Takeda's $300M Investment in Protagonist's Blood Cancer Drug Yields Positive Phase 3 Results

Takeda Pharmaceutical's strategic $300 million investment in Protagonist Therapeutics' hematology asset has proven fruitful, as the investigational drug rusfertide demonstrated significant efficacy in a pivotal phase 3 trial for polycythemia vera (PV), a rare chronic blood disorder.

Phase 3 VERIFY Trial Success

The VERIFY trial, evaluating rusfertide in PV patients, has met its primary endpoint with impressive results. 77% of patients receiving rusfertide achieved the desired response, defined as not requiring phlebotomy across weeks 20 to 32 of treatment, compared to only 33% in the placebo group. This outcome represents a substantial improvement in managing hematocrit levels, a critical factor in PV treatment.

Secondary endpoints were also met, including improved hematocrit control and a relative reduction in fatigue, further solidifying rusfertide's potential as a game-changing therapy for PV patients.

Collaborative Development and Commercialization

The success of rusfertide marks a significant milestone in the partnership between Takeda and Protagonist Therapeutics. Under their agreement, Protagonist is responsible for research and development through the phase 3 trial and potential U.S. regulatory approval. Takeda holds exclusive rights for development and commercialization activities outside the U.S., while the companies have established a 50/50 profit-sharing agreement for the U.S. market.

Dinesh Patel, Ph.D., CEO of Protagonist, expressed enthusiasm about the results, stating, "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." He added that the study results validate the company's platform and expertise in developing innovative peptide-based medicines.

Implications for Polycythemia Vera Treatment

Polycythemia vera patients face increased risks of life-threatening cardiovascular and thrombotic events due to an excess of red blood cells. Current management often involves regular phlebotomy procedures to control hematocrit levels. Rusfertide, an injectable hepcidin mimetic, offers a novel approach to treatment that could significantly reduce the need for these invasive procedures.

Andy Plump, M.D., Ph.D., Takeda's president of R&D, commented on the results: "We are encouraged by these results and excited about the potential of rusfertide to help patients living with PV. We are deeply committed to bringing additional treatment options to those living with blood cancers, including myeloid cancers such as PV."

With these positive phase 3 results, Protagonist and Takeda are poised to submit their findings to regulatory authorities, potentially bringing a new treatment option to PV patients in the near future.