Background & Aims:
Parenteral therapy using hypomethylating agents (HMAs) requires 5-7 days each month, resulting in hospital or clinic visits on a chronic basis and represents a substantial burden for this primarily elderly population and their caregivers. Magrolimab has demonstrated encouraging preliminary data and is currently being evaluated in a randomized Phase 3 study (ENHANCE, NCT04313881), comparing the efficacy and safety of magrolimab plus azacitidine with that of azacitidine plus placebo in previously untreated patients with higher-risk MDS. This phase 2 study examines the possibility of using an oral HMA (oral decitabine/cedazuridine) in combination with magrolimab which may provide the benefits of combination therapy without the burden of significant parenteral therapy.
ASTX727-10 is a phase 2, international, single-arm, open-label study investigating the safety and efficacy of combination oral decitabine/cedazuridine and magrolimab treatment in intermediate to very high-risk MDS, based on MDS International Prognostic Scoring System-Revised (IPSS-R). Secondary objectives include analysis for safety and efficacy assessments based on the IPSS- Molecular (IPSS-M) and p53 status. To be eligible, subjects with ECOG Performance Status ≤2 must have previously untreated MDS per WHO 2016 classification with < 20% bone marrow blasts and be willing to undergo red blood cell transfusions to achieve a hemoglobin >9 gm/dl at the start of study treatment. Key exclusion criteria include significant medical issues, creatinine clearance < 50 ml/min, immediate eligibility for hematopoietic stem cell transplant, or secondary MDS.
Approximately 100 subjects will be enrolled.
The study is anticipated to open soon.