Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today reported
recent business progress and financial results for the first
quarter ended March 31, 2021.
“In the first quarter, our team continued to drive adoption of
Oxbryta among sickle cell patients and healthcare providers as
the fundamentals of our launch remain strong. We introduced new and
enhanced materials to educate physicians and patients about
Oxbryta, as well as support adherence via new tools and starter
kits to set up patients for success. Despite the headwinds we
expected from the COVID-19 pandemic during the quarter, we continue
to deliver new prescriptions, and the net number of patients on
Oxbryta has increased each quarter since launch. As the year
continues, we believe we will see a gradual improvement in new
prescriptions as COVID-19 cases decrease, more people are
vaccinated, and SCD patients begin to reengage with the healthcare
system,” said Ted W. Love, M.D., president and chief executive
officer of GBT. “On the clinical front, we continue to see
real-world evidence from patients taking Oxbryta that shows
significant and sustained improvement in hemoglobin levels,
reduction in hemolysis, and improved overall health status. The
growing body of clinical evidence demonstrating the benefits of
Oxbryta supports our belief that it should become a standard
therapy in sickle cell disease. In addition, we are building an
innovative pipeline to address SCD from multiple approaches. We are
on track to start two Phase 3 trials of inclacumab by mid-year and
to generate proof-of-concept data for GBT601 in SCD patients by
year end, as we strive to make SCD a well-managed condition for
patients,” added Dr. Love.
Recent Business Progress
Commercial
- Achieved Oxbryta® (voxelotor) tablets net sales
of $39.0 million in the three months ended March 31,
2021, an increase of 177% year-over-year. On a sequential basis,
sales decreased 5% in the first quarter compared to the fourth
quarter of 2020, driven primarily by lower inventory levels and a
higher gross-to-net adjustment, partially offset by patient
demand.
- Recorded approximately 950 new prescriptions of Oxbryta in the
first quarter, despite new cases of COVID-19 reaching peak levels
in the U.S. during this period.
- New prescriptions in the first quarter reflect fewer healthcare
provider and patient interactions due to the increase in COVID-19
cases in the U.S. during this period, partially offset by
utilization of telemedicine by healthcare providers. GBT continues
to believe that when the pandemic subsides, the number of new
prescriptions will improve and surpass pre-COVID-19 levels over
time.
- Oxbryta continues to have broad payer coverage, with more than
90% of covered lives having access through their healthcare
plans.
- Launched patient starter kits and new sales materials in the
U.S. to provide deeper education on Oxbryta and increase patient
adherence, and launched www.gbtsource.com, which provides
information for patients, caregivers and health professionals on
GBT Source Solutions® - the company’s dedicated sickle cell disease
(SCD) patient support program that helps eligible patients start
and stay on Oxbryta.
- A market research study of current Oxbryta users completed
during the first quarter showed that 84% of participants reported
that it works extremely well.
Clinical
- On track to initiate two global, randomized,
placebo-controlled, pivotal Phase 3 trials evaluating the safety
and efficacy of inclacumab by mid-year, and to begin studying
GBT601 in SCD patients with a goal of providing proof-of-concept
data by the end of the year.
- In April 2021, the complete 72-week results from the Phase 3
HOPE Study of Oxbryta were published in The Lancet Haematology. The
results showed significant and sustained improvement in hemoglobin
levels, reduction in hemolysis and improved overall health status
in patients treated with Oxbryta.
- In April 2021, presented a poster at the Academy of Managed
Care Pharmacy 2021 Meeting highlighting a large-scale, longitudinal
analysis demonstrating that increased hemoglobin levels in adult
patients with SCD significantly reduced the risk of developing new
end-organ damage.
- In April 2021, results from a single-center analysis including
76 patients (age 12-70) with SCD were presented at the American
Society of Pediatric Hematology/Oncology Conference, reinforcing
the efficacy and safety of treatment with Oxbryta in a real-world
setting. 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.
- Enrolled the first patient in the Phase 4 ActIVe study that is
evaluating daily physical activity in SCD patients 12 years of age
and older.
Corporate
- Received acceptance for review from the European Medicines
Agency of Oxbryta’s Marketing Authorization Application seeking
full marketing approval to treat hemolytic anemia in patients with
SCD 12 years of age and older.
- Remain on track to submit by mid-year to seek regulatory
approval by the U.S. Food and Drug Administration to expand the
Oxbryta label for treatment of SCD in children ages 4 to 11
years.
- Entered into an agreement with Sanofi S.A. to exclusively
in-license worldwide rights to two early-stage research programs in
SCD: one that pursues a novel anti-sickling mechanism and another
that leverages a new approach to reduce inflammation and oxidative
stress.
- On May 3, 2021, Kim Smith-Whitley, M.D. officially joined GBT
as executive vice president and head of research and
development.
- Strengthened the company’s board of directors with the
appointment of Alexis A. Thompson, M.D., M.P.H., a world-renowned
hematologist and SCD expert, who brings decades of experience in
clinical research, patient care, leadership and advocacy in
hematology and will serve on the board’s research and development
committee.
Financial Results for the First Quarter
2021Total net product sales for the first quarter of 2021
was $39.0 million, resulting from sales of Oxbryta, compared to
$14.1 million for the first quarter of 2020.
Cost of sales for the three months ended March 31, 2021, was
$0.6 million, compared with $0.1 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 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 completely utilized.
