Takeda's $300M investment in Protagonist's blood cancer drug succeeds with phase 3 win

Takeda Pharmaceuticals has achieved significant progress in its collaboration with Protagonist Therapeutics, as their investigational drug, rusfertide, has shown promising results in a phase 3 clinical trial. This development follows Takeda's $300 million investment in Protagonist’s hematology asset approximately a year ago. Rusfertide, an injectable hepcidin mimetic, is designed to treat polycythemia vera (PV), a rare and chronic blood disorder. The recent trial outcomes suggest a substantial reduction in the need for bloodletting procedures among patients with this condition.

In January 2024, Takeda acquired the rights to rusfertide, which was already in the pivotal phase 3 VERIFY trial. The Japanese pharmaceutical giant reported that the study successfully achieved its primary objective. Patients with PV face heightened risks of serious cardiovascular and thrombotic events due to an excess of red blood cells, which often necessitates regular blood removal via phlebotomy to manage hematocrit levels. The trial's results indicated that 77% of participants receiving rusfertide did not require phlebotomy between weeks 20 and 32 of treatment, compared to just 33% of those on a placebo.

The trial also met its secondary goals, which included control of hematocrit levels and a noticeable reduction in fatigue. Dinesh Patel, Ph.D., CEO of Protagonist Therapeutics, expressed their intention to collaborate with Takeda to present these encouraging findings to regulatory authorities. Patel emphasized the significant clinical data demonstrating rusfertide's potential to positively impact patients suffering from PV and highlighted the successful progress of Protagonist's long-standing hepcidin program, which has further validated their innovative approach in developing peptide-based medicines.

Protagonist Therapeutics’ journey with rusfertide has not been without challenges. The U.S. Food and Drug Administration (FDA) had revoked the drug's breakthrough designation in 2022 due to concerns over observed malignancies. Nevertheless, Takeda recognized rusfertide’s potential, investing $300 million to co-develop and commercialize it in the U.S. and internationally. Protagonist is tasked with research and development up to U.S. regulatory approval, while Takeda has exclusive rights for development and commercialization outside the United States. Both companies are partners in the U.S., sharing profits equally.

Takeda executives have previously stated that securing rights to rusfertide aligns with their strategy to acquire late-stage assets in the rare disease sector. The recent trial results have strengthened this position. Andy Plump, M.D., Ph.D., Takeda’s president of R&D, expressed excitement about rusfertide's potential to benefit PV patients. He also reiterated Takeda's commitment to providing additional treatment options for individuals living with blood cancers, particularly myeloid cancers like PV.

The successful phase 3 trial marks a significant milestone for both Takeda and Protagonist Therapeutics, enhancing prospects for rusfertide as a viable treatment for polycythemia vera. The collaboration between the two companies underscores the importance of innovation and strategic partnerships in addressing unmet medical needs in rare diseases. With these promising results, they are poised to advance rusfertide towards regulatory approval and, ultimately, deliver new hope for patients affected by this challenging blood disorder.