Global Blood Therapeutics Joins Sickle Cell Disease (SCD) Coalition in Support of SCD Community with Goal to Improve Treatment and Care

SOUTH SAN FRANCISCO, Calif., Sept. 06, 2016 -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT), a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet need, today announced that in connection with Sickle Cell Disease (SCD) Awareness Month, the company has joined the Sickle Cell Disease Coalition. The Coalition is comprised of leading patient advocacy groups, researchers, clinicians, policymakers, industry, and foundations, in a collective effort to improve SCD care, early diagnosis, treatment, and research both in the United States and globally. In addition, on September 10, 2016, GBT’s employees will join the Sickle Cell Anemia Awareness Organization (SCAA) for the 3^rd Annual KMJ Sickle Cell Warrior Walk in San Francisco.

“Historically, the SCD community has been underserved. By joining forces with other key stakeholders, we believe the newly formed Sickle Cell Disease Coalition will build public awareness of this devastating disease and bring attention and resources to the SCD community that it deserves,” said Ted W. Love, M.D., president and chief executive officer of GBT. “At GBT, our continued research and development activities for GBT440 are inspired by the patients and caregivers that we serve. We are dedicated to the SCD community and will continue to work diligently to bring our potentially disease-modifying therapeutic to this community.”

SCD is a growing health problem that currently affects approximately 100,000 Americans and it is estimated that by 2050 the number of people with SCD will increase by 30 percent globallyⁱ. SCD is an inherited, lifelong disorder characterized by red blood cells that become rigid and sickle-shaped and block the flow of oxygen to the body. Beginning in childhood, SCD patients suffer unpredictable and recurrent episodes or crises of severe pain due to blocked blood flow to organs, which often lead to psychosocial and physical disabilities. This blocked blood flow, combined with hemolytic anemia, can eventually lead to stroke, multi-organ damage and premature death. 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.

Today a press conference will be held at the Knight Conference Center of the Newseum in Washington, D.C. to formally launch the Coalition. During the event, the Coalition will announce a call to action on SCD, and the American Society of Hematology and several members of the Coalition, will issue a new report, State of Sickle Cell Disease: 2016. The report 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.

In addition to joining the Sickle Cell Disease Coalition, GBT has partnered with Sickle Cell Anemia Awareness Organization to help increase awareness about the disease. In recognition of SCD Awareness Month, GBT will be sponsoring and participating in SCAA’s 3^rd Annual KMJ Sickle Cell Warrior Walk on Saturday, September 10^th from 9:30 a.m. to 1:30 p.m. This walk is a special event that brings together patients, families and caregivers. For more information or to register for the Warrior Walk, visit the event page here. Registration is free but donations are welcomed, with all proceeds going toward the Foundation of Sickle Cell Disease Research (FSCDR).

About Global Blood Therapeutics
Global Blood Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat grievous blood-based disorders with significant unmet need. GBT is developing its lead product candidate, GBT440, as an oral, once-daily therapy for sickle cell disease (SCD) and is currently evaluating GBT440 in a Phase 1/2 study in both healthy subjects and adults with SCD and a Phase 2a study in adolescents with SCD. GBT is also investigating GBT440 for the treatment of hypoxemic pulmonary disorders in an ongoing Phase 2a study in patients with idiopathic pulmonary fibrosis (IPF). In addition to GBT440, GBT is engaged in research and development activities with an oral kallikrein inhibitor for the prevention of hereditary angioedema (HAE) attacks. To learn more, please visit: www.globalbloodtx.com. 

About the Sickle Cell Disease Coalition
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.

Contact Information: 
Myesha Edwards (investors)
Global Blood Therapeutics
650-351-4730
[email protected]

Julie Normart (media)
BrewLife
415-946-1087
[email protected]