Glycomimetics : Joins New Sickle Cell Disease Coalition, Issues Call to Action to Improve Treatment and Care

Jamie Lacey-Moreira jamielacey@presscommpr.com

410-299-3310

Sickle Cell Disease Coalition launches a call to action on disease that has been neglected for far too long

SCD is an inherited, lifelong disorder characterized by red blood cells that become rigid and sickle- shaped, which causes them to stick together and block the flow of oxygen to the body, leading to intense pain and other serious issues such as stroke, organ failure, and even death. SCD affects nearly 100,000 Americans. It is a growing global health problem, and it is estimated that by 2050 the number of people with SCD will increase by 30 percent globallyi. Over the last century, several important discoveries have led to improved diagnosis and treatments, but people with the disease still have severe complications and shorter life expectancy. Many are unable to access the care they need and live with pain and disability due to a lack of effective treatment options.

Earlier this week, at a press conference and formal launch of the Coalition at the Knight Conference Center of the Newseum in Washington, D.C., the Coalition announced a call to action on SCD and the American Society of Hematology and several members of the Coalition issued a new report, State of Sickle Cell Disease: 2016 which outlines unmet needs in four priority areas: Access to care, training and professional education, research and clinical trials, and global health. While the report shows that significant improvements are needed across all areas, it also highlights strategies for change.

"Around the world, people with sickle cell disease face unique challenges - from early death and childhood stroke in countries without early screening programs to lifetimes of mental and physical disability due to lack of access to appropriate care in the United States. We can no longer accept that this is the status quo," said Charles S. Abrams, MD, President of the American Society of Hematology, the organization spearheading the Coalition. "The Sickle Cell Disease Coalition is bringing together a number of groups that care about this disease to take advantage of the opportunities we have for change."

The State of Sickle Cell Disease: 2016 Report includes several goals to help improve the state of SCD treatment and care. These goals include:

• Shifting from acute care of complications to a chronic care model that focuses on prevention of crises, early identification, and intervention for common complications;

• Increasing the availability of providers with SCD expertise through trainings of primary care providers and hematologists;

• Linking research and care more closely through robust clinical research involving a wide range of partners to find new therapies, recruit more individuals with SCD and get clear outcomes faster;

• And designing, testing and implementing sustainable care and pain management approaches for countries with limited resources.

GlycoMimetics is uniting with other organizations in the Coalition to address these goals. In particular, GlycoMimetics will continue to support efforts to advance research designed to improve treatment options for those affected by the disease.

"The Coalition provides an opportunity for the stakeholder community to identify meaningful ways to improve sickle cell disease management," said Helen Thackray, MD, Vice President of Clinical Development and Chief Medical Officer for GlycoMimetics. "We are honored to join the Coalition in its efforts to change the course of the disease."

The Coalition will provide a platform to encourage stakeholders to work together to develop and implement important projects and activities that will ultimately help the SCD community and improve outcomes for individuals with the disease. For more information on the Coalition and its mission, visit www.scdcoalition.org.

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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 of 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 is located in Rockville, MD in the BioHealth Capital Region. Learn more at www.glycomimetics.com.

The mission of the Sickle Cell Disease Coalition (SCDC) (www.scdcoalition.org) is to help amplify the voice of the SCD community, promote awareness, and improve outcomes for individuals with SCD. The Coalition is focused on promoting research, clinical care, education, training, and advocacy as well as providing a platform to encourage stakeholders to work together to develop and implement important projects and activities that will ultimately help the SCD community and improve outcomes for individuals with the disease. The Coalition is comprised of leading patient advocacy groups, people with SCD and their families, researchers, clinicians, policymakers, industry, and foundations with an interest in SCD.

i Frederic B. Piel, Simon I. Hay, Sunetra Gupta, David J. Weatherall and Thomas N. Williams, "Global Burden of Sickle Cell Anaemia in Children under Five, 2010-2050: Modelling Based on Demographics, Excess Mortality, and Interventions," PLOS 10, no. 7 (July 2013): e1001484.