Myelofibrosis
Nuvisertib, an oral, investigational, highly selective PIM1 kinase inhibitor, showed clinical activity in a phase 1/2 study.
The primary end point for RBC transfusion independence was not met but the cohort still had significant anemia improvement.
The FDA previously granted Orphan Drug Designation to nuvisertib for use in myelofibrosis in May 2022.
MANIFEST-2 compares pelabresib plus ruxolitinib with placebo plus ruxolitinib for JAK-inhibitor-naive disease.
Adrián Mosquera Orgueira, MD, PhD, spoke of his team's study into machine learning tools for improving risk stratification.
In a phase 1 trial the product performed favorably in aplastic anemia, myelofibrosis, and hypoplastic myelodysplasia.
The panel's discussion, moderated by Neil Love, MD, dove especially into the nuances of using avapritinib and ruxolitinib.
Efficacy and safety primary analysis findings are reported from the international, open-label FREEDOM2 trial.
Study identifies risks of thrombosis, hemorrhage, and leukemic transformation in patients with myelofibrosis and other MPNs.
Momelotinib and ruxolitinib improved bone marrow fibrosis, but changes were not associated with improved outcomes in MF.
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