Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported
financial results for the third quarter ended September 30, 2021
and recent business progress.
“Our third quarter results reflect progress with our ongoing
initiatives to drive Oxbryta awareness, adoption and access, and
like others in our industry, this was partially offset by the
impact of the Delta variant causing a surge in COVID-19 cases in
key states. Importantly, we continued to grow the net number of
patients on Oxbryta, and the fundamentals of our launch remain
strong,” said Ted W. Love, M.D., president and chief executive
officer of GBT. “Going forward, we have the opportunity to
significantly expand the number of patients that potentially may
benefit from Oxbryta, including approximately 16,0001 patients ages
4 to 11 years by end of year if our pediatric label expansion in
the U.S. receives approval, and approximately 52,0002 patients via
a potential marketing authorization in Europe in the first half of
2022.”
“We are also advancing a robust R&D pipeline. At the ASH
Annual Meeting in December, we will present initial results from
our Phase 1 study of GBT601, our next-generation sickle hemoglobin
polymerization inhibitor, in both healthy volunteers and sickle
cell patients – adding to our confidence in GBT601’s potential. We
will also report new Phase 1 data on inclacumab at ASH that we
believe supports its best-in-class potential as a well-tolerated,
quarterly dosed P-selectin inhibitor,” added Dr. Love.
Recent Business Progress
Commercial
- Achieved Oxbryta® (voxelotor) net sales of $52.1
million in the third quarter, an increase of 41% year over
year. On a sequential basis, sales increased 9.5%, primarily driven
by patient demand.
- Recorded approximately 850 new prescriptions for Oxbryta in the
third quarter.
- The net number of patients taking Oxbryta increased compared to
the prior quarter and has increased each quarter since launch.
- Results in the third quarter reflect headwinds from the spike
in new COVID-19 cases in August and September. GBT continues to
believe that when the pandemic subsides, the number of new Oxbryta
prescriptions will improve incrementally. GBT anticipates that in
future periods new prescriptions will eventually surpass
pre-COVID-19 levels.
- Oxbryta continues to have broad payer coverage, with more than
90% of covered lives having access through their healthcare
plans.
- Launched the first branded direct-to-consumer television
advertising campaign in sickle cell disease (SCD) to educate and
empower patients, which exceeded its targeted audience reach in the
initial months of the campaign. The commercial can be viewed on
Oxbryta.com.
- Furthered GBT’s education and brand commitment efforts,
including the launch of a video titled “Mechanism of Sickle Cell
Disease” on SickleCellSpeaks.com, to help people learn about SCD
and how it affects the body.
- Continued preparations for the potential approval of Oxbryta in
the U.S. for children ages 4-11 years, including enrolling patients
in GBT’s pediatric expanded access protocol (EAP), engaging in
medical and scientific education with pediatric hematologists, and
educating payers.
Clinical
- Received acceptance of six abstracts on GBT’s SCD programs
to be presented at the 63rd American Society of
Hematology (ASH) Annual Meeting & Exposition, which will
be held from December 11-14. The presentations will include
additional real-world experience with Oxbryta, long-term data from
the open-label extension of the Phase 3 HOPE trial, initial data
from the Phase 1 trial of GBT021601 (GBT601) in healthy volunteers
and SCD patients, and an analysis of data from the Phase 1 trial of
inclacumab in healthy volunteers.
- Presented a poster demonstrating that pediatric patients (aged
4-11 years) experience improvements in health-related quality of
life after initiating therapy with Oxbryta at The Foundation For
Sickle Cell Disease Research’s (FSCDR) Inaugural Nursing in Sickle
Cell Disease Symposium, which was held on October 1, 2021.
Corporate
- Announced that the U.S. Food and Drug Administration (FDA) has
accepted for filing and Priority Review of the company’s
supplemental New Drug Application (sNDA) seeking accelerated
approval for Oxbryta for the treatment of SCD in children ages 4 to
11 years and its New Drug Application (NDA) seeking approval for a
new age-appropriate dispersible tablet dosage form of Oxbryta
suitable for pediatric patients. The FDA assigned a Prescription
Drug User Fee Act (PDUFA) target action date for both applications
of December 25, 2021.
- Granted marketing authorization for Oxbryta by the Ministry of
Health and Prevention (MOHAP) in the United Arab Emirates (UAE) for
the treatment of SCD in adults and children 12 years of age and
older. The UAE is the first country outside of the U.S. where
Oxbryta has been approved.
- Continued progress toward potential Oxbryta marketing
authorization application approval by the European Medicines Agency
in the first half of 2022.
- Hosted the 10th Annual SCD Therapeutics Conference, which
highlighted the latest advancements and future trends for treating
patients with SCD. The event featured sessions on a variety of
critical topics, including the impact of the novel COVID-19
vaccines and the experiences of the SCD community with these
vaccines.
