Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced that it initiated the Phase 2 portion of a planned Phase
2/3 trial of GBT021601 (GBT601), the company’s investigational
next-generation sickle hemoglobin (HbS) polymerization inhibitor.
The study (NCT05431088) is a randomized, multicenter Phase 2/3
clinical trial evaluating the safety, tolerability, efficacy,
pharmacokinetics (PK) and pharmacodynamics (PD) of GBT601.
“Based on compelling preclinical and clinical data, we believe
GBT601 has the potential to be a best-in-class therapy for patients
with sickle cell disease,” said Kim Smith-Whitley, M.D., executive
vice president and head of research and development of GBT.
“Specifically, it has the potential to improve on the clinical
results achieved with Oxbryta® at a lower daily dose. The
initiation of our Phase 2/3 trial is an important milestone in our
efforts to bring GBT601 to patients. We anticipate that initial
data from this trial as well as data from the continuation of our
GBT601 Phase 1 study will be available before the end of the
year.”
The study is planned to include sites in Africa, Europe, the
Middle East, South America, and the United States, including
several sites that are expected to begin enrolling patients in the
near term. The Phase 2 portion of the study will evaluate the
safety, tolerability, and efficacy of GBT601 and enroll up to 60
patients with SCD who are 18 to 65 years of age. Patients with
hemoglobin (Hb) levels between 5.5 g/dL and 10.5 g/dL and 10 or
fewer vaso-occlusive crises in the prior year are eligible for
enrollment. Patients will be randomized 1:1:1 to a daily
maintenance dose of GBT601 of 100, 150 or up to 200 mg. The primary
outcome measure is the number of participants with a change from
baseline in Hb through Week 12. Secondary outcomes measures include
assessments of PK and PD, as well as an assessment of the
relationship between GBT601 and measures of anemia and
hemolysis.
Following the selection of the optimal safe and effective dose
of GBT601 from the Phase 2 portion of the study, the Phase 3
portion will assess the efficacy and safety of the selected optimal
dose compared to placebo in adult and pediatric SCD patients for 48
weeks. In addition, a third arm of the study will evaluate the PK
and safety of single and multiple doses of GBT601 in an open-label
single arm study with pediatric participants.
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 up to 100,000 people in
Brazil,3 and millions of people throughout the world, particularly
among those whose ancestors are from sub-Saharan Africa.4 It also
affects people of Hispanic, South Asian, Southern European and
Middle Eastern ancestry.5 SCD is a lifelong, devastating inherited
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. When sickle
hemoglobin becomes deoxygenated, it polymerizes to form rods, which
deforms the red blood cells into sickled – crescent-shaped, rigid –
cells. 3,5,6 The recurrent sickling process causes destruction of
the red blood cells, hemolysis and anemia (low hemoglobin due to
red blood cell destruction) which drives vascular inflammation
contributing to blockages in capillaries and small blood vessels
(vaso-occlusion) that impede the flow of blood and oxygen delivery
throughout the body. Episodes of painful vascular occlusions are
commonly referred to as vaso-occlusive crises (VOCs). The
diminished oxygen delivery to tissues and organs can lead to
life-threatening complications, including stroke and irreversible
organ damage. 3,6,7,8 Complications of SCD can begin in early
childhood and can include neurocognitive impairment, acute chest
syndrome, and silent and overt stroke, among other serious issues.9
Early intervention and treatment of SCD have shown potential to
modify the course of this disease, reduce symptoms and events,
prevent long-term organ damage, and extend life expectancy.10
About GBT601Discovered and designed by GBT’s
research and development team, GBT021601 (GBT601) has the same
mechanism of action as Oxbryta (voxelotor), with the potential for
improved clinical results by achieving higher hemoglobin levels and
occupancy at a lower dose. GBT601 is being studied in a Phase 1
clinical trial and Phase 2 portion of a Phase 2/3 clinical
trial.
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, starting with sickle cell disease (SCD). Founded in
2011, GBT is delivering on its goal to transform the treatment and
care of SCD, a lifelong, devastating inherited blood disorder. The
company has introduced Oxbryta (voxelotor), the first
FDA-approved medicine that directly inhibits sickle hemoglobin
(HbS) 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 HbS polymerization
inhibitor. In addition, GBT’s drug discovery teams are working on
new targets to develop the next generation 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, vision and positioning; safety, efficacy and
mechanism of action of Oxbryta and other product characteristics;
commercialization, delivery, availability, use and commercial and
medical potential of Oxbryta; significance of the initiation of the
Phase 2/3 trial of GBT601 and bringing GBT601 to patients; clinical
trials of GBT601 and other ongoing and planned studies, clinical
trials and registries, including related protocols, enrollment and
other activities, timing, data availability and other expectations;
significance of preclinical and clinical data of GBT601, including
with respect to the potential of GBT601; safety, efficacy,
mechanism of action, advancement and potential of GBT601,
inclacumab and GBT’s other drug candidates and its pipeline;
impacting the treatment, course and care of SCD; 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, 2021, 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 February 23, 2022.
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125
Accessed February 23, 2022.
- Kato GJ, et al. Nat Rev Dis Primers.
2018;4:18010.
- Centers for Disease Control and
Prevention website. Sickle Cell Disease (SCD).
https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed February
23, 2022.
- National Heart, Lung, and Blood
Institute website. Sickle Cell Disease.
https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease.
Accessed February 23, 2022.
- Rees DC, et al. Lancet.
2010;376(9757):2018-2031.
- Kato GJ, et al. J Clin Invest.
2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir
Rev. 2014;15(1):17-23.
- Kanter J, et al. Blood Rev. 2013
Nov;27(6):279-87.
- Kato GJ, et al. Nat Rev Dis Primers.
2018;4:18010.
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 Aug 2024 to Sep 2024
Global Blood Therapeutics (NASDAQ:GBT)
Historical Stock Chart
From Sep 2023 to Sep 2024