Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced that four abstracts will be presented during the virtual
edition of the 25th Annual European Hematology Association (EHA)
Congress taking place June 11-14. The abstracts include a
retrospective analysis of data from the landmark STOP 2 study
linking higher hemoglobin levels to lower transcranial Doppler
(TCD) flow velocity, a predictor of stroke risk in children with
sickle cell disease (SCD), and three encore presentations of the
pivotal Phase 3 HOPE Study of Oxbryta® (voxelotor). Oxbryta is the
first drug approved by the U.S. Food and Drug Administration that
directly inhibits sickle hemoglobin polymerization, the process
that causes red blood cells to sickle and break down.
“Children with SCD have a greatly elevated risk of stroke, and
the STOP 2 study provided critical insights that have informed
current stroke prevention strategies. We are pleased to present
data from this landmark study that further support the relationship
between increased hemoglobin levels and decreased risk of stroke,
as assessed by TCD flow velocity,” said Ted W. Love, M.D.,
president and chief executive officer of GBT. “These are important
findings that support the potential for Oxbryta to have a
clinically meaningful effect on stroke risk, which we are further
evaluating in our HOPE-KIDS 2 post-approval confirmatory
trial.”
The EHA abstracts are now available at www.ehaweb.org. Details
of GBT’s poster presentations are as follows:
Friday, June 12
Session: Sickle Cell DiseaseAbstract #EP1533: Correlation
Between Hemoglobin Levels and Transcranial Doppler Velocities: A
Retrospective STOP 2 Analysis in Children with Sickle Cell
DiseasePresenter: Jenifer Voeks, Ph.D., Medical University of South
CarolinaTime: 8:30 a.m. CEST
Session: Sickle Cell Disease Abstract #EP1540: Correlation of
Voxelotor Exposure with Hemoglobin Response and Measures of
Hemolysis in Patients from the HOPE StudyPresenter: Jo Howard, MB
BChir, MRCP, FRCPath, Guy’s and St. Thomas’ NHS Foundation Trust
and King’s College London Time: 8:30 a.m. CEST
Session: Sickle Cell DiseaseAbstract #EP1534: Concomitant
Hydroxyurea and Voxelotor: Results from the HOPE StudyPresenter:
Russell Ware, M.D., Ph.D., Cincinnati Children’s Hospital Medical
CenterTime: 8:30 a.m. CEST
Session: Sickle Cell DiseaseAbstract #EP1526: Incidence of
Vaso-occlusive Crisis Does Not Increase with Achieving Higher
Hemoglobin Levels on Voxelotor Treatment or After Discontinuation:
Analyses of the HOPE StudyPresenter: Elliott Vichinsky, M.D., UCSF
Benioff Children’s Hospital OaklandTime: 8:30 a.m. CEST
About Sickle Cell DiseaseSickle cell disease
(SCD) affects an estimated 100,000 people in the United States and
millions of people throughout the world, particularly among those
whose ancestors are from sub-Saharan Africa. It also affects people
of Hispanic, South Asian, Southern European and Middle Eastern
ancestry.1 SCD is a lifelong inherited blood disorder that impacts
hemoglobin, a protein carried by red blood cells that delivers
oxygen to tissues and organs throughout the body.2 Due to a genetic
mutation, people with SCD form abnormal hemoglobin known as sickle
hemoglobin. Through a process called hemoglobin polymerization, red
blood cells become sickled – deoxygenated, crescent-shaped and
rigid.3-5 The sickling process causes hemolytic anemia (low
hemoglobin due to red blood cell destruction) and blockages in
capillaries and small blood vessels, which impede the flow of blood
and oxygen throughout the body. The diminished oxygen delivery to
tissues and organs can lead to life-threatening complications,
including stroke and irreversible organ damage.5-7
About Oxbryta® (Voxelotor) TabletsOxbryta
(voxelotor) is an oral, once-daily therapy for patients with sickle
cell disease (SCD). Oxbryta works by increasing hemoglobin’s
affinity for oxygen. Since oxygenated sickle hemoglobin does not
polymerize, GBT believes Oxbryta blocks polymerization and the
resultant sickling and destruction of red blood cells, which are
primary pathologies faced by every single person living with SCD.
With the potential to improve hemolytic anemia and oxygen delivery,
GBT believes that Oxbryta has the potential to modify the course of
SCD. On November 25, 2019, Oxbryta received U.S. Food and
Drug Administration (FDA) accelerated approval for the
treatment of SCD in adults and children 12 years of age and
older.7 As a condition of accelerated approval, GBT will
continue to study voxelotor in the HOPE-KIDS 2 Study, a
post-approval confirmatory study using transcranial Doppler (TCD)
flow velocity to assess the ability of Oxbryta to decrease stroke
risk in children 2 to 15 years of age.
In recognition of the critical need for new SCD treatments, the
FDA granted Oxbryta Breakthrough Therapy, 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.
Important Safety InformationOxbryta should not
be taken if the patient has had an allergic reaction to voxelotor
or any of the ingredients in Oxbryta. See the end of the patient
leaflet for a list of the ingredients in Oxbryta. Oxbryta can cause
serious side effects, including serious allergic reactions.
Patients should tell their healthcare provider or get emergency
medical help right away if they get rash, hives, shortness of
breath or swelling of the face.
Patients receiving exchange transfusions should talk to their
healthcare provider about possible difficulties with the
interpretation of certain blood tests when taking Oxbryta.
