Protagonist Therapeutics and its partner Takeda presented the 52‑week results from the pivotal Phase 3 VERIFY study at the 67th American Society of Hematology (ASH) Annual Meeting on Saturday, December 6, 2025. The data confirm that 61.9 % of patients who remained on continuous rusfertide therapy were free of phlebotomy eligibility from baseline through week 52, underscoring the drug’s durable ability to control hematocrit levels in patients with polycythemia vera (PV).
The safety profile observed over the full year was consistent with earlier interim analyses: most treatment‑emergent adverse events were mild or moderate, and serious adverse events occurred in a small minority of patients, with no new safety signals identified. These findings reinforce the therapeutic promise of rusfertide and support the company’s plan to file a New Drug Application (NDA) with the U.S. Food and Drug Administration by the end of 2025.
Protagonist’s partnership with Takeda, established in 2024, grants Takeda a global license to commercialize rusfertide, while Protagonist retains responsibility for U.S. development and regulatory filing. The collaboration leverages Takeda’s worldwide commercial network and Protagonist’s focused pipeline, positioning the company to accelerate market entry once regulatory approval is obtained. The 52‑week data provide a critical evidence base for the NDA, potentially shortening the review timeline under the drug’s Fast Track and Breakthrough Therapy designations.
Financially, Protagonist reported $678.8 million in cash, cash equivalents, and marketable securities as of September 30, 2025, giving the company a runway that extends well beyond the anticipated NDA filing and into the early commercialization phase. The strong cash position mitigates short‑term funding risk and allows continued investment in the rusfertide program and other pipeline assets, such as icotrokinra, which was submitted for FDA approval in July 2025.
Management highlighted the clinical significance of the durability data. Chief Medical Officer Dr. Arturo Molina noted that “the 52‑week results confirm that rusfertide maintains hematocrit control without the need for phlebotomy in the majority of patients, a key unmet need in PV.” Lead investigator Dr. Andrew T. Kuykendall added that the sustained efficacy “provides clinicians with a reliable therapeutic option that can reduce the burden of treatment for patients.”
The presentation marks a pivotal step toward regulatory approval and commercial launch. With the 52‑week data now in the public domain, Protagonist and Takeda are poised to advance the NDA submission, potentially positioning rusfertide as a new standard of care for PV and opening a substantial market opportunity for both companies.
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