Positive Phase III VERIFY results of rusfertide in PV

US clinical-stage pharma company Protagonist Therapeutics (Nasdaq: PRGX) and Japan’s Takeda (TYO: 4502) have announced positive top-line results for the Phase III VERIFY study, in which phlebotomy-dependent patients with polycythemia vera (PV), a rare blood cancer, were randomized to treatment with either rusfertide or placebo, as an add-on to standard of care treatment.

The study met its primary endpoint and all four key secondary endpoints. Rusfertide is a first-in-class investigational hepcidin mimetic peptide therapeutic, which has received Orphan Drug and Fast Track designations from the US Food and Drug Administration (FDA).

The companies entered a collaboration on rusfertide in February last year, when Protagonist received an upfront payment of $300 million from Takeda and became eligible for additional worldwide development and regulatory milestone payments, as well as commercial milestones and tiered royalties on ex-US net sales. Under the license and collaboration, Protagonist earns a $25 million milestone payment following these positive results. The milestone is payable following completion of the VERIFY clinical study report