TRANSFORM-1 Phase 3 study: Efficacy and safety of navitoclax plus ruxolitinib in patients with untreated myelofibrosis

Blood 2026 Open access

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Authors: Naveen Pemmaraju, Adam J. Mead, Tim C. P. Somervaille, Francesca Palandri, Steffen Koschmieder, Robert Delage, Jean‐Jacques Kiladjian, Christopher Brent Benton, Srinivas Kiran Tantravahi, Noa Lavi, Su‐Peng Yeh, María Teresa Gómez‐Casares
+17 more Emanuele Ammatuna, Andrew Bruce McDonald, Sung‐Soo Yoon, Keita Kirito, Timothy Devos, Andrew Charles Perkins, Atanas Radinoff, Ömür Gökmen Sevindik, Andrija D. Bogdanovic, Rastislav Moskal, Jason G. Harb, Bryan C. Murray, Qi Jiang, Avijeet S. Chopra, Elektra J. Papadopoulos, Jalaja Potluri, Francesco Passamonti
DOI: 10.1182/blood.2025032360
Cited by: 1
References: 30

Abstract

The Phase 3 TRANSFORM-1 study (NCT04472598) evaluated ruxolitinib (RUX) in combination with navitoclax (NAV) or placebo (PBO) in Janus-kinase-inhibitor-naïve adults with intermediate-2 or high-risk myelofibrosis and Eastern Cooperative Oncology Group performance status ≤2. Patients were randomized 1:1 to NAV (200 mg/day starting dose or 100 mg escalated to 200 mg/day) or PBO, with RUX dosed per label. The primary endpoint was ≥35% spleen volume reduction (SVR) at Week 24 (SVR35W24). Secondary endpoints included change from baseline in Total Symptom Score (TSS) at Week 24 and SVR35 at any time. A total of 252 patients (NAV+RUX, n=125; PBO+RUX, n=127; median follow-up 20.3 months) were randomized; >80% had intermediate-2 risk and nearly 50% were high-molecular risk (HMR). SVR35W24 was achieved by 63.2% with NAV+RUX versus 31.5% with PBO+RUX (P

Fields of Study & Subjects

Myeloproliferative Neoplasms: Diagnosis and Treatment Platelet Disorders and Treatments Multiple Myeloma Research and Treatments

Publication details

Journal / source
Blood
Publisher
American Society of Hematology
Volume
148
Issue
16
Pages
2029-2042
Publication date
30 June 2026
Language
EN