 1b/2a combination study in HCC is ongoing where it has shown encouraging anti-cancer efficacy with a good safety and tolerability profile (ref Chon et al., ESMO 2024, Poster 986). About primary liver cancer Primary liver cancer is the third leading cause of cancer-related deaths worldwide. Hepatocellular carcinoma (HCC) is the most common cancer that arises in the liver and it is the fastest growing cancer in the USA. 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 percent [1], [2]. 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 drug candidate designed to selectively treat cancer cells in the liver 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. 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. 2) Yang, J.D., Hainaut, P., Gores, G.J. et al. A global view of hepatocellular carcinoma: trends, risk, prevention and management. Nat Rev Gastroenterol Hepatol 16, 589–604 (2019). Attachments Medivir announces finalisation of the phase 1b/2a study with fostrox + Lenvima in advanced liver cancer, remaining patients transferred to compassionate use

”The improved results presented at ESMO and the collaboration agreement with Eisai have strengthened fostrox's chances of becoming the first approved therapy in second-line liver cancer”. July – September Financial summary for the quarter Net turnover amounted to SEK 0.9 (0.8) million. The loss before interest, tax, depreciation and amortization (EBITDA) amounted to SEK -35.1 (-23.4) million. Basic and diluted earnings per share amounted to SEK -0.30 (-0.42). Cash flow from operating activities amounted to SEK -33.4 (-21.0) million. Cash and cash equivalents at the end of the period amounted to SEK 92.6 (61.1) million. Significant events during the quarter At the ESMO Cancer Congress in Barcelona in September, Medivir presented updated clinical data from the phase 1b/2a study with fostrox in the second-line treatment of advanced liver cancer. Results showed a median time to progression (TTP) of 10.9 months[1], and an Objective Response Rate (ORR) of 24% with a median duration of response of 7.0 months (4.1 – 18.1). January – September Financial summary for the period Net turnover amounted to SEK 2.5 (3.2) million. The loss before interest, tax, depreciation and amortization (EBITDA) amounted to SEK -98.4 (-68.5) million. Basic and diluted earnings per share amounted to SEK -0.85 (-1.23). Cash flow from operating activities amounted to SEK -94.8 (-55.1) million. Cash and cash equivalents at the end of the period amounted to SEK 92.6 (61.1) million. Events after the end of the period In October, Medivir received a loan facility of SEK 30 million from Linc AB. The loan will only be used if necessary, as a secondary financing option. Priority will be given to other financing options such as issues or partnering agreements. In October, Medivir's nomination committee was appointed for the 2025 annual general meeting. The nomination committee consists of Karl Tobieson, appointed by Linc AB, Richard Torgerson, appointed by Nordea Funds AB, Anders Hallberg, appointed by Hallberg Management AB and Uli Hacksell, chairman of the board of Medivir AB. In October, the results of the phase 1 study, which showed proof-of-concept with fostrox monotherapy in liver cancer, were published in the Journal of Hepatocellular Carcinoma. In November, Medivir entered into a new collaboration and supply agreement with Eisai to evaluate fostrox in combination with Lenvima in a randomized phase 2b study in advanced liver cancer. Conference call for investors, analysts and the media The Interim Report January - September 2024 will be presented by Medivir’s CEO, Jens Lindberg. Time: Wednesday, November 6, 2024, at 14.00 (CET). To access the webcast and find information about the teleconference, please klick HERE! The conference call will also be streamed via a link on the website;www.medivir.com/investors/calendar The presentation will be available on Medivir’s website after completion of the conference. CEO’s message Today, there are no approved drugs for the second-line treatment of advanced liver cancer. At the ESMO congress in Barcelona in September, we presented data indicating that the combination fostrox + Lenvima® is significantly better than the drugs used today in the second line, including Lenvima. These results position the fostrox + Lenvima combination at the forefront of becoming the first approved treatment for these patients. An opportunity strengthened by the collaboration agreement with Eisai. Results from our ongoing phase 1b/2a study of fostrox + Lenvima in advanced liver cancer are very promising. The median time to progression (TTP) of 10.9 months[1] (4.1 – 18.1) is significantly longer than previously seen in second-line liver cancer and the combination shows an Objective Response Rate (ORR) of 24% with a median duration of response of 7.0 months. This should be viewed in relation to the dismal prognosis second-line liver cancer patients generally have, with a treatment response of 5 – 10%, and an expected median TTP of only 3 – 4 months. The interest in our study results has increased as the data has matured and continuously demonstrated an improved clinical benefit. At ESMO in Barcelona, investigators from countries we had not previously had a dialogue with showed a strong interest in fostrox and the planned phase 2b study. The use of first-line immunotherapy combinations has improved treatment outcomes and resulted in more patients being in a better condition to undergo second-line treatment. However, without access to approved therapies after immunotherapy, the need for new treatment options is substantial for these patients. We have designed fostrox to target only tumor cells locally in the liver, without harming healthy cells. The tolerability profile in the study confirms the tumor-selective effect and enables patients to continue the treatment for a long time, which in itself contributes to prolonged clinical benefit. The targeted anti-tumor effect in the liver was also confirmed in the phase 1 study with fostrox monotherapy, which recently was published in the Journal of Hepatocellular Carcinoma. It is thus with strong confidence that we continue preparations to start the planned phase 2b study where fostrox + Lenvima is compared with Lenvima alone in a randomized study to confirm the effectiveness of the combination. We are very pleased with the collaboration and supply agreement with Eisai we entered into in November. In addition to Eisai contributing Lenvima to the study, we will, as part of the agreement, form a Joint Development Committee with Eisai for planning and execution of the study. The collaboration and engagement by Eisai further validate the ability of the combination to provide increased patient benefit. Preparations are proceeding according to plan and we intend to open an IND in the US in the fourth quarter of 2024. In October, Medivir received a loan facility of SEK 30 million from Linc AB. The loan will only be used if necessary, as a secondary financing option. Priority will be given to other financing options such as issues or partnering agreements. There is a clear need and an obvious position for fostrox in the treatment landscape. The data presented indicate that fostrox + Lenvima could become the first approved drug therapy in second-line liver cancer - a market worth ~$2.5 billion annually. Preparations for the planned phase 2b study are progressing according to plan. I look forward to continuing to keep you informed of Medivir's exciting developments. Jens Lindberg Chief Executive Officer This report has been subject to auditors' review. For additional information, please contact; Magnus Christensen, CFO, Medivir AB Telephone: +46 8 5468 3100 E-mail: magnus.christensen@medivir.com 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 drug candidate designed to selectively treat cancer cells in the liver 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. Medivir’s share (ticker: MVIR) is listed on Nasdaq Stockholm’s Small Cap list. www.medivir.com. 1) Chon et al., ESMO 2024, Poster 9864 Attachments Medivir AB - Interim Report, January-September 2024