Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported
recent business progress and financial results for the second
quarter ended June 30, 2020.
“In the second quarter of 2020, we continued to
make important progress on GBT’s vision of being a leading
biopharmaceutical company and the leader in sickle cell disease.
Despite the impact of COVID-19, we continued to successfully
execute the launch of Oxbryta and make significant progress on new
prescriptions, new prescribers, and payer coverage. During the
quarter, we were pleased to see increased use of virtual
engagements and telemedicine by healthcare professionals and
continued growth of Oxbryta prescriptions. In addition, we made
significant progress in our efforts to expand the approved use of
Oxbryta for more patients in the United States and in Europe, with
regulatory submissions planned by mid-2021,” said Ted W. Love,
M.D., president and CEO of GBT.
“Last quarter was also marked by a magnification
of health disparities driven by COVID-19, which is having an
outsized impact on African Americans, and a renewed focus on racial
injustice sparked by deaths due to police brutality. The sickle
cell disease community lives and breathes these issues, and GBT is
working to address them, as evidenced by our cohort of ACCEL grant
recipients. We are committed to enacting real and lasting change
for communities of color, especially when it comes to ensuring
patients have access to the care they need and deserve,” added Dr.
Love.
Recent Business Progress
Commercial
- Achieved Oxbryta® (voxelotor) net sales of $31.5 million in the
second quarter of 2020.
- Despite the impact of COVID-19, recorded approximately 1,000
new prescriptions of Oxbryta during the second quarter.
- The decrease in new prescriptions stabilized following an
initial impact of COVID-19 at the end of the first quarter. The
stabilization was primarily due to the increased use of
telemedicine by healthcare providers and increased comfort with
virtual engagements with GBT field teams. When the pandemic
subsides, GBT expects that, over time, the number of new
prescriptions will improve from current levels and surpass
pre-COVID-19 levels.
- Conducted a review of patient charts, claims, and lab data that
showed Oxbryta is being prescribed to a broad range of patients
irrespective of hemoglobin (Hb) level, history of vaso-occlusive
crisis (VOC), and current therapy for sickle cell disease (SCD).
Almost half of patients started therapy with baseline Hb greater
than 8 g/dL, and more than half take Oxbryta in combination with
another SCD therapy.
- Continued to secure Oxbryta reimbursement coverage, with 53% of
lives covered by payers either through published policies or
medical exceptions. GBT has secured fee-for-service Medicaid
coverage in 42 states, including all 17 priority states where most
SCD patients live. GBT believes it is on track to meet its goal of
obtaining broad coverage by the end of the year.
Clinical
- Collaborated with clinical trial sites to implement appropriate
protocols that would, over time, enable the resumption of patient
enrollment and participation in GBT-sponsored clinical trials. The
company believes it remains on track with the long-term timelines
for its clinical trials.
- Presented four abstracts at the virtual edition of the 25th
Annual European Hematology Association Congress:
- A retrospective analysis of data from the landmark STOP 2 study
linking higher Hb levels to lower transcranial Doppler flow
velocity, a predictor of stroke risk in children with SCD.
- Three encore presentations of the pivotal Phase 3 HOPE Study
that reinforced key attributes of Oxbryta, including that the
lowest incidence of VOCs occurred in patients who achieved the
highest Hb levels; that biomarkers of hemolysis decrease with
increasing dose; and that the treatment effect seen with Oxbryta
was independent of hydroxyurea use.
Corporate
- Announced plans to expand the potential use of Oxbryta for the
treatment of SCD in children as young as 4 years old. GBT plans to
submit a New Drug Application, which will include a new
age-appropriate formulation, to the U.S. Food and Drug
Administration by mid-2021.
- Announced plans to seek full marketing authorization in Europe
for Oxbryta to treat hemolytic anemia in SCD patients ages 12 years
and older. GBT plans to submit a Marketing Authorization
Application to the European Medicines Agency by mid-2021.
- Awarded $250,000 in grants to five nonprofit organizations 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 was established by GBT in 2019 to help
fund programs that have the potential to deliver high-quality
healthcare to people living with SCD.
Financial Results for the Second Quarter
2020Total net product sales for the second quarter of 2020
was $31.5 million, resulting from sales of Oxbryta. The company did
not generate product sales in the second quarter of 2019.
