GBT Announces Upcoming Data Presentations Supporting Voxelotor Sickle Cell Disease (SCD) Program at Foundation for Sickle Cel...
June 08 2018 - 8:30AM
Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced
that four abstracts related to the voxelotor program in sickle cell
disease will be presented during the Foundation for Sickle Cell
Disease Research’s (FSCDR) 12th Annual SCD Research and Educational
Symposium in Washington, D.C., taking place June 15-17, 2018. Four
encore abstracts have been accepted, including three for oral
presentation. Additionally, Kenneth R. Bridges, M.D., vice
president, medical affairs at GBT, will be discussing voxelotor as
part of the Investigational New Drugs, Therapeutic and Device
Symposium on Friday, June 15, from 4:30-6:30 p.m. Eastern Time
(ET).
The abstracts are now available for symposium attendees through
the FSCDR 2018 app. The app is available for download for both
iPhones and Androids. Details regarding the abstracts are as
follows:
Sunday, June 17
Oral Presentation: Breakout SessionAbstract: Opioid Utilization
Patterns in U.S. Individuals with Sickle Cell DiseaseTime:
1:45-3:15 p.m. ET
Oral Presentation: Breakout SessionAbstract: Access to Care for
Medicaid and Commercially Insured U.S. Individuals with Sickle Cell
DiseaseTime: 1:45-3:15 p.m. ET
Oral Presentation: Breakout SessionAbstract: Compassionate-Use
Voxelotor (GBT440) for Patients with Severe Sickle Cell Disease
(SCD) and Life-Threatening ComorbiditiesTime: 3:30-5:00 p.m. ET
Poster SessionAbstract: Novel Trial Design to Evaluate Oral
Voxelotor for the Treatment of Sickle Cell Disease (SCD): the Phase
3 Hemoglobin Oxygen Affinity Modulation to Inhibit Sickle
Hemoglobin Polymerization (HOPE) Trial (GBT440-031)Time: 5:15-6:30
p.m. ET
About Sickle Cell DiseaseSCD 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 GBTGBT 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 StatementsStatements 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)GBT650-351-4730investor@gbt.com
Julie Normart (media)W2O pure415-946-1087media@gbt.com
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