EntreMed, Inc. has announced that the FDA has granted orphan drug designation for the Company’s lead oncology drug candidate, ENMD-2076, for the treatment of acute myeloid leukemia (AML).
The FDA accepted EntreMed’s application upon review of preclinical data as well as initial data from its ongoing Phase 1 clinical study with ENMD-2076 in patients with relapsed or refractory acute myeloid leukemia (AML). As previously presented, initial results from the Phase 1 study demonstrated that ENMD-2076, administered orally as a single agent, was well tolerated in this patient population. The data for 15 evaluable AML patients showed that one patient achieved a CRi (complete remission with incomplete hematological recovery) and two patients achieved a morphologic leukemia-free state (MLFS). Three additional patients participating in the study experienced 11%, 14%, and 65% reductions in marrow blast count.
Carolyn F. Sidor, M.D., EntreMed Vice President and Chief Medical Officer commented on the designation, “We are pleased to have received orphan drug designation for ENMD-2076 in leukemia. Results from our Phase 1 studies along with the preclinical data continue to support the development of ENMD-2076 in both solid and hematological cancers. We previously received orphan drug designation in ovarian cancer, the initial solid tumor indication planned for Phase 2.”