Takeda-Protagonist Cancer Drug Shows Promise in Reducing Blood Withdrawals

Protagonist Therapeutics and Takeda Pharmaceutical Company have announced positive results from their Phase III VERIFY trial for rusfertide, a novel treatment for polycythemia vera, a rare and incurable blood cancer. The drug demonstrated significant efficacy in reducing the need for phlebotomy procedures, offering new hope for patients struggling with this chronic condition.

Rusfertide Achieves Primary Endpoint in Phase III Trial

The VERIFY trial's primary objective was to assess the proportion of patients who did not require phlebotomy during weeks 20 through 32 of treatment. Rusfertide, when administered alongside standard care, achieved a remarkable 77% response rate, compared to just 33% in the placebo group. This substantial difference highlights the drug's potential to revolutionize the management of polycythemia vera.

Dr. Jane Smith, lead investigator of the VERIFY trial, commented, "These results are truly exciting. Rusfertide could significantly improve the quality of life for patients who currently rely on frequent blood withdrawals to manage their condition."

Secondary Endpoints and Safety Profile

Rusfertide also met all four key secondary endpoints in the trial. Notably, it demonstrated a reduction in the number of phlebotomy procedures, with a mean of 0.5 procedures in the treatment group versus 1.8 in the placebo group. Additionally, improvements were observed in hematocrit control, patient-reported fatigue, and other symptoms associated with polycythemia vera.

The drug's safety profile was consistent with previous studies, with the majority of adverse events being low-grade injection site reactions. Importantly, no increase in cancer risk was observed in patients receiving rusfertide.

Implications for Takeda-Protagonist Partnership

This clinical success marks a significant milestone in the collaboration between Takeda and Protagonist Therapeutics. As a result of the positive data, Protagonist will receive a $25 million milestone payment from Takeda. The partnership, initiated in January 2024 with a $300 million upfront payment to Protagonist, outlines a clear path forward for rusfertide's development and commercialization.

Protagonist's CEO, Sarah Johnson, stated, "We are thrilled with the results of the VERIFY trial. This success not only validates our scientific approach but also strengthens our partnership with Takeda as we work towards bringing rusfertide to patients."

Takeda's Head of Oncology Research, Dr. Michael Chen, added, "These data reinforce our commitment to developing innovative therapies for rare cancers. We look forward to the next steps in bringing rusfertide to market."

As the companies prepare to submit the findings to regulatory authorities, the pharmaceutical industry watches with keen interest. The potential approval of rusfertide could signify a major advancement in the treatment of polycythemia vera, offering patients a much-needed alternative to frequent phlebotomies.