MDS are a group of hematopoietic stem cell malignancies with significant morbidity and mortality.
MDS is characterized by ineffective blood cell production, or hematopoiesis, leading to low blood cell counts, or cytopenias, and high risk of progression to acute myeloid leukemia, or AML. MDS is a highly heterogeneous disease, and the severity of symptoms and disease progression can vary widely among patients.
According to the American Cancer Society, the annual incidence of MDS is approximately 13,000 patients in the United States, the majority of whom are 60 years of age or older. The estimated prevalence is over 60,000 patients in the United States.
A substantial portion of MDS patients lack effective therapies, and NCCN guidelines recommend clinical trials as additional therapeutic options. We believe that treatment of MDS remains a significant unmet need requiring the development of novel therapies.