Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT), today reported
recent business progress and financial results for the third
quarter ended September 30, 2020.
“Our team continued to successfully execute on the launch of
Oxbryta during the third quarter, expanding access and adoption of
this first-in-class treatment that directly targets the underlying
cause of sickle cell disease (SCD). We delivered an increase in new
prescriptions despite a significant increase in COVID-19 infections
in the U.S., including in states with the highest number of SCD
patients – who are at high risk of serious illness from COVID-19.
We also made substantial progress in adding new prescribers and
expanding payer coverage, further establishing a strong foundation
for the Oxbryta launch, about which we continue to be very pleased.
We continue to monitor COVID-19 infection rates across the country
and prioritize the health and safety of patients, healthcare
providers, and our employees. We remain confident in the long-term
potential of Oxbryta,” said Ted W. Love, M.D., president and chief
executive officer of GBT.
“During the quarter, we took another step toward making Oxbryta
available globally with a distribution agreement aimed at providing
access to the large number of sickle cell patients in the Middle
East region,” added Dr. Love. “As part of our ongoing commitment to
raising awareness of the challenges facing SCD patients and
improving their health outcomes, we co-hosted the 9th Annual Sickle
Cell Disease Therapeutics Conference, furthering our efforts to
support the SCD community. We look forward to reinforcing GBT’s
leadership in SCD at this year’s ASH meeting, where we will present
nine abstracts, including updates on the clinical profile of
Oxbryta with longer-term data from the HOPE Study and several
real-world experience abstracts. We are also excited to share new
research on our pipeline as we continue to advance our goal of
making sickle cell disease a well-managed condition.”
Recent Business Progress
Commercial
- Achieved Oxbryta (voxelotor) net sales of $36.9 million and
$82.5 million in the three and nine months ended September 30,
2020, respectively.
- Recorded more than 1,000 new prescriptions of Oxbryta in the
quarter, despite a dramatic increase in COVID-19 cases in the U.S.
during the third quarter.
- The growth in new prescriptions from the second quarter
reflects ongoing increases in the use of telemedicine by healthcare
providers, virtual engagements with GBT field teams and in-person
visits in some geographies. New prescriptions were higher in August
and September, with some weeks nearing pre-pandemic levels. When
the pandemic subsides, GBT expects that, over time, the number of
new prescriptions will further improve and surpass pre-pandemic
levels.
- Secured broad Oxbryta reimbursement coverage one quarter ahead
of expectations, with 90% of lives covered by payers either through
published policies or verified patient adjudication by the end of
the third quarter. GBT has secured fee-for-service Medicaid
coverage in 44 states, including all 17 priority states where most
SCD patients live.
Clinical
- Received acceptance of nine abstracts on GBT’s SCD programs to
be presented at the 62nd American Society of Hematology (ASH)
Annual Meeting & Exposition, which will be held on December
5-8. The presentations will include the 72-week analysis of the
Phase 3 HOPE Study, real-world experience with Oxbryta, preclinical
data highlighting the promise of the company’s SCD pipeline,
including inclacumab and the company’s next generation hemoglobin S
polymerization inhibitor.
- Announced that the company will host a virtual Analyst &
Investor Day on December 7 to review data being presented at the
2020 ASH Annual Meeting.
- Presented two abstracts that provide greater insight into the
safety and efficacy of Oxbryta at the 15th Annual Scientific
Conference on Sickle Cell and Thalassemia (ASCAT) and 1st EHA
European Sickle Cell Conference.
Corporate
- Announced an exclusive agreement with Biopharma-Middle East and
Africa (Biopharma-MEA) to distribute Oxbryta in Bahrain, Kuwait,
Oman, Qatar, Saudi Arabia, and the United Arab Emirates,
collectively known as the Gulf Cooperation Council (GCC) region.
There are estimated to be more than 100,000 people age 12 years and
older in this region living with SCD.1
- Hosted the 9th Annual SCD Therapeutics Conference, which
highlighted recent advances and future trends in the treatment of
SCD, and the impact of COVID-19 on this vulnerable patient
population.
- Strengthened the company’s leadership team with the appointment
of Rajiv Patni, M.D., as chief medical officer.
- Dr. Patni joined GBT from Portola Pharmaceuticals, where he was
the chief medical officer. He brings 20 years of biopharmaceutical
product development experience, including 16 programs and eight
regulatory approvals, across the cardiology, diabetology,
dermato-oncology, hepatology, neurology, and hematology therapeutic
areas.
- Received the 2020 Rare Impact Award® for Industry Innovation
for Oxbryta from the National Organization for Rare Disorders
(NORD); in addition, Oxbryta was selected as Breakthrough Drug
of the Year by the 2020 National Xconomy Awards.
Financial Results for the
Third Quarter
2020Total net product sales for
the third quarter of 2020 was $36.9 million, resulting from sales
of Oxbryta. The company did not generate product sales in the third
quarter of 2019.
Cost of sales for the three months ended September 30, 2020, was
$0.5 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
third quarter of 2019 as no product sales were generated.
Research and development (R&D) expenses for the three months
ended September 30, 2020, were $40.2
million compared with $39.1 million for the same
period in 2019. The increase in R&D expense was primarily due
to increased external costs related to GBT’s inclacumab program and
preclinical research activities related to the collaboration with
Syros Pharmaceuticals, Inc., which were partially offset by
decreased manufacturing costs for Oxbryta. Following FDA approval
of Oxbryta in November 2019, GBT capitalizes manufacturing of
Oxbryta to inventory. Total R&D non-cash stock compensation
expense incurred for the three months ended September 30, 2020,
was $4.2 million compared with $5.1 million for
the same period in 2019.
