Arturo Molina, MD, Protagonist Therapeutics, discusses results from the phase 2 REVIVE clinical trial testing rusfertide in patients with polycythemia vera (PV).
PV is a myeloproliferative disorder characterized by an elevated absolute red blood cell mass caused by uncontrolled red blood cell production, frequently associated with uncontrolled white blood cell and platelet production.
The REVIVE clinical trial is a phase 2 study evaluating rusfertide in patients with PV. Rusfertide is a subcutaneous peptide hepcidin-mimetic compound. The trial consisted of three parts: part one, initial dose of 20 mg rusfertide; part two, patients were randomized to receive rusfertide or placebo; and part three, open-label extension where all patients receive rusfertide.
Of the 70 patients enrolled in part one, 59 were randomized in part two, and 58 continued to part three. The estimated average phlebotomy rate (EAPR) in patients prior to the study was 8.5/year. In part one, this was reduced to less than 1/year. In part two, the EAPR remained at less than 1/year in the rusfertide group and 6.6/year in the placebo group. An EAPR of less than 1/year remained in part three.
Rusfertide was observed to maintain hematocrit levels less than 45%, increase and stabilize platelet count, maintain stability of leukocyte counts, normalize mean serum ferritin levels, increase mean transferrin saturation, increase mean serum iron levels, and slightly increase mean corpuscular volume.
The most common treatment-emergent adverse events included injection site reactions, fatigue, COVID-19, pruritus, arthralgia, dizziness, nausea, headache, and anemia.
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To learn more about PV and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/
