Medivir provides update from supportive Type C meeting with the FDA; preparations for enhanced phase 2b in HCC with fostrox + Lenvima continue

STOCKHOLM, April 23, 2024 /PRNewswire/ -- Medivir AB (NASDAQ: MVIR) (STO: MVIR), a pharmaceutical company focused on developing innovative treatments for cancer in areas of high unmet medical need, announced today an update from a Type C meeting with the FDA, regarding the company's plans for a global phase 2b study and opening of an IND for fostroxacitabine bralpamide (fostrox) in the US.

The key outcome of the Type C meeting was that Medivir's preparations for the randomized phase 2b study, comparing fostrox + Lenvima® with Lenvima alone, continues with two key adjustments to the study design.

Firstly, as Medivir plans to use the improved capsule formulation of fostrox in the upcoming phase 2b study, an initial dose run-in as the first part of the study will be added. This means including 20-25 additional patients in an arm with a lower dose, in addition to the 30 mg dose used in the currently ongoing phase 1b/2a study. This change will also further strengthen the study design in relation to FDA's Project Optimus.

Secondly, Objective Response Rate (ORR) is a validated surrogate endpoint for Overall Survival. In prior HCC studies, ORR has supported accelerated approvals. Therefore, the primary endpoint in the enhanced study design will be changed to ORR with key secondary endpoints, including duration of response, progression free survival and overall survival.

The aim of the study, as well as size and study length, remain similar as previously planned and communicated phase 2b design.

The FDA guidance has been discussed with Scientific Advisory Council members and regulatory experts. The next step is to finalize the study design and study protocol before submitting an IND in the US to enable study start in the beginning of 2025.

- "With the outcome of the Type C meeting, we are confidently moving forward with the enhanced phase 2b study design, including the adjustments informed by the discussion with FDA. Our current phase 1b/2a study, in a similar patient population,  is still ongoing and patients are staying on treatment longer than expected. With the planned phase 2b study, we are excited to  move forward with the development of a promising treatment alternative to second line HCC patients," says Pia Baumann,  CMO at Medivir.

- "We continue to accelerate the development plan for fostrox. Engaging with FDA, to ensure best possible study design for the  next phase, is critical for the program and is an important element in partnering discussions. Clarity on study design for phase  2b also allows us to move forward and perform study feasibility with speed as well as finalize CMC preparations," says Jens  Lindberg, CEO at Medivir.

Medivir will provide additional details regarding the updated study design at the Quarterly Results webcast on April 30.

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

About fostrox

Fostrox is a type of smart chemotherapy 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. The prodrug 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.

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 smart, targeted chemotherapy designed to selectively treat liver cancer cells 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.

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