Sales, general, and administrative (SG&A) expenses for the
three months ended September 30, 2020, were $54.5
million compared with $29.7 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 September 30, 2020, was $14.9 million compared
with $7.3 million for the same period in 2019.
Net loss for the three months ended September 30, 2020,
was $59.9 million compared with $64.5
million for the same period in 2019. Basic and diluted net
loss per share for the three months ended September 30, 2020,
was $0.97 compared with $1.07 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 $535.2 million on September 30, 2020, compared
with $695.0 million on December 31, 2019.
Conference Call DetailsGBT
will host a conference call today, Thursday, November 5, 2020,
at 4:30 p.m. ET to provide a general business update and
discuss the financial results for the third quarter 2020. To
participate in the conference call, please dial 877-252-3031
(domestic) or 312-281-1210 (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,2 an
estimated 52,000 people in Europe,3 and millions of people
throughout the world, particularly among those whose ancestors are
from sub-Saharan Africa.2 It also affects people of Hispanic, South
Asian, Southern European, and Middle Eastern ancestry.2 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.4 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.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, 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.9 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.
GBT plans to seek regulatory approvals to expand the potential
use of Oxbryta in the United States for the treatment of
SCD in children age 4 to 11 years and to treat hemolytic anemia in
SCD in people age 12 years and older in Europe.
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 two
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, and
GBT021601, 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 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; increases in use of telemedicine,
virtual engagements and in-person visits; use of Oxbryta, including
new prescriptions and prescribers and related expectations; payer
coverage for Oxbryta; ongoing and planned studies of Oxbryta and
related protocols, activities, and expectations; 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; bringing Oxbryta
to patients outside the U.S.; impacting the treatment, care and
course of SCD; future presentations; the potential of GBT's
pipeline, including inclacumab and other product
candidates; 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 payer
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
- Data on file.
- 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 September 30, |
Nine Months Ended September 30, |
|
|
2020 |
|
|
2019 |
|
|
2020 |
|
|
2019 |
|
|
(Unaudited) |
(Unaudited) |
(Unaudited) |
(Unaudited) |
Product sales, net |
$ |
36,889 |
|
$ |
— |
|
$ |
82,508 |
|
$ |
— |
|
Costs and operating
expenses: |
|
|
|
|
Cost of sales |
|
513 |
|
|
— |
|
|
1,025 |
|
|
— |
|
Research and development |
|
40,196 |
|
|
39,088 |
|
|
114,054 |
|
|
109,564 |
|
Selling, general and administrative |
|
54,491 |
|
|
29,654 |
|
|
151,227 |
|
|
72,503 |
|
Total costs and operating expenses |
|
95,200 |
|
|
68,742 |
|
|
266,306 |
|
|
182,067 |
|
Loss from operations |
|
(58,311 |
) |
|
(68,742 |
) |
|
(183,798 |
) |
|
(182,067 |
) |
Other income (expense): |
|
|
|
|
Interest income |
|
881 |
|
|
4,372 |
|
|
5,251 |
|
|
11,909 |
|
Interest expense |
|
(2,291 |
) |
|
(146 |
) |
|
(6,887 |
) |
|
(487 |
) |
Other expenses, net |
|
(160 |
) |
|
(31 |
) |
|
(313 |
) |
|
(146 |
) |
Total other income (expense), net |
|
(1,570 |
) |
|
4,195 |
|
|
(1,949 |
) |
|
11,276 |
|
Net loss |
$ |
(59,881 |
) |
$ |
(64,547 |
) |
$ |
(185,747 |
) |
$ |
(170,791 |
) |
Basic and diluted net loss per
common share |
$ |
(0.97 |
) |
$ |
(1.07 |
) |
$ |
(3.04 |
) |
$ |
(2.96 |
) |
Weighted-average number of shares
used in computing basic and diluted net loss per common share |
|
61,573,877 |
|
|
60,098,093 |
|
|
61,160,984 |
|
|
57,637,318 |
|
|
|
|
|
|
GLOBAL BLOOD THERAPEUTICS,
INC.
Condensed Consolidated Balance
Sheets
(In thousands)
|
September 30, 2020 |
|
December 31, 2019 |
Assets |
(Unaudited) |
|
|
Current
assets: |
|
|
|
Cash and cash equivalents |
$ |
418,402 |
|
|
$ |
302,237 |
|
Short-term marketable securities |
|
113,523 |
|
|
|
307,732 |
|
Other current assets |
|
62,739 |
|
|
|
18,028 |
|
Total current assets |
|
594,664 |
|
|
|
627,997 |
|
Property
and equipment, net |
|
38,697 |
|
|
|
27,113 |
|
Long-term marketable securities |
|
3,275 |
|
|
|
85,030 |
|
Operating lease right-of-use assets |
|
51,337 |
|
|
|
52,775 |
|
Other
assets |
|
3,291 |
|
|
|
3,184 |
|
Total
assets |
$ |
691,264 |
|
|
$ |
796,099 |
|
Liabilities and Stockholders’ Equity |
|
|
|
Current
liabilities |
$ |
77,946 |
|
|
$ |
71,453 |
|
Long-term debt |
|
73,880 |
|
|
|
73,559 |
|
Operating lease liabilities, noncurrent |
|
80,439 |
|
|
|
72,359 |
|
Other
noncurrent liabilities |
|
1,344 |
|
|
|
34 |
|
Total
liabilities |
|
233,609 |
|
|
|
217,405 |
|
Total
stockholders’ equity |
|
457,655 |
|
|
|
578,694 |
|
Total
liabilities and stockholders’ equity |
$ |
691,264 |
|
|
$ |
796,099 |
|
|
|
|
|
|
|
|
|
Contact:Steven
Immergut 650-410-3258simmergut@gbt.com
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