Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
The Lancet Haematology has published the complete analysis of
72-week data from the Phase 3 HOPE Study of Oxbryta® (voxelotor)
tablets in patients with sickle cell disease (SCD). The results
showed significant and sustained improvement in hemoglobin levels,
reduction in hemolysis and improved overall health status in
patients treated with Oxbryta. These findings support the long-term
use of Oxbryta to reduce hemolytic anemia and hemolysis in SCD,
potentially mitigating life-threatening complications of the
condition.
Oxbryta, a first-in-class oral, once-daily therapy, directly
inhibits hemoglobin polymerization, the root cause of the sickling
and destruction of red blood cells in SCD. Oxbryta is approved in
the United States for the treatment of SCD in patients ages 12
years and older.
“Sickle cell disease is a devastating disease that can lead to
organ damage and a shortened life expectancy and is complicated by
significant disparities in access to quality care,” said lead
author Professor Jo Howard of Guy’s and St. Thomas’ NHS Foundation
Trust and King’s College London. “Fortunately, we have entered a
new era of treatment. The HOPE Study is the longest registrational
trial to date among recently approved therapies for sickle cell
disease, and these results further demonstrate that by sustainably
improving both the hemolysis and anemia manifestations of the
disease, Oxbryta has the potential to be a safe and effective
disease-modifying treatment in patients with sickle cell
disease.”
The HOPE Study, a randomized, double-blind, placebo-controlled,
international, multicenter trial in 274 patients ages 12 to 65
years with SCD, showed treatment with the U.S. FDA-approved dose of
Oxbryta 1500 mg resulted in rapid and durable improvements in
hemoglobin levels throughout 72 weeks of treatment. Approximately
90 percent of patients treated with Oxbryta achieved a hemoglobin
improvement of >1 g/dL from baseline at one or more time points
during the study compared to placebo (25 percent). In addition,
approximately 59 percent of patients treated with Oxbryta 1500 mg
were able to achieve a hemoglobin increase of >2 g/dL and 20
percent achieved >3 g/dL at one or more time points,
compared to approximately 3 percent and no patients in the placebo
group, respectively. The analysis also showed that study
participants treated with Oxbryta had numerically fewer
vaso-occlusive crises (VOCs), consistent with the trends at 24
weeks, and were three times less likely to experience an acute
anemic episode (decrease in hemoglobin >2 g/dL from
baseline).
Additionally, approximately 74 percent of patients (39 of 53)
taking Oxbryta had their overall clinical status rated as
“moderately improved” or “very much improved” by their clinician
compared with approximately 47 percent (24 of 51) of the placebo
group, a statistically significant difference. Treatment with
Oxbryta remained generally well tolerated, and rates of adverse
events were similar between treatment groups over 72 weeks.
“The sickle cell disease community, which for decades has been
dramatically underserved, deserves treatments that address the
sickling and destruction of red blood cells due to hemoglobin
polymerization – the root cause of this disease,” said Ted W. Love,
M.D., president and chief executive officer of GBT. “The HOPE Study
represents a significant milestone in advancing the treatment of
SCD, and we are building on this groundbreaking trial with our
commitment to increase access to Oxbryta and develop novel
therapeutics that can transform SCD into a well-managed
disease.”
Findings from a post hoc analysis of the HOPE Study published
recently in the American Journal of Hematology evaluated the
incidence and outcomes of leg ulcers in SCD patients and further
support the foundational role of hemoglobin S polymerization
inhibition in SCD treatment.1 Results of the analysis showed leg
ulcers improved or resolved by week 72 in all patients (5 of 5)
receiving Oxbryta 1500 mg compared with 63 percent of patients (5
of 8) in the placebo group. Resolution of leg ulcers was associated
with increases in hemoglobin levels and decreases in hemolysis.
Patients who experienced a hemoglobin increase of >1.0 g/dL
while treated with Oxbryta were most likely to experience
resolution of their leg ulcers within 24 weeks. These results
highlight the potential of Oxbryta to meaningfully impact major
patient outcomes.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects an estimated 100,000 people in the United
States,2 an estimated 52,000 people in Europe,3 and
millions of people throughout the world, particularly among those
whose ancestors are from sub-Saharan Africa.2 It also affects
people of Hispanic, South Asian, Southern European and Middle
Eastern ancestry.2 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.4-6 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 throughout the
body. The diminished oxygen delivery to tissues and organs can lead
to life-threatening complications, including stroke and
irreversible organ damage.5-8
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, GBT believes Oxbryta blocks
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.9
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), 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 hemolysis and raising hemoglobin; commercialization,
delivery, availability, use and commercial and medical potential of
Oxbryta; significance of the HOPE Study results, including support
for the long-term use of Oxbryta; 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; 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 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 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 December 31, 2020, 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
- Minniti CP, et al. The impact of voxelotor treatment on leg
ulcers in patients with sickle cell disease. Am J Hematol. 2021.
https://doi.org/10.1002/ajh.26101. Accessed March 2, 2021.
- 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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