The outcome of HMA-refractory patients with MDS or AML is dismal with a median survival of 5 months after failure, representing a significant unmet medical need due to the very limited treatment options. In this context, a specific targeting of the leukemic stem cell (LSC) seems a promising option to selectively combat the leukemic progenitor cells. In fact, CD123 is overexpressed in AML and MDS progenitors making it an attractive target for immunotherapy-based approaches. JNJ-56022473 is a promising compound that has been engineered with regard to this strategy and the current phase II trial has the aim to evaluate the overall hematological response rate at 3 months in HMA refractory/relapsed AML and MDS patients.

NCT02992860

Conditions

1. Myelodysplastic Syndrome (MDS)
2. Acute Myeloid Leukemia (AML)

Interventions

1. Drug: JNJ-56022473
2. Procedure: Bone marrow analyses and CBC with differential
3. Other: Flow cytometry analyses
4. Other: Central biobanking
5. Procedure: Histopathology analysis
6. Genetic: Cytogenetic analysis
7. Procedure: Serum chemistry
8. Procedure: Automated CBC
9. Procedure: Pregnancy Test

MeSH:Leukemia, Myeloid Leukemia, Myeloid, Acute Preleukemia Myelodysplastic Syndromes

HPO:Acute megakaryocytic leukemia Acute myeloid leukemia Myelodysplasia Myeloid leukemia

To enhance the cytotoxicity of the first-generation antibody CSL360, the proprietary Xencor (Xmab®) technology was applied and two amino acid mutations (S239D and I332E) were introduced into the Fc region. --- S239D --- --- I332E ---