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Pelabresib is an oral, small-molecule BET inhibitor capable of modifying critical components of MF pathobiology. Updated results from the MANIFEST-2 trial (NCT04603495) evaluating the long-term efficacy and safety of pelabresib + ruxolitinib vs ruxolitinib + placebo in JAKi-naïve patients with MF were presented by John Mascarenhas at the 66th ASH Annual Meeting and Exposition.1 |
Key learnings |
A higher proportion of patients receiving PELA + RUX arm achieved SVR35 responses at Week 48 vs those receiving RUX + PBO (57.0% vs 37.5%). |
At Week 48, TSS50 responses were achieved by 45.3% vs 39.4% of patients receiving PELA + RUX vs RUX + PBO, respectively. Grade ≥1 BM fibrosis improvement was higher with PELA + RUX vs RUX + PBO (41.0% vs 15.0%). |
Hb responses were achieved by 13.1% and 7.9% of patients receiving PELA + RUX vs RUX + PBO, respectively. Grade ≥3 anemia was reported in 25.9% vs 38.3% of patients in the PELA + RUX vs RUX + PBO arms, respectively. |
These findings suggest that PELA + RUX results in deep and sustained responses and continues to improve outcomes in JAKi-naïve patients with MF. Longer-term follow-up results will be presented at future conferences. |
Abbreviations: ASH, American Society of Hematology; BET, bromodomain and extra-terminal; BM, bone marrow; Hb, hemoglobin; JAKi, Janus kinase inhibitor; MF, myelofibrosis; PELA, pelabresib; PBO, placebo; RUX, ruxolitinib; SVR35, spleen volume reduction ≥35%; TSS, total symptom score.
References
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