Patients with primary liver cancer, hepatocellular carcinoma (HCC), are typically at an advanced stage of the disease when diagnosed. The good news is that today's first-line treatment, Tecentriq/Avastin, has improved the outlook for many patients. The negative is that many patients stop responding to treatment after a while. For these, there are currently no approved treatments available, that is – no second-line option. This is worrying given that HCC is the third most common cause of cancer-related deaths globally.
Despite this, many pharmaceutical companies continue to focus on developing more first-line options. One exception is Huddinge-based Medivir, who has seen the potential for their drug candidate fostrox in the second line, where competition is largely non-existent.
The goal is to establish fostrox – which has orphan drug status for the treatment of HCC in the US and Europe – as the first approved option for patients where current first-line treatment is ineffective or not tolerated.
Progress in 2023
The safety and tolerability of Fostrox is currently being evaluated in a fully recruited Phase Ib/IIa study in combination with the cancer drug Lenvima. The first patient in the expansion phase was dosed in March last year. Thanks to the great interest among investigators and patients to participate in the study, patients were recruited quickly and after the summer the last patient was included.
The first clinical results indicated good tolerability and promising tumor control, followed by further evaluation that showed, among other things, a complete tumor response and two partial tumor responses in the first six patients.
During the autumn, more mature data from the 18 patients in phase Ib/IIa who had undergone at least 12 weeks of follow-up were presented. The results continued to show clear patient benefit with the fostrox + Lenvima combination. The year ended with a positive so-called Type D meeting with the US Food and Drug Administration FDA regarding the development plan for fostrox before the first data were presented at the ASCO-GI conference in San Francisco. Data that showed further clear signs of clinical benefit and improvement the longer patients are on treatment.
Based on these promising data, combined with the unmet medical need in HCC, the ambition is to next conduct a phase IIb study with the goal of applying for accelerated approval from the FDA and thus a shorter time to market approval.
CEO comments
In addition to all the clinical progress achieved in 2023, Medivir also conducted a rights issue in late autumn that raised approximately SEK 129 million for the company, followed by a directed issue of approximately SEK 20 million as we turned the page in 2024.
BioStock contacted the company's CEO Jens Lindberg for a look back and a status update.
Jens, what stands out as most important for the continued development of fostrox?
– A couple of things. First, the great interest among investigators and patients to be part of our ongoing study. It is clear that fostrox's unique, liver-targeted mechanism of action is seen as an attractive treatment for advanced liver cancer, which creates the conditions for rapid recruitment in our upcoming phase IIb study. The second thing is of course the promising data we have been able to show with the combination, and above all that we see a clearly higher proportion who respond to the treatment and that they benefit from the treatment for longer than can be expected from the treatment options available today.
What conclusions do you draw from the fact that patients continue to benefit the longer they are on treatment?
– First, the combination of fostrox + Lenvima is very well tolerated, which is especially important so that patients can stay on and benefit from the treatment for as long as possible. But also that it gives clear hope to patients that even though this is advanced liver cancer in second-line treatment, there is clear potential for patients to have long-term benefit from the treatment.
You have been successful in raising capital despite tough economic times, especially for a risky industry like life science. What do you think has been the key to this?
– Of course, the data we have shown with Fostrox in liver cancer looks so promising, but I also believe that our positioning of Fostrox in second-line treatment where there are currently no approved treatment alternatives has also had a positive impact. Then, of course, I hope that those who have chosen to invest in Medivir also do so because they have confidence in Medivir's management and employees.
What milestones do you have ahead of you in the coming period?
– First, to confirm the study design of the planned phase IIb study with the regulatory authority in the US and our investigators, while also selecting a CRO partner to conduct the study.
– Another important issue is the establishment of partnerships with a focus on Asia for the development and commercialization of fostrox, where we began the work of finding the right partner(s) at the end of 2023.
– Finally, we very much look forward to presenting additional data from the ongoing study at a couple of scientific conferences in 2024, given that such a large proportion of patients continue to be on treatment longer than expected and benefit from the combination.