On May 17, 2017 GlycoMimetics, Inc. (NASDAQ: GLYC) reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for treatment of adult relapsed/refractory acute myeloid leukemia (AML) to the company’s drug candidate GMI-1271, an E-selectin antagonist currently being evaluated in the Phase 2 portion of a Phase 1/2 clinical trial in patients with AML (Press release, GlycoMimetics, MAY 17, 2017, View Source [SID1234617412]). The U.S. Food and Drug Administration (FDA) had previously granted Orphan Drug designation and Fast Track Status for GMI-1271 in AML.
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In the ongoing clinical trial, GMI-1271 is being administered, along with chemotherapy, to patients with relapsed or refractory AML as well as those 60 years of age and older with newly diagnosed disease. Data from this trial were presented in 2016 at meetings of the European Hematology Association (EHA) (Free EHA Whitepaper) and the American Society of Hematology (ASH) (Free ASH Whitepaper). In the trial, patients treated with GMI-1271 achieved higher than expected remission rates and lower than expected 30- and 60-day mortality rates in early evaluations of patients with relapsed/refractory AML as well as in newly diagnosed patients. In March 2017, the Company announced that the first of two patient cohorts in the Phase 2 portion of the trial of GMI-1271 had completed enrollment. In April 2017, the Company announced plans to present further data updates on both patient populations in the ongoing AML trial at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June.
The FDA grants Breakthrough Therapy designation to companies to help accelerate development and review of drug candidates when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies. The designation is designed to expedite the development and review of designated therapies, without changing FDA standards for new drug approval.
"The FDA’s granting to GMI-1271 of Breakthrough Therapy designation will further help GlycoMimetics to accelerate the development of GMI-1271 as a treatment for this very difficult-to-treat patient population," said Helen Thackray, MD, Chief Medical Officer of GlycoMimetics. "We believe GMI-1271 when combined with chemotherapy has the potential to address an unmet therapeutic need for individuals living with AML. We are encouraged by our clinical results to date, and look forward to working closely with the FDA to bring this novel therapy to patients as quickly as possible."
About AML
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. AML is the most common type of acute leukemia in adults. Each year in the United States, about 19,900 people (usually older than 45 years of age) are diagnosed, and about 10,400 people die from all forms of the disease, according to the American Cancer Society. Unlike other cancers that start in an organ and spread to the bone marrow, AML is known for rapid growth of abnormal white blood cells that gather in the bone marrow, getting in the way of normal blood cell production. The lack of normal blood cells can cause some of the symptoms of AML, including anemia (shortage of red blood cells resulting in tiredness and weakness), neutropenia (shortage of white blood cells that may lead to increased infections), and thrombocytopenia (shortage of platelets in the blood that may lead to excessive bleeding). Current treatment options for AML consist of reducing and eliminating cancer cells mainly through chemotherapy, radiation therapy, and stem cell transplantation.