Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced five abstracts related to its sickle cell disease (SCD)
programs will be presented at the European Hematology Association
(EHA) 2022 Hybrid Congress. Presentations include new real-world
evidence data on Oxbryta® (voxelotor) as well as an oral
presentation of additional data from the Phase 1 study of GBT021601
(GBT601), the company’s next generation sickle hemoglobin (HbS)
polymerization inhibitor. In addition, initial findings from the
Sickle Cell Health Awareness, Perspectives and Experiences (SHAPE)
survey, a global burden of disease study assessing healthcare
professional (HCP), patient and caregiver perspectives on the unmet
needs in SCD, will be presented at the Congress, which will be held
from June 9-12 in Vienna, Austria and online.
“Our data presentations at EHA2022 support the sustained use of
Oxbryta for the treatment of hemolytic anemia in sickle cell
disease, and the promise of our pipeline to improve clinical
outcomes for people living with this devastating condition.
Real-world evidence continues to provide valuable insights into
what happens every day in clinical practice and supports important
decisions regarding care,” said Kim Smith-Whitley, M.D., executive
vice president and head of research and development at GBT. “Data
presented at this year’s Congress will contribute to the growing
body of evidence that could transform the treatment of sickle cell
disease.”
Following are highlights of GBT’s presentations at the
Congress.
Data from a large retrospective study of 216 patients in the
United States treated with Oxbryta in a real-world setting further
supports its utility in treating patients with SCD:
- New data from the multicenter
Retrospective Study to Evaluate
Outcomes in Patients with Sickle Cell Disease
Treated with Oxbryta (RETRO)
provide further support that treatment with Oxbryta is associated
with increased hemoglobin (Hb) levels and decreased hemolytic
markers. These data are consistent with those from the Phase 3 HOPE
Study that led to the approval of Oxbryta.
Data from the Phase 1 studies of GBT601, which has shown promise
in the clinic, supports its further development:
- Multiple daily doses of GBT601 were
well tolerated in both healthy volunteers and adult SCD patients.
In SCD patients, the 100 mg maintenance dose studied resulted in a
mean Hb occupancy of more than 30%, increased Hb levels, reduced
markers of hemolysis, and improved red blood cell (RBC) health
(including improved deformability and less sickling). Additional
data will be included with the oral presentation.
Data presented from two abstracts add greater insight into the
disease burden and effect of Hb levels on people with SCD:
- The multinational
Sickle Cell Health
Awareness, Perspectives and
Experiences Survey (SHAPE) assessed HCP
perspectives on treating SCD and the patient burden of the disease.
Results of the SHAPE survey underscore the negative impact of SCD
on quality of life and highlight health inequities in SCD and the
need for improved awareness, education and treatments that fully
address end-organ damage in SCD.
- A retrospective analysis of 12 years
of data from the Clinical Practice Research Datalink and the
Hospital Episode Statistics databases in the UK was sufficient to
observe end-organ damage events in patients with SCD. It showed
that an increase in Hb of 1 g/dL was associated with a significant
reduction in the risk for stroke, pulmonary hypertension, chronic
kidney disease, end-stage renal disease, leg ulcers, and pneumonia,
supporting the use of therapeutics that increase Hb levels in
patients with SCD to protect against organ damage.
A presentation on GBT’s pivotal Phase 3 studies of inclacumab,
the company’s P-selectin inhibitor:
- Currently in progress and enrolling
patients, the two pivotal Phase 3 THRIVE studies are evaluating the
safety and efficacy of inclacumab in reducing vaso-occlusive crises
(VOCs) and readmissions due to VOCs. An additional THRIVE
open-label expansion (OLE) study will examine the long-term safety
of inclacumab in individuals with SCD.
All abstracts are now available at www.ehaweb.org. Details of
the GBT presentations, which will be available in the poster hall
and via the virtual event platform, are as follows:
Oral Presentation: Sickle Cell DiseaseAbstract
# S268: Safety, Tolerability, and Pharmacokinetic/Pharmacodynamic
Results from Phase 1 Studies of GBT021601, a Next-Generation HbS
Polymerization Inhibitor for Treatment of Sickle Cell
DiseasePresenter: Clark Brown, M.D., Ph.D., Aflac Cancer and Blood
Disorders Center of Children’s Healthcare of Atlanta and Department
of Pediatrics, Emory School of Medicine, Atlanta, GA, USASunday,
June 12, 2022, 11:30 -12:45 CEST
Poster Session: Sickle Cell DiseaseAbstract #
P1487: Sickle Cell Health Awareness, Perspectives and Experiences
(SHAPE) Survey: Findings on the Burden of Sickle Cell Disease on
Patients and Their Unmet Needs as Reported by Healthcare
ProfessionalsPresenter: Baba Inusa, M.D., Department of Paediatric
Haematology, Guy’s and St Thomas’ Hospital, London, UKFriday, June
10, 2022, 16:30 - 17:45 CEST
Abstract # P1486: Trials in Progress - The THRIVE Studies
Evaluating the Efficacy, Safety, and Long-Term Treatment with
Inclacumab, a P-Selectin Inhibitor, in Patients with Sickle Cell
DiseasePresenter: Biree Andemariam, M.D., New England Sickle Cell
Institute, University of Connecticut Health, Farmington, CT,
USAFriday, June 10, 2022, 16:30 - 17:45 CEST
Abstract # P1485: A Multicenter, Retrospective Study on
Real-World Experience of Patients with Sickle Cell Disease Treated
with VoxelotorPresenter: Biree Andemariam, M.D., New England Sickle
Cell Institute, University of Connecticut Health, Farmington, CT,
USAFriday, June 10, 2022, 16:30 - 17:45 CEST
Abstract # P1488: Association Between Hemoglobin Levels and
End-Organ Damage in Sickle Cell Disease: A Retrospective Linked
Primary and Secondary Care Database Analysis in EnglandPresenter:
