GlycoMimetics’ GMI-1271 Receives FDA Breakthrough Therapy Designation for Adult Relapsed/Refractory Acute
Myeloid Leukemia
GlycoMimetics, Inc. (NASDAQ: GLYC) today announced 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. The U.S. Food and Drug Administration (FDA) had previously granted Orphan Drug designation and Fast Track
Status for GMI-1271 in AML.
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) and the American Society of Hematology (ASH). 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) 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.
About GlycoMimetics, Inc.
GlycoMimetics is a clinical-stage biotechnology company focused on cancer and sickle cell disease. GlycoMimetics' most
advanced drug candidate, rivipansel, a pan-selectin antagonist, is being developed for the treatment of vaso-occlusive crisis in
sickle cell disease and is being evaluated in a Phase 3 clinical trial being conducted by its strategic collaborator, Pfizer.
GlycoMimetics' wholly-owned drug candidate, GMI-1271, an E-selectin antagonist, is being evaluated in an ongoing Phase 1/2 clinical
trial as a potential treatment for AML and in a Phase 1 clinical trial in multiple myeloma. GlycoMimetics has also
recently initiated a clinical trial with a third drug candidate, GMI-1359, a combined CXCR4 and E-selectin
antagonist. GlycoMimetics is located in Rockville, MD in the BioHealth Capital Region. Learn more
at www.glycomimetics.com.
Forward-Looking Statements
This press release contains forward-looking statements regarding GlycoMimetics’ planned activities with respect to the clinical
development of its drug candidate, GMI-1271. Actual results may differ materially from those indicated by such forward-looking
statements as a result of various important factors, including the availability and timing of data from ongoing clinical trials,
the uncertainties inherent in the initiation of future clinical trials, whether interim results from a clinical trial will be
predictive of the final results of the trial or results of early clinical trials will be indicative of the results of future
trials, expectations for regulatory approvals, availability of funding sufficient for GlycoMimetics’ foreseeable and unforeseeable
operating expenses and capital expenditure requirements, other matters that could affect the availability or commercial potential
of GlycoMimetics’ drug candidates and other factors discussed in the “Risk Factors” section of GlycoMimetics’ Annual Report on Form
10-K that was filed with the U.S. Securities and Exchange Commission on March 1, 2017 and other filings GlycoMimetics makes with
the Securities and Exchange Commission from time to time. In addition, the forward-looking statements included in this press
release represent GlycoMimetics’ views as of the date hereof. GlycoMimetics anticipates that subsequent events and developments may
cause its views to change. However, while GlycoMimetics may elect to update these forward-looking statements at some point in the
future, GlycoMimetics specifically disclaims any obligation to do so, except as may be required by law. These forward-looking
statements should not be relied upon as representing GlycoMimetics’ views as of any date subsequent to the date hereof.
GlycoMimetics, Inc.
Investor Contact:
Shari Annes, 650-888-0902
sannes@annesassociates.com
or
Media Contact:
Jamie Lacey-Moreira, 410-299-3310
jamielacey@presscommpr.com
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