Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced positive results from real-world and long-term studies
with Oxbryta® (voxelotor) tablets for the treatment of sickle cell
disease (SCD). A first-in-class oral, once-daily therapy, Oxbryta
directly inhibits sickle hemoglobin polymerization, the root cause
of the sickling and destruction of red blood cells in SCD. Results
from a large retrospective analysis of 3,128 SCD patients treated
with Oxbryta showed a statistically significant improvement in
hemoglobin (Hb) levels, and statistically significant reductions in
transfusions, vaso-occlusive crises (VOCs) and hospitalizations.
These data, as well as Phase 1 results for inclacumab, GBT’s
investigational P-selectin inhibitor, were presented at the
63rd American Society of Hematology (ASH) Annual Meeting
& Exposition, taking place from December 11-14, 2021 in
Atlanta, Georgia and online.
“We’re thrilled with the data presented at ASH, which
demonstrate the clinically meaningful impact of Oxbryta for
patients living with sickle cell disease. The benefits of Oxbryta
were reinforced in this large retrospective study to evaluate the
impact of an SCD medicine on clinical outcomes and healthcare
resource utilization in a real-world setting,” said Kim
Smith-Whitley, M.D., executive vice president and head of research
and development of GBT. “We continue our commitment to the sickle
cell disease community in our pursuit of developing innovative
treatments that address the urgent needs of patients.”
Oxbryta Real-World ExperienceA total of 3,128
SCD patients in the United States ages 12 and older were included
in the retrospective analysis (Poster #2052) from the Symphony
Health claims database. This study of medical and pharmacy claims
for patients who initiated Oxbryta treatment between November 2019
and June 2021 compared annualized rates per patient-year (PPY) for
transfusions, VOCs and VOC-related and all-cause hospitalizations
for the three months before Oxbryta initiation versus the period
after beginning the treatment. Among patients with at least one
recent transfusion prior to initiating treatment, there was a 52%
mean reduction in the number of transfusions after beginning
Oxbryta. Patients with VOCs in the pre-study period experienced a
mean reduction of 23% in the number of VOCs after initiating
treatment. After starting Oxbryta, the mean number of VOC-related
hospitalizations decreased by 34%, while the mean number of
all-cause hospitalizations decreased by 37% among patients who were
recently hospitalized. Approximately 61% of patients for whom Hb
lab data were available showed increased Hb levels of greater than
1 g/dL during follow up – consistent with the results from the
Phase 3 HOPE Study.
“These data presented at ASH contribute to the growing body of
evidence on the real-world clinical benefit of Oxbryta, providing
additional support for its use in the treatment of sickle cell
disease and management of associated complications,” said Nirmish
Shah, M.D., associate professor of medicine, pediatrics, Duke
University School of Medicine. “It is encouraging that patients
have an available innovative therapeutic option that has the
potential to modify the course of this devastating disease.”
Additional Oxbryta Studies at ASH 2021 An
analysis of an ongoing open-label extension (OLE) of the Phase 3
HOPE Study (Poster #3114; will be presented on December 13)
confirmed the safety and efficacy of long-term Oxbryta use in SCD
patients ages 12 and older. The improvements in Hb and markers of
hemolysis that were observed in the HOPE study were sustained in
the OLE period for patients who previously received 900 mg and
1,500 mg of Oxbryta, demonstrating a durability of response.
Patients who switched from placebo to Oxbryta saw improvements in
Hb levels and measures of hemolysis from the start of the OLE
through week 48, consistent with the HOPE Study results.
Data from the ongoing Retrospective Study to
Evaluate Outcomes in Patients with Sickle Cell
Disease Treated with Oxbryta
(RETRO) (Poster #3100; will be presented on December 13), 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. Additional
findings are expected to be presented in 2022 to enable a deeper
understanding of the long-term efficacy and safety of Oxbryta.
Safety data across the HOPE OLE and RETRO studies of Oxbryta
were consistent with those from the Phase 3 HOPE Study of SCD
patients ages 12 years and older.
Inclacumab Phase 1 Dosing Analysis in Healthy
Volunteers An analysis (Poster #977) 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, showed a
well-tolerated safety profile for up to 29 weeks following a single
dose of 20 or 40 mg/kg in 15 healthy subjects. Plasma inclacumab
exposures were dose-proportional over the dose range tested and
demonstrated expected pharmacokinetics for healthy subjects, with
apparent nonlinearity below approximately 10 µg/mL, suggesting
target-mediated drug disposition.
Target concentration for both doses at 12 weeks was greater than
the target activity threshold of 10 μg/mL, which was associated in
prior studies of inclacumab with full inhibition of the formation
of platelet-leukocyte aggregate (PLA), a known factor in the
development of vascular lesions and cardiovascular events. The
results support best-in-class potential for inclacumab at the dose
of 30 mg/kg every 12 weeks in patients with SCD-related VOCs, which
is the dose being studied in GBT’s two ongoing Phase 3 THRIVE
(THerapy for Reduction with
Inclacumab of VOC
Episodes) trials (NCT04935879 and
NCT04927247).
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.3 Complications of SCD begin in early childhood
and can include neurocognitive impairment, acute chest syndrome,
and silent and overt stroke, among other serious issues.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.5 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.5-7 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.6-9
About
Oxbryta® (voxelotor)
Tablets 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, 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.10
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 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 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. 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.
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 healthcare 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
healthcare 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 healthcare
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 healthcare 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 InclacumabInclacumab is a novel, fully
human monoclonal antibody that selectively targets P-selectin, a
protein that mediates cell adhesion and is clinically validated to
reduce pain crises,11 known as vaso-occlusive crises or VOCs, in
people with sickle cell disease (SCD). Preclinical results suggest
that inclacumab has the potential to be a best-in-class option for
reducing VOCs in people with SCD, with the potential for quarterly,
rather than monthly dosing. GBT has exclusive worldwide rights to
inclacumab as part of the company’s licensing agreement with F.
Hoffmann-La Roche Ltd. The safety, tolerability and
pharmacokinetics of inclacumab have been evaluated by Roche in more
than 700 non-SCD patients.
About Global Blood TherapeuticsGlobal
Blood Therapeutics, Inc. (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 medicine 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, 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, commitment,
focus, goals, mission and vision; safety, efficacy and mechanism of
action of Oxbryta and other product characteristics; 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; potential future
findings from RETRO, including timing and significance; ongoing and
planned studies, clinical trials and registries, and related
protocols, activities, timing and other 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 regulatory review, timing and approval; altering the
treatment, course and care of SCD and mitigating related
complications; safety, efficacy, mechanism of action, advancement
and potential of GBT’s drug candidates and 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 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
Research. https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-understanding-sickle-cell-disease.html.
Accessed December 1, 2021.
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed June 12, 2020.
- Centers for Disease Control and Prevention website. Sickle
Cell Disease
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed June 3, 2019.
- Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87.
- 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.
- Ataga K. et al. N Engl J
Med. 2017;376(5):429-439.
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 Mar 2024 to Apr 2024
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Apr 2023 to Apr 2024