Patient Information

For GMI-1070, currently being studied in vaso-occlusive crisis (VOC) of sickle cell disease

For GMI-1271, currently being studied for its potential in improving treatment for blood cancers such as Acute Myeloid Leukemia (AML), and other cancers that metastasize (or spread) from one part of the body to another

GlycoMimetics has reported results in its Phase 2 clinical trial studying its lead investigational drug GMI-1070 in vaso-occlusive crisis (VOC) of sickle cell disease.

About Sickle Cell Disease
Sickle cell disease is one of the most prevalent genetic disorders, affecting between 90,000 and 100,000 people in the U.S. alone. It is a chronic condition with substantial illness and death, responsible for more than 70,000 hospitalizations per year in the U.S. One of the most debilitating effects of sickle cell disease is vaso-occlusive crisis (VOC), where changes in blood protein cause red blood cells to become rigid and stick inside small blood vessels.  This causes blockages in blood flow and results in pain, which can require medical care and hospital stays for pain medication. Other medical problems can also occur during vaso-occlusive crisis, which can complicate treatment and lead to longer hospital stays.

GMI-1070 for Treatment of Vaso-occlusive Crisis of Sickle Cell Disease
The first potential treatment use for GMI-1070, the lead investigational drug candidate in the GlycoMimetics pipeline, may be in “vaso-occlusive crisis of sickle cell disease.” GMI-1070 is intended to treat vaso-occlusive crisis by reducing the cell adhesion and inflammation that is believed to contribute to blocking blood flow. GlycoMimetics has begun to evaluate if this may reduce the pain experienced by individuals during sickle cell crisis and shorten their time in the hospital. GlycoMimetics, along with its partner Pfizer, is also studying whether this may reduce other medical problems that result from blood flow blockages.

GMI-1070 has received Orphan Drug status and Fast Track status from the U.S. Food and Drug Administration (FDA), which may lead to speedier delivery to people living with sickle cell disease, if the treatment is found to be effective.

In January 2013, GlycoMimetics, Inc. announced it had completed enrolling patients in a Phase 2 sickle cell disease trial with GMI-1070.  GlycoMimetics enrolled 76 patients ages 12 to 60 at 22 trial sites in the United States and Canada. The company reported topline data from the trial in April 2013. In this trial, patients treated with GMI-1070 experienced reductions in time to reach resolution of VOC, length of hospital stay and use of opioid analgesics for pain management, in each case as compared to patients receiving placebo.

For More Information
GlycoMimetics posts information for all ongoing clinical trials on the National Institutes of Health’s (NIH) clinicaltrials.gov website. To learn more, please visit http://clinicaltrials.gov/ct2/show/NCT01119833?term=glycomimetics&rank=1 or email: clinicaltrials@glycomimetics.com.

For Community Organizations
GlycoMimetics, Inc. is committed to partnering with those affected by sickle cell disease and the organizations that support them. Please click on this link to learn more: Information for Sickle Cell Disease Community Organizations »

GMI-1271 is currently being studied for its potential in improving treatment for blood cancers such as Acute Myeloid Leukemia (AML), and other cancers that metastasize (or spread) from one part of the body to another.

About Acute Myeloid Leukemia
Adult 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 will be 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 known for rapid growth of abnormal white blood cells that gather in the bone marrow and in most cases move into the blood, getting in the way of normal blood cell production. The lack of normal blood cells can cause some of the symptoms of acute myeloid leukemia, 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.

GMI-1271
GMI-1271 is currently being studied for its potential in improving treatment for blood cancers such as AML, and other cancers that metastasize, or spread from one part of the body to another. The drug is also being investigated for thromboembolic complications (those occurring when a blood vessel is blocked by a blood clot) and for its potential to reduce some of the side effects of chemotherapy, including mucositis (i.e., the painful inflammation and ulceration of the mucous membranes lining the digestive tract) and neutropenia (i.e., the presence of abnormally few neutrophils in the blood, leading to increased susceptibility to infection).

The first clinical program with GMI-1271 will address treatment of acute myeloid leukemia (AML). GlycoMimetics recently initiated a Phase 1 trial of GMI-1271 in healthy volunteers.

For More Information
GlycoMimetics posts information for all ongoing clinical trials on the National Institutes of Health’s (NIH) clinicaltrials.gov website. If you are interested in receiving updates or more information, please email: clinicaltrials@glycomimetics.com.