Global Blood Therapeutics Announces Upcoming Presentation of GBT440 Data in Adolescents with Sickle Cell Disease at European ...
May 18 2017 - 4:00PM
Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced
that new data supporting its GBT440 program in sickle cell disease
(SCD) will be presented at the upcoming 22nd Congress of the
European Hematology Association (EHA), which will be held June
22-25, 2017 in Madrid. The poster presentation will highlight
preliminary results from the single dose arm of the ongoing
HOPE-KIDS 1 Study, a Phase 2a open-label study of GBT440 in
adolescents ages 12 to 17.
The EHA abstracts are now available at www.ehaweb.org. The
poster presentation will include additional data not available in
the abstract. Presentation details are as follows:
Poster Session: Enzymes and sickle cell diseaseAbstract #P620:
The Pharmacokinetics (PK) of GBT440 are Similar in Adolescents and
Adults with Sickle Cell Disease (SCD)Date: Saturday, June 24,
2017Time: 5:30-7:00 p.m. CEST / 11:30 a.m.-1:00 p.m. ET Location:
Poster area (Hall 7)
About GBT440 in Sickle Cell DiseaseGBT440 is
being developed as an oral, once-daily therapy for patients with
SCD. GBT440 works by increasing hemoglobin's affinity for oxygen.
Since oxygenated sickle hemoglobin does not polymerize, GBT
believes GBT440 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 GBT440 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 GBT440 for the
treatment of patients with SCD both fast track and orphan drug
designations, and the European Commission (EC) has designated
GBT440 for the treatment of patients with SCD as an orphan
medicinal product. GBT is currently evaluating GBT440 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, GBT440 is being studied in the
ongoing Phase 1/2 GBT440-001 trial and in the ongoing HOPE-KIDS 1
Study, an open-label, single and multiple dose study in adolescents
(age 12 to 17) with SCD designed to assess the safety,
tolerability, pharmacokinetics and exploratory treatment effect of
GBT440.
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 (or HbS). In its
deoxygenated state, HbS has a propensity to polymerize, or bind
together, forming long, rigid rods within a red blood cell (or
RBC). The polymer rods deform RBCs to assume a sickled shape and to
become inflexible, which can cause blockage in capillaries 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 Global Blood TherapeuticsGlobal 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 lead product
candidate, GBT440, as an oral, once-daily therapy for sickle cell
disease. GBT is also investigating GBT440 for the treatment of
hypoxemic pulmonary disorders in two ongoing Phase 2a studies in
patients with idiopathic pulmonary fibrosis. 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 GBT440,
our ability to implement our enrollment and other clinical
development plans for GBT440 in both SCD and hypoxemic pulmonary
disorders, our ability to generate and report data from our ongoing
studies of GBT440, 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 regulatory authorities may disagree
with our clinical development plans or require additional studies
or data to support further clinical investigation of our product
candidates, and that drug-related adverse events may be observed in
later stages of clinical development, 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 March 31, 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
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