Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced that six abstracts related to its sickle cell disease
(SCD) programs, including data from the growing body of real-world
evidence on Oxbryta® (voxelotor) tablets and new data on GBT’s
pipeline, will be presented at the 63rd American Society of
Hematology (ASH) Annual Meeting & Exposition. The meeting
is taking place online and in person at the Georgia World Congress
Center in Atlanta, Georgia from December 11-14, 2021.
“Our data presentations at ASH 2021 include three new analyses
from the growing body of data on Oxbryta that reinforce our belief
in the benefits of this innovative and potentially
disease-modifying treatment for sickle cell disease,” said Kim
Smith-Whitley, M.D., executive vice president and head of research
and development at GBT. “Additionally, we’re very excited to
present new Phase 1 data from our pipeline relating to GBT601 and
inclacumab – both of which we believe have the potential to be
best-in-class therapies – as we work on behalf of patients to
achieve our goal of transforming sickle cell disease into a
well-managed chronic condition.”
The three Oxbryta analyses provide greater insight into its
efficacy and safety in both the real-world and long-term clinical
trial settings:
- An analysis of nearly 2,700 patients ages 12 years or older
from the Symphony Health claims database who initiated Oxbryta
treatment between November 2019 and March 2021 demonstrated that,
in real-world practice, Oxbryta increased hemoglobin (Hb),
consistent with the results of the Phase 3 HOPE Study. These data
show statistically significant reductions in transfusions,
vaso-occlusive crises (VOCs), and all-cause and VOC-related
hospitalizations after Oxbryta use.
- An evaluation of data from an open-label extension of the Phase
3 HOPE Study demonstrated that long-term use of Oxbryta is safe,
well tolerated, and has a durable effect in reducing anemia and
hemolysis in patients with SCD.
- Data from the Retrospective Study to Evaluate Outcomes in
Patients with Sickle Cell Disease Treated with Oxbryta (RETRO), the
first multicenter, retrospective study to examine the real-world
effectiveness of Oxbryta, showed the treatment was associated with
increased Hb levels and decreased hemolytic markers. The safety
data are consistent with those from the Phase 3 HOPE Study of SCD
patients ages 12 years and older.
Data presented from two key Phase 1 studies from GBT’s R&D
pipeline in SCD include:
- An analysis of a Phase 1 study of GBT021601 (GBT601), GBT’s
next-generation sickle hemoglobin polymerization inhibitor, will
highlight data in both healthy volunteers and a cohort of six
adults with SCD. Single ascending doses in healthy volunteers and
single doses of GBT601 in patients with SCD were well tolerated in
healthy volunteers and patients with SCD. In addition,
multiple-dose data will be presented and will help to evaluate
GBT601’s potential as a best-in-class, oral, disease-modifying
therapy. GBT believes GBT601 has the capacity to achieve a targeted
Hb occupancy and attain the desired hematological effect at low
doses, therefore reducing pill burden and improving clinical
outcomes for individuals living with SCD.
- An analysis of a Phase 1 study of inclacumab, GBT’s fully
human P-selectin monoclonal antibody in development for the
reduction of VOCs in SCD patients, displayed a well-tolerated
safety profile for up to 29 weeks following a single dose of 20 or
40 mg/kg in healthy subjects. Durable inhibition of
platelet-leukocyte aggregate (PLA) formation was observed through
at least 12 weeks, consistent with prior observations. The results
support a dose of 30 mg/kg every 12 weeks in patients with
SCD-related VOCs being studied in GBT’s two Phase 3 THRIVE
(THerapy for Reduction
with Inclacumab
of VOC Episodes) trials
(NCT04935879 and NCT04927247), which the company initiated in June
2021.
An additional abstract features data from a natural history
cohort of pediatric patients with SCD:
- An analysis of a natural history cohort of pediatric SCD
patients showed an inverse relationship between Hb level and
transcranial Doppler (TCD) velocity in patients with SCD,
supporting the beneficial effect of higher Hb levels in SCD
patients. TCD is a validated screening tool to identify pediatric
SCD patients at risk of cerebrovascular
events.
