• The study provides clinical proof-of-concept for fostrox monotherapy in patients with cancer in the liver.

• The results show that fostrox is safe and tolerable with preliminary anti-tumor activity.

• Confirmation of fostrox’ liver-targeted mechanism inducing DNA damage selectively in tumor cells.

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 1 study of fostrox monotherapy in patients with cancers in the liver has been published in the Journal of Hepatocellular Carcinoma (online first doi: https://doi.org/10.2147/JHC.S481410). The title of the article is “A phase 1a/1b study of fostroxacitabine bralpamide (fostrox) monotherapy in hepatocellular carcinoma and solid tumor liver metastases”, author Plummer R et al. Fostrox is currently in the final phase of the ongoing phase 1b/2a combination study with Lenvima in advanced liver cancer.
 
The published study evaluated safety and preliminary efficacy of fostrox as a novel, oral drug candidate designed to maximise exposure in the liver while minimizing systemic adverse events. The study established safety and tolerability with clinical proof-of-concept for fostrox monotherapy, including biopsy confirmed selective induction of DNA damage in tumor cells.
 
"Primary liver cancer (HCC) has a particularly poor prognosis, and the incidence is projected to increase dramatically, attributed to life style factors such as obesity and fatty liver disease. Despite progress in development of new treatments, there are currently no approved second line treatments in liver cancer post immunotherapy. Fostrox, with its liver-targeted mechanism has, in this first monotherapy study, shown to be safe and tolerable with preliminary anti-tumor activity”, says Dr Jeff Evans, Beatson West of Scotland Cancer Centre, one of the investigators in the study.
 
Study results confirmed that fostrox had a liver targeted distribution. Most patients could stay on the recommended phase 2 dose of 40 mg without dose modification or discontinuation. In liver cancer patients, the clinical benefit rate was 63%, establishing preliminary efficacy with fostrox monotherapy in patients that had exhausted all approved treatment options. The data supported the next step in clinical development of fostrox in combination with other modes of action in HCC.
 
"In primary liver cancer, it is critical to maximize the anti-tumor effect locally in the liver as the combination of tumor burden and underlying liver disease increases the risk of liver failure. Fostrox has been designed with this challenge in mind and the study results confirm a liver-directed delivery where fostrox induces DNA damage selectively in tumors cells, to ensure optimal efficacy and safety. This targeted mechanism is unique to fostrox and enables patients to stay on treatment long-term as it avoids harming healthy liver cells. The data reinforces our confidence in fostrox as a potential treatment option for patients with advanced liver cancer. Fostrox in combination with Lenvima is currently being investigated in a phase 1b/2a study”, says Dr. Pia Baumann, CMO at Medivir.