Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, today reported financial
results for the fourth quarter and full-year 2020.
“In the midst of a challenging pandemic, in 2020
the Sarepta team executed and stayed focused on the patients we
serve. We advanced our multi-platform portfolio and achieved a
number of important milestones, both in our gene therapy and in our
RNA platform, including the submission of our FDA application for
the approval of AMONDYS 45™ (casimersen), which resulted in the
approval we reported last week. This is our third internally
developed RNA therapy approved in the U.S. to treat Duchenne
muscular dystrophy and we are now able to offer treatment to the 8%
of Duchenne patients with a confirmed exon 45 amenable mutation. By
providing a treatment option for patients with an exon 45 amenable
mutation, AMONDYS 45 will contribute to the steady growth of our
RNA product revenue, which in the fourth quarter of 2020 achieved
net product sales of $122.6 million, a 23% increase over the same
quarter last year and $455.9 million for full year 2020, a nearly
20% increase over the prior year,” stated Doug Ingram, Sarepta’s
president and CEO. “This approval moves us closer to our goal of
treating the greatest percentage of the Duchenne community as
possible. Our three therapies together can treat nearly 30% of
Duchenne patients in the U.S. and with our RNA-PMO technology, or
its next generation version, the PPMO, we could build constructs to
ultimately treat over 80% of Duchenne mutations.”
Mr. Ingram continued, “In January and at the end
of the fourth quarter we reported results from two clinical trials
for two of our key value-driving pipeline assets in development for
Duchenne: Part 1 of Study 102 for our gene therapy SRP-9001, and
Part A of the MOMENTUM study for SRP-5051, our first candidate
developed using our next-generation PPMO technology. Although Study
102 did not achieve statistical significance on the primary
functional endpoint, the top-line results reinforce our confidence
in the potentially transformative benefits of SRP-9001 and
generated key insights that will inform the protocol for our
upcoming trial, Study 301. Additionally, we plan to report data in
the second quarter from Study 103 called ENDEAVOR, our open-label
study that is testing commercially representative SRP-9001
material. For SRP-5051 we are pleased with the higher tissue
concentration, exon skipping and dystrophin production in the 20
mg/kg dosing group observed at an early 12-week timepoint at an
order of magnitude lower cumulative drug exposure compared to our
current PMO technology. We are on track to report data from the 30
mg/kg arm of the MOMENTUM trial in the second quarter.
Additionally, later this month at the 2021 MDA Clinical and
Scientific Conference, we will present the data for SRP-9001-102
Part 1 and new long-term functional data from study SRP-9003-101,
our gene therapy in development for limb-girdle muscular dystrophy
type 2E.”
Fourth Quarter 2020 and Recent Corporate
Developments:
- Received FDA Approval of AMONDYS 45 (casimersen)
Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in
Patients Amenable to Skipping Exon 45, Sarepta’s third RNA
exon-skipping treatment for DMD approved in the U.S.:
AMONDYS 45 is an antisense oligonucleotide from Sarepta’s
phosphorodiamidate morpholino oligomer (PMO) platform, indicated
for the treatment of DMD in patients with a confirmed mutation
amenable to exon 45 skipping. This indication is based on a
statistically significant increase in dystrophin production in
skeletal muscle observed in patients treated with AMONDYS 45, which
is reasonably likely to predict clinical benefit for those patients
who are exon 45 amenable. Consistent with the accelerated approval
pathway, the continued approval of AMONDYS 45 may be contingent on
confirmation of a clinical benefit in confirmatory trials. The
ESSENCE trial, a placebo-controlled, confirmatory trial to support
both the AMONDYS 45 and VYONDYS 53 approvals is ongoing and
expected to conclude in 2024.
