Global Blood Therapeutics Announces Enrollment of First Patient in Phase 3 HOPE Study in Sickle Cell Disease
19 January 2017 - 12:30AM
Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today
announced enrollment of the first patient in the HOPE
(
Hemoglobin
Oxygen Affinity
Modulation to Inhibit HbS
Polym
Erization) Study, a pivotal
Phase 3 clinical trial of GBT440 in people with sickle cell disease
(SCD). The HOPE Study will be conducted at leading SCD sites
globally and is expected to enroll up to 400 adults and adolescents
with SCD who have had at least one episode of vaso-occlusive crisis
(VOC) in the previous year.
"Enrolling the first patient in our pivotal Phase 3 HOPE Study
is an important milestone for GBT as we continue to work to bring
GBT440 to the SCD community,” said Ted W. Love, M.D., president and
chief executive officer of GBT. “The innovative HOPE Study trial
design has strong grounding in the mechanism of action of GBT440
and is designed to assess the efficacy of GBT440 in producing
meaningful clinical improvements in anemia and daily symptoms.
GBT440 has the potential to fundamentally modify the course of this
devastating disease by inhibiting sickle hemoglobin polymerization,
the fundamental cause of SCD pathophysiology.”
HOPE Study DesignThe randomized, double-blind,
placebo-controlled, multi-national trial will enroll patients age
12 and older with SCD who have had at least one episode of VOC in
the previous year. The study will be conducted in two parts: Part A
will compare GBT440 administered at doses of 900 or 1,500 mg per
day vs. placebo in up to 150 patients treated for at least 12
weeks, and Part B will include 250 patients randomized to placebo
or a dose of GBT440 selected from Part A. The main objectives of
Part A are to select the optimal dose, define the final secondary
endpoints for Part B, and qualify the patient-reported outcome
(PRO) instrument. The primary efficacy endpoint of the HOPE Study
is the proportion of patients who achieve a greater than 1 g/dL
increase in hemoglobin at 24 weeks of treatment compared with
baseline. Key secondary efficacy endpoints include the effect of
GBT440 on SCD symptom exacerbation (as measured by the HOPE PRO
instrument), overall SCD symptoms, traditionally defined VOCs,
hospitalizations and red blood cell transfusions.
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
dramatically 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 and the European Commission
(EC) has designated GBT440 as an orphan medicinal product, in each
case for the treatment of patients with SCD. In addition to the
Phase 3 HOPE Study and the ongoing Phase 1/2 GBT440-001 trial,
GBT440 is being evaluated in an open-label, single and multiple
dose study in adolescents (age 12 to 17) with SCD, 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, leading to 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 (RBC). The polymer
rods deform RBCs to assume a sickled shape and to become
inflexible, which can cause blockage in 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.
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,
the timing of, and our ability to generate data from our ongoing
clinical trials of GBT440 for sickle cell disease, including our
ability to enroll patients in, conduct and complete our HOPE Study,
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, 2015, and in
our Quarterly Reports on Form 10-Q for the quarters ended March 31,
2016, June 30, 2016 and September 30, 2016, 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)
BrewLife
415-946-1087
media@globalbloodtx.com
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