October 15, 2026BloodTRANSFORM-1 (randomized, double-blind, placebo-controlled phase 3), NCT04472598
Navitoclax Plus Ruxolitinib Doubles Spleen Response in Untreated Myelofibrosis but Misses the Symptom Endpoint, With Substantially Higher Hematologic Toxicity: Phase 3 TRANSFORM-1
TRANSFORM-1 randomized 252 JAK inhibitor–naïve adults with intermediate-2 or high-risk myelofibrosis 1:1 to ruxolitinib plus the BCL-XL/BCL-2/BCL-W inhibitor navitoclax (NAV+RUX) or ruxolitinib plus placebo (PBO+RUX). The primary endpoint was met: a ≥35% spleen volume reduction at week 24 (SVR35W24) was reached by 63.2% (n = 79) versus 31.5% (n = 40); P < .0001.