Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT), today
announced that nine abstracts related to its sickle cell disease
(SCD) programs will be presented at the 62nd American Society of
Hematology (ASH) Annual Meeting & Exposition, taking place
online Dec. 5-8, 2020. The studies to be presented include the
72-week analysis of the Phase 3 HOPE Study of Oxbryta® (voxelotor)
tablets and new research from GBT’s pipeline.
“Nearly one year after the FDA approval of Oxbryta, we are
pleased to share new clinical data supporting both the long-term
use of this first-in-class therapy to reduce anemia and hemolysis
in people with sickle cell disease and its effectiveness in the
real-world setting,” said Ted W. Love, M.D., president and chief
executive officer of GBT. “As part of our strategy to be the leader
in sickle cell disease and our deep commitment to this community,
we are also excited to unveil new preclinical data from our SCD
pipeline programs, including our novel fully human monoclonal
antibody that has the potential to be a best-in-class P-selectin
inhibitor, and our next generation hemoglobin S polymerization
inhibitor.”
Highlights of the data to be presented at ASH include:
Oxbryta
- An analysis of the Phase 3 HOPE Study showed that Oxbryta
(1,500 mg) treatment demonstrated sustained improvements in
hemoglobin (Hb) levels and markers of hemolysis over 72 weeks,
consistent with previously published 24-week analyses. These
results support durability of Oxbryta longer-term use to reduce
anemia and hemolysis and potentially mitigate the associated
morbidity and mortality of SCD.
- Consistent with the 24-week results analysis, patients in the
Phase 3 HOPE Study who achieved the greatest average Hb levels over
72 weeks with Oxbryta experienced numerically fewer vaso-occlusive
crises (VOCs), with a stepwise reduction in VOC rate with each
increase in Hb stratum (up to 12 to ≤ 13.3 g/dL).
- In the Phase 3 HOPE Study, the Clinical Global Impression of
Change (CGI-C) outcome measure showed that treatment with Oxbryta
compared to placebo resulted in a significantly higher rating of
improved overall patient health status after 72 weeks of treatment,
regardless of baseline Hb and hemolysis markers. CGI provides an
overall clinician-determined summary measure that takes into
account available information, including knowledge of the patient’s
history, psychosocial circumstances, symptoms and behavior, and the
impact of the symptoms on the patient’s ability to function.
- An analysis of Symphony Health claims data from a subset of
1,275 patients treated with Oxbryta showed that the increase in Hb
levels observed in a real-world setting was consistent with
published results from the Phase 3 HOPE Study. The data further
demonstrated favorable trends in decreasing transfusion and
annualized rates of VOCs after the initiation of Oxbryta
therapy.
- An analysis of data from Clinical and Patient Global Impression
of Improvement scales (CGI-I and PGI-I) collected from 27 patients
treated with Oxbryta at The University of Texas Comprehensive
Sickle Cell Center demonstrated that the majority of patients
reported substantial improvement in overall clinical status after
treatment with Oxbryta, which correlated with the clinician
impression of their status.
Pipeline
- An in vitro study of inclacumab, a novel P-selectin inhibitor
in development to reduce VOCs in SCD, demonstrated the potential
for a substantially longer duration of exposure and more convenient
dosing compared to crizanlizumab.
- A preclinical analysis demonstrated that GBT021601, GBT’s next
generation HbS polymerization inhibitor, tested at lower doses than
Oxbryta, was highly effective in reducing hemolysis, increasing
hemoglobin levels, prolonging red blood cell (RBC) half-life and
improving RBC health as well as potentially improving organ
function in SCD transgenic mice.
The ASH abstracts are now available at www.hematology.org.
Details of the GBT presentations are as follows:
Saturday, Dec.
5, available virtually
from 7 a.m. to 3:30 p.m.
P.T.
