Global Blood Therapeutics Announces Upcoming Presentation Of Data Supporting Sickle Cell Disease Program At The 58th American Society Of Hematology (ASH) Annual Meeting & Exposition Conference

SOUTH SAN FRANCISCO, Calif., Nov. 03, 2016 (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 need, today announced that new data supporting its GBT440 program in sickle cell disease (SCD) will be presented at the 58 th American Society of Hematology (ASH) Annual Meeting & Exposition in San Diego. The poster presentations will include additional data from the ongoing Phase 1/2 GBT440-001 trial of GBT440 including dosing of 900 mg per day in SCD patients for up to 6 months, and the metabolism of GBT440 in healthy subjects. The poster presentations will also include the methodology utilized to develop the Patient Reported Outcome (PRO) tool that will be used in the HOPE ( Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polym Erization) Study.  Patient enrollment of variant SCD genotypes in the GBT440-001 trial is ongoing, and will support the inclusion of patients with variant genotypes in the HOPE Study.

GBT440 is being developed as an oral, once-daily therapy for patients with sickle cell disease (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. With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT believes that GBT440 may be capable of modifying the progression of SCD. The U.S. Food and Drug Administration (FDA) has granted GBT440 both fast track and orphan drug designations for the treatment of patients with SCD, in recognition of the critical need for new treatments.  The pivotal HOPE Study will begin screening patients by December 2016.

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

Poster Session: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical Poster II Abstract #2488: Long-Term Dosing in Sickle Cell Disease Subjects with GBT440, a Novel HbS Polymerization Inhibitor Presenter: Dr. Jo Howard, Guy's and St Thomas' NHS Foundation Trust, London, UK Date: Sunday, December 4, 2016 Time: 6:00-8:00 p.m. PT/9:00 p.m.-11:00 p.m. ET  Location: San Diego Convention Center, Hall GH

Poster Session: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical Poster II Abstract #2487: Absorption, Metabolism and Excretion of GBT440, a Novel Hemoglobin S (HbS) Polymerization Inhibitor for the Treatment of Sickle Cell Disease (SCD), in Healthy Male Subjects Presenter: Dr. Peter Rademacher, Global Blood Therapeutics Date: Sunday, December 4, 2016 Time: 6:00-8:00 p.m. PT/9:00 p.m.-11:00 p.m. ET  Location: San Diego Convention Center, Hall GH

Poster Session: 903. Outcomes Research—Non-Malignant Conditions: Poster III Abstract #4760: The 10-Item Sickle Cell Disease Severity Measure (SCDSM-10): A Novel Measure of Daily SCD Symptom Severity Developed to Assess Benefit of GBT440, an Experimental HbS Polymerization Inhibitor Presenter: Dr. Jeremy Hobart, Peninsula Schools of Medicine and Dentistry and Dr. Ken Bridges, Global Blood Therapeutics Date: Monday, December 5, 2016 Time: 6:00-8:00 p.m. PT/9:00 p.m.-11:00 p.m. ET  Location: San Diego Convention Center, Hall GH

About Sickle Cell Disease (SCD)SCD is a lifelong 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 TherapeuticsGlobal 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 and will initiate its Phase 3 clinical trial by the end of 2016. GBT is also investigating GBT440 for the treatment of hypoxemic pulmonary disorders in an ongoing Phase 2a study in patients with idiopathic pulmonary fibrosis. To learn more, please visit:

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 implement our clinical development plans for GBT440, the timing of, and our ability to initiate our Phase 3 clinical trial of GBT440 and our ability to enroll patients in and begin screening in our HOPE Study, 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 candidates, and that drug-related adverse events may be observed in later stages of clinical development, along with those risks 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 June 30, 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.
Contact Information: Myesha Edwards (investors)Global Blood Therapeutics650-351-4730investor@globalbloodtx.comJulie Normart (media)

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