Protagonist and Takeda Report Positive Results from Phase 3 PV Study

Rare Daily Staff

Protagonist Therapeutics and Takeda reported the phase 3 VERIFY clinical trial of rusfertide to treat people with polycythemia vera, a rare blood disease, met its primary and all four secondary endpoints.

Under the 2024 license and collaboration agreement between Protagonist and Takeda, Protagonist will earn a $25 million milestone payment following completion of the VERIFY clinical study report. Takeda received a $300 million payment under the agreement.

People with polycythemia vera (PV) are at increased risk for life-threatening cardiovascular and thrombotic events. Many people with PV require regular phlebotomy, a process of removing blood to manage elevated hematocrit levels caused by excess red blood cells, and treatment with cytoreductive therapies. Phlebotomy can be burdensome and exacerbate symptoms, including severe fatigue, visual disturbances and iron deficiency, which impact patients’ quality of life. The reduction of hematocrit below 45 percent is a primary treatment goal for patients with PV as recommended by current treatment guidelines.

Rusfertide is a first-in-class investigational hepcidin mimetic peptide therapeutic, in development to reduce erythrocytosis in people with PV who do not require further treatment for thrombocytosis and/or leukocytosis.

The study met its primary endpoint, with a significantly higher proportion of clinical responders 1 among rusfertide-treated patients with PV (77 percent) compared to those who received placebo (33 percent) during weeks 20-32. The study’s primary endpoint was the proportion of patients achieving a response, which was defined as the absence of phlebotomy eligibility.

Rusfertide was generally well tolerated in the phase 3 VERIFY trial, and safety was in line with previous rusfertide clinical studies. No new safety findings were observed in the study.

“The totality of impressive clinical data to date shows that rusfertide has the potential for meaningful positive impact on the lives of patients with PV,” said Dinesh Patel, president and CEO of Protagonist. “Today’s study results also mark a critical inflection point in Protagonist’s decade-long journey in the hepcidin program and further validates our platform and expertise in innovating highly differentiated peptide-based medicines to fulfill unmet medical needs.”

Photo: Dinesh Patel, president and CEO of Protagonist