- Reported top-line results
for Part 1 of Study SRP-9001-102 (Study 102) evaluating SRP-9001,
Sarepta’s investigational gene transfer therapy for the treatment
of Duchenne muscular dystrophy: Study 102 is a
double-blind, 1:1 randomized, placebo-controlled clinical trial of
SRP-9001 in 41 participants with DMD, ages of 4-7, using clinical
process SRP-9001. Primary endpoints are micro-dystrophin expression
at 12 weeks and change in NSAA total score at 48 weeks compared to
placebo. In Part 1 results from the treatment and placebo groups
are compared through 48 weeks following treatment. Data from the
Part 1 of the study showed the following:
- Met the primary biological endpoint
of micro-dystrophin protein expression at 12 weeks post-treatment
(P<0.0001), as measured by western blot, in SRP-9001-treated
participants versus placebo with mean micro-dystrophin expression
of 28.1% for treated participants.
- SRP-9001-treated participants
showed an increase in North Star Ambulatory Assessment (NSAA) total
score compared to placebo at 48 weeks.
- Study did not achieve statistical
significance (P=0.37) on the primary functional endpoint of
improvement in NSAA total score compared to placebo at 48 weeks
post-treatment.
- No new safety signals for SRP-9001
were identified.In the pre-specified analysis of participants aged
4-5 (n=16) at the time of treatment, the treatment group
demonstrated a statistically significant improvement on NSAA
compared to the age-matched placebo group (P=0.0172). The
functional status at baseline for the 4-5 age group was balanced
across the placebo and treatment cohorts. A statistically
significant imbalance (P=0.0046) in baseline NSAA total score was
present in the cohort of 6-7-year-old participants (n=25),
resulting in milder participants in the placebo arm (n=13) than in
the treated arm (n=12). The significantly different baseline
characteristics between treatment and control groups in the 6-7 age
group may have contributed to the inability to observe a treatment
effect in the 6-7 age group at the week 48 timepoint in Part
1.Study 102 is ongoing and remains blinded with all participants
having entered the Part 2 crossover phase. Participants continue to
be monitored for safety and will undergo another biopsy at week 12
in Part 2 to assess expression and biological markers, in addition
to longer-term assessments of functional outcomes.
-
Announced positive clinical results from MOMENTUM, a global
Phase 2 clinical trial of SRP-5051 in patients with Duchenne
muscular dystrophy amenable to skipping exon 51: The
multiple-ascending dose trial of Sarepta’s investigational peptide
phosphorodiamidate morpholino oligomer (PPMO), its next-generation
technology for the treatment of Duchenne, demonstrated
proof-of-concept for SRP-5051 and supports continued dose
escalation. At a total dose exposure approximately 10x lower than
eteplirsen, SRP-5051 at 20 mg/kg showed enhanced tissue exposure,
greater exon skipping, and greater dystrophin production with no
negative renal or other laboratory findings. When compared to a
control group of Duchenne patients from the PROMOVI study who
received biopsies at 24 weeks after taking a weekly 30 mg/kg dose
of eteplirsen, once-monthly dosing of SRP-5051 resulted in higher
muscle concentration, increased exon-skipping and dystrophin at 12
weeks. A dose-dependent increase in exon-skipping and dystrophin
was observed, with patients in the 20 mg/kg dose group of SRP-5051
seeing a 1.6-fold increase in exon skipping (n=4) and a 5-fold
increase in the % of normal dystrophin (n=2) when compared to the
group taking eteplirsen at 24 weeks.
- Entered
into research collaboration with Genevant Sciences for lipid
nanoparticle (LNP) -based gene editing therapeutics: LNPs
offer the potential for a non-viral approach to gene editing and
can provide both optimal uptake into desired cells and efficient
release, resulting in functional delivery of gene editing cargo,
such as CRISPR-Cas, to target tissues. Genevant will design and
collaborate with Sarepta in the development of muscle targeted LNPs
to be applied to gene editing targets in early-stage development.
Sarepta has rights to an exclusive license to Genevant’s LNP
technology for up to four neuromuscular indications, including
Duchenne muscular dystrophy.
