Core Insights - Protagonist Therapeutics and Takeda announced new 52-week results from the Phase 3 VERIFY study, highlighting the efficacy and safety of rusfertide in treating polycythemia vera (PV) patients, with 61.9% of patients maintaining absence of phlebotomy eligibility from baseline to Week 52 [1][2] Efficacy and Safety - The 52-week data demonstrated sustained efficacy of rusfertide, significantly reducing the need for phlebotomy while maintaining hematocrit control [2] - 61.9% of patients treated with rusfertide maintained a durable clinical response, defined as absence of phlebotomy eligibility [4] - 84.1% of patients who had a clinical response in the Part 1a assessment maintained their response [4] - The median time to first phlebotomy was not reached in the rusfertide group, indicating effective treatment [4] Patient Outcomes - Patients treated with rusfertide reported improvements in quality of life, as measured by PROMIS Fatigue SF-8 and MFSAF TSS [4] - The mean hematocrit remained below 43% through Week 52 for patients treated with rusfertide [4] - Serious adverse events occurred in 8.1% of rusfertide-treated patients, indicating a generally well-tolerated safety profile [5] Long-term Study Insights - The THRIVE study showed a greater than 13-fold reduction in the estimated annual therapeutic phlebotomy rate compared to baseline, with a mean annualized phlebotomy rate dropping from 9.2 to 0.7 phlebotomies/year [6] - The THRIVE study is designed to assess the long-term durability of response and safety profile of rusfertide over an additional two-year treatment period [10] Regulatory and Development Status - Rusfertide has received Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation from the FDA, indicating its potential as a significant treatment option for PV [7] - The Phase 3 VERIFY study is ongoing, evaluating rusfertide in 293 patients over a 156-week period [8]