DNA Damage Response
Sapacitabine is a DNA damage response inhibitor (DDRi). It is an oral prodrug of CNDAC (2’-C-cyano-2’-deoxy-1-β-D-arabino-pentofuranosylcytosine). Both sapacitabine and CNDAC have demonstrated potent antitumor activity in preclinical models.
Sapacitabine has been evaluated in both hematologic cancers and solid tumors. More than 1,000 patients have received sapacitabine in phase 1, 2, and 3 studies. A Phase 3, multicenter, randomized trial (SEAMLESS) has been conducted as a frontline treatment in elderly patients 70 years or older with newly diagnosed acute myeloid leukemia (AML) who are not candidates for or have refused intensive induction chemotherapy. Sapacitabine has been granted orphan drug designation for the treatment of both AML and myelodysplastic syndrome from the European Medicines Agency and the US Food and Drug Administration.
Phase 1/2 in Leukemias:
Clinical trials are registered with ClinicalTrials.gov. Learn more about sapacitabine trial(s).