Research and development (R&D) expenses for the three months
ended March 31, 2021, were $50.9 million compared
with $39.8 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, including an upfront payment
related to the Sanofi in-license of two early-stage research
programs in SCD, and the company’s inclacumab program. Total
R&D non-cash stock compensation expense incurred for the three
months ended March 31, 2021, was $4.9 million compared
with $5.4 million for the same period in 2020.
Sales, general and administrative (SG&A) expenses for the
three months ended March 31, 2021, were $59.0
million compared with $47.7 million for the same
period in 2020. The increase in SG&A expense was primarily
attributable to increased employee-related costs, including
non-cash stock compensation expense, and increased professional and
consulting services associated with the company’s commercial
operations for Oxbryta. Total SG&A non-cash stock compensation
expense incurred in the three months ended March 31, 2021,
was $15.1 million compared with $11.0 million for
the same period in 2020.
Net loss for the three months ended March 31, 2021,
was $74.9 million compared with $73.0
million for the same period in 2020. Basic and diluted net
loss per share for the three months ended March 31, 2021,
was $1.21 compared with $1.20 for the same
period in 2020. First 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 second quarter of 2021 as the
company continues to ramp up these efforts.
Cash, cash equivalents, and marketable securities
totaled $482.0 million at March 31, 2021, compared
with $560.9 million at December 31, 2020.
Conference Call DetailsGBT will host a
conference call today, Wednesday, May 5, 2021, at 4:30 p.m. ET to
provide a general business update and discuss the financial results
for the first quarter 2021. 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 rare 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, 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.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)
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. 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 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.8
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.
The EMA has accepted for review GBT’s Marketing Authorization
Application seeking full marketing authorization of Oxbryta
in Europe to treat hemolytic anemia in SCD patients ages 12
years and older. GBT also plans to seek 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), 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 (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 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, use, adoption, and
commercial and medical potential of Oxbryta; significance of
patient starter kits, enhanced educational materials and related
information in supporting commercialization efforts; potential of
Oxbryta as a standard therapy in SCD; payer coverage for Oxbryta;
ongoing and planned studies and related protocols, activities,
timing and other expectations; GBT’s financial position, outlook,
guidance, and expectations; the COVID-19 pandemic and related
expectations; providing access to Oxbryta to more patients;
regulatory submissions, review and approval to potentially expand
the approved use of Oxbryta for more patients in the U.S. and to
treat patients in Europe; impacting the treatment, care and course
of SCD and making SCD a well-managed condition; the potential and
advancement of GBT’s pipeline, including inclacumab and other
product candidates; 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 development, manufacture,
distribution 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
obligations under the Pharmakon loan; and the timing and progress
of activities under GBT’s research collaborations; 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
- 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 March 31, |
|
|
2021 |
|
2020 |
|
|
(Unaudited) |
|
(Unaudited) |
|
Product sales, net |
$ |
39,043 |
|
|
$ |
14,118 |
|
|
Costs and operating
expenses: |
|
|
|
|
|
|
Cost of sales |
|
584 |
|
|
|
135 |
|
|
Research and development |
|
50,857 |
|
|
|
39,773 |
|
|
Selling, general and administrative |
58,966 |
|
|
47,662 |
|
|
Total costs and operating expenses |
110,407 |
|
|
87,570 |
|
|
Loss from operations |
(71,364 |
) |
|
(73,452 |
) |
|
Other income (expense): |
|
|
|
|
|
|
Interest income |
329 |
|
|
2,856 |
|
|
Interest expenses |
(3,689 |
) |
|
(2,314 |
) |
|
Other expenses, net |
(206 |
) |
|
(116 |
) |
|
Total other income (expense), net |
(3,566 |
) |
|
426 |
|
|
Net loss |
$ |
(74,930 |
) |
|
$ |
(73,026 |
) |
|
Basic and diluted net loss per
common share |
$ |
(1.21 |
) |
|
$ |
(1.20 |
) |
|
Weighted-average number of shares
used in computing basic and diluted net loss per common share |
62,101,070 |
|
|
60,787,710 |
|
|
GLOBAL BLOOD THERAPEUTICS,
INC.
Condensed Consolidated Balance
Sheets
(In thousands)
|
|
March 31, 2021 |
|
December 31, 2020 |
Assets |
|
(Unaudited) |
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
445,310 |
|
|
$ |
494,766 |
|
Short-term marketable securities |
|
|
36,736 |
|
|
|
66,126 |
|
Other current assets |
|
|
76,086 |
|
|
|
71,271 |
|
Total current assets |
|
|
558,132 |
|
|
|
632,163 |
|
Property and equipment, net |
|
|
38,050 |
|
|
|
37,882 |
|
Operating lease right-of-use
assets |
|
|
50,085 |
|
|
|
50,722 |
|
Other assets |
|
|
3,631 |
|
|
|
3,235 |
|
Total assets |
|
$ |
649,898 |
|
|
$ |
724,002 |
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
Current liabilities |
|
$ |
58,967 |
|
|
$ |
79,032 |
|
Long-term debt |
|
|
149,052 |
|
|
|
148,815 |
|
Operating lease liabilities,
noncurrent |
|
|
77,862 |
|
|
|
79,176 |
|
Other noncurrent liabilities |
|
|
822 |
|
|
|
822 |
|
Total liabilities |
|
|
286,703 |
|
|
|
307,845 |
|
Total stockholders’ equity |
|
|
363,195 |
|
|
|
416,157 |
|
Total liabilities and
stockholders’ equity |
|
$ |
649,898 |
|
|
$ |
724,002 |
|
Contact: Steven
Immergut (media)650-410-3258simmergut@gbt.com
Courtney
Roberts (investors)650-351-7881croberts@gbt.com
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