Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported
financial results for the fourth quarter and year ended December
31, 2021, and recent business progress.
“2021 was a momentous year for GBT as we continued to grow the
net number of patients on Oxbryta in the U.S., while making
progress on our work to expand access to this innovative medicine
to more patients and advance our R&D pipeline in sickle cell
disease,” said Ted W. Love, M.D., president and chief executive
officer of GBT. “Despite ongoing headwinds from COVID-19, including
surges in cases caused by new variants, the fundamentals of our
launch are strong and reflect the dedication of our team. We are
building positive momentum with several key growth initiatives for
Oxbryta, highlighted by new real-world evidence presented at
December’s ASH annual meeting, the FDA approval expanding Oxbryta’s
label to include patients as young as 4 years old in the U.S., and
a groundbreaking marketing authorization in the EU.”
“We believe GBT has the most comprehensive pipeline in SCD and
we are making excellent progress with our lead development programs
based on the compelling data that we generated in 2021. This
included positive Phase 1 proof-of-concept data for GBT601
presented at ASH, which we believe demonstrated its potential to
improve clinical outcomes in people living with SCD, while reducing
pill burden. We plan to initiate a Phase 2 trial for GBT601 by
mid-2022 as we work to rapidly advance this exciting program. In
addition, patient enrollment is ongoing in our two Phase 3 trials
of inclacumab, our P-selectin inhibitor that is being studied as a
quarterly dosed intervention to reduce both the frequency of
painful vaso-occlusive crises and related hospital readmissions,”
added Dr. Love.
Recent Business Progress
Commercial
- Achieved Oxbryta® (voxelotor) net sales of $56.1
million in the fourth quarter, an increase of 36% year over
year. On a sequential basis, sales increased 8%, primarily driven
by patient demand. Net sales for the full year 2021 were $194.7
million, an increase of 57% year over year. The net number of
patients taking Oxbryta increased compared to the prior quarter and
has increased each quarter since launch.
- Recorded approximately 800 new prescriptions for Oxbryta in the
fourth quarter. GBT continues to believe that when the COVID-19
pandemic subsides, the number of new Oxbryta prescriptions will
improve incrementally. GBT anticipates that eventually, in future
periods, new prescriptions will surpass pre-COVID-19 levels.
- Received U.S. Food and Drug Administration (FDA) approval of a
supplemental New Drug Application (sNDA) for Oxbryta for the
treatment of SCD in children ages 4 to less than 12 years and of a
separate New Drug Application (NDA) for a new dispersible,
once-daily tablet dosage form suitable for patients ages 4 to less
than 12 years, which became available to patients in early January.
With the expanded label, Oxbryta now has the potential to reach
more than 100,000 SCD patients in the U.S.1
- 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 anticipates achieving broad coverage by
mid-2022.
Clinical
- Presented six posters at the American Society of Hematology
(ASH) Annual Meeting & Exposition in December 2021, including
positive results of a large real-world experience study of Oxbryta
and long-term data from the open-label extension of the Phase 3
HOPE trial.
- Also at ASH, presented proof of concept results from a Phase 1
trial of GBT021601 (GBT601), the company’s investigational
next-generation sickle hemoglobin (HbS) polymerization inhibitor.
GBT believes these positive data support the continued development
of GBT601 and anticipates initiating a Phase 2 trial of GBT601 by
mid-2022.
- GBT’s ASH posters also covered an analysis of data from the
Phase 1 trial of inclacumab in healthy volunteers.
- 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 February 2022, initiated two randomized, double-blind,
placebo-controlled, multicenter trials studying the treatment
effect of Oxbryta. The Phase 3b trial will evaluate neurocognitive
function in pediatric and adolescent SCD patients (ages 8 to 18
years) and the Phase 4 trial will evaluate cerebral blood flow in
adult and adolescent SCD patients (ages 12 to 30
years).
Corporate
- Received European Commission (EC) marketing authorization for
Oxbryta for the treatment of hemolytic anemia due to sickle cell
disease in adult and pediatric patients 12 years of age and older
as monotherapy or in combination with hydroxycarbamide
(hydroxyurea). This approval means Oxbryta has received marketing
authorization in all European Union member states, as well as in
Iceland, Liechtenstein and Norway. In the United Kingdom, GBT has
submitted a Marketing Authorization Application (MAA) to the
Medicines and Healthcare products Regulatory Agency (MHRA) for a
Great Britain marketing authorization using the EC Decision
Reliance Procedure.
- Received a positive scientific opinion for Oxbryta under the
Early Access to Medicines Scheme (EAMS) from the United Kingdom’s
MHRA, which means that those patients living with SCD and meeting
the eligibility criteria may gain early, pre-license access to
Oxbryta, while the MHRA completes its review of the MAA.
- Strengthened the company’s leadership team with the appointment
of Alain Romero, Ph.D., an accomplished leader with 30 years of
development and clinical experience, as senior vice president, head
of medical affairs.
