On March 5, 2018 GlycoMimetics, Inc. (NASDAQ: GLY) reported its design for a randomized, double-blind, placebo-controlled Phase 3 clinical trial to evaluate GMI-1271 in combination with MEC (Mitoxantrone, etoposide and Ara-C) or in combination with FAI (fludarabine, cytosine arabinoside and idarubicin) in individuals with relapsed/refractory acute myeloid leukemia (AML) (Press release, GlycoMimetics, MAR 5, 2018, View Source [SID1234524393]). The design is aligned with guidance received from the U.S. Food and Drug Administration (FDA). The single pivotal trial is planned to enroll 380 adult patients worldwide and is expected to begin in the third quarter of 2018. The primary endpoint will be overall survival, and censoring for transplant in the primary efficacy analysis will not be required. Key secondary endpoints will include incidence of severe mucositis and remission rate, which will be assessed in a hierarchical fashion for potential inclusion in the product labeling, if GMI-1271 is approved by the FDA. In 2017, GMI-1271 received Breakthrough Therapy Designation.
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"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. "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. 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. 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."
"Our development strategy now sets us up for multiple, value-creating clinical data readouts, the first of which is topline data from the ongoing Phase 3 trial of rivipansel in sickle cell disease in the second half of 2018," Ms. King added. "In early 2019, we anticipate topline data from our proof-of-concept trial of GMI-1271 in multiple myeloma, and now, by the end of 2020, we expect to have topline data from our pivotal trial of GMI-1271 in patients with relapsed/refractory AML."
Additional details regarding the Phase 3 trial will be provided in the company’s fourth quarter and fiscal year 2017 financial results teleconference on Tuesday, March 6, 2018, at 8:30 a.m. ET. The dial-in number for the conference call is (844) 413-7154 for domestic participants and (216) 562-0466 for international participants, with participant code 1453008. A webcast replay will be available via the "Investors" tab on the GlycoMimetics website for 30 days following the call. A dial-in phone replay will be available for 24 hours after the close of the call by dialing (855) 859-2056 for domestic participants and (404) 537-3406 for international participants, participant code 1453008