Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
that five U.S. nonprofit organizations serving the sickle cell
disease (SCD) community have been awarded a total of $250,000
through the company’s 2020 Access to Excellent Care for Sickle Cell
Patients (ACCEL) Grant Program. For the second year, the ACCEL
program has funded innovative programs that have the potential to
deliver high-quality healthcare to people living with SCD.
“Individuals living with sickle cell disease face significant
challenges every day, including accessing quality healthcare in
their communities. These challenges are greater than ever given the
impact of COVID-19 and the healthcare disparities the pandemic has
brought harshly to light,” said Jung E. Choi, chief business and
strategy officer, and head of patient advocacy and government
affairs at GBT. “We selected this year’s grant recipients based on
their potential to make an outsized difference on improving access
to care for patients. With the additional funding from the ACCEL
program, these organizations have the opportunity to speed the
development and amplify the reach of their programs.”
The 2020 ACCEL grant recipients are:
- Atrium Health Foundation for “Expanding Access
to Care for Pediatric Sickle Cell Patients by Educating and
Empowering Their Primary Care Providers (PCPs)”
- Augusta University Sickle Cell Transition
Program for “Transition to Adult Sickle Cell Disease Care
in Rural South Georgia”
- East Carolina University Comprehensive Sickle Cell
Program for “Improving Transition Education and
Facilitating Access to Care for Underserved Youth with SCD”
- OSF Saint Francis Medical Center for
“Enhancing Rural Sickle Cell Outreach Clinics and Easing Transition
to Adult Care”
- University of Rochester for “Improving
Transition from Pediatric Specialty Care to Adult Health Care for
Individuals with Sickle Cell Disease”
“The passion and innovation in the ACCEL grant proposals that we
received this year gives us hope that critical gaps in sickle cell
care can be narrowed and, one day, closed,” said Payal Desai, M.D.,
a member of the review panel and director of sickle cell research
at The Ohio State University. “We are thankful to have received so
many compelling proposals and look forward to the continued
innovation in years to come. Together, we can make a tremendous
difference for the sickle cell community.”
GBT launched the ACCEL program in February 2019 to fund
U.S.-based nonprofit organizations that serve patients with SCD and
their families and seek to improve their access to high-quality
healthcare. Proposals received this year were reviewed by a panel
of external and GBT stakeholders familiar with the issues affecting
people with SCD. The panel chose grant recipients based on the
strength of their submission, level of innovation, and greatest
potential impact to patient care.
For more information about GBT’s grants program and other
corporate giving,
visit https://www.gbt.com/our-commitment/corporate-giving/.
About Sickle Cell Disease Sickle cell disease
(SCD) affects an estimated 100,000 people in the United States,1 an
estimated 52,000 people in Europe,2 and millions of people
throughout the world, particularly among those whose ancestors are
from sub-Saharan Africa.1 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.3 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.4-7
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,
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, 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 and
potential impact of the ACCEL program and the grant recipients;
transforming the treatment and care of SCD and making a difference
for the SCD community; 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.
- 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.
Contact Information:Steven Immergut
(media)650-410-3258simmergut@gbt.com
Stephanie Yao (investors)650-741-7730syao@gbt.com
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