Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported
financial results for the first quarter ended March 31, 2022, and
recent business progress.
“In the first quarter, we achieved two major milestones in our
journey to expand patient access to Oxbryta,” said Ted W. Love,
M.D., president and chief executive officer of GBT. “In the U.S.,
we launched Oxbryta for patients ages 4 to 11, including a new
age-appropriate formulation, giving us the potential to expand our
reach to more SCD patients. We’ve received positive feedback on the
launch, with encouraging trends during the quarter in new
prescriptions for this age group as well as some incremental growth
for the ages 12 and older population. In addition, we received
marketing authorization from the European Commission for Oxbryta,
making it the first medicine approved in the EU that directly
inhibits the molecular basis of sickling and destruction of red
blood cells in SCD. This milestone is a key step on our path to
reach patients in Europe, with an initial focus on launching in
Germany and establishing reimbursement in key countries.”
“On the clinical front, we are enrolling patients in our two
Phase 3 trials evaluating inclacumab for the potential reduction of
the frequency of vaso-occlusive crises and related hospital
readmissions. In addition, we expect to soon restart our
GBT601 Phase 1 trial due to interest from the patients, which will
also give us the opportunity to study an increased dose of 150 mg,
and we plan to advance GBT601 into a Phase 2/3 trial by
mid-year. We continue to be excited about GBT601, which we believe
has the potential to improve clinical outcomes for people living
with SCD, in a low-dose, once-daily pill,” added Dr. Love.
Recent Business Progress
Commercial
- Recorded Oxbryta® (voxelotor) net sales of $55.2
million in the first quarter, an increase of 41% year over
year. On a sequential basis, sales decreased 2%, driven primarily
by distributors reducing inventory in the first quarter following
purchases at the end of 2021 and a higher gross-to-net adjustment,
partially offset by higher patient demand. The net number of
patients taking Oxbryta increased compared to the prior quarter and
has increased each quarter since launch in 2019.
- Achieved more than 1,200 new prescriptions for Oxbryta in the
first quarter, including a strong launch in children ages 4 to less
than 12 years, which began in early January 2022. GBT continues to
believe that as the COVID-19 pandemic subsides, the number of new
Oxbryta prescriptions will improve. GBT anticipates that
eventually, in future periods, new prescriptions will surpass
pre-COVID-19 levels.
- Oxbryta continues to have broad payer coverage for patients
ages 12 years and older, with more than 90% of covered lives having
access through their healthcare plans. For patients ages 4 to less
than 12 years, GBT continues to work towards securing Oxbryta
reimbursement coverage and is on track to achieve its goal of broad
payer coverage in this age group by mid-year.
- Launched Oxbryta in the United Arab Emirates (UAE)
via GBT's distributor partnership with Biopharma-MEA. This is
the first commercial launch of Oxbryta outside of the U.S.
- Following the receipt of European Commission (EC) marketing
authorization in February 2022, GBT is actively working to
commercially launch Oxbryta in Germany, which is anticipated by
mid-May. In addition, GBT initiated reimbursement discussions in
Germany, France and England, and is educating healthcare providers
across Europe on Oxbryta.
Clinical
- Enrollment is ongoing in two global, randomized,
placebo-controlled, pivotal Phase 3 trials, collectively named
THRIVE, evaluating the safety and efficacy of inclacumab, GBT’s
P-selectin inhibitor, for the potential reduction of vaso-occlusive
crisis (VOC) frequency and VOC-related hospital readmissions.
- In April, data from the Phase 2a HOPE-KIDS 1 study was
published in Pediatric Blood & Cancer, an online peer-reviewed
journal.
- GBT anticipates restarting in May the Phase 1 study of
GBT021601 (GBT601), the company’s investigational next-generation
sickle hemoglobin (HbS) polymerization inhibitor, to study a 150 mg
daily dose, which is an increase over the 100 mg daily maintenance
dose GBT previously studied. All six SCD patients that participated
in the study in 2021 have expressed interest in participating in
the restarted study. Upon completion, the company plans to submit
this data for presentation at a medical meeting later this year.
