Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported
financial results for the second quarter ended June 30, 2021 and
recent business progress.
“In the second quarter we continued to grow the net number of
patients on Oxbryta, reflecting the positive real-world experience
reported by physicians and patients presented at major medical
meetings and shown in our market research,” said Ted W. Love, M.D.,
president and chief executive officer of GBT. “The fundamentals of
the Oxbryta launch remain strong, and we have several initiatives
ongoing and planned that are intended to drive awareness, adoption,
and access. Although overall visits in the second quarter remained
below pre-pandemic levels, we believe in-person reengagement will
drive a gradual improvement in new prescriptions if the COVID-19
pandemic consistently improves.”
“We believe GBT is well positioned for long-term success. As the
body of real-world evidence for Oxbryta grows, we initiated
multi-center, prospective and retrospective data registries that
will track hematologic and clinical responses to treatment with the
medicine. In addition, we submitted our applications seeking FDA
approval of a pediatric formulation of Oxbryta in patients age 4 to
11, and we continue to execute our regulatory strategies in Europe
and the Gulf Cooperation Council (GCC) countries in the Middle
East,” continued Dr. Love. “In addition, our R&D teams are
advancing a portfolio of complementary product candidates that we
believe could enhance care across a range of sickle cell disease
(SCD) clinical manifestations. We initiated two pivotal Phase 3
studies for inclacumab and our Phase 1 study for GBT601 in SCD
patients, giving us confidence that we will achieve our goal to
present proof-of-concept results for GBT601 by the end of
year.”
Recent Business Progress
Commercial
- Achieved Oxbryta® (voxelotor) tablets net sales
of $47.6 million in the second quarter, an increase of
51% year-over-year. On a sequential basis, sales increased 22%,
primarily driven by patient demand.
- Recorded approximately 925 new prescriptions for Oxbryta in the
second quarter.
- The net number of patients taking Oxbryta increased compared to
the prior quarter and has increased each quarter since launch.
- GBT believes that if the pandemic subsides in the second half
of 2021, the number of new prescriptions will improve
incrementally. In addition, GBT anticipates that in future periods
new prescriptions will eventually surpass pre-COVID-19 levels.
- Most recent healthcare provider survey (n = 278, completed in
June-July 2021) confirms continued high levels of satisfaction with
Oxbryta, including its ability to keep SCD under control for
patients. Nearly all prescribers aware of Oxbryta have or would
prescribe it in the future.
- Oxbryta continues to have broad payer coverage, with more than
90% of covered lives having access through their healthcare
plans.
- In July, launched the first branded direct-to-consumer
television advertising campaign in sickle cell disease to educate
and empower patients.
Clinical
- Presented new data from the Phase 2a HOPE-KIDS 1 Study at the
European Hematology Association (EHA) 2021 Virtual Congress that
showed children with SCD ages 4 to 11 years treated with Oxbryta
achieved significant improvements in hemoglobin levels.
- Initiated the RETRO and PROSPECT data registries to enable
deeper understanding of Oxbryta’s long-term efficacy and safety.
Initial results from the RETRO study were presented at EHA,
demonstrating that after 12 months, 50% of patients had a greater
than 1 g/dL increase in hemoglobin.
- Results from a single-center analysis including 77 patients
(age 12-70) were presented at EHA, reinforcing the efficacy and
safety of treatment with Oxbryta in a real-world setting, including
with and without the use of hydroxyurea. In addition, when measured
by the patient and clinical global impressions of change scale, the
majority of patients were rated much improved or very much
improved.
- In July 2021, initiated two global, randomized,
placebo-controlled, pivotal Phase 3 trials evaluating the safety
and efficacy of inclacumab, GBT’s P-selectin inhibitor, for the
reduction of vaso-occlusive crisis (VOC) frequency and VOC-related
hospital readmissions, respectively.
- Initiated a Phase 1 trial of GBT601 in SCD patients, with a
goal of providing proof-of-concept data by the end of the
year.
- Enrollment of pediatric expanded access protocol (EAP) is
exceeding expectations and GBT plans to increase the number of
patients from 50 to up to 150. The EAP is designed to provide
access to Oxbryta prior to potential approval for children ages 4
to 11 years with SCD in the United States who have no alternative
treatment options and are ineligible to participate in clinical
trials of Oxbryta.
