Press Release

Global Blood Therapeutics Announces New GBT440 Data in Sickle Cell Disease to be Presented at the American Society of Hematology (ASH) Annual Meeting

Nov 05, 2015 at 9:20 AM EST

SOUTH SAN FRANCISCO, Calif., Nov. 05, 2015 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT), a biopharmaceutical company developing novel therapeutics for the treatment of grievous blood-based disorders with significant unmet needs, today announced that data from the ongoing Phase 1/2 GBT440-001 study of GBT440 will be presented at the 57thAmerican Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida. Data will include an oral presentation of clinical data from the GBT440-001 study in sickle cell disease (SCD) including data from SCD patients receiving additional dosing regimens beyond those described in the abstract, as well as a poster presentation of pharmacokinetic data.  

GBT440 is an oral, once-daily therapy for the treatment of SCD that works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT440 blocks polymerization and the resultant sickling of red blood cells (RBCs). With the potential to restore normal hemoglobin function, GBT440 may be capable of modifying the progression of SCD. Currently, GBT440 is being investigated in an ongoing Phase 1/2 clinical trial in both healthy subjects and SCD patients.

The abstracts are now available at Following are details of the presentations, which will include additional data not currently available in the published abstracts.

Oral Session: 114. Hemoglobinopathies, Excluding Thalassemia – Clinical: Promising Therapies in Sickle Cell Disease 
Abstract #542: GBT440, a Potent Anti-Sickling Hemoglobin Modifier Reduces Hemolysis, Improves Anemia and Nearly Eliminates Sickle Cells in Peripheral Blood of Patients with Sickle Cell Disease
Presenter: Dr. Claire Hemmaway, MA (Hons) Cantab, MBBS, FRCP, FRCPATH, Queens Hospital, Essex, United Kingdom
Session Date: Monday, December 7, 2015
Session Time: 10:30 a.m.12:00 p.m. Eastern Time (ET)
Presentation Time: 10:45 a.m. ET
Location: Orange County Convention Center, W340

Poster Session: 113. Hemoglobinopathies, Excluding Thalassemia – Basic and Translational Science: Poster II
Abstract #2172: GBT440 Demonstrates High Specificity for Red Blood Cells in Nonclinical Species
Session Date/Time: Sunday, December 6, 2015, 6 – 8 p.m. ET
Location: Orange County Convention Center, Hall A

About Sickle Cell Disease (SCD)
Sickle cell disease (SCD) is an inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, leading to formation of abnormal hemoglobin known as sickle hemoglobin, or HbS. In its deoxygenated state, HbS has a propensity to polymerize, or bind together forming long, rigid rods within a red blood cell (RBC). The polymer rods deform RBCs to assume a sickled shape and to become inflexible, which can cause blockage in small blood vessels. 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 (the destruction of RBCs), can eventually lead to multi-organ damage and early death.

About Global Blood Therapeutics
Global Blood Therapeutics, Inc. (GBT) 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 initial product candidate, GBT440, as an oral, once-daily therapy for sickle cell disease (SCD) and is currently evaluating GBT440 in both healthy subjects and SCD patients in a randomized, placebo-controlled, double-blind Phase 1/2 clinical trial. In addition to GBT440 for the treatment of SCD, GBT is engaged in research and development activities targeted toward hypoxemic pulmonary disorders, including idiopathic pulmonary fibrosis (IPF) and hereditary angioedema (HAE). To learn more, please visit:  

Forward-Looking Statements
Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act and Section 21E of the Securities Exchange Act. We intend these forward-looking statements, including statements regarding the therapeutic potential of GBT440 and any other product candidates that we may identify and develop, to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. We can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved, and furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, those set forth in the prospectus for our initial public offering of common stock that was filed with the U.S. Securities and Exchange Commission (the "SEC") on August 12, 2015, as well as discussions of potential risks, uncertainties and other important factors in our subsequent filings with the SEC. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.


Contact Information:
Joey Fleury (investors)

Ryan Flinn (media)

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