It has been shown in preclinical experiments with bone marrow from patients with
myelodysplastic syndrome that APG101 rescues erythrocytes from premature cell death. This is
expected to translate in an improved erythropoiesis and ameliorated anemia in MDS patients.
APG101 might, therefore, be a valuable addition to current treatments of low- or intermediate
MDS patients suffering from anaemia.
Transfusion-dependent patients with low or intermediate risk MDS according to WHO Prognostic
Scoring Scale (WPSS) can be included in this study.
Treatment consists of 100mg APG101 intravenous as a weekly treatment over 12 weeks + 6 months
follow up phase.
Primary objective of the trial is safety and tolerability of APG101; secondary objectives are
- Hematologic, cytologic and cytogenetic response rate using modified International
Working Group (IWG) response criteria
- Incidence and time to leukemic progression at 37 weeks
- OS (Overall survival) at 37 weeks