GlycoMimetics Inc.'s drug candidate GMI-1271 showed high remission and low mortality rates in patients with acute myeloid leukemia in the phase 2 portion of its ongoing phase 1/2 clinical trial.
The trial showed that patients treated with GMI-1271 together with standard chemotherapy continue to achieve higher-than-expected remission rates based on historical controls, as well as lower-than-expected induction-related mortality rates. Treatment also continues to be well tolerated in 79 patients.
Data showed that among 54 relapsed/refractory acute myeloid leukemia patients, the overall response rate was 41% and the 60-day induction related mortality rate was 7%. The median overall survival time for phase 1 trial patients was 7.6 months.
Remissions were durable enough to allow 9 patients to receive stem cell transplants. More than half of patients who responded with a remission survived for at least a year after treatment.
Oral mucositis, or inflammation with mouth ulcers that is a sign of adverse effects of chemotherapy, was seen at low rates and severity with only one grade 3/4 event observed.
Further, among 25 newly diagnosed elderly patients, the overall response rate was 68%, with 73% in patients with de novo acute myeloid leukemia and 64% in patients with secondary acute myeloid leukemia.
The 60-day mortality rate was 8% and there were no cases of grade 3 or 4 mucositis.
For nine evaluable patients achieving complete remission or complete remission with incomplete marrow recovery, disease-free survival was 100% at six months after treatment.
GlycoMimetics submitted the data from the trial to the U.S. Food and Drug Administration. In May, the FDA granted GMI-1271 breakthrough therapy designation for the treatment of adult patients with relapsed/refractory acute myeloid leukemia.
The agency also granted fast-track status to the drug candidate. In addition, GMI-1271 has been granted orphan drug designation by the FDA and the European Commission.