“To our knowledge, the MANIFEST trial in JAKi treatment-naïve patients is the first study with a rational combination of BETi pelabresib and ruxolitinib that showed clinically meaningful durable improvements in splenomegaly and symptoms, was associated with biomarker findings indicating potential disease modification, and demonstrated a generally favorable safety profile. This combination has the potential to improve the standard of care for treatment-naïve patients with myelofibrosis and warrants further investigation. A randomized, double-blind, phase III study (MANIFEST-2) compares safety and efficacy of pelabresib in combination with ruxolitinib with those of ruxolitinib monotherapy in JAKi treatment-naïve patients (ClinicalTrails.gov identifier: NCT04603495)”
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CONCLUSIONThe rational combination of the BETi pelabresib and ruxolitinib in JAKi-naïve patients with myelofibrosis was well tolerated and showed durable improvements in spleen and symptom burden, with associated biomarker findings of potential disease-modifying activity.