GBT is also planning to advance GBT601 into a Phase 2/3 trial, and
anticipates initiating the Phase 2 portion by mid-2022.
- Results from the Oxbryta expanded access program for children
(ages 4 to 11) with SCD will be presented in early May at the
American Society of Pediatric Hematology/Oncology Conference,
reinforcing the efficacy and safety of treatment with Oxbryta in
these patients. In addition, the majority of patients had improved
scores as measured by the patient and clinical global impressions
of change scale.
Corporate
- Anticipates receiving marketing authorization for Oxbryta in
Great Britain by mid-2022. There are approximately 15,0001 SCD
patients in Great Britain.
- Strengthened the company’s research and development (R&D)
team with the appointment of R. Clark Brown, M.D., Ph.D., a
world-renowned pediatric hematologist-oncologist and SCD expert, as
vice president of integrated science and clinical site excellence,
starting in July 2022. In this new role, Dr. Brown will lead
clinical research planning and implementation including initiatives
to drive increased engagement at new and existing clinical trial
sites. Previously, Dr. Brown served as the director of clinical
research at the Aflac Cancer and Blood Disorders Center of
Children’s Healthcare of Atlanta, the largest comprehensive SCD
program in the U.S.
- Published inaugural environmental, social and governance (ESG)
report, demonstrating GBT’s progress with its ESG priorities, which
include improving access to affordable care; investing in GBT
employees and communities; increasing environmental sustainability;
and upholding GBT’s ethics and values.
Financial Results for the First Quarter
2022Total net product sales for the first quarter of 2022
were $55.2 million, resulting from sales of Oxbryta, compared to
$39.0 million for the first quarter of 2021.
Cost of sales for the three months ended March 31, 2022, was
$1.1 million, compared with $0.6 million for the same period in
2021. Manufacturing costs incurred prior to FDA approval of Oxbryta
in November 2019 were previously recorded as R&D expense in the
company’s consolidated statement of operations. GBT expects the
cost of Oxbryta sales as a percentage of revenue will increase in
future periods as product manufactured prior to FDA approval, and
therefore previously expensed, is completely utilized.
R&D expenses for the three months ended March 31, 2022,
were $52.8 million, compared with $50.9
million for the same period in 2021. The increase in R&D
expenses for the period was primarily due to an increase in
external costs related to the GBT601 program, partially offset by a
decrease in external costs related to Oxbryta. Total R&D
non-cash stock compensation expense incurred for the three months
ended March 31, 2022, was $5.5 million, compared
with $4.9 million for the same period in 2021.
Sales, general, and administrative (SG&A) expenses for the
three months ended March 31, 2022, were $74.5
million, compared with $59.0 million for the same
period in 2021. The increase in SG&A expenses for the period
was primarily attributable to increased professional and consulting
services associated with the company’s expanded commercial and
medical affairs operations for Oxbryta, including supporting the
pediatric launch in the U.S. and launch readiness in geographies
outside of the U.S.; increased employee-related costs; and
operational growth, partially offset by a decrease in non-cash
stock compensation expense. Total SG&A non-cash stock
compensation expense incurred in the three months ended March
31, 2022, was $13.6 million, compared with $15.1
million for the same period in 2021.
Net loss for the three months ended March 31, 2022,
was $81.4 million, compared with $74.9
million for the same period in 2021. Basic and diluted net
loss per share for the three months ended March 31, 2022,
was $1.26, compared with $1.21 for the same
period in 2021.
Cash, cash equivalents, and marketable securities
totaled $662.1 million at March 31, 2022, compared
with $734.8 million at December 31, 2021.
