Medivir Advances HCC Treatment with FDA-Supported Phase 2b Study Design

Swedish pharmaceutical firm Medivir AB is advancing its plans for a phase 2b clinical trial in hepatocellular carcinoma (HCC), a type of liver cancer, following a productive Type C meeting with the U.S. Food and Drug Administration (FDA). The company intends to refine the study design for its investigational drug fostroxacitabine bralpamide, known as fostrox, which is being studied in combination with Lenvima®.

The Type C meeting's outcome highlighted two pivotal modifications to the study's approach. Initially, Medivir will incorporate an initial dose run-in phase, adding 20-25 patients to receive a lower dosage of fostrox, beyond the 30 mg dose from the ongoing phase 1b/2a trial. This adjustment is expected to bolster the study's alignment with the FDA's Project Optimus initiative. Secondly, the primary endpoint will shift to the Objective Response Rate (ORR), recognized as a valid surrogate for Overall Survival and instrumental in previous HCC studies for fast-tracking approvals. Supporting endpoints will encompass the duration of response, progression-free survival, and overall survival.

The overarching goal, scale, and duration of the study are set to mirror the previously outlined phase 2b framework. Medivir has engaged with its Scientific Advisory Council and regulatory authorities to dissect the FDA's advice. The subsequent phase involves finalizing the study's blueprint and protocol, preceding the IND filing in the U.S. to kick off the study in early 2025.

Pia Baumann, Medivir's Chief Medical Officer, expressed confidence in the enhanced phase 2b study design, citing ongoing positive results from the phase 1b/2a study. Jens Lindberg, the company's CEO, emphasized the importance of collaboration with the FDA in shaping an optimal study design, which is crucial for partnership discussions and speeding up study feasibility and CMC preparations.

Fostrox represents a novel chemotherapy approach, selectively targeting tumors while limiting damage to healthy cells. It is an oral drug that carries the active ingredient troxacitabine directly to the liver, where it is released. This targeted delivery system positions fostrox as a potential pioneering liver-targeted oral treatment for liver cancer. A phase 1b monotherapy study for fostrox has concluded, and a phase 1b/2a combination study in HCC is in progress.

HCC stands as the third most common cause of cancer-related mortality globally. With over 660,000 new cases diagnosed annually and a five-year survival rate of less than 20 percent, there is a significant need for improved therapies. Medivir is dedicated to addressing this medical shortfall with fostrox, which is part of the company's broader mission to innovate in areas of cancer with high unmet needs.

Medivir's business strategy heavily relies on collaborations and partnerships, focusing on developing drugs for cancer indications where current treatments are inadequate. The company's stock (ticker: MVIR) is publicly traded on the Nasdaq Stockholm's Small Cap list.