Global Blood Therapeutics Expands Ongoing Phase 2a HOPE-KIDS 1 Study into Younger Pediatric Population
July 10 2017 - 8:00AM
Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced
that it has expanded the ongoing HOPE-KIDS 1 Study (GBT440-007), a
Phase 2a open-label study of GBT440 in pediatric patients with
sickle cell disease (SCD), to include a new single-dose cohort in
children age 6 to 11.
“Following positive pharmacokinetic and safety results from the
single-dose cohort of adolescent patients age 12 to 17, which were
recently presented at the Congress of the European Hematology
Association, we have dosed the first patient in a new cohort of
younger children, age 6 to 11,” said Ted W. Love, M.D., president
and chief executive officer of GBT. “We believe a new treatment
option for SCD is desperately needed for patients of all ages and
have designed our development program to generate data across age
groups. We anticipate data from this new single-dose cohort in
younger pediatric participants later this year, which will help
inform future GBT440 dose selections for continued evaluation in
children.”
About the HOPE-KIDS 1 StudyThe open-label,
single- and multiple-dose HOPE-KIDS 1 Study (GBT440-007) is
evaluating the safety, tolerability, pharmacokinetics and
exploratory treatment effect of GBT440 in a pediatric population
(age 6 to 17) with SCD. The study is being conducted in two
parts:
- The single-dose Part A portion includes two cohorts of patients
who will receive a single oral dose of 600 mg of GBT440. Cohort 1
is complete, with seven patients age 12 to 17 enrolled. Cohort 2,
which is the recently initiated portion of the study, will enroll
patients age 6 to 11.
- Part B will explore the safety of multiple doses of GBT440
administered to patients age 12 to 17. The doses being evaluated,
900 mg per day and 1500 mg per day, are consistent with those
currently being administered in GBT’s ongoing Phase 3 HOPE Study in
patients age 12 and older.
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,
the European Medicines Agency (EMA) has included GBT440 in its
Priority Medicines (PRIME) program, 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 pediatric patients
(age 6 to 17) with SCD. HOPE-KIDS 1 is assessing 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,
our ability to implement our 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,
regulatory review and actions relating to GBT440, our ability to
generate and report data from the new single-dose cohort in younger
pediatric participants 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:
Joey Fleury (investors and media)
Pure Communications
415-946-1090
investor@globalboodtx.com
media@globalbloodtx.com
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