Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT), today
announced its participation in the 15th Annual Scientific
Conference on Sickle Cell and Thalassemia (ASCAT) and 1st EHA
European Sickle Cell Conference, taking place online on Oct. 26-31,
2020. Two abstracts have been accepted for presentation, including
real-world effectiveness data of Oxbryta® (voxelotor) tablets in
the treatment of sickle cell disease (SCD).
“GBT is focused on addressing sickle cell disease
at its root cause to seek to modify the course of the disease, and,
ultimately, mitigate the serious and life-threatening complications
that often lead to long-term damage and early death,” said Ted W.
Love, M.D., president and CEO of GBT. “We’re pleased to be at ASCAT
2020, where we are sharing real-world effectiveness data
demonstrating that the benefits of treatment of sickle cell
patients with Oxbryta were consistent with the results of our Phase
3 HOPE Study.”
The two abstracts presented at the conference provide greater
insight into the safety and efficacy of Oxbryta:
- A retrospective chart review study to assess the real-world
effectiveness of voxelotor based on data during the first several
months post-U.S. FDA approval. In a sample of charts from 56
patients with SCD, voxelotor increased hemoglobin by more than 1
g/dL on average and decreased hemolysis markers to a degree
consistent with the randomized controlled HOPE trial results.
Evidence also suggests that voxelotor treatment was associated with
improvement in important symptoms of SCD, such as pain and fatigue,
and other aspects of quality of life.
- The case report of a patient with
SCD treated with voxelotor after presenting with a significant drop
in hemoglobin, who was not responsive to transfusion with red blood
cells in association with hospitalization for COVID-19. In this
case, the patient's hemoglobin and overall clinical status improved
rapidly with voxelotor treatment, thereby avoiding exchange
transfusion, sparing red blood cell units and decreasing exposure
of health care providers to COVID-19, all of which are important
considerations during this era of pandemic and limited blood
supply.
Details of the ASCAT presentations are as follows:
Wednesday,
Oct. 28Abstract
Session: New TherapiesReal-World Effectiveness of Voxelotor for the
Treatment of Sickle Cell Disease: A Chart Review StudyPresenter:
Kenneth Bridges, M.D., GBTTime: 11 a.m. GMT
Virtual Presentation
in Poster RoomSickle Cell Anemia
and COVID-19: Use of Voxelotor to Avoid TransfusionPresenter:
William B. Ershler, M.D., Inova Schar Cancer Institute
As part of its presence at ASCAT, GBT will host an educational
online symposium, “Getting to the Root Cause: Understanding the
Devastating Impact of SCD on Patients,” on Monday, Oct. 26, 7-8
p.m. GMT. The symposium will include presentations by Caterina
Minniti, M.D., professor of clinical medicine and pediatrics at
Einstein College of Medicine and director of the Sickle Cell Center
for Adults at Montefiore Medical Center; and Bart Biemond, M.D.,
Ph.D., professor of internal medicine and hematologist at the
Faculty of Medicine of the University of Amsterdam. More
information about the symposium, which is available to registered
attendees of the ASCAT meeting, can be found in the conference
program.
Coinciding with ASCAT, today GBT released a comprehensive
report, “Overview of the Sickle Cell Disease Environment in Select
European Countries,” in order to help raise awareness of SCD
in Europe. The report is based on research conducted in five
European countries and explores the burden of the disease on
patients, families and health care systems. In addition, the report
examines existing public policy efforts to address and alleviate
the challenges faced by those living with SCD. SCD is one of the
most prevalent genetic rare diseases in Europe, and despite a
clinical understanding of SCD, estimated life expectancy for those
affected by the disease is significantly lower than the European
average, indicating an urgent need to improve the quality of care
for these patients.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects an estimated 100,000 people in the United States,1 an
estimated 52,000 people in Europe2 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 rare 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,
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.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
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 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.
GBT 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.
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), 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 potential expansion of the
approved use of Oxbryta for more patients in the U.S., and
potential regulatory approval for Oxbryta to treat patients in
Europe; raising awareness of SCD in Europe; altering the treatment,
course and care of SCD and mitigating related complications; need
to improve the quality of care for SCD patients in Europe; 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.
- Oxbryta
(voxelotor) tablets prescribing information. South San
Francisco, Calif. Global Blood Therapeutics,
Inc.; November 2019.
Contact: Steven
ImmergutGBT650-410-3258media@gbt.com
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