- Supported the Sickle Cell Disease Association of America’s
(SCDAA) 49th Annual National Convention as the Diamond sponsor, to
help address the multifactorial aspects of SCD and foster the
exchange of the latest scientific and clinical information.
- Launched The GBT Foundation, a 501(c)(3) organization and
source of charitable giving that will work to improve the health
and well-being of underserved patient communities around the world.
It will fund programs that will support people within the SCD
community and beyond through education, empowerment, improved
healthcare access and enhanced health equity.
- GBT and Oxbryta received the prestigious 2021 Prix Galien USA
award for “Best Biotechnology Product” from The Galien Foundation.
Oxbryta was selected among 19 nominees by a committee of 12 leaders
from the biotech industry and academia, including five Nobel
Laureates.
Financial Results for the Third Quarter
2021Total net product sales for the third quarter of 2021
were $52.1 million, resulting from sales of Oxbryta, compared to
$36.9 million for the third quarter of 2020.
Cost of sales for the three months ended September 30, 2021, was
$0.8 million, compared with $0.5 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 September 30, 2021, were $50.5
million, compared with $40.2 million for the same
period in 2020. The increase was primarily due to an increase in
external costs related to the company’s preclinical programs and
Oxbryta and inclacumab programs. Total R&D non-cash stock
compensation expense incurred for the three months ended September
30, 2021, was $4.6 million, compared with $4.2
million for the same period in 2020.
Sales, general, and administrative (SG&A) expenses for the
three months ended September 30, 2021, were $68.0
million, compared with $54.5 million for the same
period in 2020. The increase in SG&A expense 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 September 30, 2021,
was $13.5 million, compared with $14.9 million for
the same period in 2020.
Net loss for the three months ended September 30, 2021,
was $71.0 million, compared with $59.9
million for the same period in 2020. Basic and diluted net
loss per share for the three months ended September 30, 2021,
was $1.13, compared with $0.97 for the same
period in 2020.
Cash, cash equivalents, and marketable securities
totaled $416.8 million at September 30, 2021,
compared with $560.9 million at December 31,
2020.
Conference Call DetailsGBT will host a
conference call today, Thursday, November 4, 2021,
at 4:30 p.m. ET to discuss the financial results for the third
quarter 2021 and provide a general business update. To participate
in the conference call, please dial 855-327-6838 (domestic) or +1
604-235-2082 (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.
GBT R&D Day DetailsGBT will host its
R&D Day on Thursday, November 11, 2021, in New York City from
9:00 a.m. to 12:00 p.m. ET. The company will provide an update to
the investment community on Oxbryta, including the growing body of
real-world experience with the product, and its R&D pipeline,
including inclacumab and GBT601. A live video webcast can be
accessed on GBT’s website at www.gbt.com in the Investors section.
The archived video webcast will be available for three months
following the event.
About Sickle Cell DiseaseSickle cell disease
(SCD) affects an estimated 100,000 people in the United
States,3 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.3 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. With the potential to improve hemolytic
anemia and oxygen delivery, GBT believes that Oxbryta has the
potential to modify the course of SCD. On November 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), 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).
The EMA has accepted for review GBT’s Marketing Authorization
Application (MAA) seeking full marketing authorization of Oxbryta
in the European Union to treat hemolytic anemia in SCD patients
ages 12 years and older. GBT is also seeking regulatory approval to
expand the potential use of Oxbryta in the United States for the
treatment of SCD in children as young as 4 years old.
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 healthcare 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
healthcare 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 healthcare
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 2
weeks after the last dose.
Patients should tell their healthcare 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 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) tablets, 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
Phase 3 development to address pain crises associated with the
disease, and GBT021601 (GBT601), 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 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; the content, timing and significance of data and
abstracts to be presented at ASH; payer coverage for Oxbryta; the
EAP for Oxbryta and other initiatives to provide early access,
including the availability, enrollment, use and impact; ongoing and
planned studies, clinical trials and registries, and related
protocols, activities, timing, and other expectations; GBT’s
financial position, outlook, guidance, and expectations; the
COVID-19 pandemic and related expectations, including the potential
impact on prescriptions as the pandemic subsides; expanding access
to Oxbryta, including related strategies, activities and
expectations; regulatory submissions to potentially expand the
approved use of Oxbryta for more patients and in a pediatric
formulation in the U.S. and to treat patients in Europe and other
territories, including potential review, timing and approval; the
GBT Foundation, including the related activities and expectations;
impacting the treatment, care, and course of SCD and mitigating
related complications; safety, efficacy, mechanism of action,
potential and advancement of GBT’s drug candidates and 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 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
- Symphony Health Claims Data, May 2021.
- European Medicines
Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed February 24, 2021.
- Centers for Disease Control and Prevention website.
Sickle Cell Disease
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed February 24, 2021.
- 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.