All of these abstracts are now available
at www.hematology.org. Details of the GBT presentations,
which will be available in the poster hall and via the virtual
event platform, are as follows:
Saturday, Dec. 11, 5:30 p.m. to 7:30
p.m. ETPoster Session: Preliminary Results of a Phase
1 Study in Healthy Subjects Administered Inclacumab, a Fully Human
IgG4 Anti-P-Selectin Monoclonal Antibody in Development for
Treatment of Sickle Cell DiseaseAbstract
#977Presenter: Christina Mayer, PharmD, Semivida Research,
Dallas, TX, (formerly with GBT)
Sunday, Dec. 12, 6:00 p.m. to 8:00
p.m. ETPoster Session: Real-World Experience of
Voxelotor for the Management of Complications in Sickle Cell
DiseaseAbstract #2052Presenter: Nirmish Shah, M.D., Duke
University School of Medicine, Durham, NC
Monday, Dec. 13, 6:00 p.m. to 8:00
p.m. ETPoster Session: Real-World Experience of
Patients with Sickle Cell Disease Treated with Voxelotor: A
Multicenter, Retrospective StudyAbstract #3100Presenter: Biree
Andemariam, M.D., New England Sickle Cell Institute, University of
Connecticut Health, Farmington, CT
Poster Session: Long-Term Safety and Efficacy of Voxelotor for
Patients with Sickle Cell Disease: Results from an Open-Label
Extension of the Phase 3 HOPE TrialAbstract
#3114Presenter: Maureen M. Achebe, M.D., MPH, Brigham and
Women’s Hospital, Harvard Medical School, Boston, MA
Poster Session: The Role of Hemoglobin and Hemolysis on
Transcranial Doppler Velocities in Children with Sickle Cell
Disease: Data from a Natural History CohortAbstract
#3092Presenter: Raffaella Colombatti, M.D., Ph.D., Clinic of
Pediatric Hematology Oncology, Department of Women’s and Child’s
Health, Azienda Ospedale – Università di Padova, Padua, Italy
Poster Session: GBT021601, a Next Generation HbS Polymerization
Inhibitor: Results of Safety, Tolerability, Pharmacokinetics and
Pharmacodynamics in Adults Living with Sickle Cell Disease and
Healthy VolunteersAbstract #3099Presenter: 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
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 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, Oxbryta inhibits sickle
hemoglobin polymerization and the resultant sickling and
destruction of red blood cells, 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. 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.8
As a condition of accelerated approval, GBT is studying 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 was 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).
The EMA has accepted for review GBT’s Marketing Authorization
Application (MAA) seeking full marketing authorization of Oxbryta
in Europe to treat hemolytic anemia in SCD patients ages
12 years and older. GBT is also seeking regulatory approval to
expand the potential use of Oxbryta in the United States for the
treatment of SCD in children as young as 4 years old.
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) 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 Phase 3 development to address pain
crises associated with the disease, and GBT021601 (GBT601), 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 potential treatments for SCD. To learn
more, 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, commitment,
focus, goals, mission and vision; safety, efficacy and mechanism of
action of Oxbryta and other product characteristics; potential of
Oxbryta, including in being a disease-modifying treatment;
significance of reducing sickling and hemolysis and raising
hemoglobin; commercialization, delivery, availability, use and
commercial and medical potential of Oxbryta; the content, timing
and significance of data and abstracts to be presented at ASH;
ongoing and planned studies, clinical trials and registries, and
related protocols, activities and expectations; regulatory
submissions to potentially expand the approved use of Oxbryta for
more patients and in a pediatric formulation in the U.S. and
to treat patients in Europe and other territories, including
potential review, timing and approval; altering the treatment,
course and care of SCD and mitigating related complications;
safety, efficacy, mechanism of action, potential and advancement 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, 2020, 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 (media)+1.650.410.3258simmergut@gbt.com
Courtney
Roberts (investors)+1.650.351.7881croberts@gbt.com
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Sep 2024 to Oct 2024
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Oct 2023 to Oct 2024