Takeda’s $300 million bet on Protagonist Therapeutics’ hematology asset a year ago appears to have paid off as the drug reduced blood procedures among patients with a rare type of cancer in a phase 3 trial.
The Japanese pharma licensed rusfertide, an investigational injectable hepcidin mimetic designed to treat a rare chronic blood disorder called polycythemia vera (PV), back in January 2024. Rusfertide was already undergoing the pivotal phase 3 VERIFY trial, and Takeda announced this morning that the study has hit its primary endpoint.
Patients with PV are at increased risk from life-threatening cardiovascular and thrombotic events, explained Takeda, meaning they may require the regular removal of blood via phlebotomy in order to manage hematocrit levels caused by an excess of red blood cells.
The trial demonstrated that 77% of patients receiving rusfertide achieved a response—defined as not requiring phlebotomy across weeks 20 to 32 of treatment—compared to 33% of those on placebo. Secondary endpoints were also met, including hematocrit control and a relative reduction in fatigue.
Protagonist’s CEO Dinesh Patel, Ph.D., said the biotech will work with Takeda to submit today’s findings to regulators.
“The totality of impressive clinical data to date shows that rusfertide has the potential for meaningful positive impact on the lives of patients with PV,” Patel added in a March 3 release. “Today’s study results also mark a critical inflection point in Protagonist’s decade-long journey in the hepcidin program and further validates our platform and expertise in innovating highly differentiated peptide-based medicines to fulfill unmet medical needs.”
That journey hasn’t always been straightforward, with the FDA rescinding rusfertide’s breakthrough designation in 2022, citing “observed malignancies.” But Takeda was able to see the drug’s potential, handing over $300 million to Protagonist to codevelop and commercialize rusfertide both in the U.S. and globally.
While Protagonist is responsible for R&D through the phase 3 trial and potential U.S. regulatory approval, Takeda holds exclusive rights for all development and commercialization activities outside the U.S. Stateside, the companies are co-commercialization partners, having signed a 50/50 profit sharing agreement.
Last year, Takeda execs said the move to secure rights to rusfertide represented a fresh page in the company's strategy to acquire late-stage assets in the rare disease space.
“We are encouraged by these results and excited about the potential of rusfertide to help patients living with PV,” Takeda’s president of R&D, Andy Plump, M.D., Ph.D., said in this morning’s release. “We are deeply committed to bringing additional treatment options to those living with blood cancers, including myeloid cancers such as PV.”