Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported
recent business progress and financial results for the first
quarter ended March 31, 2020.
“Our first quarter performance exceeded our expectations and
demonstrates the quality of execution across our entire
organization as well as our employees’ commitment to deliver
Oxbryta, a first-in-class therapy that directly inhibits the
sickling and destruction of red blood cells. We have received
positive feedback from patients and physicians, with increased
awareness of Oxbryta and many patients responding well,” said Ted
W. Love, M.D., president and chief executive officer of GBT. “We
are pleased that a growing number of sickle cell disease patients
have started Oxbryta therapy and are enrolling in GBT Source, our
patient support program. Our payer education activities are also
going well, and we believe we are on track to meet our goal of
obtaining broad payer coverage by the end of the year.
“With the emergence of the COVID-19 pandemic, we quickly adapted
our business activities and launch tactics. We focused on ensuring
our field teams were fully equipped to support our key stakeholders
in an environment with restrictions on in-person meetings. We are
encouraged that many healthcare professionals are embracing
telemedicine to provide ongoing care for their patients.
Additionally, in this challenging environment, I am proud that we
expanded our support for the sickle cell disease community with
direct donations to organizations providing immediate relief to
patients and families across the country.”
Recent Business Progress
Commercial
- Achieved Oxbryta net sales of $14.1 million in the first
quarter of 2020.
- Recorded approximately 1,650 new patient prescriptions of
Oxbryta during the first quarter, for a total of approximately
2,000 since launch. The vast majority of new patient prescriptions
were enrollments into GBT Source™, the company’s patient support
program. The number of new patient prescriptions includes patients
whose treatment was reimbursed and patients who received free
medicine under GBT’s patient assistance program due to insurance
not providing coverage or not having insurance.
- Observed a decline in new patient prescriptions beginning in
the second half of March due to the COVID-19 pandemic. GBT believes
this decline is temporary and expects new patient prescriptions to
return to pre-COVID levels once the pandemic subsides.
- Continued to put Oxbryta reimbursement coverage in place, with
payers representing 25% of covered lives. GBT has secured coverage
policies with Medicaid fee-for-service plans in 11 of 17 priority
states. Most payer meetings to review Oxbryta remain on track for
completion during the first half of 2020, and GBT believes it will
meet the goal of obtaining broad coverage by the end of the
year.
Clinical
- Paused new patient screening and enrollment in
company-sponsored clinical trials due to the COVID-19 pandemic,
while continuing to support patients already enrolled in studies
and conduct study-related administrative activities. GBT
anticipates restarting enrollment in these clinical trials at an
appropriate time and currently does not expect this pause to impact
long-term timelines for its clinical trials, including the
completion of the HOPE-KIDS 2 post-approval confirmatory
study.
Corporate
- Proactively took action intended to support public health
efforts to address the COVID-19 pandemic and to ensure
uninterrupted access and support for patients who are prescribed
Oxbryta. This included temporarily suspending the company’s field
team from all in-person interactions to help slow the spread of the
virus, and ensuring that GBT Source remained available and fully
functional.
- Contributed more than $350,000 to support the sickle cell
disease (SCD) community during the COVID-19 pandemic through grants
from the newly created GBT Community Fund to U.S. non-profit
organizations, a donation to the Sickle Cell Disease Association of
America and personal contributions from GBT’s board of directors
and employees.
Financial Results for the First Quarter
2020
Total product sales, net for the first quarter of 2020 was $14.1
million, resulting from sales of Oxbryta. The company did not
generate product sales in the first quarter of 2019.
Cost of sales for the three months ended March 31, 2020, was
$135,000. 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
first quarter of 2019 as no product sales were generated.
Research and development (R&D) expenses for the three months
ended March 31, 2020, were $39.8 million compared
with $34.5 million for the same period in 2019. The
increase in R&D expenses for this comparative period was
primarily attributable to increased costs related to preclinical
research and manufacturing activities for inclacumab, increased
employee-related costs, including non-cash stock compensation
expense, and increased costs for other preclinical research
activities related to the collaboration that GBT established with
Syros Pharmaceuticals, Inc., in December 2019. The increase was
partially offset by a decrease in manufacturing costs for Oxbryta.
Following the approval of Oxbryta by the FDA in November 2019, GBT
now capitalizes manufacturing of the product to inventory. Total
R&D non-cash stock compensation expense incurred for the three
months ended March 31, 2020, was $5.4 million compared
with $4.0 million for the same period in 2019.
Sales, general and administrative (SG&A) expenses for the
three months ended March 31, 2020, were $47.7
million compared with $18.1 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 March
31, 2020, was $11.0 million compared with $5.4
million for the same period in 2019.
Net loss for the three months ended March 31, 2020,
was $73.0 million compared with $48.9
million for the same period in 2019. Basic and diluted net
loss per share for the three months ended March 31, 2020,
was $1.20 compared with $0.87 for the same
period in 2019. The company expects its operating costs to increase
in subsequent quarters due to costs associated with
commercialization activities as well as costs associated with the
advancement of its clinical pipeline.