Conference Call DetailsGBT will host a
conference call today, Wednesday, May 4, 2022, at 4:30
p.m. ET to discuss the financial results for the first quarter 2022
and provide a business update. To participate in the conference
call, please dial 877-407-3982 (domestic) or +1 201-493-6780
(international). A live audio webcast of the conference call can be
accessed on GBT’s website at www.gbt.com in the Investors section.
An archived audio webcast will be available for one month following
the event.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects more than 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.4 It also affects people of Hispanic, South
Asian, Southern European and Middle Eastern ancestry.4 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.5 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.5,6,7 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 delivery throughout the body. The diminished oxygen
delivery to tissues and organs can lead to life-threatening
complications, including stroke and irreversible organ
damage.6,7,8,9 Complications of SCD begin in early childhood and
can include neurocognitive impairment, acute chest syndrome, and
silent and overt stroke, among other serious issues.10
About
Oxbryta® (voxelotor) Tablets
and Tablets for Oral SuspensionOxbryta (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 leading to hemolysis
and hemolytic anemia, 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.
In November 2019, the U.S. Food and Drug Administration (FDA)
granted accelerated approval for Oxbryta tablets for the treatment
of SCD in adults and children 12 years of age and older, and in
December 2021, the FDA expanded the approved use of Oxbryta for the
treatment of SCD in patients 4 years of age and older in the United
States.11 As a condition of accelerated approval for patients ages
4 and older in the United States, 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 14
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 received the prestigious
2021 Prix Galien USA award for “Best Biotechnology Product” from
The Galien Foundation.
Oxbryta has been granted Priority Medicines (PRIME) designation
from the European Medicines Agency (EMA), Oxbryta was designated by
the European Commission (EC) as an orphan medicinal product for the
treatment of patients with SCD, and Oxbryta was granted Promising
Innovative Medicine (PIM) designation in the United Kingdom from
the Medicines and Healthcare products Regulatory Agency (MHRA). In
February 2022, the European Commission (EC) granted Marketing
Authorization for Oxbryta for the treatment of hemolytic anemia due
to SCD in adult and pediatric patients 12 years of age and older as
monotherapy or in combination with hydroxycarbamide (hydroxyurea).
In addition, the Ministry of Health and Prevention (MOHAP) in the
United Arab Emirates (UAE) has granted marketing authorization for
Oxbryta for the treatment of SCD in adults and children 12 years of
age and older.
Please click here for Important Safety Information and full
Prescribing Information including Patient Information for Oxbryta
in the U.S.
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 wave of potential
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, commitment, dedication,
focus, goals, mission, vision, and positioning; the safety,
efficacy, and mechanism of action of Oxbryta, and other product
characteristics; the commercialization, awareness, delivery,
availability, use, and commercial and medical potential of Oxbryta,
including the use, significance and potential of related
initiatives; expanding patient access to Oxbryta; potential launch
of Oxbryta in Europe, including strategy and timing; significance
of the FDA’s approval of regulatory submissions to expand the
approved use of Oxbryta for more patients and in a pediatric
formulation in the U.S. and the marketing authorization for Oxbryta
by the EC, including with respect to patient access and use; payer
coverage for Oxbryta, including coverage for patients aged 4 to
less than 12 and establishing reimbursement in Europe; ongoing and
planned studies, clinical trials and registries, and related
protocols, activities, timing, and other expectations; future
submission and presentation of data; GBT’s financial position,
outlook, guidance, and expectations; the COVID-19 pandemic and
related expectations, including the potential impact on
prescriptions; expanding the approved use of Oxbryta, including
through the potential marketing authorization in Great Britain and
related timing; Dr. Brown’s potential impact, including driving
engagement at clinical trial sites; GBT’s ESG priorities and
related progress; impacting the treatment, care, and course of SCD
and mitigating related complications; 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, 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
- NHS England.
https://www.england.nhs.uk/wp-content/uploads/2018/08/Specialised-services-for-haemoglobinopathy-care-all-ages.pdf.
Last accessed April 2022.
- 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.
- 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.
- 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(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.
