This phase 1b/2a open-label study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of DISC-0974 as well as categorize the effects on anemia response in subjects with myelofibrosis or myelodysplastic syndrome and anemia.
Age 18 years or older at the time of signing the informed consent form (ICF).
For Phase 1b: DIPSS score of 3 to 4 (intermediate 2 risk) or ≥5 (high-risk) primary MF, post PV MF, and/or post ET MF, as confirmed in the most recent local bone marrow biopsy report, according to WHO 2016 criteria.55
Androgens
EPO
Hereditary hemochromatosis
Hemoglobinopathy or intrinsic RBC defect associated with anemia
Total splenectomy
Hematopoietic cell transplant within the past 2 years or graft vs host disease requiring immunosuppression
Sequential
DISC-0974
DrugDISC-0974 is administered subcutaneously.
Incidence of treatment-emergent adverse events (Phase 1b only)
Incidence of clinically abnormal vital signs (Phase 1b only)
Incidence of clinically abnormal physical exam (Phase 1b only)
Incidence of clinically abnormal electrocardiograms (Phase 1b only)
Anemia response defined per IWG-MRT criteria (Phase 1b only)
TD high and TD low participants will be evaluated for absence of PRBC transfusions for a consecutive, "rolling" 12 week interval during the treatment period (Phase 1b only)
TD high participants will be evaluated for absence of PRBC transfusions with minimum Hgb of 7 g/dL during a terminal 12 week interval during the treatment period (Phase 1b only)
TD low participants will be evaluated for absence of PRBC transfusions with minimum Hgb of 7 g/dL during any rolling 16-week interval during the treatment period (Phase 1b only)
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