The most common side effects of Oxbryta include headache,
diarrhea, stomach (abdominal) pain, nausea, tiredness, rash and
fever. These are not all the possible side effects of Oxbryta.
Before taking Oxbryta, patients should tell their healthcare
provider about all medical conditions, including if they have liver
problems; if they are pregnant or plan to become pregnant as it is
not known if Oxbryta can harm an unborn baby; or if they are
breastfeeding or plan to breastfeed as it is not known if Oxbryta
can pass into breastmilk or if it can harm a baby. Patients should
not breastfeed during treatment with Oxbryta and for at least 2
weeks after the last dose.
Patients should tell their healthcare provider about all the
medicines they take, including prescription and over-the-counter
medicines, vitamins and herbal supplements. Some medicines may
affect how Oxbryta works. Oxbryta may also affect how other
medicines work.
Patients are advised to call their doctor for medical advice
about side effects. Side effects can be reported to FDA at
1-800-FDA-1088. Side effects can also be reported to Global
Blood Therapeutics at 1-833-428-4968 (1-833-GBT-4YOU).
Full Prescribing Information for Oxbryta is available
at Oxbryta.com.
About Global Blood TherapeuticsGlobal
Blood Therapeutics (GBT) is a biopharmaceutical company
dedicated to the discovery, development and delivery of
life-changing treatments that provide hope to underserved patient
communities. Founded in 2011, GBT is delivering on its goal to
transform the treatment and care of sickle cell disease (SCD), a
lifelong, devastating inherited blood disorder. The company has
introduced Oxbryta® (voxelotor), the first FDA-approved
treatment that directly inhibits sickle hemoglobin polymerization,
an underlying cause of SCD. GBT is also advancing its pipeline
program in SCD with inclacumab, a p-selectin inhibitor in
development to address pain crises associated with the disease. In
addition, GBT’s drug discovery teams are working on new targets to
develop the next generation of treatments for SCD. To learn more,
please visit www.gbt.com and follow the company on
Twitter @GBT_news.
Forward-Looking StatementsCertain statements in
this press release are forward-looking within the meaning of the
Private Securities Litigation Reform Act of 1995, including
statements containing the words “will,” “anticipates,” “plans,”
“believes,” “forecast,” “estimates,” “expects” and “intends,” or
similar expressions. These forward-looking statements are based on
GBT’s current expectations and actual results could differ
materially. Statements 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. GBT intends these forward-looking statements, including
statements regarding GBT’s priorities, dedication, focus, goals and
vision; the safety, efficacy and mechanism of action of Oxbryta and
other product characteristics; the commercialization, delivery,
availability, use, and commercial and medical potential of Oxbryta;
ongoing and planned studies of Oxbryta and related protocols,
activities and expectations; the significance of abstracts to be
presented; transforming the treatment and care of SCD; the
potential of inclacumab; and advancing GBT’s pipeline, working on
new targets and discovering, developing and delivering treatments,
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 GBT makes this
statement for purposes of complying with those safe harbor
provisions. These forward-looking statements reflect GBT’s current
views about its plans, intentions, expectations, strategies and
prospects, which are based on the information currently available
to the company and on assumptions the company has made. GBT 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 GBT’s control including, without
limitation, risks and uncertainties relating to the COVID-19
pandemic, including the extent and duration of the impact on GBT’s
business, including commercialization activities, regulatory
efforts, research and development, corporate development
activities, and operating results, which will depend on future
developments that are highly uncertain and cannot be accurately
predicted, such as the ultimate duration of the pandemic, travel
restrictions, quarantines, social distancing and business closure
requirements in the U.S. and in other countries, and the
effectiveness of actions taken globally to contain and treat the
disease; the risks that GBT has only recently established its
commercialization capabilities and may not be able to successfully
commercialize Oxbryta; risks associated with GBT’s dependence on
third parties for development, manufacture and commercialization
activities related to Oxbryta; government and third-party payor
actions, including those relating to reimbursement and pricing;
risks and uncertainties relating to competitive products and other
changes that may limit demand for Oxbryta; the risks regulatory
authorities may require additional studies or data to support
continued commercialization of Oxbryta; the risks that drug-related
adverse events may be observed during commercialization or clinical
development; data and results may not meet regulatory requirements
or otherwise be sufficient for further development, regulatory
review or approval; compliance with the funding and other
obligations under the Pharmakon loan; and the timing and progress
of GBT’s and Syros’ research and development activities under their
collaboration; along with those risks set forth in GBT’s Annual
Report on Form 10-K for the fiscal year ended December 31,
2019, and in GBT’s most recent Quarterly Report on Form 10-Q filed
with the U.S. Securities and Exchange Commission, as well as
discussions of potential risks, uncertainties and other important
factors in GBT’s subsequent filings with the U.S. Securities
and Exchange Commission. Except as required by law, GBT assumes no
obligation to update publicly any forward-looking statements,
whether as a result of new information, future events or
otherwise.
References
- Centers for Disease Control and Prevention website.
Sickle Cell Disease
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed June 3, 2019.
- National Heart, Lung, and Blood Institute website.
Sickle Cell
Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease.
Accessed August 5, 2019.
- Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
- Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- Kato GJ, et al. J Clin Invest.
2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir Rev.
2014;15(1):17-23.
- Oxbryta (voxelotor) tablets prescribing
information. South San Francisco, Calif. Global Blood
Therapeutics, Inc.; November 2019.
Contact Information:Steven Immergut
(media)650-410-3258simmergut@gbt.com
Stephanie Yao (investors)650-741-7730syao@gbt.com
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