Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced
new preclinical data on its sickle cell disease (SCD) pipeline
therapies – inclacumab, a novel P-selectin inhibitor in development
to reduce the frequency of vaso-occlusive crises (VOCs) in patients
with SCD, and GBT021601, a next-generation hemoglobin S (HbS)
polymerization inhibitor. These data are being presented at the
all-virtual 62nd American Society of Hematology (ASH) Annual
Meeting and Exposition.
“In pursuit of our mission to transform the treatment of and
care for people living with sickle cell disease, our research and
development pipeline is targeting multiple pathologies, including
vascular occlusion and hemoglobin polymerization,” said Ted W.
Love, M.D., president and chief executive officer of GBT. “We’re
very excited about the best-in-class potential of both inclacumab
and GBT021601. In 2021, we plan to initiate two pivotal Phase 3
clinical trials evaluating inclacumab for its ability to reduce the
frequency of VOCs and hospital readmissions caused by VOCs. In
addition, this is the first time we are presenting data on
GBT021601. These preclinical data are very promising, and we look
forward to studying the safety and efficacy of this potentially
innovative therapy in SCD patients once we enter the clinic as
planned in the near future.”
Inclacumab: In
Vitro Analysis and Phase
3 Clinical Study ProgramIn vitro study
results (Abstract #1707) demonstrated that inclacumab has the
potential to be a best-in-class P-selectin inhibitor for reducing
the frequency of VOCs in patients with SCD. When characterized
alongside crizanlizumab, an FDA-approved P-selectin inhibitor for
treatment of VOCs, inclacumab:
- Binds P-selectin at the natural ligand binding site and has an
affinity similar to crizanlizumab,
- Demonstrated rapid binding kinetics to P-selectin and remained
bound for longer, and
- Inhibited platelet-leukocyte aggregation to a greater extent
than crizanlizumab.
Additionally, prior clinical experience with inclacumab in more
than 700 non-SCD participants demonstrated the potential for a
substantially longer duration of exposure and near complete
inhibition of platelet-leukocyte aggregation over a 12-week period.
Taken together, we believe these characteristics will translate
into quarterly dosing, improved patient adherence, and the
potential to expand use to a broader patient population.
In 2021, GBT plans to initiate two global, randomized,
placebo-controlled pivotal Phase 3 trials evaluating safety and
efficacy of inclacumab. These trials are designed to enhance
understanding of how P-selectin inhibitors could provide clinical
benefit for patients with SCD and reduce overall healthcare
utilization. One study is designed to reduce the frequency of VOCs
over one year in patients with SCD when treated with inclacumab (30
mg/kg) or placebo every 12 weeks. The second study will evaluate
inclacumab based on a primary endpoint of 90-day hospital
readmission rates following a VOC hospitalization. Participants in
that trial will receive either a single dose of inclacumab (30
mg/kg) or placebo, peri-discharge following a VOC hospitalization.
Approximately 50 percent of U.S. SCD patients with least two annual
VOC events are re-admitted within 90 days following a VOC
hospitalization.1 Initiation of both trials is expected in the
first half of 2021.
GBT021601:
Preclinical Analysis of Next
Generation HbS Polymerization
InhibitorPreclinical data (Abstract #1704) on GBT021601, a
molecule discovered and designed by scientists at GBT, demonstrated
its potential as a potent next-generation HbS polymerization
inhibitor. GBT021601 has the same mechanism of action as Oxbryta®
(voxelotor) tablets, but with the potential for greater efficacy by
achieving higher hemoglobin (Hb) occupancy at significantly lower
doses. The study showed that GBT021601 normalized Hb levels in
Townes sickle cell mice. In addition, in this study GBT021601 was
highly effective in:
- Reducing hemolysis,
- Prolonging red blood cell (RBC) lifespan,
- Improving RBC health, and
- Potentially improving organ function.
Following treatment with GBT021601, levels of erythropoietin
(EPO), a hormone that plays a key role in the production of RBCs,
did not change – indicating that the observed increase in Hb levels
was safe and was not due to hypoxic response.
A Phase 1 clinical study on the safety and tolerability of
GBT021601 in SCD patients is expected to begin by mid-2021.
GBT presentations from the ASH Annual Meeting will be available
on the GBT website.
GBT Analyst & Investor Day Webcast
DetailsGBT is hosting a virtual Analyst & Investor Day
event tomorrow, Monday, December 7, at 4 p.m. PT to review data on
Oxbryta in patients with SCD and preclinical studies on inclacumab
and GBT021601 presented at the 62nd ASH Annual Meeting and
Exposition. The event will provide additional background on the
Phase 3 study program for inclacumab. The webcast can be accessed
directly at www.gbtinvestorday.virtualeventsite.com. Participants
are requested to register in advance. A replay will be available
for three months following the event on GBT’s investor webpage at
www.gbt.com.
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.
GBT plans to seek regulatory approvals to expand the potential
use of Oxbryta in the United States for the treatment of SCD in
children as young as 4 years old, and to treat hemolytic anemia in
SCD patients ages 12 years and older in Europe.
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, 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 https://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, focus, goals,
mission and vision; safety, efficacy and mechanism of action of
Oxbryta and other product characteristics; commercialization,
delivery, availability, use, and commercial and medical potential
of Oxbryta; inferences drawn from study results and related
analyses, including with respect to the potential of inclacumab and
GBT021601; ongoing and planned studies of Oxbryta and drug
candidates and related protocols, activities, timing and other
expectations; potential expansion of the approved use of Oxbryta
for more patients in the U.S., and potential regulatory approval
for Oxbryta to treat patients in Europe; altering the treatment,
course and care of SCD and mitigating related complications;
potential of GBT’s pipeline, including inclacumab and GBT021601;
and advancing GBT’s 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 development, manufacture 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, 2019,
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
- GBT Analysis of Symphony Health Solutions Deidentified Patient
Claims Data from Aug’16 – Jul’19
- 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)GBT650.410.3258simmergut@gbt.com
Courtney Roberts (investors)GBT650.351.7881croberts@gbt.com
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