Global Blood Therapeutics Announces Completion of Safety Review by Independent Data and Safety Monitoring Board (DSMB) for Ph...
November 13 2017 - 8:00AM
Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) announced
today that the independent Data and Safety Monitoring Board (DSMB)
for the Phase 3 HOPE (
Hemoglobin
Oxygen Affinity Modulation to Inhibit HbS
Polym
Erization) Study evaluating
voxelotor (previously called GBT440) for the treatment of sickle
cell disease (SCD), has completed its first scheduled clinical
safety review. No drug-related serious or severe adverse events
were reported in either of the voxelotor treatment arms (900 mg and
1500 mg), and there were no discontinuations due to adverse events.
Based on this review, the DSMB did not identify safety concerns
with voxelotor at either dose level across all patients dosed in
the ongoing SCD clinical program, including adult patients in the
Phase 3 HOPE Study and adolescent patients in the HOPE-KIDS 1
(GBT440-007) Study. Based on the available safety data to date,
including at the high dose level of 1500 mg, the DSMB recommended
that the pivotal study continue as planned and approved initiation
of enrollment in adolescent patients age 12 to 17.
“This first DSMB review, which opens up enrollment to younger
patients, is an important milestone for the HOPE Study and we are
encouraged that this independent committee has determined that
voxelotor continues to be well tolerated,” said Ted W. Love, M.D.,
president and chief executive officer of GBT. “The opportunity to
include adolescent SCD patients in the HOPE Study is a great step
forward as we believe voxelotor has the potential to be a
disease-modifying therapy that is needed in all age groups who
suffer from this devastating disease. We remain on track to
announce top-line data for our HOPE Study in the first half of
2019, and we look forward to providing an interim update on our
ongoing HOPE-KIDS 1 Study at the upcoming American Society of
Hematology Annual Meeting in December.”
The HOPE Study protocol, agreed to by the U.S. Food and Drug
Administration (FDA), required that an initial group of patients be
treated with 1500 mg of voxelotor to allow assessment by the DSMB
prior to enrolling adolescent patients age 12 to 17 into this
ongoing Phase 3 study.
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 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 an 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 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, voxelotor, as an oral, once-daily therapy for sickle
cell disease. 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 voxelotor
(previously called GBT440), 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, 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, 2016 and in our Quarterly
Report on Form 10-Q for the quarter ended June 30, 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)GBT650-351-4730investor@globalbloodtx.com
Julie Normart (media)Pure
Communications415-946-1087media@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