Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today
announced results from the
Sickle Cell
Health
Awareness,
Perspectives and
Experiences
(SHAPE) survey, a multinational survey on the burden of sickle cell
disease (SCD) and unmet needs as reported by more than 1,300
patients, caregivers and healthcare professionals (HCPs) in 10
countries. The findings highlight the significant impact of SCD on
everyday life for patients, including fatigue, pain and mental
health problems, in addition to impacting their ability to attend
and be successful at school or work, and, thereby, reducing their
earning potential.
One of the largest global burden of disease surveys conducted in
SCD, SHAPE identified long-term health complications of SCD as a
key concern among patients and HCPs. The survey also revealed that
SCD patient caregivers face profound physical, psychosocial, and
economic burdens resulting from taking care of people living with
the disease.1,2
“Sickle cell disease is a lifelong condition that causes damage
in the body and has a profound impact on the quality of life of
those who suffer from it and their caregivers. The SHAPE survey is
important because it illustrates how vital it is that we understand
our patients’ needs, and it suggests what we within the medical
community can do to help change perspectives, increase education
and awareness, and improve care,” said Dr. Baba Inusa, professor
and consultant of paediatric haematology, Guy’s and St Thomas’ NHS
Foundation Trust, London and chair of the National
Haemoglobinopathy Panel in England. “These results are a wake-up
call, and I believe that the actions that follow can enable us to
help drive a better dialogue and improved conversations around the
management and care of this long-neglected and devastating
disease.”
SHAPE Survey HCP ResultsThe findings from the
HCP portion of the survey were presented during a poster
presentation on Friday, June 10, 2022, at the European Hematology
Association (EHA) 2022 Hybrid Congress.1 SHAPE highlighted the
complex environment of treating people living with SCD. The survey
results suggest the need for more education and tools to address
the long-term impact of the disease and underpin the need for
improved overall awareness and understanding of SCD. Key results
from this portion of the study include:
- Despite fatigue/tiredness being identified as having the
biggest impact on patients’ lives (84%), only half (53%) of HCPs
felt they have effective tools to treat this symptom.
- Most HCPs (84%) believed that treating hemolytic anemia (low
hemoglobin due to red blood cell destruction) can improve most
aspects of SCD, including reducing organ damage.
- Most HCPs (78%) felt confident in their knowledge of the
long-term effects of SCD, but 83% wanted more support in educating
their patients on this topic.
- Nearly one third (31%) of HCPs found it challenging to
understand patients’ concerns, with 43% citing difficulties due to
having different ethnic background from their patients.
- Two-thirds (66%) of HCPs felt that patients do not always
understand the long-term effects of SCD, and more than one third
(37%) were reluctant to raise potential future challenges or
complications with a patient or their family.
- Most HCPs (82%) agreed that SCD causes financial difficulties
for patients, which may consequently affect their access to
care.
SHAPE Survey Patient and Caregiver Results The
findings from patients and caregivers highlight that the realities
of SCD for both patients and caregivers extend beyond the
significant pain often experienced by patients and comprise a
broad, lifelong series of physical, emotional and social
barriers.
“SCD is the reality of so many people around the world, and yet
patients often experience poor care, especially by non-specialist
healthcare providers who may lack the training to provide good
comprehensive care. In recent years, we’ve started to see some
positive change, but it is still not enough. The challenges
for patients seem endless and this survey highlights that, in spite
of this, patients do not feel heard. They continue to face
acute physical but also emotional damage, stigma and
discrimination.” said Dianaba Ba, who lives with SCD and is
director of operations, SOS GLOBI: The Federation of Sickle Cell
and Thalassemic Patients, an SCD patient advocacy organization in
France.
Results from the patient and caregiver portions of the survey,
which will be featured in an oral presentation on Friday, June 17,
2022 (16:00 – 17:45 CEST), at the 4th Global Congress on Sickle
Cell Disease,2 include:
- Most patients (93%) agreed that reducing their risk of
long-term health issues due to SCD, such as organ damage, was
important to them.
- Fatigue/tiredness was a symptom experienced by most patients
(84%) in the past year, which also had the greatest negative impact
on their ability to attend and be successful at school or work,
and, thereby, on their earning potential.
- Patients reported that due to SCD they missed an average of 7.5
days of school/work in the past month.
- Mental health symptoms affected the majority of patients with
SCD in the past year, including 62% who reported feeling depressed,
low mood or down, and 57% who indicated they felt worry or
nervousness.
- Patients reported more positive experiences with specialist
HCPs as compared with emergency departments and general HCPs.
- More than half (54%) of SCD patients reported they believe they
had experienced poor care in the past from emergency medicine
professionals due to such providers’ lack of knowledge about
SCD.
- Over half of caregivers reported that patient symptoms
negatively impact their own lives, such as their career and
education (56%), long-term health prospects (55%) and overall
well-being (53%).
“Our aim for this survey is to add to the body of knowledge
around better understanding and characterizing the misunderstood,
under-reported experiences and unmet needs of people living with
SCD, as well as those caring for and treating people with the
condition,” said Kim Smith-Whitley, M.D., executive vice president
and head of research and development of GBT. “These results provide
the opportunity to leverage multinational perspectives and
experiences to help drive more attention about sickle cell disease
and improve dialogue around the treatment, management and care of
these patients. GBT is committed to being part of shaping change
within the global SCD community, and we are grateful to all those
who participated in and contributed to this important
research.”
