Press Release

GBT Announces Upcoming Data Presentations at 60th American Society of Hematology (ASH) Annual Meeting & Exposition

Company to Present Three Oral Presentations, Including Results Beyond 12 Weeks from Part A of the HOPE Study

Company to Host Investor Webcast on Monday, December 3

SOUTH SAN FRANCISCO, Calif., Nov. 01, 2018 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that four abstracts related to its sickle cell disease (SCD) development programs, including voxelotor, have been accepted for oral and poster presentation during the 60th American Society of Hematology (ASH) Annual Meeting & Exposition, taking place December 1-4, at the San Diego Convention Center.

“Our data presentations at ASH 2018, which include a meta-analysis of published literature showing that chronic anemia increases the risk for serious negative clinical outcomes like stroke, kidney failure and premature death in SCD patients, support that raising hemoglobin is meaningful, even at increases below 1 g/dL. These findings further strengthen the scientific rationale that a drug like voxelotor, that safely raises hemoglobin and improves tissue oxygenation, has the potential to be a disease-modifying treatment to reduce the morbidity and mortality of SCD,” said Ted W. Love, M.D., president and chief executive officer of GBT. “We look forward to presenting new longer-term exposure and safety data from Part A of our pivotal Phase 3 HOPE Study and data from the 1500 mg cohort of the HOPE-KIDS 1 Study as we continue to advance the clinical development of voxelotor with the goal of bringing it to children, adolescents and adults with this severe and debilitating disease.”

The voxelotor results that will be presented at the meeting will include longer-term exposure and safety data not available at the time the abstracts were submitted to the Congress, and, as such, remain under embargo until the time of presentation.

The systematic literature review and meta-analysis identified and evaluated data from approximately 140 SCD studies published in peer-reviewed journals over the past 20 years. This comprehensive evaluation showed a significant relationship between chronic anemia and negative clinical outcomes in SCD patients. Specifically, low hemoglobin levels increased the risk for:

  • Stroke, silent cerebral infarct and increased transcranial doppler-measured cerebral artery velocity (based on 10 studies of 3,397 patients);
  • Kidney disease as determined by the presence of microalbuminuria (based on eight studies of 1,579 pediatric patients);
  • Elevated estimated pulmonary artery systolic pressure (based on 12 studies of 1,465 patients); and
  • Premature death (based on six studies of 3,196 patients).

The meta-analysis found that hemoglobin was significantly lower by approximately 0.4 to 1 g/dL among patients experiencing negative outcomes. Further modeling of these data predicted that an increase in hemoglobin of 1 g/dL or greater might reduce the risk of stroke by 41 percent and of mortality by 64 percent.

The ASH abstracts are now available at www.hematology.org. Details of the presentations are as follows:

Saturday, December 1

Oral Session: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical
Abstract #117261: Low Hemoglobin Increases Risk for Stroke, Kidney Disease, Elevated Estimated Pulmonary Artery Systolic Pressure, and Premature Death in Sickle Cell Disease: A Systematic Literature Review and Meta-Analysis
Presenter: Kenneth Ataga, MBBS, Director, University of Tennessee Health Science Center’s (UTHSC) Center for Sickle Cell Disease, Memphis                                                
Time: 8:45 a.m. PT                                                                                                                            
Location: Room 25B

Monday, December 3                                                                                                

Oral Session: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical                             
Abstract #118508: Results from Part A of the Hemoglobin Oxygen Affinity Modulation to Inhibit HbS Polymerization (HOPE) Trial (GBT440-031), a Placebo-Controlled Randomized Study Evaluating Voxelotor (GBT440) in Adults and Adolescents with Sickle Cell Disease                                      
Presenter: Elliott Vichinsky, M.D., Director of Hematology/Oncology, UCSF Benioff Children’s Hospital, Oakland, Calif.
Time: 7:00 a.m. PT                                                                                                                               
Location: Room 28D

Oral Session: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical                      
Abstract #117510: Efficacy and Safety of 1500mg Voxelotor in a Phase 2a Study (GBT440-007) in Adolescents with Sickle Cell Disease                                                                                                 
Presenter: Clark Brown, M.D., Ph.D., Clinical Director, Children's Healthcare of Atlanta
Time: 8:00 a.m. PT                                                                                                                               
Location: Room 28D

Poster Session: 901. Health Services Research—Non-Malignant Conditions
Abstract #119420: Societal Costs of Sickle Cell Disease in the United States                                       
Presenter: Kartik Pappu, Ph.D., Associate Director, Commercial Planning, GBT, South San Francisco, Calif.
Time: 6:00-8:00 p.m. PT                                                                                                                           
Location: Hall GH

Investor Webcast Details                                                                                                            
GBT will host an investor event webcast on Monday, December 3, 2018, at 12:00 p.m. PT/3:00 p.m. ET to review the data being presented at the 2018 ASH Annual Meeting. The event will be webcast live and available for replay from GBT's website at www.gbt.com in the Investors section.

About Sickle Cell Disease
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 causes hemolytic anemia (the destruction of RBCs) that can lead to multi-organ damage and early death. This sickling process also causes blockage in capillaries and small blood vessels. Beginning in childhood, SCD patients typically 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.

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 improve hemolytic anemia and 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 two therapies for the potential treatment of sickle cell disease, including its late-stage product candidate, voxelotor, as an oral, once-daily therapy. 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 ability to generate additional data from patients enrolled in our ongoing Phase 3 HOPE Study), the potential for an increase in hemoglobin of 1 g/dL or greater to significantly reduce the risk of stroke and mortality in patients with SCD, the sufficiency of our data to support an application for regulatory approval, 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, including the sufficiency of our clinical data and of our primary and other key endpoints in our Phase 3 HOPE Study of voxelotor to support approval, or require additional studies or data to support approval or further clinical investigation of voxelotor, that drug-related adverse events may be observed in clinical development, that data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review or approval, and that we may need to devote additional time and resources to meet these regulatory requirements, 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 June 30, 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
559-974-3245
media@gbt.com

 

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Global Blood Therapeutics, Inc.