Takeda and Protagonist Therapeutics have entered a global collaboration and licensing agreement to advance the development and commercialization of rusfertide, Protagonist's late-stage rare hematology asset.
Rusfertide, an investigational injectable hepcidin mimetic peptide, targets the treatment of polycythemia vera (PV). PV a is a rare chronic blood disorder impacting up to 160,000 patients in the U.S. and Europe. It is characterized by an overproduction of red blood cells, elevating the risk of cardiovascular and thrombotic events like heart attacks and strokes.
In the partnership, Takeda takes on the roles of co-developer and U.S. co-commercialization partner, with profits shared with California-based Protagonist. Takeda secures exclusive ex-U.S. global rights for rusfertide, making an initial upfront payment of $300 million. Additionally, Protagonist is poised to receive extra payments linked to global development, regulatory milestones, and tiered royalties based on ex-U.S. net sales.
This collaboration capitalizes on Protagonist's expertise in pharmaceutical peptide drug development, illustrated by rusfertide's mechanism of action uncovered through Protagonist's peptide technology platform. The effectiveness of rusfertide is validated by the phase 2 REVIVE study, demonstrating its ability to control hematocrit levels and reduce phlebotomy use in PV patients.
Rusfertide was granted breakthrough designation by the U.S. FDA for treating patients with polycythemia vera back in June 2021. Less than a year later, the FDA sent a letter to Protagonist, saying it wants to revoke rusfertide’s status as a breakthrough therapy.
