GlycoMimetics Doses First Patient in Phase 2 Portion of Clinical Trial of GMI-1271 in Newly Diagnosed Acute Myeloid Leukemia

ROCKVILLE, Md.–(BUSINESS WIRE)–GlycoMimetics, Inc. (NASDAQ: GLYC) today announced dosing of the first
patient with newly diagnosed acute myeloid leukemia (AML) in the Phase 2
portion of its ongoing Phase 1/2 study evaluating its novel E-selectin
antagonist, GMI-1271, combined with chemotherapy. Earlier this month, the
company announced it had received Fast Track designation
from the
U.S. Food and Drug Administration (FDA) for GMI-1271 for treatment of
adult patients with relapsed or refractory AML and elderly patients aged
60 years or older with AML. In addition, GlycoMimetics recently
announced that the first patient with relapsed or refractory AML has
been dosed
in the other arm of the Phase 2 portion of this study.

For the study’s Phase 2 portion, the optimal dose of GMI-1271 has been
determined, and in this arm of the study clinical investigators will
study the effects on newly diagnosed patients receiving the drug
candidate to obtain additional safety and efficacy data. Study
enrollment in this arm is limited to patients at least 60 years of age
who have been newly diagnosed with AML and are eligible to receive
treatment with the chemotherapy agents cytarabine and idarubicin
(‘7+3’). All patients must be eligible to receive this intensive
chemotherapy regimen, and will be given GMI-1271 in addition to this
combination chemotherapy. During the Phase 1 portion of the study,
patients received a single cycle of treatment including GMI-1271. During
this Phase 2 portion, certain patients will be eligible to receive
additional cycles of treatment.

“The data from the first cohorts point to both the safety and potential
efficacy of GMI-1271 as a treatment for AML,” said Helen
Thackray
, M.D., Chief Medical Officer of GlycoMimetics. “We are now
enrolling a new group of study participants to evaluate the effects of
GMI-1271 on newly diagnosed patients who also are receiving
chemotherapy. If the second half of the trial confirms our earlier
preclinical and clinical findings, we believe that GMI-1271 could well
address the unmet needs of AML patients, beyond what is currently
possible with available therapies.”

This clinical trial is a multinational open-label study evaluating
endpoints for safety, pharmacokinetics (PK) and efficacy of GMI-1271 in
combination with induction chemotherapy in patients with high-risk AML.
This trial is being conducted at a number of academic medical
institutions in the United States, Ireland, and Australia. While the
primary objective is to assess safety, additional endpoints include
overall response rate, biomarkers of activity, durability of response
and overall survival. This Phase 2 portion of the study in newly
diagnosed patients is expected to include approximately 25 participants.

GlycoMimetics announced
on June 10
, presentation of data in patients with
relapsed/refractory acute AML from the Phase 1 portion of this ongoing
study. Data were reported at the European Hematology Association 21st
Congress
in Copenhagen, Denmark in a poster entitled “Results of a
Phase 1 study of GMI-1271, a potent E-selectin antagonist in combination
with induction chemotherapy in relapsed/refractory AML: a novel,
well-tolerated regimen with a high remission rate.”

About GMI-1271

GMI-1271 is designed to block E-selectin (an adhesion molecule on cells
in the bone marrow) from binding with AML cells as a targeted approach
to disrupting well-established mechanisms of leukemic cell resistance
within the bone marrow microenvironment. Preclinical research points to
the drug’s potential role in moving cancerous cells out of the
protective environment of the bone marrow where they hide and escape the
effects of chemotherapy. In preclinical studies using animal models of
AML, the results of which were presented at meetings of the American
Society of Hematology (ASH), GMI-1271 was also associated with a
reduction of chemotherapy-induced neutropenia and chemotherapy-induced
mucositis.

About GlycoMimetics, Inc.

GlycoMimetics is a clinical-stage biotechnology company focused on
sickle cell disease and cancer. 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.
GlycoMimetics expects to file an IND with the FDA for a third drug
candidate, GMI-1359, a combined CXCR4 and E-selectin antagonist, in the
third quarter of 2016. 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 February
29, 2016, 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.

Contacts

GlycoMimetics, Inc.
Investor Contact:
Shari Annes,
650-888-0902
sannes@annesassociates.com
or
Media
Contact:

Jamie Lacey-Moreira, 410-299-3310
jamielacey@presscommpr.com