Cost of sales for the three months ended June
30, 2020, was $0.4 million. 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 that 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 utilized. GBT did not incur cost of sales for Oxbryta
in the second quarter of 2019 as no product sales were
generated.
Research and development (R&D) expenses for
the three months ended June 30, 2020, were $34.1
million compared with $36.0 million for the same
period in 2019. The decrease in R&D expenses for this
comparative period was primarily attributable to a decrease in
manufacturing costs for Oxbryta. Following FDA approval of Oxbryta
in November 2019, GBT now capitalizes manufacturing of the product
to inventory. The decrease in R&D expenses was partially offset
by increased costs related to preclinical research and
manufacturing activities for inclacumab, increased employee-related
costs, and increased costs for other preclinical research
activities related to the collaboration with Syros Pharmaceuticals,
Inc. Total R&D non-cash stock compensation expense incurred for
the three months ended June 30, 2020, was $3.4
million compared with $4.7 million for the same
period in 2019.
Sales, general, and administrative (SG&A)
expenses for the three months ended June 30, 2020,
were $49.1 million compared with $24.8
million for the same period in 2019. The increase in SG&A
expenses for this comparative period was primarily attributable to
increased employee-related costs, including non-cash stock
compensation expense, and increased professional and consulting
services associated with the build-out of the company’s commercial
operations and launch of Oxbryta. Total SG&A non-cash stock
compensation expense incurred in the three months ended June
30, 2020, was $13.1 million compared with $6.2
million for the same period in 2019.
Net loss for the three months ended June
30, 2020, was $52.8 million compared with $57.3
million for the same period in 2019. Basic and diluted net
loss per share for the three months ended June 30, 2020,
was $0.86 compared with $1.01 for the same
period in 2019. The company expects its operating costs to increase
in subsequent quarters due to costs associated with expanding
commercialization activities as well as costs associated with the
advancement of its clinical pipeline.
Cash, cash equivalents, and marketable
securities totaled $574.2 million at June 30, 2020,
compared with $695.0 million at December 31,
2019.
Conference Call DetailsGBT will
host a conference call today, Wednesday, August 5, 2020,
at 4:30 p.m. ET to provide a general business update and
discuss the financial results for the second quarter 2020. To
participate in the conference call, please dial 877-407-3982
(domestic) or 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,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.1 It also affects people of
Hispanic, South Asian, Southern European, and Middle Eastern
ancestry.1 SCD is a lifelong inherited blood disorder that impacts
hemoglobin, a protein carried by red blood cells that delivers
oxygen to tissues and organs throughout the body.3 Due to a genetic
mutation, people 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.3-5 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.4-7
About Oxbryta® (voxelotor)
tablets 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. Through addressing hemolytic anemia
and improving oxygen delivery throughout the body, 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.8 As a
condition of accelerated approval, GBT will continue to study
voxelotor in the HOPE-KIDS 2 Study, a post-approval confirmatory
study using transcranial Doppler (TCD) flow velocity to assess the
ability of Oxbryta 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. The European Medicines
Agency (EMA) has included Oxbryta in its Priority Medicines
(PRIME) program, and the European Commission (EC) has
designated Oxbryta as an orphan medicinal product for the treatment
of patients with SCD.
Important Safety Information
Oxbryta 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), 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 development to address pain crises
associated with the disease. 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, and vision; the
safety, efficacy, and mechanism of action of Oxbryta, and other
product characteristics; the commercialization, delivery,
availability, and commercial and medical potential of Oxbryta; use
of Oxbryta, including new prescriptions and related expectations;
payer coverage for Oxbryta; ongoing and planned studies of Oxbryta
and related protocols, activities, and expectations, including with
respect to resumption of enrollment and participation in clinical
trials and the impact on related timelines; GBT’s financial
position, outlook, guidance, and expectations; the COVID-19
pandemic and related expectations; the potential expansion of the
approved use of Oxbryta for more patients in the U.S. and potential
approval of Oxbryta to treat patients in Europe, including the
submission of regulatory filings, the type and contents of such
filings, and related timing; impacting the treatment, care and
course of SCD; GBT’s efforts with respect to health disparities,
racial injustice, and enacting change; the significance and
potential impact of the ACCEL program and the grant recipients; the
potential of inclacumab; and advancing GBT’s 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 has only recently established its
commercialization capabilities and may not be able to successfully
commercialize Oxbryta; risks associated with GBT’s dependence on
third parties for development, manufacture, and commercialization
activities related to Oxbryta; government and third-party payor
actions, including those relating to reimbursement and pricing;
risks and uncertainties relating to competitive products 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 the funding and other
obligations under the Pharmakon loan; and the timing and progress
of GBT’s and Syros’ research and development activities under their
collaboration; along with those risks set forth in GBT’s Annual
Report on Form 10-K for the fiscal year ended December 31, 2019,
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
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed June 3, 2019.
