About Tosedostat

Tosedostat (toe-SEE-doe-stat) is a selective, oral inhibitor of aminopeptidases, enzymes which are required by tumor cells to extract from proteins amino acids necessary for growth and tumor cell survival. Tosedostat has demonstrated significant anti-tumor responses in blood-related cancers and solid tumors in Phase 1 and 2 clinical trials.

Development Status

There are several ongoing Phase 2 cooperative group-sponsored trials and investigator-sponsored trials evaluating the clinical activity of tosedostat in combination with standard agents in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Data from these signal-finding trials may help inform the appropriate design for a Phase 3 trial. Please see for more information.

Tosedostat in Combination With Cytarabine or Decitabine in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome This study is ongoing but is not recruiting participants.

Combination Study of Cytarabine and Tosedostat in Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndromes (MDS) This study is ongoing but is not recruiting participants.



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