- Oxbryta (voxelotor) tablets and tablets for oral suspension
prescribing information. South San Francisco,
Calif. Global Blood Therapeutics, Inc.; December
2021.
GLOBAL BLOOD THERAPEUTICS, INC. |
|
Condensed Consolidated Statements of
Operations |
(Unaudited) |
(In thousands, except share and per share
amounts) |
|
|
|
Three Months Ended March 31, |
|
|
2022 |
|
2021 |
Product sales, net |
|
$ |
55,160 |
|
|
$ |
39,043 |
|
Costs and operating
expenses: |
|
|
|
|
Cost of sales |
|
|
1,055 |
|
|
|
584 |
|
Research and development |
|
|
52,833 |
|
|
|
50,857 |
|
Selling, general and administrative |
|
|
74,533 |
|
|
|
58,966 |
|
Total costs and operating expenses |
|
|
128,421 |
|
|
|
110,407 |
|
Loss from operations |
|
|
(73,261 |
) |
|
|
(71,364 |
) |
Other income (expense): |
|
|
|
|
Interest income (expense), net |
|
|
(7,705 |
) |
|
|
(3,360 |
) |
Other expenses, net |
|
|
(286 |
) |
|
|
(83 |
) |
Total other expense, net |
|
|
(7,991 |
) |
|
|
(3,443 |
) |
Loss before income taxes |
|
|
(81,252 |
) |
|
|
(74,807 |
) |
Provision for income
taxes |
|
|
171 |
|
|
|
123 |
|
Net loss |
|
|
(81,423 |
) |
|
|
(74,930 |
) |
Other comprehensive
expense: |
|
|
|
|
Net unrealized loss on marketable securities, net of tax |
|
|
(1,915 |
) |
|
|
(181 |
) |
Cumulative translation adjustment |
|
|
(147 |
) |
|
|
— |
|
Comprehensive loss |
|
$ |
(83,485 |
) |
|
$ |
(75,111 |
) |
Basic and diluted net loss per
common share |
|
$ |
(1.26 |
) |
|
$ |
(1.21 |
) |
Weighted-average number of
shares used in computing basic and diluted net loss per common
share |
|
|
64,842,086 |
|
|
|
62,101,070 |
|
GLOBAL BLOOD THERAPEUTICS, INC. |
|
Condensed Consolidated Balance Sheets |
(In thousands) |
|
|
|
March 31, 2022 |
|
December 31, 2021 |
Assets |
|
(Unaudited) |
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
391,803 |
|
$ |
684,717 |
Short-term marketable securities |
|
|
161,614 |
|
|
— |
Other current assets |
|
|
122,231 |
|
|
117,253 |
Total current assets |
|
|
675,648 |
|
|
801,970 |
Long-term marketable
securities |
|
|
108,667 |
|
|
50,057 |
Property and equipment,
net |
|
|
33,598 |
|
|
34,918 |
Operating lease right-of-use
assets |
|
|
47,271 |
|
|
48,015 |
Other assets, noncurrent |
|
|
4,359 |
|
|
4,248 |
Total
assets |
|
$ |
869,543 |
|
$ |
939,208 |
Liabilities and
Stockholders’ Equity |
|
|
|
|
Current liabilities |
|
|
79,305 |
|
|
87,998 |
Long-term debt, net |
|
|
246,738 |
|
|
246,352 |
Convertible debt, net |
|
|
334,451 |
|
|
334,089 |
Other liabilities,
noncurrent |
|
|
75,047 |
|
|
74,359 |
Total
liabilities |
|
|
735,541 |
|
|
742,798 |
Total stockholders’
equity |
|
|
134,002 |
|
|
196,410 |
Total liabilities and
stockholders’ equity |
|
$ |
869,543 |
|
$ |
939,208 |
Contact Information: Steven
Immergut (media)+1 650-410-3258simmergut@gbt.com
Courtney Roberts (investors)+1
650-351-7881croberts@gbt.com
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