Conference CallThe Company will
be hosting a conference call at 4:30 p.m. Eastern Time to discuss
Sarepta’s financial results and provide a corporate update. The
conference call may be accessed by dialing (844) 534-7313 for
domestic callers and (574) 990-1451 for international callers. The
passcode for the call is 9672289. Please specify to the operator
that you would like to join the “Sarepta Fourth Quarter and
Full-Year 2020 Earnings Call.” The conference call will be webcast
live under the investor relations section of Sarepta's website at
www.sarepta.com and will be archived there following the call for
90 days. Please connect to Sarepta's website several minutes prior
to the start of the broadcast to ensure adequate time for any
software download that may be necessary.
Financial Results
On a GAAP basis, for the three months ended
December 31, 2020 and 2019, the Company reported a net loss of
$189.3 million and $235.7 million, or $2.40 and $3.16 per basic and
diluted share, respectively. On a non-GAAP basis, the net loss for
the three months ended December 31, 2020 and 2019 was $145.1
million and $116.9 million, or $1.84 and $1.57 per basic and
diluted share, respectively.
On a GAAP basis, for the twelve months ended
December 31, 2020 and 2019, the Company reported a net loss of
$554.1 million and $715.1 million, or $7.11 and $9.71 per basic and
diluted share, respectively. On a non-GAAP basis, the net loss for
the twelve months ended December 31, 2020 and 2019 was $454.3
million and $316.3 million, or $5.83 and $4.30 per basic and
diluted share, respectively.
Revenues
For the three months ended December 31, 2020,
the Company recorded net product revenues of $122.6 million,
compared to net product revenues of $100.1 million for the same
period of 2019, an increase of $22.5 million. For the twelve months
ended December 31, 2020, the Company recorded net product revenues
of $455.9 million, compared to net product revenues of $380.8
million for the twelve months ended December 31, 2019, an increase
of $75.1 million. The increase primarily reflects the continuing
increase in demand for EXONDYS 51 and the launch of VYONDYS 53 in
the U.S.
For the three and twelve months ended December
31, 2020, the Company recognized $22.5 million and $84.2 million of
collaboration revenue, respectively, which primarily relates to the
Company’s collaboration arrangement with F. Hoffman-La Roche Ltd.
(Roche). In February 2020, the Company received an aggregate of
approximately $1.2 billion in cash consideration from Roche,
consisting of an up-front payment for the rights granted to Roche
and pre-paid development costs, and an equity investment in the
Company. Of that amount, $348.7 million is recorded as deferred
revenue and is being recognized as collaboration revenue on a
straight-line basis over the performance period, estimated to be
through the fourth quarter of 2023. There was no such transaction
in 2019.
Cost and Operating Expenses
Cost of sales (excluding amortization of
in-licensed rights)
For the three months ended December 31, 2020,
cost of sales (excluding amortization of in-licensed rights) was
$22.4 million, compared to $15.6 million for the same period of
2019, an increase of $6.8 million. For the twelve months ended
December 31, 2020, cost of sales (excluding amortization of
in-licensed rights) was approximately $63.4 million, compared to
$56.6 million for the same period of 2019, an increase of $6.8
million. The increase in cost of sales is primarily due to an
increase in royalty payments to BioMarin Pharmaceutical (BioMarin)
and University of Western Australia (UWA) that reflects increasing
demand for the Company’s products.
