A pivotal Phase III clinical trial is currently evaluating treatment with rusfertide, a potential first-in-class treatment for polycythemia vera.
Takeda and Protagonist Therapeutics have entered a global license and collaboration agreement to develop and commercialize rusfertide, a potential first-in-class treatment for polycythemia vera (PV), a rare blood disorder. Under terms of the agreement, Protagonist will receive an upfront payment of $300 million, with potential for additional payments based on development, regulatory, and commercial milestones, plus royalties on sales outside the United States. Protagonist retains responsibility for the drug's development and US regulatory approval, while Takeda will lead global commercialization efforts and development outside the United States.1
According to the release, the partnership combines Protagonist's expertise in peptide drug development and Takeda's global commercial capabilities, aiming to optimize rusfertide's market potential, especially in the United States, where profits are expected to be split down the middle. The agreement also includes provisions for Protagonist to potentially opt out of the profit-sharing arrangement in favor of receiving enhanced payments.1
“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, US business unit, US country head, Takeda, in a company press release. “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.”1
Last year, Protagonist discovered positive results from its Revive study. According to the data, patients receiving rusfertide achieved highly statistically significant improvements versus placebo in the primary endpoint. Other key findings included:
PV is a type of blood cancer that causes bone marrow to produce excess red blood cells, which can lead to serious medical issues, including blood clots. Without treatment, PV can lead to death; however, life proper medical care can reduce the signs, symptoms and complications of the disease.3
"The new randomized withdrawal data confirm our previous efficacy and safety findings of rusfertide in PV and support our strong conviction that rusfertide can be a potentially transformational therapeutic option for polycythemia vera," said Dinesh V. Patel, PhD, president, CEO, Protagonist. "With the completion of the REVIVE study, the Company's topmost priority continues to be execution of the 250-patient global, pivotal, Phase III VERIFY study in PV. The Protagonist team continues to work with full dedication alongside investigators, site staff and other partners with the shared aim of bringing this important potential therapy to PV patients."
1. Takeda and Protagonist Therapeutics, Inc. Enter into Worldwide License and Collaboration Agreement for Rusfertide, a Late-Stage Rare Hematology Asset. Takeda. January 31, 2024. Accessed February 1, 2024. https://www.takeda.com/newsroom/newsreleases/2024/takeda-and-protagonist-therapeutics-inc-enter-into-worldwide-license-and-collaboration-agreement-for-rusfertide-a-late-stage-rare-hematology-asset/
2. Protagonist Therapeutics Announces Highly Statistically Significant Results from the Randomized Withdrawal Portion of the REVIVE Study of Rusfertide in Polycythemia Vera. Accesswire. March 15, 2023. Accessed February 1, 2024. https://www.accesswire.com/743817/Protagonist-Therapeutics-Announces-Highly-Statistically-Significant-Results-from-the-Randomized-Withdrawal-Portion-of-the-REVIVE-Study-of-Rusfertide-in-Polycythemia-Vera
3. Polycythemia vera. Mayo Clinic. Webpage. Updated February 11, 2022. Accessed February 1, 2024. https://www.mayoclinic.org/diseases-conditions/polycythemia-vera/symptoms-causes/syc-20355850