Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
that six abstracts related to its sickle cell disease (SCD)
programs will be presented at the European Hematology Association
(EHA) 2021 Virtual Congress, taking place online June 9-17, 2021.
Results from an analysis of the Phase 2a HOPE-KIDS 1 Study
(GBT440-007) in children with SCD ages 4 to 11 years treated with
Oxbryta® (voxelotor) tablets will be presented for the first time
in an oral session.
The HOPE-KIDS 1 analysis, which evaluated 45
children treated with Oxbryta (1,500 mg or weight-based
equivalent), found that 47 percent achieved a hemoglobin increase
of >1 g/dL at 24 weeks. Reductions in markers of hemolysis were
also observed. Oxbryta was well tolerated, and no new adverse
safety signals were detected. The most commonly reported
treatment-related adverse events were diarrhea (11 percent),
vomiting (11 percent) and rash (11 percent).
These findings from the HOPE-KIDS 1 Study are
consistent with data from the Phase 3 HOPE Study of patients with
SCD ages 12 years and older, which was published in The New England
Journal of Medicine and presented as part of the Presidential
Symposium at the EHA Congress in June 2019. The complete analysis
of 72-week data from the HOPE Study was recently published in The
Lancet Haematology, showing durable improvement in hemoglobin
levels and reduction in hemolysis for patients receiving
Oxbryta.
“New treatment options for younger children with
sickle cell disease are urgently needed. We believe that reducing
the sickling and destruction of red blood cells, thereby improving
the anemia and hemolysis that characterize this devastating
inherited condition early in life, could modify the course of the
disease and alleviate serious and life-threatening complications,”
said Ted W. Love, M.D., president and chief executive officer of
GBT. “We are encouraged by the extensive body of new data being
presented at this year’s EHA Congress, supporting the use of
Oxbryta in both real-world and clinical trial settings. We look
forward to potentially expanding access to Oxbryta over time across
a broad range of sickle cell disease patients in the U.S., Europe,
the Gulf Cooperation Council region and other areas.”
The European Medicines Agency (EMA) has accepted
for review GBT’s Marketing Authorization Application (MAA) seeking
full marketing authorization of Oxbryta in Europe to treat
hemolytic anemia in SCD patients ages 12 years and older. GBT also
plans to seek regulatory approval to expand the potential use of
Oxbryta in the United States for the treatment of SCD in children
as young as 4 years old.
The EHA abstracts are available at
https://ehaweb.org. All oral and poster presentations will be
available on the EHA website on Friday, June 11, at 9:00 a.m.
CEST/3:00 a.m. EDT. Other abstracts to be presented include
real-world evidence supporting the use of Oxbryta in patients ages
12 and older. Full details are as follows:
Oral Session: Changing the scene on sickle
cell diseaseAbstract #S260: Safety and Efficacy of
Voxelotor in Pediatric Patients with Sickle Cell Disease Aged 4-11
YearsPresenter: Clark Brown, M.D., Ph.D., Children’s Healthcare of
Atlanta Live Q&A: Tuesday, June 15, 16:00-16:45
CEST/10:00-10:45 a.m. EDT
Poster Session: Sickle cell
diseaseAbstract #EP1209: Real-World Experience of
Voxelotor for the Treatment of Patients with Sickle Cell Disease –
A Single-Center StudyPresenter: Alan R. Anderson, M.D., University
of South Carolina School of Medicine Greenville
Abstract #EP1206: Real-World Experience of Patients
with Sickle Cell Disease Treated with Voxelotor: A Multicenter,
Retrospective StudyPresenter: Biree Andemariam, M.D., University of
Connecticut Health
Abstract #EP1198: GBT021601 Improves the
Pathophysiology of Sickle Cell Disease in a Murine ModelPresenter:
Kobina Dufu, Ph.D., GBT
Abstract #EP1201: Silent Cerebral Infarcts in
Pediatric Sickle Cell Disease Natural History Cohort: Too Many, Too
Soon?Presenter: Raffaella Colombatti, M.D., Ph.D., University of
Padova, Italy
Publication OnlyAbstract #PB1769:
Pediatric Patient Reported Outcomes in Patients Receiving Voxelotor
for Sickle Cell DiseaseFirst Author: Clark Brown, M.D., Ph.D.,
Director of Sickle Cell Clinical Research, Children’s Healthcare of
Atlanta
About Sickle Cell DiseaseSickle
cell disease (SCD) affects an estimated 100,000 people
in the United States,2 an estimated 52,000 people
in Europe,3 and millions of people throughout the world,
particularly among those whose ancestors are from sub-Saharan
Africa.2 It also affects people of Hispanic, South Asian,
Southern European and Middle Eastern ancestry.2 SCD is a
lifelong inherited rare blood disorder that impacts hemoglobin, a
protein carried by red blood cells that delivers oxygen to tissues
and organs throughout the body.4 Due to a genetic mutation,
individuals 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.4-6 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.5-8
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, Oxbryta inhibits sickle
hemoglobin polymerization and the resultant sickling and
destruction of red blood cells, which are primary pathologies faced
by every single person living with SCD. Through addressing
hemolytic anemia and improving oxygen delivery throughout the body,
GBT believes that Oxbryta has the potential to modify the course of
SCD. On Nov. 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.8
As a condition of accelerated approval, GBT will
continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval
confirmatory study using transcranial Doppler (TCD) flow velocity
to assess the ability of the therapy 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. Additionally, Oxbryta has been
granted Priority Medicines (PRIME) designation from the European
Medicines Agency (EMA), and the European Commission (EC) has
designated Oxbryta 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
health care 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 health care 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
health care 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 two weeks after the last dose.
Patients should tell their health care 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
the 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) tablets, the
first FDA-approved treatment that directly inhibits sickle
hemoglobin polymerization, the root cause of red blood cell
sickling in 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, and GBT021601 (GBT601),
the company’s next- generation hemoglobin S polymerization
inhibitor. In addition, GBT’s drug discovery teams are working on
new targets to develop the next wave 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, commitment, focus, goals, mission and vision; safety,
efficacy and mechanism of action of Oxbryta and other product
characteristics; significance of reducing sickling and hemolysis
and raising hemoglobin; commercialization, delivery, availability,
use and commercial and medical potential of Oxbryta; significance
of data to be presented at the EHA Congress, including support for
the use of Oxbryta; ongoing and planned studies and related
protocols, activities and expectations; regulatory submissions,
review and approval to potentially expand the approved use of
Oxbryta for more patients in the U.S. and to treat patients
in Europe and other territories; altering the treatment,
course and care of SCD and mitigating related complications;
potential and advancement of GBT’s pipeline, including inclacumab
and other product candidates; and 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 is continuing to establish its commercialization
capabilities and may not be able to successfully commercialize
Oxbryta; risks associated with GBT’s dependence on third parties
for development, manufacture, distribution and commercialization
activities related to Oxbryta; government and third-party payer
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 obligations under the Pharmakon
loan; and the timing and progress of activities under GBT’s
collaborations and license agreements; along with those risks set
forth in GBT’s Annual Report on Form 10-K for the fiscal year
ended December 31, 2020, 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.
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed June 12, 2020.
- 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:Steven
Immergut (media)650-410-3258simmergut@gbt.com
Courtney
Roberts (investors)650-351-7881croberts@gbt.com
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