Paul Telfer, D.M., F.R.C.P., Queen Mary, University of London,
UKFriday, June 10, 2022, 16:30 - 17:45 CEST
About Sickle Cell DiseaseSickle cell disease
(SCD) affects more than 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.3 It also affects people of Hispanic, South
Asian, Southern European and Middle Eastern ancestry.4 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.4,5,6 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 delivery throughout the body. The diminished oxygen
delivery to tissues and organs can lead to life-threatening
complications, including stroke and irreversible organ
damage.5,6,7,8 Complications of SCD begin in early childhood and
can include neurocognitive impairment, acute chest syndrome, and
silent and overt stroke, among other serious issues.9
About
Oxbryta® (voxelotor)
Oxbryta (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 leading to hemolysis and hemolytic anemia, 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.In November 2019, the U.S. Food
and Drug Administration (FDA) granted accelerated approval for
Oxbryta tablets for the treatment of SCD in adults and children 12
years of age and older, and in December 2021, the FDA
expanded the approved use of Oxbryta for the treatment of SCD in
patients 4 years of age and older in the United
States.10 As a condition of accelerated approval for patients
ages 4 and older in the United States, 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 14 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 received the prestigious 2021 Prix Galien
USA award for “Best Biotechnology Product” from The
Galien Foundation.
Oxbryta has been granted Priority Medicines (PRIME) designation
from the European Medicines Agency (EMA), Oxbryta was designated by
the European Commission (EC) as an orphan medicinal product for the
treatment of patients with SCD, and Oxbryta was granted Promising
Innovative Medicine (PIM) designation in the United Kingdom from
the Medicines and Healthcare products Regulatory Agency (MHRA). In
February 2022, the European Commission (EC) granted Marketing
Authorization for Oxbryta for the treatment of hemolytic anemia due
to SCD in adult and pediatric patients 12 years of age and older as
monotherapy or in combination with hydroxycarbamide (hydroxyurea).
In addition, the Ministry of Health and Prevention (MOHAP) in the
United Arab Emirates (UAE) has granted marketing authorization for
Oxbryta for the treatment of SCD in adults and children 12 years of
age and older.Please click here for Important Safety
Information and full Prescribing Information including
Patient Information for Oxbryta in the U.S.
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, starting with sickle cell disease (SCD). Founded in
2011, GBT is delivering on its goal to transform the treatment and
care of SCD, a lifelong, devastating inherited blood disorder. The
company has introduced Oxbryta® (voxelotor), the first
FDA-approved medicine that directly inhibits sickle hemoglobin
(HbS) 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 Phase 3 development to
address pain crises associated with the disease, and GBT021601
(GBT601), the company’s next generation HbS polymerization
inhibitor. 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, vision, and positioning; the safety,
efficacy, and mechanism of action of Oxbryta, and other product
characteristics; the commercialization, awareness, delivery,
availability, use, and commercial and medical potential of Oxbryta,
including the use, significance and potential of related
initiatives; presentation of data at EHA and their significance,
including with respect to the use of Oxbryta, the potential of
GBT’s pipeline, the further development of GBT601, and the use of
therapeutics that increase Hb levels; the significance and use of
real-world evidence; the impact of this year’s Congress, including
in contributing to evidence to transform the treatment of SCD;
ongoing and planned studies, clinical trials and registries, and
related protocols, activities, timing, and other expectations;
impacting the treatment, care, and course of SCD and mitigating
related complications; safety, efficacy, mechanism of action,
advancement and potential of GBT’s drug candidates and pipeline;
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 research, development, manufacture, distribution,
and commercialization activities; government and third-party payer
actions, including those relating to reimbursement and pricing;
risks and uncertainties relating to competitive treatments 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 collaboration, license and distribution
agreements; along with those risks set forth in GBT’s Annual Report
on Form 10-K for the fiscal year ended December 31, 2021, 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
Research. https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-understanding-sickle-cell-disease.html.
Accessed February 23, 2022.
- European Medicines
Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125
Accessed February 23, 2022.
- Centers for Disease Control and Prevention website.
Sickle Cell Disease
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed February 23, 2022.
- National Heart, Lung, and Blood Institute website.
Sickle Cell
Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease.
Accessed February 23, 2022.
- Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
- Kato GJ, et al. J Clin Invest.
2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir Rev.
2014;15(1):17-23.
- Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87.
- Oxbryta (voxelotor) tablets and tablets for oral suspension
prescribing information. South San Francisco,
Calif. Global Blood Therapeutics, Inc.; December
2021.
- Ataga K. et al. N Engl J
Med. 2017;376(5):429-439.
Contact Information:Steven Immergut (media)+1
650-410-3258simmergut@gbt.com
Courtney Roberts (investors)+1
650-351-7881croberts@gbt.com
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