Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
new data from the Phase 2a HOPE-KIDS 1 Study that showed children
with sickle cell disease (SCD) ages 4 to 11 years treated with
Oxbryta® (voxelotor) tablets experienced significant improvements
in hemoglobin levels. These data, along with results from two
real-world evidence studies of Oxbryta, will be presented at the
European Hematology Association (EHA) 2021 Virtual Congress, taking
place online June 9-17, 2021.
“The encouraging results seen with Oxbryta treatment in younger
patients living with sickle cell disease support our belief that
Oxbryta can have a positive impact across a wide age range of
people living with this devastating, lifelong disease by reducing
the sickling and destruction of red blood cells, thereby improving
anemia and hemolysis – hallmarks of the condition,” said Ted W.
Love, M.D., president and chief executive officer of GBT.
“Additionally, we are pleased by the growing body of real-world
evidence demonstrating the meaningful benefits of Oxbryta in
patients ages 12 and older. Collectively, these data reinforce our
commitment to seek broad access for patients and add to our
confidence in the potential of this innovative medicine to address
the urgent needs of sickle cell disease patients in the U.S.,
Europe, the Middle East and beyond.”
New Data Analysis from Phase 2a HOPE-KIDS 1 Study (Oral
Abstract #S260)A new analysis of data from 45 children
with SCD ages 4 to 11 years enrolled in the open-label Phase 2a
HOPE-KIDS 1 Study (GBT440-007) showed that treatment with Oxbryta
(1,500 mg or weight-based equivalent dispersed in a
pediatric-appropriate formulation) resulted in rapid and sustained
improvements in hemoglobin. An increase in hemoglobin of greater
than 1 g/dL from baseline was observed in 47% of patients as early
as two weeks and sustained through 24 weeks, consistent with
results in patients ages 12 years and older in the Phase 3 HOPE
Study. Concurrent improvements in markers of hemolysis were also
observed.
“The potentially life-threatening complications of sickle cell
disease are the cumulative result of damage caused by the sickling
and destruction of red blood cells that begins at a very young age.
While the goal is to treat sickle cell disease early in life to
prevent potential long-term consequences, current therapeutic
options for younger patients are limited and do not adequately
address the underlying cause of this devastating disease,” said
Clark Brown, M.D., Ph.D., director of sickle cell clinical research
at the Aflac Cancer and Blood Disorders Center of Children’s
Healthcare of Atlanta, a primary investigator of the study. “With
consistent results to published data in adults, the data from the
HOPE-KIDS 1 Study underscore the potential for Oxbryta to provide
important benefits in children as young as 4 years old, with a
favorable safety profile.”
The findings support the equivalency of weight-based dosing in
children ages 4 to 11 years and the 1,500 mg dose of Oxbryta in
adolescents ages 12 to 17 years. The mean hemoglobin occupancy was
26% in children ages 4 to 11, which is consistent with results in
the Phase 3 HOPE Study. In the HOPE-KIDS 1 study, Oxbryta was well
tolerated and no new adverse safety signals were detected. The most
commonly reported treatment-related adverse events were transient
and self-limiting (diarrhea (11%), vomiting (11%) and rash
(11%)).
Real-World Experience with Oxbryta Findings
from two new analyses of real-world experience with Oxbryta
demonstrated improvements in hemoglobin consistent with results
observed in the Phase 3 HOPE Study, which were previously published
in The New England Journal of Medicine.
- An analysis of real-world outcomes in 77 patients with SCD
treated in a single-center case series (Abstract #EP1209) showed
that hemoglobin levels increased by an average of 2 g/dL after
treatment with Oxbryta along with corresponding improvements in
markers of hemolysis. A hemoglobin response greater than 1 g/dL
with Oxbryta was observed in 62% of patients without the use of
hydroxyurea and 87% of patients treated with hydroxyurea and
Oxbryta. The robust hematologic response was associated with
improved clinical status, as measured by the Clinical Global
Impression of Change (CGI-C) and Patient Global Impression of
Change (PGI-C) scales, validated outcomes measures that provide
holistic assessments of the effect of treatment by the physicians
and patients, respectively. Few adverse events were recorded, and
all were resolved with dose modification. The safety profile
observed in this analysis was consistent with the approved label
for Oxbryta.
- A separate study (Abstract #EP1206) provides a study design
overview of RETRO, GBT’s multicenter retrospective data collection
and analysis registry, which will collect real-world outcomes in up
to 300 adults and adolescents with SCD treated with Oxbryta at 10
sites across the United States. The study will incorporate medical
records for each patient one year before and up to one year after
starting Oxbryta and will capture clinical outcome measures, health
resource utilization data, and laboratory measures. An initial
analysis of outcomes in 20 patients enrolled in the RETRO study at
a single study site was reported. After 12 months of treatment with
Oxbryta, 50% of patients had increased hemoglobin greater than 1
g/dL.
Patient registration and data collection in the RETRO Study is
ongoing. GBT also recently initiated the prospective PROSPECT
Study, which is designed to enroll up to 750 patients at
approximately 25 U.S. sites. GBT intends to collect data from these
registries to enable deeper understanding of the long-term efficacy
and safety of Oxbryta.
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 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 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.
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 also plans to seek 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 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 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; GBT’s commitment to seek broad
patient access to Oxbryta; significance of data to be presented at
the EHA Congress, including support for the use of Oxbryta and
manner of dosing in children; ongoing and planned studies and
related protocols, activities and expectations; regulatory
submissions, review and approval to potentially expand the approved
use of Oxbryta for more patients in the U.S. and to treat
patients in Europe and other territories; altering the
treatment, course and care of SCD and mitigating related
complications; potential and advancement of GBT’s pipeline,
including inclacumab and other product candidates; 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 development, manufacture, distribution and commercialization
activities related to Oxbryta; government and third-party payer
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 obligations under the Pharmakon
loan; and the timing and progress of activities under GBT’s
collaborations and license 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)650.410.3258simmergut@gbt.com
Courtney
Roberts (investors)650.351.7881croberts@gbt.com
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