FDA Policy/General

Rusfertide Phase 3 Data Meet Endpoints

Protagonist Therapeutics and Takeda are announcing “positive topline results” for the Phase 3 VERIFY study, in which phlebotomy-dependent patients with polycythemia vera (PV) received rusfertide or placebo as an add-on to standard of care treatment. The study met its primary endpoint and all four key secondary endpoints, according to the companies. Rusfertide is described as a first-in-class investigational hepcidin mimetic peptide therapeutic. It has previously been awarded FDA orphan drug and fast track designations.

In meeting the primary endpoint, the companies say there was a significantly higher proportion of clinical responders among rusfertide-treated patients with PV (77%) compared to those who received placebo (33%) during weeks 20-32. The primary endpoint was the proportion of patients achieving a response, which was defined as the absence of phlebotomy eligibility, they say.

“The positive results of the Phase 3 VERIFY study across the primary and all key secondary endpoints provide compelling evidence of the potential for rusfertide as a first-in-class erythrocytosis-specific agent to address unmet medical needs in patients with PV who are unable to achieve adequate hematocrit control despite standard of care treatments,” Protagonist chief medical officer Arturo Molina is quoted in a release as saying.

The companies say that patients with PV are at increased risk for life-threatening cardiovascular and thrombotic events. “Many patients with PV require regular phlebotomy, a process of removing blood to manage elevated hematocrit levels caused by an excess of red blood cells, as well as treatment with cytoreductive therapies,” they explain.

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