Medivir has received FDA approval for its Investigational New Drug (IND) application to evaluate fostrox in combination with Lenvima in a randomized phase IIb study. The study targets patients with advanced hepatocellular carcinoma (HCC) who have previously been treated with immunotherapy.
The Phase IIb study builds on promising results from the recently completed Phase Ib/IIa study, where the combination demonstrated manageable safety and encouraging antitumor activity, including a median time to progression (TTP) of 10,9 months. Medivir plans to recruit participants from eight countries in the US, Europe and Asia, with results expected in 2027.
The company's focus on liver cancer is based on the disease's growing global impact, with HCC being the fastest growing cancer in the United States and among the leading causes of cancer-related deaths worldwide. Current HCC treatments provide limited results, with a five-year survival rate of less than 20 percent, highlighting the need for new treatment approaches.
What the Phase IIb study involves
The study will compare fostrox plus Lenvima to Lenvima alone in patients with advanced liver cancer who are no longer responding to first-line immunotherapy. The primary endpoint of the study is Objective Response Rate, with secondary endpoints including duration of response (DoR), progression-free survival (PFS), overall survival (OS), and safety and quality of life.
The study will be led by the principal investigator Prof. Maria Reig, a globally recognized expert and head of Barcelona Clinic Liver CancerProf. Reig emphasizes the critical need for effective and liver-tolerable therapies in this patient population, and that fostrox shows potential to fill this gap without compromising liver function.
CEO comments
We contacted Medivir's CEO Jens Lindberg to discuss the significance of the IND approval and its effect on partnering discussions.
Jens, can you tell us about the significance of the FDA's IND approval?
– The IND approval is significant because it means the FDA has approved our proposed study design and it allows us to enroll US patients in the Phase IIb study. The FDA is at the forefront of enabling innovative treatments that fill significant unmet medical needs and can reach the market faster. However, to qualify for programs such as Breakthrough Therapy Designation and Accelerated Approval, the study must enroll US patients, which is why this IND approval is so important.
How does fostrox address the challenges of preserving liver function in HCC patients?
– Fostrox has been designed to selectively kill cancer cells in the liver. The cell-killing effect of fostrox will only affect cells that are dividing, which is key. The reason for this is that while healthy liver cells divide very rarely, cancer cells in the liver divide much more frequently. As a result, cancer cells will be killed as soon as they divide and are exposed to fostrox, while healthy cells that do not divide are not affected. As the liver is a critical organ that, among other things, metabolizes food and transports toxins from the body, it is especially important not to damage normal liver function.
What led to the decision to conduct the study in several countries on three continents?
– The great medical need is a global challenge. It is one of the deadliest and fastest growing cancers, largely driven by obesity and fatty liver, which significantly increases the risk of liver cancer. It has therefore been important for us to design a global study that includes patients of all ethnicities to ensure that we maximize the chance of early regulatory approvals in all regions. Including patients in the US, Europe and Asia will also make fostrox more attractive for potential partner discussions.
What role do you think fostrox will play in the broader treatment landscape for HCC?
– Fostrox is unique in several ways and has the potential to change the second-line treatment of liver cancer. It is the only oral, liver-targeted treatment in development and its mechanism of action that selectively kills cancer cells locally in the liver is also unique. It therefore has the potential to become the first, approved treatment option in combination with Lenvima in second-line liver cancer. Since fostrox is the only drug that targets the liver, it has the potential to be combined with all other treatment options used in liver cancer, including previous lines of treatment.
Finally, what impact could IND approval have on your partnering processes?
– This is an important step. The commercial opportunity in the US is significant and it is the best way to reach the market as quickly as possible. Therefore, the ability to include US patients and the potential for accelerated approval will also be very valuable components for a potential partner.