Cash, cash equivalents and marketable securities
totaled $615.2 million at March 31, 2020, compared
with $695.0 million at December 31, 2019. The
decrease of $79.8 million from December 31, 2019 includes the
one-time upfront payment of $20 million paid in January 2020
related to the company’s collaboration with Syros.
Conference Call Details
GBT will host a conference call today, Wednesday, May 6,
2020, at 4:30 p.m. ET to provide a general business update and
discuss the financial results for the first 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 Disease
Sickle cell disease (SCD) affects an estimated 100,000 people
in the United States and millions of people throughout
the world, particularly among those whose ancestors are from
sub-Saharan Africa. 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.2 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.5-7
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, GBT believes Oxbryta blocks
polymerization and the resultant sickling and destruction of red
blood cells. 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.7 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 voxelotor in its Priority Medicines (PRIME) program,
and the European Commission (EC) has designated voxelotor
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 Therapeutics
Global 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,
an underlying cause of 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, focus, goals and vision; the safety,
efficacy and mechanism of action of Oxbryta and other product
characteristics; the delivery, availability, use, and commercial
and medical potential of Oxbryta; the commercialization of Oxbryta,
including the activities and resources of the commercial field team
and healthcare professionals; patient use of Oxbryta and GBT
Source, including the recent decline in new patient prescriptions
and related expectations; access and support for patients
prescribed Oxbryta; payer education, meetings and coverage for
Oxbryta; ongoing and planned studies of Oxbryta and related
protocols, activities and expectations, including with respect to
restarting enrollment and the impact on related timelines; GBT’s
financial position, outlook, guidance and expectations;
transforming the treatment and care of SCD; 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,
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.
- 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 March 31, |
|
|
2020 |
|
2019 |
|
|
(Unaudited) |
|
(Unaudited) |
Product sales, net |
|
$ |
14,118 |
|
|
$ |
— |
|
Costs and operating
expenses: |
|
|
|
|
|
|
Cost of sales |
|
|
135 |
|
|
|
— |
|
Research and development |
|
|
39,773 |
|
|
|
34,468 |
|
Selling, general and administrative |
|
47,662 |
|
|
18,055 |
|
Total costs and operating expenses |
|
87,570 |
|
|
52,523 |
|
Loss from operations |
|
(73,452 |
) |
|
(52,523 |
) |
Other income (expense): |
|
|
|
|
|
|
Interest income |
|
2,856 |
|
|
3,831 |
|
Interest expenses |
|
(2,314 |
) |
|
(181 |
) |
Other expenses, net |
|
(116 |
) |
|
(50 |
) |
Total other income, net |
|
426 |
|
|
3,600 |
|
Net loss |
|
$ |
(73,026 |
) |
|
$ |
(48,923 |
) |
Basic and diluted net loss per
common share |
|
$ |
(1.20 |
) |
|
$ |
(0.87 |
) |
Weighted-average number of
shares used in computing basic and diluted net loss per common
share |
|
60,787,710 |
|
|
56,231,587 |
|
GLOBAL BLOOD THERAPEUTICS,
INC.
Condensed Consolidated Balance
Sheets
(In thousands)
|
|
March 31, 2020 |
|
December 31, 2019 |
Assets |
|
(Unaudited) |
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
315,525 |
|
|
$ |
302,237 |
|
Short-term marketable securities |
|
|
215,295 |
|
|
|
307,732 |
|
Other current assets |
|
|
33,649 |
|
|
|
18,028 |
|
Total current assets |
|
|
564,469 |
|
|
|
627,997 |
|
Property and equipment,
net |
|
|
37,329 |
|
|
|
27,113 |
|
Long-term marketable
securities |
|
|
84,378 |
|
|
|
85,030 |
|
Operating lease right-of-use
assets |
|
|
52,082 |
|
|
|
52,775 |
|
Other assets |
|
|
2,888 |
|
|
|
3,184 |
|
Total assets |
|
$ |
741,146 |
|
|
$ |
796,099 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
Current liabilities |
|
$ |
62,248 |
|
|
$ |
71,453 |
|
Long-term debt |
|
|
73,688 |
|
|
|
73,559 |
|
Operating lease liabilities,
noncurrent |
|
|
81,638 |
|
|
|
72,359 |
|
Other noncurrent
liabilities |
|
|
— |
|
|
|
34 |
|
Total liabilities |
|
|
217,574 |
|
|
|
217,405 |
|
Total stockholders’
equity |
|
|
523,572 |
|
|
|
578,694 |
|
Total liabilities and
stockholders’ equity |
|
$ |
741,146 |
|
|
$ |
796,099 |
|
Contact Information:Steven
Immergut (media)650-410-3258simmergut@gbt.com
Stephanie Yao
(investors)650-741-7730syao@gbt.com
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