Corporate
- In July 2021, announced submission of a supplemental New Drug
Application (sNDA) to the U.S. Food and Drug Administration (FDA)
seeking accelerated approval for Oxbryta for the treatment of SCD
in children ages 4 to 11 years, together with a related separate
NDA required to seek approval for a pediatric weight-based
formulation of Oxbryta. GBT requested priority review for the sNDA
and NDA, which, if granted, could result in a six-month review
process. The FDA has a 60-day filing review period to determine
whether the sNDA and the NDA are complete and acceptable for filing
and review.
- Oxbryta became the first and only SCD treatment to receive the
Promising Innovative Medicine (PIM) designation from the Medicines
and Healthcare Products Regulatory Agency (MHRA) in the United
Kingdom, where approximately 15,000 people live with SCD.1
- In France, Oxbryta was granted Temporary Authorization for Use
(cATU), providing early access to patients beginning in the third
quarter 2021. There are an estimated 20,000 patients diagnosed with
SCD in France.2
- In Germany, a compassionate use program for Oxbryta has been
approved by the Federal Institute for Drugs and Medical Devices
(BfArM). There are an estimated 3,000 patients diagnosed with SCD
in Germany.3
- In the GCC countries, submitted and received validation of an
application for approval of Oxbryta tablets in the United Arab
Emirates (UAE).
- Awarded approximately $450,000 in grants to U.S.
community-based organizations and institutions to advance access to
care for people living with SCD through the company’s Access to
Excellent Care for Sickle Cell Patients (ACCEL) program. The ACCEL
program, which is in its third year and increased the total funding
and number of grantees from 2020, provides support to accelerate
the development of sustainable access-to-care programs for people
living with SCD.
- Strengthened the company’s leadership team with the appointment
of industry veteran Carrie Krehlik as chief human resources
officer, who brings more than 30 years of experience in human
resources, primarily in the biopharmaceutical and technology
industries. Previously, Ms. Krehlik served as senior vice president
and chief human resources officer at Adicet Bio and in leadership
roles at ZS Pharma, Hyperion Therapeutics, and InterMune.
Financial Results for the Second Quarter
2021Total net product sales for the second quarter of 2021
were $47.6 million, resulting from sales of Oxbryta, compared to
$31.5 million for the second quarter of 2020.
Cost of sales for the three months ended June 30, 2021, was $0.7
million, compared with $0.4 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 June 30, 2021, were $51.8 million compared
with $34.1 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 June 30, 2021, was $4.9
million compared with $3.4 million for the same
period in 2020.
Sales, general, and administrative (SG&A) expenses for the
three months ended June 30, 2021, were $61.1
million compared with $49.1 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 June 30, 2021,
was $15.1 million compared with $13.1 million for
the same period in 2020.
Net loss for the three months ended June 30, 2021,
was $69.6 million compared with $52.8
million for the same period in 2020. Basic and diluted net
loss per share for the three months ended June 30, 2021,
was $1.12 compared with $0.86 for the same
period in 2020. Second quarter loss per share included an
anticipated increase in operating costs driven by expanding
commercialization activities related to Oxbryta and the advancement
of the company’s clinical pipeline. GBT anticipates a sequential
increase in operating expenses in the third quarter of 2021 as the
company continues to ramp up these efforts.
Cash, cash equivalents, and marketable securities
totaled $437.4 million at June 30, 2021, compared
with $560.9 million at December 31, 2020.
Conference Call DetailsGBT will host a
conference call today, Tuesday, August 3, 2021, at 4:30
p.m. ET to discuss the financial results for the second quarter
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
under the Investors section. An archived audio webcast will be
available for one month 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,4 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.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.5-7 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 throughout the
body. The diminished oxygen delivery to tissues and organs can lead
to life-threatening complications, including stroke and
irreversible organ damage.6-9
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 Nov. 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.11
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), and the European Commission (EC) has
designated Oxbryta as an orphan medicinal product for the treatment
of patients with SCD. Also, in May 2021, 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 seeking full marketing authorization of Oxbryta in the
European Union to treat hemolytic anemia in SCD patients ages 12
years and older.
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, delivery, availability,
use, and commercial and medical potential of Oxbryta, including the
use, significance and potential of related initiatives; engagement
with patients for Oxbryta, including impact of increased in-person
engagement; significance of data presented at the EHA Congress;
payer coverage for Oxbryta; the expanded access protocol 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 ACCEL Grant
Program, 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
- NHS England.
https://www.england.nhs.uk/wp-content/uploads/2018/08/Specialised-services-for-haemoglobinopathy-care-all-ages.pdf.
