Press Release

Global Blood Therapeutics Announces Presentation of New GBT440 Data in Sickle Cell Disease at the European Hematology Association's 21st Congress
-- Presentations to Include Additional and Longer-Term Data from Ongoing Phase 1/2 Trial --
-- Company to Host Investor Webcast to Review the Data on Friday, June 10 --

SOUTH SAN FRANCISCO, Calif., May 19, 2016 /PRNewswire/ -- 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 new GBT440 data will be presented in a poster session at the European Hematology Association's (EHA) 21st Congress in Copenhagen. The presentations will include additional data from the ongoing Phase 1/2 GBT440-001 study in sickle cell disease (SCD), including 90-day data from a cohort of patients taking 700 mg of GBT440, 28-day results from three dosing cohorts of GBT440 and additional data on the pharmacokinetics and pharmacodynamics of GBT440.

GBT440 is being developed as an oral, once-daily therapy for patients with SCD. GBT440 works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes 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.

The EHA abstracts are now available at www.ehaweb.org. The poster presentations will include additional data not available in the abstracts. Presentation details are as follows:

Poster Session: Red blood cells and iron -- Clinical 1
Abstract #P371: GBT440, A Novel HbS Polymerization Inhibitor, Increases Hb Oxygen Affinity And Results In A Rapid Improvement In Hemolysis And Anemia
Presenter: Dr. Paul Telfer, Barts Health NHS Trust and Queen Mary, University of London
Date: Friday, June 10, 2016
Time: 5:15-6:45 p.m. CEST/11:15 a.m.-12:45 p.m. ET
Location: Poster area (Hall H)

Poster Session:  Red blood cells and iron -- Clinical 1
Abstract #P370: Pharmacokinetics (PK) and Pharmacodynamics (PD) Of GBT440, A Novel Hemoglobin S (HbS) Polymerization Inhibitor for the Treatment Of Sickle Cell Disease (SCD), In Healthy Volunteers and SCD Patients
Presenter: Mira Patel, Global Blood Therapeutics
Date: Friday, June 10, 2016
Time: 5:15-6:45 p.m. CEST/11:15 a.m.-12:45 p.m. ET
Location: Poster area (Hall H)

Investor Event Webcast Details
GBT will host an investor webcast on Friday, June 10, 2016 at 1:30 p.m. CEST/7:30 a.m. ET, during which members of GBT's management team and distinguished experts Dr. H. Franklin Bunn of Harvard Medical School and Brigham and Women's Hospital, Dr. Paul Telfer of Barts Health NHS Trust and Queen Mary, University of London, and Dr. Wally R. Smith of Virginia Commonwealth University will review the GBT440 data presented at EHA.

The investor event will be webcast live and available for replay from GBT's website at www.globalbloodtx.com in the Investors section.

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 lead product candidate, GBT440, as an oral, anti-polymerization therapy for sickle cell disease (SCD) and is currently evaluating GBT440 in SCD patients in an ongoing Phase 1/2 clinical trial. GBT is also evaluating GBT440 for the treatment of hypoxemic pulmonary disorders, including idiopathic pulmonary fibrosis, and is engaged in other research and development activities targeted toward hereditary angioedema (HAE). To learn more, please visit www.globalbloodtx.com.

Forward-Looking Statements
Statements we make in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. We intend these forward-looking statements, including statements regarding the therapeutic potential and safety profile of GBT440, our ability to receive data from our ongoing Phase 1/2 clinical study of GBT440, and our ability to initiate our pivotal program for GBT440 in SCD, and the timing of these events, 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, the risks that our clinical and preclinical development activities may be delayed or terminated for a variety of reasons, that regulatory authorities may disagree with our clinical development plans or require additional studies or data to support further clinical investigation of our product candidate, and that drug-related adverse events may be observed in later stages of clinical development, along with those set forth in our Annual Report on Form 10-K for the fiscal year ended December 31, 2015 and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2016, as well as discussions of potential risks, uncertainties and other important factors in our subsequent filings with the U.S. Securities and Exchange Commission. 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.

 

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SOURCE Global Blood Therapeutics, Inc.

Myesha Edwards (investors), Global Blood Therapeutics, 650-351-4730, medwards@globalbloodtx.com, Ryan Flinn (media), BrewLife, 415-946-1059, media@globalbloodtx.com