Poster Session: 114. Hemoglobinopathies, Excluding
Thalassemia—Clinical: Poster IAbstract #795: Higher Hemoglobin
Levels Achieved with Voxelotor Are Associated with Lower
Vaso-occlusive Crisis Incidence: 72-Week Analysis from the HOPE
Study Presenter: Elliott Vichinsky, M.D., UCSF Benioff Children’s
Hospital Oakland
Poster Session: 114. Hemoglobinopathies, Excluding
Thalassemia—Clinical: Poster IAbstract #802: Improvement in the
Clinical Global Impression of Change with Voxelotor in Patients
with Sickle Cell Disease in the Phase 3 HOPE Trial Presenter: Wally
Smith, M.D., Virginia Commonwealth University
Sunday, Dec.
6, available virtually
from 7 a.m. to 3:30 p.m.
P.T. Poster
Session: 114. Hemoglobinopathies, Excluding Thalassemia—Clinical:
Poster IIAbstract #1716: Efficacy and Safety of Voxelotor in
Adolescents and Adults with Sickle Cell Disease: HOPE Trial 72-Week
Analysis Presenter: Jo Howard, MB BChir, MRCP, FRCPath, Guy’s and
St. Thomas’ NHS Foundation Trust and King’s College London
Poster Session: 114. Hemoglobinopathies, Excluding
Thalassemia—Clinical: Poster IIAbstract #1723: Patient Perception
of Oxbryta Treatment Benefit Presenter: Modupe Idowu, M.D.,
McGovern Medical School at The University of Texas Health Science
Center at Houston
Poster Session: 113. Hemoglobinopathies, Excluding
Thalassemia—Basic and Translational Science: Poster II Abstract
#1704: GBT021601 Inhibits HbS Polymerization, Prevents RBC Sickling
and Improves the Pathophysiology of Sickle Cell Disease in a Murine
Model Presenter: Kobina Dufu, Ph.D., GBT
Poster Session: 113. Hemoglobinopathies, Excluding
Thalassemia—Basic and Translational Science: Poster II Abstract
#1707: Inclacumab, a Fully Human Anti-P-selectin Antibody, Directly
Binds to PSGL-1 Binding Region and Demonstrates Robust and Durable
Inhibition of Cell Adhesion Presenter: Xin Geng, Ph.D., GBT
Poster Session: 113. Hemoglobinopathies, Excluding
Thalassemia—Basic and Translational Science: Poster II Abstract
#1706: Rheological Impact of GBT1118 Cessation in a Sickle Mouse
Model Presenters: Danitza Nebor, Ph.D., Baylor College of Medicine
Vivian Sheehan, M.D., Ph.D., Baylor College of Medicine
Poster Session: 114. Hemoglobinopathies, Excluding
Thalassemia—Clinical: Poster II Abstract #1724: The Impact of
Hemoglobin Level on Risk of End-Organ Damage Among Patients with
Sickle Cell Disease – A Large-Scale, Longitudinal
AnalysisPresenter: William Ershler, M.D., Inova Schar Cancer
Institute
Monday, Dec.
7, available virtually
from 7
a.m.
to 3
p.m.
P.T.
Poster Session: 113. Hemoglobinopathies, Excluding
Thalassemia—Basic and Translational Science: Poster III Abstract
#2627: Real-World Effectiveness of Voxelotor for Treating Sickle
Cell Disease in the U.S.Presenter: Ahmar Zaidi, M.D., Children’s
Hospital of Michigan, Central Michigan University, College of
Medicine
GBT Analyst & Investor Day Webcast
DetailsGBT will host a virtual Analyst & Investor Day
event on Monday, Dec. 7, at 4 p.m. P.T. to review data being
presented at the 2020 ASH Annual Meeting. The event will also
provide an overview of the company’s SCD development pipeline,
including inclacumab and GBT021601. The webcast can also be
accessed directly at
http://www.gbtinvestorday.virtualeventsite.com/. Participants are
requested to register in advance. A replay will be available
for three months following the event on GBT’s investor webpage at
www.gbt.com.
About Sickle Cell DiseaseSickle 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
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. 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
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 Oxbryta in its Priority Medicines (PRIME) program, and
the European Commission (E.C.) 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, and GBT021601, 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 Statements
Certain 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, strategy, 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; impacting the treatment, care, course and outcome of SCD;
future presentations and the significance of related results; the
potential of GBT’s pipeline, including inclacumab and other product
candidates; 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
Dec. 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
Immergut 650-410-3258simmergut@gbt.com
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