On June 17, 2026 Deciphera Pharmaceuticals, LLC, a member of Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; "Ono"), reported the first patient was dosed in the global pivotal Phase 3 INTREPID study evaluating sapablursen for the treatment of polycythemia vera (PV). Sapablursen is an investigational drug that has the potential to offer a once-monthly treatment option for patients with PV, a rare and potentially life-threatening hematologic disease.
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"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."
INTREPID is a pivotal global Phase 3 study in patients with phlebotomy-dependent PV designed to compare the efficacy and safety of sapablursen to 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 are number of phlebotomies, which is the primary endpoint for the European Medicines Agency for potential regulatory approval, hematocrit control, and improvement in the Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form Total T-score and the Myelofibrosis Symptom Assessment Form (MFSAF) Total Symptom Score. 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 clinical trial has initiated in the United States and is planned in additional regions including North America, Latin America, Asia Pacific and Europe. For more information on the INTREPID study, please visit View Source
Sapablursen received Fast Track designation and Orphan Drug Designation in 2024 and Breakthrough Therapy Designation in 2025 by the U.S. Food and Drug Administration (FDA).
Sapablursen was discovered and advanced through Phase 2 clinical development by Ionis Pharmaceuticals. Ono obtained exclusive global rights for the development and commercialization of sapablursen after entering into a license agreement with Ionis in March 2025.
About Sapablursen
Sapablursen is designed to reduce the production of TMPRSS6 resulting in increased expression of hepcidin, the key regulator of iron homeostasis. By increasing the production of hepcidin, sapablursen has the potential to positively impact PV by decreasing hematocrit, reducing the need for phlebotomy, and improving quality of life.
About Polycythemia Vera
Polycythemia vera (PV) is a rare and potentially life-threatening hematologic disease characterized by the overproduction of red blood cells, which significantly increases the risk of serious blood clots, heart attack, stroke, and death. The primary treatment goal in PV is to maintain blood hematocrit levels <45% to prevent thrombotic events and alleviate burdensome symptoms, such as severe fatigue, difficulty concentrating, night sweats, and pruritus. Current treatment options often worsen symptoms and inadequately maintain hematocrit control.
(Press release, Deciphera Pharmaceuticals, JUN 17, 2026, View Source [SID1234668788])