There is one clinical trial.
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.
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 ---
Description: Overall hematological response rate at 3 months (either CR, PR, marrow-CR, HI, SD)Measure: Overall hematological response rate Time: 3 months
Description: Toxicity as measured by NCI CTCAE 4.03Measure: Toxicity Time: 3 or 12 months
Description: measured by EORTC-QLQ30Measure: Quality of life EORTC-QLQ30 Time: 9 or 15 months