The key outcome of the Type C meeting was that
Firstly, as
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
- "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
- "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
For additional information, please contact
Telephone: +46 8 5468 3100
E-mail: magnus.christensen@medivir.com
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
1) Rumgay et al.,
https://news.cision.com/medivir/r/medivir-provides-update-from-supportive-type-c-meeting-with-the-fda--preparations-for-enhanced-phase,c3965751
https://mb.cision.com/Main/652/3965751/2751026.pdf
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