Deciphera Pharmaceuticals Announces First Patient Dosed in Pivotal Phase 3 INTREPID Study of Sapablursen in Polycythemia Vera

• Global pivotal Phase 3 study will evaluate the efficacy and safety over a 52-week treatment period
• Polycythemia Vera (PV) is a rare and potentially life-threatening hematologic disease with significant persistent unmet need
• Sapablursen received Fast Track, Orphan Drug and Breakthrough Therapy designations by U.S. Food and Drug Administration (FDA)

WALTHAM, Mass.--(BUSINESS WIRE)--Deciphera Pharmaceuticals, LLC, a member of Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”), announced 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.

“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 https://clinicaltrials.gov/study/NCT07429266.

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.

About Deciphera Pharmaceuticals, LLC

Deciphera, a member of Ono Pharmaceutical Co., Ltd., is a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines and providing hope to people living with cancer, neurologic, and autoimmune disease. Deciphera is leveraging its proprietary switch-control kinase inhibitor platform and deep expertise in kinase biology to develop a broad portfolio of innovative medicines. In addition to advancing multiple product candidates from Deciphera’s platform in clinical studies, QINLOCK^® (ripretinib) is Deciphera’s switch-control kinase inhibitor approved in many countries including the European Union and the United States for the treatment of adult patients with advanced gastrointestinal stromal tumor (GIST) who have received prior treatment with 3 or more kinase inhibitors, including imatinib. ROMVIMZA^TM (vimseltinib) is a kinase inhibitor approved in the United States for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity, and in the European Union for adult patients with TGCT associated with clinically relevant physical function deterioration and in whom surgical options have been exhausted or would induce unacceptable morbidity or disability. For more information, visit www.deciphera.com and follow us on LinkedIn and X (@Deciphera).

About Ono Pharmaceutical Co., Ltd.

Ono Pharmaceutical Co., Ltd. delivers innovative medicines for patients worldwide. Upholding its philosophy of “Dedicated to the Fight against Disease and Pain,” Ono targets areas with unmet medical needs including oncology, immunology & inflammation, and neurology, and fosters partnerships with academic and biotech organizations to accelerate drug discovery. Through its affiliate, Deciphera Pharmaceuticals, Ono is accelerating clinical development and commercial operations in the US and Europe to drive global business expansion and further its commitment to patient care. For more information, please visit the company's website at https://www.ono-pharma.com/en.

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