Japan-based pharmaceutical firm Takeda and US-based Protagonist Therapeutics have inked a global license and collaboration agreement for the development and commercialisation of rusfertide, an investigational injectable hepcidin mimetic peptide designed to mimic the natural hormone hepcidin.

Rusfertide is currently undergoing a pivotal Phase 3 trial, VERIFY, for the treatment of Polycythemia Vera (PV), a rare chronic blood disorder characterised by the overproduction of red blood cells, affecting approximately 160,000 patients in the US and a similar number in Europe.

As per the agreement's terms, Protagonist will receive an upfront payment of $300 million, along with potential additional payments linked to global development and regulatory milestones, as well as commercial milestones and royalties on ex-US net sales. Protagonist will continue to handle research and development until the completion of the Phase 3 clinical trial and US regulatory approval. Takeda will take charge of ex-US development and lead global commercialisation efforts.

The deal allows Protagonist to focus on completing Phase 3 while leveraging Takeda’s global commercialisation capabilities.

Rusfertide, discovered through Protagonist's peptide technology platform, is designed to regulate iron homeostasis, controlling the absorption, storage, and distribution of iron in the body. 

This collaboration aligns with Takeda's focus on Rare Haematology and follows the recent FDA approval of ADZYNMA, Takeda’s treatment for congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.