Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
that it has entered into an exclusive agreement with
Biopharma-Middle East and Africa (Biopharma-MEA) to distribute
Oxbryta® (voxelotor) tablets in Bahrain, Kuwait, Oman, Qatar, Saudi
Arabia, and the United Arab Emirates, collectively known as the
Gulf Cooperation Council (GCC) region. There are estimated to be
more than 100,000 people age 12 years and older in this region
living with sickle cell disease (SCD).1
Oxbryta is a once-daily, oral therapy that directly
inhibits hemoglobin polymerization, the root cause of the sickling
and destruction of red blood cells in SCD. The sickling process
causes hemolytic anemia (low hemoglobin due to red blood cell
destruction), which impairs adequate oxygen delivery to the tissues
and organs in the body.
“GBT is driven by our mission to help the millions
of people around the world affected by sickle cell disease,” said
Ted W. Love, M.D., president and chief executive officer of GBT.
“With this agreement, we are taking an important step in expanding
access to Oxbryta, a first-in-class treatment that directly targets
the underlying cause of this devastating disease, by making it
available to physicians and thousands of patients in need in these
countries. We’re pleased to partner with Biopharma-MEA, which has
an impressive track record of success in reaching patients in the
Middle East suffering from serious and rare conditions.”
In the GCC region, the U.S. approval of Oxbryta can
be referenced to allow for access to the medicine while health
authorities conduct their reviews. Oxbryta received U.S. Food and
Drug Administration accelerated approval in November 2019 for the
treatment of SCD in adults and children 12 years of age and
older.2
Earlier this year, GBT announced plans to seek
regulatory approvals to expand the potential use of Oxbryta in the
United States for the treatment of SCD in children age 4 to 11
years and to treat hemolytic anemia in SCD in people age 12 years
and older in Europe.
About Sickle Cell Disease Sickle
cell disease (SCD) affects an estimated 100,000 people in the
United States,3 more than 100,000 people age 12 years and
older in the Gulf Cooperation Council region,1 and millions of
people throughout the world, particularly among those whose
ancestors are from sub-Saharan Africa.3 It also affects people
of Hispanic, South Asian, Southern European, and Middle Eastern
ancestry.3 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.4 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.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 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.2 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
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) 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. 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,
commitment, dedication, focus, goals, mission, and vision; the
agreement with Biopharma-MEA, including related activities and
expectations; referencing FDA approval to allow access to Oxbryta
in the GCC region and review of Oxbryta by health authorities; 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 potential expansion of the
approved use of Oxbryta for more patients in the U.S. and potential
approval of Oxbryta to treat patients in Europe; impacting the
treatment, care, and course 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; the
progress of activities under the distribution agreement with
Biopharma-MEA, and the 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
- Data on file.
- Oxbryta (voxelotor) tablets prescribing information. South San
Francisco, Calif. Global Blood Therapeutics, Inc.; November
2019.
- 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.
Contact Information:Steven
Immergut (media)GBT650-410-3258simmergut@gbt.com
Stephanie Yao
(investors)GBT650-741-7730syao@gbt.com
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