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GBT Announces Upcoming Data Presentations Supporting Voxelotor Sickle Cell Disease Program at 23rd European Hematology Association (EHA) Congress

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SOUTH SAN FRANCISCO, Calif., May 17, 2018 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced that data supporting its voxelotor program in sickle cell disease (SCD) will be presented during the 23rd European Hematology Association (EHA) Congress in Stockholm, taking place June 14-17, 2018. A total of five abstracts have been accepted for poster presentation including 24-week data on patients treated with the 900 mg dose of voxelotor in Part B of the HOPE-KIDS 1 Study (GBT440-007), a Phase 2a open-label study in adolescents ages 12 to 17.

The EHA abstracts are now available at www.ehaweb.org. Details are as follows:

Friday, June 15

Poster Session: Sickle Cell Disease
Abstract #PF709: Results from a Phase 2a Study (GBT440-007) Evaluating Adolescents with Sickle Cell Disease Treated with Multiple Doses of Voxelotor (GBT440), a HbS Polymerization Inhibitor
Time: 5:30-7:00 p.m. CEST / 11:30 a.m.-1:00 p.m. ET
Location: Poster Area (Hall A)

Poster Session: Sickle Cell Disease
Abstract #PF711: Compassionate-Use Voxelotor (GBT440) for Patients with Severe Sickle Cell Disease (SCD) and Life-Threatening Comorbidities
Time: 5:30-7:00 p.m. CEST / 11:30 a.m.-1:00 p.m. ET
Location: Poster Area (Hall A)

Poster Session: Sickle Cell Disease
Abstract #PF713: Pharmacokinetics (PK) of Voxelotor (GBT440) Using Population Pharmacokinetic (PPK) and Physiologically Based Pharmacokinetic (PBPK) Modeling in Pediatric Subjects with Sickle Cell Disease (SCD)
Time: 5:30-7:00 p.m. CEST / 11:30 a.m.-1:00 p.m. ET
Location: Poster Area (Hall A)

Saturday, June 16

Poster Session: Sickle Cell Disease
Abstract #PS1455: Sickle Cell Disease Severity Measure: Development, Translation, and Patient Cultural Sensitivity Validation
Time: 5:30-7:00 p.m. CEST / 11:30 a.m.-1:00 p.m. ET
Location: Poster Area (Hall A)

Poster Session: Sickle Cell Disease
Abstract #PS1461: Novel Trial Design to Evaluate Oral Voxelotor for the Treatment of Sickle Cell Disease: The Phase 3 Hemoglobin Oxygen Affinity Modulation to Inhibit Sickle Hemoglobin Polymerization (HOPE) Trial
Time: 5:30-7:00 p.m. CEST / 11:30 a.m.-1:00 p.m. ET
Location: Poster Area (Hall A)

About Sickle Cell Disease (SCD)
SCD is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, which leads to the formation of abnormal hemoglobin known as sickle hemoglobin (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 capillaries and 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 Voxelotor in Sickle Cell Disease
Voxelotor (previously called GBT440) is being developed as an oral, once-daily therapy for patients with SCD. Voxelotor works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes voxelotor 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 voxelotor may potentially modify the course of SCD. In recognition of the critical need for new SCD treatments, the U.S. Food and Drug Administration (FDA) has granted voxelotor Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD. The European Medicines Agency (EMA) has included voxelotor in its Priority Medicines (PRIME) program, and the European Commission (EC) has designated voxelotor as an orphan medicinal product for the treatment of patients with SCD.

GBT is currently evaluating voxelotor in the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a Phase 3 clinical study in patients age 12 and older with SCD. Additionally, voxelotor is being studied in the ongoing Phase 2a HOPE-KIDS 1 Study, an open-label, single- and multiple-dose study in pediatric patients (age 6 to 17) with SCD. HOPE-KIDS 1 is assessing the safety, tolerability, pharmacokinetics and exploratory treatment effect of voxelotor.

About GBT
GBT is a clinical-stage biopharmaceutical company determined to discover, develop and deliver innovative treatments that provide hope to underserved patient communities. GBT is developing its lead product candidate, voxelotor, as an oral, once-daily therapy for sickle cell disease. To learn more, please visit www.gbt.com and follow the company on Twitter @GBT_news.

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 voxelotor, our ability to implement and complete our clinical development plans for voxelotor, our ability to generate and report data from our ongoing and potential future studies of voxelotor (including our ongoing Phase 3 HOPE Study and our ongoing Phase 2a HOPE-KIDS 1 Study), regulatory review and actions relating to voxelotor, 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 results of clinical trials may be subject to differing interpretations, 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, that drug-related adverse events may be observed in clinical development, and that data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review or approval, along with those risks set forth in our Annual Report on Form 10-K for the fiscal year ended December 31, 2017 and our Quarterly Report on Form 10-Q for the quarter ended March 31, 2018, 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 Lacy (investors)
GBT
650-351-4730
investor@gbt.com

Julie Normart (media)
W2O pure
415-946-1087
media@gbt.com

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