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed June 12, 2020.
- 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 June 30,
|
Six Months Ended June 30, |
|
|
2020 |
|
|
2019 |
|
|
2020 |
|
|
2019 |
|
|
(Unaudited) |
(Unaudited) |
(Unaudited) |
(Unaudited) |
Product sales, net |
$ |
31,501 |
|
$ |
— |
|
$ |
45,619 |
|
$ |
— |
|
Costs and operating
expenses: |
|
|
|
|
Cost of sales |
|
377 |
|
|
— |
|
|
512 |
|
|
— |
|
Research and development |
|
34,085 |
|
|
36,010 |
|
|
73,858 |
|
|
70,476 |
|
Selling, general and administrative |
|
49,075 |
|
|
24,794 |
|
|
96,736 |
|
|
42,849 |
|
Total costs and operating expenses |
|
83,537 |
|
|
60,804 |
|
|
171,106 |
|
|
113,325 |
|
Loss from operations |
|
(52,036 |
) |
|
(60,804 |
) |
|
(125,487 |
) |
|
(113,325 |
) |
Other income (expense): |
|
|
|
|
Interest income |
|
1,514 |
|
|
3,706 |
|
|
4,370 |
|
|
7,537 |
|
Interest expense |
|
(2,282 |
) |
|
(160 |
) |
|
(4,596 |
) |
|
(341 |
) |
Other expenses, net |
|
(36 |
) |
|
(63 |
) |
|
(153 |
) |
|
(115 |
) |
Total other income, net |
|
(804 |
) |
|
3,483 |
|
|
(379 |
) |
|
7,081 |
|
Net loss |
$ |
(52,840 |
) |
$ |
(57,321 |
) |
$ |
(125,866 |
) |
$ |
(106,244 |
) |
Basic and diluted net loss per
common share |
$ |
(0.86 |
) |
$ |
(1.01 |
) |
$ |
(2.06 |
) |
$ |
(1.88 |
) |
Weighted-average number of shares
used in computing basic and diluted net loss per common
share |
|
61,116,707 |
|
|
56,539,760 |
|
|
60,952,269 |
|
|
56,386,560 |
|
|
|
|
|
|
GLOBAL BLOOD THERAPEUTICS,
INC.Condensed Consolidated Balance
Sheets(In thousands)
|
June 30, 2020 |
|
December 31, 2019 |
Assets |
(Unaudited) |
|
|
Current assets: |
|
|
|
Cash and cash equivalents |
$ |
384,716 |
|
|
$ |
302,237 |
|
Short-term marketable securities |
|
156,014 |
|
|
|
307,732 |
|
Other current assets |
|
53,303 |
|
|
|
18,028 |
|
Total current assets |
|
594,033 |
|
|
|
627,997 |
|
Property and equipment, net |
|
39,668 |
|
|
|
27,113 |
|
Long-term marketable
securities |
|
33,479 |
|
|
|
85,030 |
|
Operating lease right-of-use
assets |
|
51,580 |
|
|
|
52,775 |
|
Other assets |
|
2,907 |
|
|
|
3,184 |
|
Total assets |
$ |
721,667 |
|
|
$ |
796,099 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
Current liabilities |
$ |
69,402 |
|
|
$ |
71,453 |
|
Long-term debt |
|
73,775 |
|
|
|
73,559 |
|
Operating lease liabilities,
noncurrent |
|
81,903 |
|
|
|
72,359 |
|
Other noncurrent liabilities |
|
771 |
|
|
|
34 |
|
Total liabilities |
|
225,851 |
|
|
|
217,405 |
|
Total stockholders’ equity |
|
495,816 |
|
|
|
578,694 |
|
Total liabilities and
stockholders’ equity |
$ |
721,667 |
|
|
$ |
796,099 |
|
Contact Information:Steven Immergut
(media)650-410-3258simmergut@gbt.com
Stephanie Yao (investors)650-741-7730syao@gbt.com
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