- Supported the ASH Research Collaborative Data Hub SCD program
with a grant of $400,000 to help expand SCD real-world data
gathering in the U.S.
- Strengthened the company’s balance sheet by executing financing
activities that raised net cash proceeds of approximately $384
million. GBT completed an offering of $345 million of Convertible
Senior Notes due 2028 and entered into privately negotiated capped
call transactions to reduce potential dilution to GBT’s common
stock. The company also amended its term loan with funds managed by
Pharmakon Advisors LP to increase the loan by an additional $100
million and to extend the interest-only payment period to March 31,
2025 and the maturity date to December 17, 2027.
- Included in the 2022 Bloomberg Gender-Equality Index (GEI) for
the second consecutive year. The GEI measures gender equality
across five pillars: female leadership and talent pipeline, equal
pay and gender pay parity, inclusive culture, anti-sexual
harassment policies, and pro-women brand. GBT was one of 418
companies across 11 sectors globally, including one of 23 in
healthcare, to be included.
Financial Results for the Fourth Quarter and Year-End
2021Total net product sales for the fourth quarter of 2021
were $56.1 million, resulting from sales of Oxbryta, compared to
$41.3 million for the fourth quarter of 2020. Total net product
sales for the full year 2021 were $194.7 million, compared with
$123.8 million for the same period in 2020.
Cost of sales for the three months ended December 31, 2021, was
$1.2 million, compared with $1.0 million for the same period in
2020. Total cost of sales for the full year 2021 was $3.3 million,
compared with $2.0 million for the same period in 2020.
Manufacturing costs incurred prior to FDA approval of Oxbryta in
November 2019 were previously recorded as research and development
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 fully expensed, is completely utilized.
Research and development (R&D) expenses for the three months
ended December 31, 2021, were $59.0
million, compared with $41.1 million for the same
period in 2020. R&D expenses for the year ended December 31,
2021, were $212.1 million, compared with $155.1 million for the
same period in 2020. The increase in R&D expenses for both
comparative periods was primarily due to an increase in external
costs related to the company’s inclacumab program, including a
clinical milestone payment of $5.3 million incurred in the fourth
quarter of 2021, as well as the preclinical, Oxbryta and GBT601
programs. Total R&D non-cash stock compensation expense
incurred for the three months ended December 31, 2021,
was $4.4 million, compared with $3.7
million for the same period in 2020. Total R&D non-cash
stock compensation expense incurred for the year ended December 31,
2021, was $21.3 million, compared with $18.1 million for the same
period in 2020.
Sales, general, and administrative (SG&A) expenses for the
three months ended December 31, 2021, were $78.9
million, compared with $58.6 million for the same
period in 2020. SG&A expenses for the year ended December 31,
2021, were $267.0 million, compared with $210.9 million for the
same period in 2020. The increase in SG&A expenses for both
comparative periods was primarily attributable to increased
professional and consulting services associated with the company’s
commercial operations for Oxbryta and employee-related costs,
including non-cash stock compensation expense. Total SG&A
non-cash stock compensation expense incurred in the three months
ended December 31, 2021, was $13.7 million, compared
with $14.3 million for the same period in 2020. Total
SG&A non-cash stock compensation expense incurred in the year
ended December 31, 2021, was $57.4 million, compared with $53.4
million for the same period in 2020.
Net loss for the three months ended December 31, 2021,
was $87.6 million, compared with $61.8
million for the same period in 2020. Basic and diluted net
loss per share for the three months ended December 31, 2021,
was $1.36, compared with $1.00 for the same
period in 2020. Net loss for the year ended December 31, 2021, was
$303.1 million, compared with $247.6 million for the same period in
2020. Basic and diluted net loss per share for the year ended
December 31, 2021, was $4.81, compared with $4.04 for the same
period in 2020.
Cash, cash equivalents, and marketable securities
totaled $734.8 million at December 31, 2021,
compared with $560.9 million at December 31,
2020.
Conference Call DetailsGBT will host a
conference call today, Wednesday, February 23, 2022,
at 4:30 p.m. ET to discuss the financial results for the
fourth quarter and full year 2021 and provide a general 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,1 an estimated 52,000 people in Europe,2 and
millions of people throughout the world, particularly among those
whose ancestors are from sub-Saharan Africa.3 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.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.4,5,6 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 delivery
throughout the body. The diminished oxygen delivery to tissues and
organs can lead to life-threatening complications, including stroke
and irreversible organ damage.5,6,7,8 Complications of SCD
begin in early childhood and can include neurocognitive impairment,
acute chest syndrome, and silent and overt stroke, among other
serious issues.9
About
Oxbryta® (voxelotor)
Oxbryta (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.10 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. 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 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, 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; 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 the potential to
reach more patients; significance of data generated in 2021 and
presented at ASH; payer coverage for Oxbryta, including coverage
for patients aged 4 to less than 12; significance of the positive
scientific opinion under EAMS, including patient access; ongoing
and planned studies, clinical trials and registries, and related
protocols, activities, timing, and other expectations; GBT’s
financial position, outlook, guidance, and expectations;
significance of activities and achievements in 2021 and
opportunities and initiatives for potential growth; the COVID-19
pandemic and related expectations, including the potential impact
on prescriptions; expanding the approved use of Oxbryta, including
related regulatory and other activities and expectations; the
potential impact of the capped call transactions, including with
respect to dilution; 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, 2020, 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.
