Takeda and Protagonist Therapeutics Announce Global License and Collaboration Agreement for Rusfertide, an Advanced Rare Hematology Asset

Takeda and Protagonist Therapeutics, Inc., (Nasdaq: PTGX) have inked a global license and collaboration agreement for the development and commercialization 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 characterized by the overproduction of red blood cells, affecting approximately 160,000 patients in the U.S. 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-U.S. net sales. Protagonist will continue to handle research and development until the completion of the Phase 3 clinical trial and U.S. regulatory approval. Takeda will take charge of ex-U.S. development and lead global commercialization efforts.

Protagonist Therapeutics, expressed the company's belief in finding the right partner at the right time to maximize the impact of their innovation. The deal allows Protagonist to focus on completing Phase 3 while leveraging Takeda’s global commercialization capabilities. Patel highlighted the strategic advantages of mitigating execution risks during the first-time commercial launch, optimizing timing, and enhancing the potential sales scope for rusfertide. Protagonist will maintain a 50:50 profit split in the U.S. market, allowing active participation in the commercial experience and economics.

Takeda's U.S. Business Unit, emphasized the strategic move as part of Takeda's rare disease strategy. Takeda aims to leverage its infrastructure, commercial organization, and expertise to deliver rusfertide as a first-in-class therapy for Polycythemia Vera patients, addressing significant needs in the community. Kim highlighted Takeda's commitment to supporting patients through their medical journey, building on the company's more than 70 years of innovation in rare diseases and hematology.

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. The Phase 2 REVIVE study demonstrated positive results in Polycythemia Vera, achieving its primary endpoint. The long-term follow-up data from the 2-year open label extension showed durable hematocrit control, decreased phlebotomy use, long-term tolerability, and no new safety signals.

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

After the U.S. regulatory filing, Takeda will lead the commercialization of rusfertide, and Protagonist will have the option to co-detail in the U.S. The agreement allows Protagonist to opt-out of the 50:50 profit share, in which case, it would receive opt-out payments and enhanced milestone and royalty payments, while Takeda would retain full ex-U.S. rights under both scenarios.

Protagonist Therapeutics has filed additional details related to the agreement with the U.S. Securities and Exchange Commission on Form 8-K, and the effectiveness of the agreement is subject to the termination or expiration of any applicable waiting periods under the Hart-Scott-Rodino Act.

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