Deciphera Pharmaceuticals has dosed the first patient in its global pivotal Phase III INTREPID study, evaluating sapablursen for the treatment of polycythaemia vera (PV).
Sapablursen is being investigated as a potential once-monthly treatment for PV, a rare and potentially life-threatening haematologic disease.
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The study is designed for patients with phlebotomy-dependent PV and compares sapablursen to placebo in a 32-week double-blind treatment period. This is followed by up to 124 weeks of open-label treatment.
The primary endpoint is response, defined by the absence of phlebotomy eligibility.
Secondary endpoints include the number of phlebotomies, which is the primary endpoint for regulatory approval from the European Medicines Agency.
Other endpoints are haematocrit control and improvements in 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 is evaluated over 52 weeks in patients randomised to sapablursen.
The INTREPID trial is enrolling in the US and will include regions in the Asia Pacific, Europe, Latin America, and North America.
Sapablursen received fast track and orphan drug designations from the US Food and Drug Administration (FDA) in 2024, as well as breakthrough therapy designation in 2025.
Ionis Pharmaceuticals discovered and advanced sapablursen through Phase II, with Ono acquiring exclusive global rights for development and commercialisation in March 2025.
Sapablursen targets transmembrane serine protease 6 (TMPRSS6), leading to increased hepcidin, the central regulator of iron homeostasis.
PV is marked by excessive red blood cell production, increasing risks of blood clots, heart attack, stroke, and death.
Deciphera Pharmaceuticals chief medical officer Matthew Sherman said: “We look forward to building upon the positive efficacy and safety results from the Phase IIa IMPRSSION study, which demonstrated the ability of sapablursen to reduce the frequency of phlebotomy, control haematocrit, and improve PV symptoms in patients treated with phlebotomy alone and those on cytoreductive therapies.
“Sapablursen has the potential to be an important new treatment option for patients with PV, and we are excited to begin our Phase III INTREPID study, which brings us one step closer to addressing the unmet needs of these patients.”