Members of the SHAPE survey Steering Committee of SCD expert
clinicians and patient advocates include:
- Alan Anderson, M.D., University of South Carolina School of
Medicine Greenville; Comprehensive Sickle Cell Program,
Prisma-Health Upstate, Greenville, SC, USA
- Ashley Clark, Sickle Cell Association of America (SCDAA)
(formerly)
- Baba Inusa, M.D., Paediatric Haematology, Evelina Children's
Hospital, Guy's and St Thomas' Hospital, London, United
Kingdom
- Biba Tinga, Sickle Cell Disease Association of Canada
- Dianaba Ba, SOS GLOBI: The Federation of Sickle Cell and
Thalassemic Patients, France; sickle cell patient
- Elvie Ingoli, German Sickle Cell Disease and Thalassaemia
Association
- Fernando Ferreira Costa, M.D., Ph.D., Haematology and
Haemotherapy Centre, School of Medicine, University of Campinas -
UNICAMP, Campinas, São Paulo, Brazil
- Isaac Odame, MB, ChB, Department of Hematology/Oncology, The
Hospital for Sick Children, Toronto, Canada
- Joachim B. Kunz, M.D., Department of Pediatric Oncology,
Hematology and Immunology, Hopp Children’s Cancer Center (KiTZ)
Heidelberg, University of Heidelberg, Heidelberg, Germany
- John James, OBE, Sickle Cell Society, London, UK
- Mariane de Montalembert, M.D., Hôpital universitaire
Necker-Enfants maladies, Paris, France
- Wasil Jastaniah, MBBS, King Faisal Specialist Hospital &
Research Center, Jeddah, Kingdom of Saudi Arabia
- Zakareya Al Kadhem, Bahrain Society for Sickle Cell Anaemia
Patient Care
About the SHAPE Survey Commissioned
by GBT, the SHAPE survey was conducted by Ipsos Healthcare, a
global health research agency dedicated to understanding the
motivations, behaviors and influences of the multiple actors in the
healthcare sector. Quantitative surveys of 919 patients, 207
caregivers and 219 HCPs were conducted in 10 countries (Bahrain,
Brazil, Canada, France, Germany, Oman, Saudi Arabia, United Arab
Emirates, United Kingdom, and the United States of America),
with fieldwork conducted between September 2021 and February 2022.
Eligible patients had to be aged 12 or over, caregivers had to be
18 or over and HCPs had to care for at least 10 SCD patients.
For more information on the SHAPE survey visit
https://www.gbt.com/shape.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects more than 100,000 people in the United States,3
an estimated 52,000 people in Europe,4 up to 100,000
people in Brazil,5 and millions of people throughout the world,
particularly among those whose ancestors are from sub-Saharan
Africa.6 It also affects people of Hispanic, South Asian,
Southern European and Middle Eastern ancestry.7 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.7 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.7,8,9 The recurrent
sickling process causes destruction of the red blood cells and
hemolytic anemia (low hemoglobin due to red blood cell destruction)
and blockages in capillaries and small blood vessels
(vaso-occlusion), which impede the flow of blood and oxygen
delivery throughout the body, 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.8,9,10,11
Complications of SCD begin in early childhood and can include
neurocognitive impairment, acute chest syndrome, and silent and
overt stroke, among other serious issues.12
About Global Blood Therapeutics Global
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 Statements Certain 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;
significance of the SHAPE survey and related findings and possible
future activities; commercialization, delivery, availability, use
and commercial and medical potential of Oxbryta; impacting the
treatment, course and care of SCD; safety, efficacy, mechanism of
action, advancement and potential of GBT’s drug candidates and
pipeline; 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.
Contact:
Steven Immergut (media) +1 650 410 3258
simmergut@gbt.com
Claudia Nabaie (media Europe) +41 79 906 5814 cnabaie@gbt.com
Courtney Roberts (Investors) +1
650-351-7881croberts@gbt.com
References
- De Montalembert. M et al. Sickle Cell Health Awareness,
Perspectives and Experiences (SHAPE) Survey: Findings on the Burden
of Sickle Disease on Patients and Their Unmet Needs As Reported by
Healthcare Professionals. Presented at the European Hematology
Association 2022 Hybrid Congress; June 9-12, 2022, Vienna, Austria,
and Virtual.
- Odama I et al. Sickle Cell Health Awareness, Perspectives and
Experiences (SHAPE) Survey: Findings on the Burden of Sickle Cell
Disease an Unmet Needs as Reported by Patients and Caregivers. To
be presented at the 4th Global Congress on Sickle Cell Disease;
June 16-18, 2022, Paris, France.
- Centers for Disease Control and Prevention website. Sickle Cell
Disease Research.
https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-understanding-sickle-cell-disease.html.
Last accessed June 2022.
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Last accessed June 2022.
- Ministério da Saúde (Brasil), Protocolo Clínico e Diretrizes
Terapêuticas da Doença Falciforme, Feb. 19, 2018
- Centers for Disease Control and Prevention website. Sickle
Cell Disease
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Last
accessed June 2022.
- National Heart, Lung, and Blood Institute website.
Sickle Cell
Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease.
Last accessed June 2022.
- Kato GJ et al. Nat Rev Dis Primers. 2018;4:18010
- Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
- Kato GJ et al. J Clin Invest. 2017;127:750-760
- Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23
- Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87.
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