- 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 December 31, |
|
Year Ended December 31, |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
(Unaudited) |
|
(Unaudited) |
|
(Unaudited) |
|
|
Product sales, net |
|
$ |
56,099 |
|
|
$ |
41,295 |
|
|
$ |
194,749 |
|
|
$ |
123,803 |
|
Costs and operating
expenses: |
|
|
|
|
|
|
|
|
Cost of sales |
|
|
1,154 |
|
|
|
960 |
|
|
|
3,316 |
|
|
|
1,986 |
|
Research and development |
|
|
58,965 |
|
|
|
41,068 |
|
|
|
212,135 |
|
|
|
155,122 |
|
Selling, general and administrative |
|
|
78,942 |
|
|
|
58,640 |
|
|
|
266,988 |
|
|
|
210,851 |
|
Gain on lease modification |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(984 |
) |
Total costs and operating expenses |
|
|
139,061 |
|
|
|
100,668 |
|
|
|
482,439 |
|
|
|
366,975 |
|
Loss from operations |
|
|
(82,962 |
) |
|
|
(59,373 |
) |
|
|
(287,690 |
) |
|
|
(243,172 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
Interest income (expense), net |
|
|
(4,250 |
) |
|
|
(2,340 |
) |
|
|
(14,747 |
) |
|
|
(3,975 |
) |
Other income (expense), net |
|
|
(369 |
) |
|
|
(93 |
) |
|
|
(654 |
) |
|
|
(406 |
) |
Total other income (expense), net |
|
|
(4,619 |
) |
|
|
(2,433 |
) |
|
|
(15,401 |
) |
|
|
(4,381 |
) |
Net loss |
|
|
(87,581 |
) |
|
|
(61,806 |
) |
|
|
(303,091 |
) |
|
|
(247,553 |
) |
Other comprehensive income
(loss): |
|
|
|
|
|
|
|
|
Net unrealized loss on
marketable securities, net of tax |
|
|
(220 |
) |
|
|
(417 |
) |
|
|
(521 |
) |
|
|
(452 |
) |
Cumulative translation
adjustment |
|
|
99 |
|
|
|
— |
|
|
|
319 |
|
|
|
— |
|
Comprehensive loss |
|
$ |
(87,702 |
) |
|
$ |
(61,806 |
) |
|
$ |
(303,293 |
) |
|
$ |
(248,005 |
) |
Basic and diluted net loss per
common share |
|
$ |
(1.36 |
) |
|
$ |
(1.00 |
) |
|
$ |
(4.81 |
) |
|
$ |
(4.04 |
) |
Weighted-average number of
shares used in computing basic and diluted net loss per common
share |
|
|
64,488,886 |
|
|
|
61,849,456 |
|
|
|
62,963,317 |
|
|
|
61,334,037 |
|
|
|
GLOBAL BLOOD THERAPEUTICS,
INC.
Condensed Consolidated Balance
Sheets
(In thousands)
|
|
|
December 31, |
|
|
2021 |
|
2020 |
|
|
(Unaudited) |
|
|
Assets |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
684,717 |
|
$ |
494,766 |
Short-term marketable securities |
|
|
— |
|
|
66,126 |
Other current assets |
|
|
117,253 |
|
|
71,271 |
Total current assets |
|
|
801,970 |
|
|
632,163 |
Long-term marketable
securities |
|
|
50,057 |
|
|
— |
Property and equipment,
net |
|
|
34,918 |
|
|
37,882 |
Operating lease right-of-use
assets |
|
|
48,015 |
|
|
50,722 |
Other assets, noncurrent |
|
|
4,248 |
|
|
3,235 |
Total
assets |
|
$ |
939,208 |
|
$ |
724,002 |
Liabilities and
Stockholders’ Equity |
|
|
|
|
Current liabilities: |
|
|
87,998 |
|
|
79,032 |
Long-term debt, net |
|
|
246,352 |
|
|
148,815 |
Convertible debt, net |
|
|
334,089 |
|
|
— |
Other noncurrent
liabilities |
|
|
74,359 |
|
|
79,998 |
Total
liabilities |
|
|
742,798 |
|
|
307,845 |
Total stockholders’
equity |
|
|
196,410 |
|
|
416,157 |
Total liabilities and
stockholders’ equity |
|
$ |
939,208 |
|
$ |
724,002 |
|
Contact Information: Steven
Immergut (media)+1 650-410-3258simmergut@gbt.com
Courtney Roberts (investors)+1
650-351-7881croberts@gbt.com
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