GlycoMimetics,
Inc. (NASDAQ: GLYC) announced today that the U.S.
Food and Drug Administration (FDA) has granted Orphan Drug
designation to GMI-1271,
a novel and proprietary E-selectin antagonist in the company’s pipeline
for treatment of patients with acute myeloid leukemia (AML).
GlycoMimetics is currently recruiting patients in a Phase 1/2,
open-label multicenter study designed to evaluate the safety,
pharmacokinetics and efficacy of GMI-1271 in combination with
chemotherapy in adult patients with AML; a
Phase 1 study in healthy volunteers was completed last year.
“Having the FDA designate GMI-1271 as an orphan drug for the treatment
of AML is an important accomplishment for GlycoMimetics. This is a
significant regulatory milestone for our program,” said Helen
Thackray, M.D., FAAP, Vice President of Clinical Development and
Chief Medical Officer, GlycoMimetics. “We look forward to advancing
GMI-1271 through clinical trials targeting E-selectin for multiple
blood, or hematologic, cancers.”
The FDA’s Orphan Drug designation program is designed to promote the
development of promising therapeutics for the treatment of rare diseases
affecting fewer than 200,000 people in the United States. Orphan Drug
designation includes benefits such as a potential seven-year period of
U.S. marketing exclusivity after approval. Other potential advantages
include protocol assistance, the ability to apply for research funding,
tax credits for certain research expenses, and regulatory fee waivers.
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, and it is
estimated that there were over 18,000 new cases and over 10,000 deaths
from the disease in 2014. Unlike other cancers that start in an organ
and spread to the bone marrow, AML is associated with rapid growth of
abnormal white blood cells that gather in the bone marrow and, as a
result, impede normal blood cell production. While leukemic cells move
into the blood, the lack of normal blood cells can lead to a number of
symptoms, 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 include chemotherapy and stem cell
transplantation, both of which can destroy cancer cells but do not
reduce the related side effects.
About GlycoMimetics, Inc.
GlycoMimetics is a clinical stage biotechnology company focused on the
discovery and development of novel glycomimetic drugs to address unmet
medical needs resulting from diseases in which carbohydrate biology
plays a key role. Pfizer is the company's development partner for
rivipansel, a GlycoMimetics-discovered investigational therapy for pain
crisis associated with sickle cell disease, and is preparing to conduct
a Phase 3 clinical study. A GlycoMimetics wholly-owned drug candidate
(GMI-1271) for acute myeloid leukemia (AML) and other blood disorders is
also in clinical trials. Glycomimetics are molecules that mimic the
structure of carbohydrates involved in important biological processes.
Using its expertise in carbohydrate chemistry and knowledge of
carbohydrate biology, GlycoMimetics is developing a pipeline of
glycomimetic drug candidates that inhibit disease-related functions of
carbohydrates, such as the roles they play in inflammation, cancer and
infection. Learn more at www.glycomimetics.com.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements regarding the
clinical development of GMI-1271. Actual results may differ materially
from those in these forward-looking statements. For a further
description of the risks associated with these statements, as well as
other risks facing GlycoMimetics, please see the risk factors described
in the Company’s annual report on Form 10-K that was filed with the U.S.
Securities and Exchange Commission on March 16, 2015, and other filings
the Company makes with the SEC from time to time. Forward-looking
statements speak only as of the date of this release, and GlycoMimetics
undertakes no obligation to update or revise these statements, except as
may be required by law.
Copyright Business Wire 2015