Waltham, Mass. — Deciphera Pharmaceuticals, LLC, a member of Ono Pharmaceutical Co., Ltd., announced that the first patient has been dosed in the global pivotal Phase 3 INTREPID study evaluating sapablursen for the treatment of polycythemia vera.
Sapablursen is an investigational drug that could offer a once-monthly treatment option for patients with polycythemia vera, or PV, a rare and potentially life-threatening hematologic disease.
“We look forward to building upon the positive efficacy and safety results from the Phase 2a IMPRSSION study, which demonstrated the ability of sapablursen to reduce the frequency of phlebotomy, control hematocrit, and improve PV symptoms in patients treated with phlebotomy alone and those on cytoreductive therapies,” said Matthew L. Sherman, M.D., Chief Medical Officer of Deciphera. “Sapablursen has the potential to be an important new treatment option for patients with PV, and we are excited to begin our Phase 3 INTREPID study, which brings us one step closer to addressing the unmet needs of these patients.”
The INTREPID study is a pivotal global Phase 3 trial in patients with phlebotomy-dependent PV. It is designed to compare the efficacy and safety of sapablursen with placebo over a 32-week double-blind treatment period, followed by up to 124 weeks of open-label treatment.
The primary endpoint is response, defined by the absence of phlebotomy eligibility. Key secondary endpoints include the number of phlebotomies, which is the primary endpoint for the European Medicines Agency for potential regulatory approval, hematocrit control, and improvement in fatigue and symptom scores.
Durability of response will be evaluated in patients randomized to sapablursen over 52 weeks of study treatment, including 32 weeks of blinded treatment and 20 weeks of open-label treatment.
The INTREPID trial has begun in the United States and is planned for additional regions, including North America, Latin America, Asia Pacific and Europe.
Sapablursen received Fast Track designation and Orphan Drug Designation from the U.S. Food and Drug Administration in 2024 and Breakthrough Therapy Designation in 2025.
Sapablursen was discovered and advanced through Phase 2 clinical development by Ionis Pharmaceuticals. Ono obtained exclusive global rights to develop and commercialize sapablursen after entering into a license agreement with Ionis in March 2025.
