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Press Release

Global Blood Therapeutics Announces Upcoming Data Presentations Supporting Sickle Cell Disease Program at 59th American Society of Hematology (ASH) Annual Meeting


Posted on: 01 Nov 17

Results from HOPE-Kids 1 Study Expanded Dose Cohort Selected for Inclusion in ASH Press Program

Company to Host Investor Webcast on Monday, December 11

SOUTH SAN FRANCISCO, Calif., Nov. 01, 2017 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced that new data supporting the clinical development of voxelotor (previously called GBT440) for the treatment of sickle cell disease (SCD) will be presented during the 59th American Society of Hematology (ASH) Annual Meeting & Exposition, taking place December 9-12 at the Georgia World Congress Center in Atlanta. A total of seven abstracts have been accepted for presentation. One of the two oral presentations – updated data from the ongoing Phase 2a Study (GBT440-007) Evaluating Adolescents with Sickle Cell Disease Treated with Multiple Doses of GBT440, a HbS Polymerization Inhibitor – has been selected to be featured in an ASH press briefing.

“Our data presentations at this year’s ASH meeting reinforce why we remain confident about voxelotor as a potential disease-modifying treatment for children and adults living with SCD and provide insight on challenges faced by the community related to opioid use, access to care and quality of care,” said Ted W. Love, M.D., president and chief executive officer of GBT. “We look forward to sharing a more extensive data set from the expanded dose cohort of our ongoing HOPE-KIDS 1 Study in adolescents including the first longitudinal experience with the patient-reported outcome tool that we are utilizing in our pivotal HOPE Study. Updated findings from seven adult SCD patients with severe anemia and multiple co-morbidities who received voxelotor through compassionate access will also be presented.”

The ASH abstracts are now available at www.hematology.org. The oral and poster presentations and ASH press briefing will include additional data not available in the abstracts. Details are as follows:

Saturday, December 9

ASH Press Briefing: Engineered Approaches
Date: Saturday, December 9
Time: 10:00-11:00 a.m. ET

Oral Session: 901. Health Services Research—Non-Malignant Conditions: Sickle Cell Disease
Abstract #130: Opioid Utilization Patterns in United States Patients with Sickle Cell Disease
Presenter: Samir K. Ballas, MD, FACP, Thomas Jefferson University, Philadelphia, PA
Date: Saturday, December 9
Time: 10:15 a.m. ET
Location: Building B, Level 2, B216-B217

Poster Session: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical: Poster I
Abstract #980: The Pharmacokinetics (PK) of GBT440 Following Single Doses in Pediatric Patients with Sickle Cell Disease (SCD)
Presenter: Carla B Washington, PhD, Global Blood Therapeutics, South San Francisco, CA
Date: Saturday, December 9
Time: 5:30-7:30 p.m. ET
Location: Building A, Level 1, Hall A2

Poster Session: 901. Health Services Research—Non-Malignant Conditions: Poster I 
Abstract #2098: Quality of Care in United States Children with Sickle Cell Anemia
Presenter: Julie Kanter, MD, Lifespan Comprehensive Sickle Cell Center, Medical University Of South Carolina
Date: Saturday, December 9
Time: 5:30-7:30 p.m. ET
Location: Building A, Level 1, Hall A2

Monday, December 11

Oral Session: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical II
Abstract #689: Initial Results from a Cohort in a Phase 2a Study (GBT440-007) Evaluating Adolescents with Sickle Cell Disease Treated with Multiple Doses of GBT440, a HbS Polymerization Inhibitor
Presenter: Carolyn Hoppe, M.D., Associate Hematologist, UCSF Benioff Children’s Hospital, Oakland, Calif.
Date: Monday, December 11
Time: 3:45-4:00 p.m. ET
Location: Building B, Level 3, B308-B309

Poster Session: 113. Hemoglobinopathies, Excluding Thalassemia—Basic and Translational Science: Poster III
Abstract #3516: GBT1118 Diminishes Vaso-Occlusion in Sickle Cell Disease Mice
Presenter: Carsten Alt, PhD, Global Blood Therapeutics, South San Francisco, CA
Date: Monday, December 11
Time: 6:00-8:00 p.m. ET
Location: Building A, Level 1, Hall A2

Poster Session: 114. Hemoglobinopathies, Excluding Thalassemia – Clinical: Poster III
Abstract #3545: A Single Center Experience of GBT440 Treatment of Severe Anemia in Sickle Cell Disease (SCD)
Presenter: Lanetta Bronté, M.D., M.P.H., M.S.P.H., founder of the Foundation for Sickle Cell Disease Research (FSCDR)
Date: Monday, December 11
Time: 6:00-8:00 p.m. ET
Location: Building A, Level 1, Hall A2

Poster Session: 901. Health Services Research—Non-Malignant Conditions: Poster I 
Abstract #4660: Access to Care for Medicaid and Commercially-Insured United States Patients with Sickle Cell Disease
Presenter: Carlton Dampier, MD, Emory University, Atlanta, GA
Date: Monday, December 11
Time: 6:00-8:00 p.m. ET
Location: Building A, Level 1, Hall A2

The following voxelotor data will be published online only in the December 8 supplemental volume of Blood:

Abstract #4779: Impact of GBT440 on Unconjugated Bilirubin and Jaundice in Sickle Cell Disease: A Patient Case Report
Authors: Paul Telfer, M.D., Josh Lehrer, M.D., MPhil, FACC, Irene Agodoa, M.D., Marzena Jurek, MS, Kathleen M Fox, Ph.D., Laurie Burke, BPharm, M.P.H., Noel Landsman, Dip.Nur (SA) and Timothy Mant, MB, FFPM, FRCP

Investor Webcast Details
GBT will host an investor event webcast on Monday, December 11, 2017 at 8:15 p.m. ET/5:15 p.m. PT to review the data being presented at the ASH Annual Meeting. The investor event will feature members of GBT’s management team and SCD experts. The 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)
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 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 trial in patients age 12 and older with SCD. Additionally, voxelotor is being studied in the ongoing Phase 1/2 GBT440-001 trial and 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 Global Blood Therapeutics
Global 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 late-stage product candidate, voxelotor, as an oral, once-daily therapy for sickle cell disease. To learn more, please visit: www.globalbloodtx.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 (previously called GBT440), our ability to implement our clinical development plans for voxelotor, our ability to generate and report data from our ongoing and potential future studies of voxelotor, regulatory review and actions relating to voxelotor, our ability to adequately obtain and protect our intellectual property rights, 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, 2016 and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2017, 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@globalbloodtx.com

Julie Normart (media)
Pure Communications
415-946-1087
media@globalbloodtx.com

GlobeNewswire
globenewswire.com

Last updated on: 01/11/2017

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