GBT Supports the ASH Research Collaborative’s Data Hub Sickle Cell Disease Program

Grant of $400,000 to help expand sickle cell disease real-world data gathering in the U.S.

WASHINGTON and SOUTH SAN FRANCISCO, Calif., Dec. 02, 2021 (GLOBE NEWSWIRE) -- The ASH Research Collaborative (ASH RC), a non-profit organization established in 2018 by the American Society of Hematology (ASH), and Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that GBT will provide a grant of $400,000 to the ASH RC in support of the ASH RC Data Hub Sickle Cell Disease (SCD) program. With the Data Hub SCD program, the ASH RC seeks to advance research and outcomes for people living with SCD by expanding its current data collection of real-world, research-grade data to include more than half of people living with SCD in the United States.

"We have a shared mission with GBT to help transform the treatment of sickle cell disease," said Martin S. Tallman, M.D., of Memorial Sloan Kettering Cancer Center, who serves as president of the ASH Research Collaborative and the American Society of Hematology. "Working closely with the SCD community, the ASH RC is committed to efficiently capturing longitudinal real-world data to better understand the natural history of sickle cell disease and to create a national repository of high-quality data to accelerate research and enhance clinical care. We aim to give the SCD community a meaningful voice in the creation of one of the world's largest repositories of real-world, research-grade data in SCD. We appreciate GBT's support to help us work toward improving care and outcomes for our patients with this rare disease."

The ASH RC fosters collaborative partnerships to advance progress in hematology and improve the lives of people affected by blood diseases. The foundation of the ASH RC is its Data Hub, a multi-stakeholder, real-world data program that facilitates the exchange of information on hematologic conditions to support scientific inquiry, discovery, and quality improvement. Nearly 30 U.S. clinical sites providing SCD care are currently participating in the Data Hub and are submitting HIPAA-compliant data representing 6,000 individuals living with SCD. The Data Hub is actively enrolling up to an additional 110 clinical sites, and the ASH RC believes it is well-positioned to capture data on more than 50,000 people in the U.S. living with SCD.

GBT is the first biopharmaceutical company to provide the ASH RC with financial support through a grant that will help onboard clinical sites and integrate their data to the Data Hub. These sites are organized across 19 consortia also enrolled with the ASH RC SCD Clinical Trials Network. The purpose of the SCD Clinical Trials Network is to accelerate research that advances treatment and care of those affected by SCD.

"Comprehensive real-world data can empower people with sickle cell disease and their healthcare providers to make decisions regarding their care, while also guiding the development of new treatments and guidelines that we hope will transform SCD into a well-managed disease," said Kim Smith-Whitley, M.D., executive vice president and head of research and development at GBT. "GBT is proud to support the ASH RC and its critical mission to collect real-world data to help address the urgent needs of people with sickle cell disease. There has never been a more important time to advance research initiatives with the potential to improve care and address long-standing gaps in health equity for the SCD community."

The Data Hub can collect a wide variety of data, including electronic medical record data, clinical and laboratory data, genomic or molecular correlates, patient-reported outcomes, and aggregated population data. These data are obtained from inpatient and outpatient clinical sites, industry or government datasets, registries, other U.S. or international sources, and directly from patients. By using state-of-the-art technology to automate data clinical capture and linkages to other data sources, the Data Hub is focused on minimizing data capture burden. Patient information collected in the Data Hub complies with national and local privacy laws and regulations to protect patient privacy.

To learn more, please visit www.ashresearchcollaborative.org.

About Sickle Cell Disease
Sickle cell disease affects more than 100,000 people in the United States,¹ an estimated 52,000 people in Europe,² and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa.³ It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.³ SCD is a lifelong inherited rare blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.⁴ Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled - deoxygenated, crescent-shaped and rigid.^4-6 The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.^5-8

About the ASH Research Collaborative
The ASH Research Collaborative (ASH RC) is a non-profit organization established by the American Society of Hematology (ASH) to improve the lives of people affected by blood diseases by fostering collaborative partnerships to accelerate progress in hematology. The foundation of the ASH RC is its Data Hub and Clinical Trials Network. Through the ASH RC's state-of-the-art data-sharing platforms and patient-centric approach to enrollment, design, and execution of clinical trials for SCD, the ASH RC is making it quicker and more efficient for companies to develop new treatments to help those suffering from hematologic conditions, and to maximize the value of those treatments post-approval. The ASH RC aims to transform research and practice in malignant and non-malignant hematologic diseases throughout the world, for the benefit of patients and the hematology community.

About Global Blood Therapeutics
Global Blood Therapeutics, Inc. (GBT) is a biopharmaceutical company dedicated to the discovery, development and delivery of life-changing treatments that provide hope to underserved patient communities. Founded in 2011, GBT is delivering on its goal to transform the treatment and care of sickle cell disease (SCD), a lifelong, devastating inherited blood disorder. The company has introduced Oxbryta^® (voxelotor) tablets, the first FDA-approved treatment that directly inhibits sickle hemoglobin polymerization, the root cause of red blood cell sickling in SCD. GBT is also advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in Phase 3 development to address pain crises associated with the disease, and GBT021601 (GBT601), the company's next-generation hemoglobin S polymerization inhibitor. In addition, GBT's drug discovery teams are working on new targets to develop the next wave of potential treatments for SCD. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news.

References

1. Centers for Disease Control and Prevention website. Sickle Cell Disease Research. https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-understanding-sickle-cell-disease.html. Accessed December 1, 2021.
2. European Medicines Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed June 12, 2020.
3. Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed June 3, 2019.
4. National Heart, Lung, and Blood Institute website. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed August 5, 2019.
5. Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
6. Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
7. Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
8. Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.

ASH Contact:
Andrea Fischer, American Society of Hematology
202-823-4884
[email protected]

GBT Contact:

Steven Immergut (media)
650-410-3258
[email protected]

Courtney Roberts (investors)
650-351-7881
[email protected]

Source: Global Blood Therapeutics, Inc.

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