Last accessed July 2021.
- Arlet et al. Lancet Haematology (2020)
10.1016/S2352-3026(20)30204-0
- Onkopedia.
https://www.onkopedia.com/de/onkopedia/guidelines/sichelzellkrankheiten/@@guideline/html/index.html
- Centers for Disease Control and Prevention website.
Sickle Cell Disease
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed February 24, 2021.
- European Medicines
Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
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
(In thousands, except share and per share
amounts)
|
|
Three Months Ended |
|
Six Months Ended |
|
|
June 30, |
June 30, |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
Product sales, net |
|
$ |
47,555 |
|
|
$ |
31,501 |
|
|
$ |
86,598 |
|
|
$ |
45,619 |
|
Costs and operating
expenses: |
|
|
|
|
|
|
|
|
Cost of sales |
|
|
748 |
|
|
|
377 |
|
|
|
1,332 |
|
|
|
512 |
|
Research and development |
|
|
51,784 |
|
|
|
34,085 |
|
|
|
102,641 |
|
|
|
73,858 |
|
Selling, general and administrative |
|
|
61,093 |
|
|
|
49,075 |
|
|
|
120,059 |
|
|
|
96,736 |
|
Total costs and operating expenses |
|
|
113,625 |
|
|
|
83,537 |
|
|
|
224,032 |
|
|
|
171,106 |
|
Loss from operations |
|
|
(66,070 |
) |
|
|
(52,036 |
) |
|
|
(137,434 |
) |
|
|
(125,487 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
Interest income |
|
|
164 |
|
|
|
1,514 |
|
|
|
493 |
|
|
|
4,370 |
|
Interest expense |
|
|
(3,677 |
) |
|
|
(2,282 |
) |
|
|
(7,366 |
) |
|
|
(4,596 |
) |
Other expenses, net |
|
|
(9 |
) |
|
|
(36 |
) |
|
|
(215 |
) |
|
|
(153 |
) |
Total other income, net |
|
|
(3,522 |
) |
|
|
(804 |
) |
|
|
(7,088 |
) |
|
|
(379 |
) |
Net loss |
|
|
(69,592 |
) |
|
|
(52,840 |
) |
|
|
(144,522 |
) |
|
|
(125,866 |
) |
Basic and diluted net loss per
common share |
|
$ |
(1.12 |
) |
|
$ |
0.86 |
|
|
$ |
(2.32 |
) |
|
$ |
(2.06 |
) |
Weighted-average number of
shares used in computing basic and diluted net loss per common
share |
|
|
62,312,418 |
|
|
|
61,116,707 |
|
|
|
62,207,328 |
|
|
|
60,952,269 |
|
|
|
|
|
|
|
|
|
|
GLOBAL BLOOD THERAPEUTICS,
INC.
Condensed Consolidated Balance
Sheets
(In thousands)
|
|
June 30, 2021 |
|
December 31, 2020 |
Assets |
|
(Unaudited) |
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
419,311 |
|
|
$ |
494,766 |
|
Short-term marketable securities |
|
|
18,085 |
|
|
|
66,126 |
|
Other current assets |
|
|
87,463 |
|
|
|
71,271 |
|
Total current assets |
|
|
524,859 |
|
|
|
632,163 |
|
Property and equipment, net |
|
|
36,775 |
|
|
|
37,882 |
|
Operating lease right-of-use
assets |
|
|
49,421 |
|
|
|
50,722 |
|
Other assets |
|
|
3,917 |
|
|
|
3,235 |
|
Total assets |
|
$ |
614,972 |
|
|
$ |
724,002 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
Current liabilities |
|
$ |
72,675 |
|
|
$ |
79,032 |
|
Long-term debt |
|
|
149,268 |
|
|
|
148,815 |
|
Operating lease liabilities,
noncurrent |
|
|
76,441 |
|
|
|
79,176 |
|
Other noncurrent liabilities |
|
|
822 |
|
|
|
822 |
|
Total liabilities |
|
|
299,206 |
|
|
|
307,845 |
|
Total stockholders’ equity |
|
|
315,766 |
|
|
|
416,157 |
|
Total liabilities and
stockholders’ equity |
|
$ |
614,972 |
|
|
$ |
724,002 |
|
Contact: Steven
Immergut (media)650-410-3258simmergut@gbt.com
Courtney
Roberts (investors)650-351-7881croberts@gbt.com
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