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SOUTH SAN FRANCISCO, Calif., Oct. 02, 2019 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that four abstracts related to its sickle cell disease (SCD) research have been accepted for oral and poster presentations during the 47th Annual National Sickle Cell Disease Association of America (SCDAA) Convention in Baltimore, taking place October 9-12, 2019. Additionally, GBT will host a lunch symposium at the conference, “Sickle Cell Disease and Brain Health,” at 11:45 a.m. ET on Friday, October 11. The symposium will focus on helping attendees understand how SCD impacts neurocognition and the function of the brain over a patient’s lifetime.
“Since the SCDAA was formed nearly 50 years ago, it has been a driving force in advocating for the SCD community and improving the lives of people living with SCD across the country and around the world,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Over the past five decades, we have seen an incredible amount of positive momentum, especially in the areas of education, research and access to high-quality medical care. Yet, we realize there is still more progress to be made and know that the only way to fully realize our goals is by bringing together the SCD community at events such as the SCDAA 2019 convention, where I am honored to speak during the opening ceremony and join attendees in celebrating the theme of ‘Sickle Cell Community Embracing Change Together.’”
Details of GBT’s oral and poster presentations are as follows:
Thursday, October 10
|Abstract:||Treatment of Moderate to Severe Anemia in Sickle Cell Disease: Results from a 2018 U.S. Patient Chart Analysis|
|Presenter:||Robin Howard, Vice President, Commercial Planning, GBT|
|Time:||6:00-7:00 p.m. ET|
Friday, October 11
|Oral Session:||Public Health|
|Abstract:||Evaluation of Variables Affecting the Care of Patients with Sickle Cell Disease by U.S. Location|
|Presenter:||Julie Kanter, M.D., Associate Professor, University of Alabama at Birmingham|
|Time:||1:45-2:00 p.m. ET|
|Oral Session:||Community Based/Psychosocial Research|
|Abstract:||Beyond Pain: The Symptoms and Impacts of Sickle Cell Disease on Children and Their Caregivers|
|Presenter:||Clark Brown, M.D., Ph.D., Clinical Director, Children's Healthcare of Atlanta|
|Time:||3:00-3:15 p.m. ET|
|Oral Session:||Clinical Research|
|Abstract:||Results from the Randomized Placebo-controlled Phase 3 HOPE Trial of Voxelotor in Adults and Adolescents with Sickle Cell Disease|
|Presenter:||Carolyn Hoppe, M.D., Senior Medical Director, GBT|
|Time:||3:30-3:45 p.m. ET|
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 (low hemoglobin due to RBC destruction) 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, voxelotor blocks polymerization and the resultant sickling and destruction 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 4 to 17) with SCD. The HOPE-KIDS 1 Study is assessing the safety, tolerability, pharmacokinetics and exploratory treatment effect of voxelotor.
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.
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995, including statements about GBT’s development plans for voxelotor and the potential benefits of voxelotor for SCD patients and other statements containing the words “anticipate,” “planned,” “believe,” “forecast,” “estimated,” “expected,” and “intend,” among others. These forward-looking statements are based on GBT’s current expectations and actual results could differ materially. 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 availability of, and sufficiency of our data to support, accelerated regulatory approval, the therapeutic potential and safety profile of voxelotor, including the potential to be a disease-modifying therapy for SCD, the potential for voxelotor to be approved and to become a new standard of care for treating adolescents and adults with SCD, 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 data from patients enrolled in our Phase 3 HOPE Study, and data from our ongoing Phase 2a HOPE-KIDS 1 Study), regulatory review and actions relating to voxelotor, our potential commercial launch of 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, 2018, and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2019, 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.
Stephanie Yao (investors & media)