Global Blood Therapeutics Announces New Data Supporting Ongoing Clinical Development of GBT440 in Adolescents with Sickle Ce...
June 23 2017 - 2:30AM
Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced
the presentation of preliminary results from the single-dose
adolescent (age 12 to 17) cohort of the ongoing HOPE-KIDS 1 Study,
a Phase 2a open-label study of GBT440 in adolescents with sickle
cell disease (SCD). The results, which demonstrated that the
pharmacokinetics of GBT440 are similar in adolescents and adults,
were presented at the 22nd Congress of the European Hematology
Association (EHA) in Madrid.
“Because sickle cell disease is a lifelong inherited condition
that begins manifestation in childhood, it is critical that
adolescent and pediatric patients are included in ongoing research
efforts. We believe GBT440 could be particularly impactful in these
young patients before much of the irreversible damage associated
with SCD occurs,” said Ted W. Love, M.D., president and chief
executive officer of GBT. “Based on these encouraging
pharmacokinetic and safety results, we are expanding our HOPE-KIDS
1 trial to evaluate GBT440 administered at doses of 900 mg and
1,500 mg per day, consistent with the doses currently being
administered in the Phase 3 HOPE Study. These data will also be
used to support enrolling adolescent patients in the HOPE Study and
to inform future GBT440 dose selections for evaluation in a younger
pediatric population of children age 6 to 11.”
Abstract #P620: The Pharmacokinetics (PK) of GBT440 Are
Similar in Adolescents and Adults with Sickle Cell Disease
(SCD) HOPE-KIDS 1 (GBT440-007) is an open-label, single-
and multiple-dose study that is evaluating the safety,
tolerability, pharmacokinetics and exploratory treatment effect of
GBT440 in adolescents age 12 to 17 years with SCD. Results
presented at EHA showed that, in adolescents who received a single
oral dose of 600 mg of GBT440:
- The pharmacokinetics and half-life of GBT440 were similar in
adolescents and adults, with results supporting once-daily dosing
and a high specificity for hemoglobin.
- GBT440 was well tolerated, with no serious or severe adverse
events related to study drug observed.
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 GBT440 in Sickle Cell Disease GBT440 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 both Fast Track and
Orphan Drug designations for the treatment of patients with SCD,
and the European Commission (EC) has designated GBT440 as
an orphan medicinal product for the treatment of patients with SCD.
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 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 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
including in particular patient populations, 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
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Mar 2024 to Apr 2024
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Apr 2023 to Apr 2024