The design for Phase 3 clinical trial to evaluate GMI-1271 in combination with mitoxantrone, etoposide and Ara-C (MEC) or in combination with fludarabine, cytosine arabinoside and idarubicin (FAI) in individuals with relapsed/refractory acute myeloid leukemia (AML) was revealed by GlycoMimetics, Inc. yesterday.
The randomized, double-blind, placebo-controlled study is aligned with guidance received from the U.S. Food and Drug Administration (FDA) and is anticipated to enroll 380 patients worldwide, beginning in the third quarter of 2018. The primary endpoint for the single pivotal trial will be overall survival (OS), and censoring for transplant in the primary efficacy analysis will not be obligatory.
AML first presents in the bone marrow and rapidly spreads to the blood. It is characterized by the swift expansion of white blood cells, and the enflamed cells impede the creation and development of classic blood cells. GMI-1271 is being developed as a targeted approach to block an adhesion molecule on cells in the bone marrow, known as E-selectin, from binding with blood cancer cells.
GMI-1271 was granted orphan drug designation by the FDA in May 2015. In 2017, GMI-1271 received Breakthrough Therapy Designation.
“Reaching alignment with the FDA on overall survival as the primary endpoint for the trial, without statistical censoring for transplant, positions GMI-1271 well for a potential successful outcome,” said Rachel King, Chief Executive Officer of GlycoMimetics in a press release. “Getting more patients to transplant following treatment with GMI-1271 is one of our goals for this therapy. If we accomplish this, we hope GMI-1271 will contribute to prolonged overall survival for relapsed/refractory AML patients.
Key secondary endpoints will include incidence of severe mucositis and remission rate, which will be assessed in a tiered approach for potential inclusion in the product labeling, should GMI-1271 be approved by the FDA.
“We believe this is a rigorously designed Phase 3 trial that has the potential to bring us one step closer to meeting the significant unmet needs of this patient population,” said King. “In addition, we believe that our trial design should streamline the path to data on overall survival, considered the ‘gold standard’ of clinical benefit, and that if this primary endpoint is achieved, it should position GMI-1271 optimally with U.S. and European regulatory agencies, as well as in the marketplace.”
King has noted that she expects the company to have multiple clinical readouts planned starting at the end of 2018 and through 2019 and 2020. Top-line data from the trial of GMI-127 in patients with relapsed/refractory AML are expected by the end of 2020.
Additional details regarding the Phase 3 trial will be provided in the company’s fourth quarter and fiscal year 2017 financial results teleconference today.
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