GLOBAL BLOOD THERAPEUTICS,
INC.
Condensed Consolidated Statements of
Operations (Unaudited) (In
thousands, except share and per share amounts)
|
Three Months Ended |
|
Nine Months Ended |
|
September 30, |
September 30, |
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
Product sales, net |
$ |
52,052 |
|
|
$ |
36,889 |
|
|
$ |
138,650 |
|
|
$ |
82,508 |
|
Costs and operating
expenses: |
|
|
|
|
|
|
|
Cost of sales |
|
830 |
|
|
|
513 |
|
|
|
2,162 |
|
|
|
1,025 |
|
Research and development |
|
50,529 |
|
|
|
40,196 |
|
|
|
153,170 |
|
|
|
114,054 |
|
Selling, general and administrative |
|
67,987 |
|
|
|
54,491 |
|
|
|
188,046 |
|
|
|
151,227 |
|
Total costs and operating expenses |
|
119,346 |
|
|
|
95,200 |
|
|
|
343,378 |
|
|
|
266,306 |
|
Loss from operations |
|
(67,294 |
) |
|
|
(58,311 |
) |
|
|
(204,728 |
) |
|
|
(183,798 |
) |
Other income (expense): |
|
|
|
|
|
|
|
Interest income |
|
85 |
|
|
|
881 |
|
|
|
578 |
|
|
|
5,251 |
|
Interest expense |
|
(3,709 |
) |
|
|
(2,291 |
) |
|
|
(11,075 |
) |
|
|
(6,887 |
) |
Other expenses, net |
|
(70 |
) |
|
|
(160 |
) |
|
|
(285 |
) |
|
|
(313 |
) |
Total other income (expense), net |
|
(3,694 |
) |
|
|
(1,570 |
) |
|
|
(10,782 |
) |
|
|
(1,949 |
) |
Net loss |
|
(70,988 |
) |
|
|
(59,881 |
) |
|
|
(215,510 |
) |
|
|
(185,747 |
) |
Other comprehensive loss: |
|
|
|
|
|
|
|
Net unrealized gain on
marketable securities, net of tax |
|
(42 |
) |
|
|
(507 |
) |
|
|
(301 |
) |
|
|
(35 |
) |
Cumulative translation
adjustment |
|
(39 |
) |
|
|
— |
|
|
|
220 |
|
|
|
— |
|
Comprehensive loss |
$ |
(71,069 |
) |
|
$ |
(60,388 |
) |
|
$ |
(215,591 |
) |
|
$ |
(185,782 |
) |
Basic and diluted net loss per
common share |
$ |
(1.13 |
) |
|
$ |
(0.97 |
) |
|
$ |
(3.45 |
) |
|
$ |
(3.04 |
) |
Weighted-average number of
shares used in computing basic and diluted net loss per common
share |
|
62,925,081 |
|
|
|
61,573,877 |
|
|
|
62,449,207 |
|
|
|
61,160,984 |
|
|
|
|
|
|
|
|
|
GLOBAL BLOOD THERAPEUTICS,
INC.
Condensed Consolidated Balance
Sheets
(In thousands)
|
|
September 30, 2021 |
|
December 31, 2020 |
|
|
(Unaudited) |
|
|
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
382,869 |
|
|
$ |
494,766 |
|
Short-term marketable securities |
|
|
3,205 |
|
|
|
66,126 |
|
Other current assets |
|
|
102,790 |
|
|
|
71,271 |
|
Total current assets |
|
|
488,864 |
|
|
|
632,163 |
|
Long-term marketable securities |
|
|
30,692 |
|
|
|
|
— |
|
Property
and equipment, net |
|
|
35,415 |
|
|
|
37,882 |
|
Operating lease right-of-use assets |
|
|
48,732 |
|
|
|
50,722 |
|
Other
assets |
|
|
3,975 |
|
|
|
3,235 |
|
Total
assets |
|
$ |
607,678 |
|
|
$ |
724,002 |
|
Liabilities and Stockholders’ Equity |
|
|
|
|
Current
liabilities |
|
$ |
74,058 |
|
|
$ |
79,032 |
|
Long-term debt |
|
|
149,508 |
|
|
|
148,815 |
|
Operating lease liabilities, noncurrent |
|
|
74,990 |
|
|
|
79,176 |
|
Other
noncurrent liabilities |
|
|
822 |
|
|
|
822 |
|
Total
liabilities |
|
|
299,378 |
|
|
|
307,845 |
|
Total
stockholders’ equity |
|
|
308,300 |
|
|
|
416,157 |
|
Total
liabilities and stockholders’ equity |
|
$ |
607,678 |
|
|
$ |
724,002 |
|
|
|
|
|
|
|
|
|
|
Contact: Steven
Immergut (media)650-410-3258simmergut@gbt.com
Courtney
Roberts (investors)650-351-7881croberts@gbt.com
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