Takeda and Protagonist Therapeutics enter into worldwide license and collaboration agreement for late-stage rare hematology asset

Takeda and Protagonist Therapeutics have signed a worldwide license and collaboration agreement for the development and commercialization of rusfertide, an investigational injectable hepcidin mimetic peptide of the natural hormone hepcidin, currently in a pivotal Phase 3 trial, VERIFY, for the treatment of Polycythemia Vera (PV).

PV is a rare chronic blood disorder characterized by excessive production of red blood cells that affects as many as 160,000 patients in the U.S., with a similar prevalence in Europe. The hallmark manifestations of PV are increased red blood cell count and iron deficiency, putting patients at higher risk of cardiovascular and thrombotic events, such as heart attack and stroke, often impacting quality-of-life with symptoms such as fatigue and confusion.

Under the terms of the agreement, Protagonist will receive an upfront payment of $300 million and is eligible to receive additional worldwide development and regulatory milestone payments, as well as commercial milestones and tiered royalties on ex-U.S. net sales. Protagonist will remain responsible for research and development through the completion of the Phase 3 clinical trial and U.S. regulatory approval. Takeda has rights for ex-U.S. development and is responsible for leading global commercialization activities.

“As pioneers in the field of pharmaceutical peptide drug discovery and development, we believe that the maximum impact from our innovation and development acumen can be best achieved with the right partner at the right time,” said Dinesh V. Patel, Ph.D., President and CEO of Protagonist Therapeutics.

“This transformational deal allows Protagonist to focus on completion of Phase 3, while leveraging Takeda’s exceptional global commercialization capabilities to immediately commence pre-commercial activities with a first-in-class new chemical entity.

“As we progress towards a fully integrated pharmaceutical company, this deal mitigates the inherent execution risks of a first-time commercial launch, optimizes the timing and enhances the scope for peak potential sales of rusfertide, while still allowing us to actively participate in the commercial experience and economics with a 50:50 profit split in the U.S. market.”

“The agreement with Protagonist represents an important step forward in our strategy of acquiring late-stage assets in the rare disease space to leverage our existing infrastructure and expertise. It combines the strength of their science with the reach of our commercial organization, understanding of rare diseases, and most importantly, our focus on supporting patients through their journey,” said Julie Kim, president, U.S. Business Unit and U.S. country head, Takeda.

“This is an exciting opportunity to leverage our more than 70 years of innovation and commitment to rare diseases and hematology to deliver a first-in-class therapy to Polycythemia Vera patients, helping to address significant needs in the community. We look forward to working closely with Protagonist as rusfertide completes its registrational clinical program.”

Discovered through Protagonist’s peptide technology platform, rusfertide’s mechanism of action is thought to regulate iron homeostasis and control the absorption, storage and distribution of iron in the body. The randomized portion of the Phase 2 REVIVE study of rusfertide in Polycythemia Vera achieved its primary endpoint. The long-term follow-up data from the 2-year open label extension were presented at the American Society of Hematology 2023 Annual Meeting in early December, which showed durable hematocrit control, decreased phlebotomy use, long-term tolerability and no new safety signals in patients with PV.

The announcement builds upon Takeda’s heritage in Rare Hematology and follows the recent U.S. Food and Drug Administration (FDA) approval of ADZYNMA, Takeda’s treatment for congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

Following U.S. regulatory filing, Takeda will lead commercialization of rusfertide with Protagonist holding an option to co-detail in the U.S. Under the terms of the agreement, Protagonist has the right to opt-out of the 50:50 profit share. In that event, Protagonist would be eligible to receive opt-out payments and enhanced milestone and royalty payments. Takeda would maintain full ex-U.S. rights under either scenario.

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