Protagonist Therapeutics (PTGX.US) and Takeda's Phase 3 VERIFY study preliminary results are positive, helping patients with PV to overcome bleeding treatment.

Protagonist Therapeutics (PTGX.US) and Takeda announced the preliminary positive results of the Phase 3 study VERIFY today. Analysis shows that the study met its primary endpoint as well as all four key secondary endpoints, with the potential "first-in-class" therapy rusfertide as an adjunct to standard therapy, leading to nearly 80% of patients with polycythemia vera (PV) no longer needing phlebotomy treatment. Patients with PV are at increased risk of fatal cardiovascular events and thrombosis. Many PV patients require regular phlebotomy treatment to control the increased blood volume caused by excess red blood cells, along with cytoreductive therapy. Regular phlebotomy treatment may burden patients and exacerbate symptoms including severe fatigue, visual disturbances, and iron deficiency, affecting the quality of life of patients. Current treatment guidelines recommend reducing blood volume to below 45% as the main treatment goal for PV patients. Analysis shows that the study met its primary endpoint. In the 20-32 week period, the clinical response rate in the rusfertide treatment group was 77%, significantly higher than the placebo group's 33% (p<0.0001), with the primary endpoint defined as patients not needing phlebotomy treatment. Additionally, the study also met the first key secondary endpoint (the primary endpoint predetermined by EU regulatory agencies). In the 0-32 week period, the average number of phlebotomies for the rusfertide group was 0.5 times, compared to 1.8 times for the placebo group (p<0.0001). The results for the other three predetermined key secondary endpoints, including blood volume control and fatigue assessment indicators, also reached statistical significance.