Research and development
Research and development expenses were $207.2
million for the three months ended December 31, 2020, compared to
$223.1 million for the same period of 2019, a decrease of $15.9
million. The decrease in research and development expenses
primarily reflects the following:
- $58.1 million increase in manufacturing expenses primarily due
to a continuing ramp-up of the Company’s micro-dystrophin gene
therapy program as well as other gene therapy programs in the
Company’s pipeline;
- $9.8 million increase in clinical trial expenses primarily due
to increased patient enrollment for the Company’s ESSENCE program
as well as certain start-up activities for the Company’s
micro-dystrophin program;
- $2.6 million increase in facility- and technology-related
expenses due to the Company’s continuing global expansion
efforts;
- $2.2 million increase in compensation and other personnel
expenses primarily due to a net increase in headcount;
- $2.2 million increase in pre-clinical expenses primarily due to
an increase of toxicology studies in the Company’s gene therapy
platforms during the fourth quarter of 2020, offset by the
completion of certain toxicology studies in the Company’s PPMO
platform;
- $1.9 million increase in stock-based compensation expense
primarily driven by increases in headcount and stock price;
- $1.4 million decrease in professional service expenses
primarily due to a decrease in reliance on third-party research and
development contractors as a result of an increase in hiring and
headcount;
- $5.8 million decrease in collaboration cost sharing with
Genethon on its micro-dystrophin drug candidates and Lysogene on
its MPS IIIA drug candidate;
- $64.2 million decrease in up-front, milestone and other
expenses primarily due to a $46.9 million up-front payment to
StrideBio, Inc. (StrideBio) and a $28.0 million up-front payment to
Genethon as a result of the execution of a license and
collaboration agreement with each company, respectively, in the
fourth quarter of 2019. This was offset primarily by $10.6 million
of up-front payments as a result of the execution of certain
research, option and license agreements during the fourth quarter
of 2020; and
- $24.0 million offset to expense incurred in the fourth quarter
of 2020 associated with a collaboration reimbursement from Roche
related to the micro-dystrophin project.
Research and development expenses were $722.3
million for the twelve months ended December 31, 2020, compared to
$560.9 million for the same period of 2019, an increase of $161.4
million. The increase in research and development expenses
primarily reflects the following:
- $223.9 million increase in manufacturing expenses primarily due
to a continuing ramp-up of the Company’s micro-dystrophin gene
therapy program as well as other gene therapy programs in the
Company’s pipeline;
- $17.5 million increase in compensation and other personnel
expenses primarily due to a net increase in headcount;
- $16.1 million increase in clinical expenses primarily due to
increased patient enrollment in the Company’s ESSENCE program as
well as certain start-up activities for the Company’s
micro-dystrophin program;
- $14.0 million increase in stock-based compensation expense
primarily driven by increases in headcount and stock price;
- $8.8 million increase in facility- and technology-related
expenses due to the Company’s continuing global expansion
efforts;
- $5.4 million increase in research and other primarily driven by
an increase in sponsored research with academic institutions during
2020;
- $3.7 million increase in collaboration cost-sharing with
Genethon on its micro-dystrophin drug candidates and Lysogene on
its MPS IIIA drug candidate;
- $1.6 million decrease in pre-clinical expenses primarily due to
completion of certain toxicology studies in the Company’s PPMO
platform, offset by an increase of toxicology studies in the
Company’s gene therapy programs;
- $4.6 million decrease in professional services primarily due to
a decrease in reliance on third-party research and development
contractors as a result of an increase in hiring and
headcount;
- $55.9 million decrease in up-front, milestone, and other
expenses, primarily due to $9.3 million of milestone expense
related to payments accrued to an academic institution and $38.0
million of up-front payments as a result of the execution of
certain research, option and license agreements during 2020. This
was offset primarily by $46.9 million of up-front payments to
StrideBio and a $28.0 million up-front payment to Genethon as a
result of the execution of a license and collaboration agreement
with each company, respectively, and $25.6 million of up-front and
milestone payments made to various academic institutions throughout
2019; and
- $65.9 million offset to expense incurred during 2020 associated
with a collaboration reimbursement from Roche related to the
micro-dystrophin project.
Non-GAAP research and development expenses were
$180.8 million and $135.4 million for the three months ended
December 31, 2020 and 2019, respectively, an increase of $45.4
million. Non-GAAP research and development expenses were $615.3
million and $414.8 million for the twelve months ended December 31,
2020 and 2019, respectively, an increase of $200.5 million.
Selling, general and administration
Selling general and administrative expenses were
approximately $86.0 million for the three months ended December 31,
2020, compared to $81.4 million for the same period in 2019, an
increase of $4.6 million. The increase in selling, general and
administrative expenses primarily reflects the following:
- $5.5 million
increase in stock-based compensation partially due to increases in
headcount and stock price;
- $3.9 million
increase in professional services primarily due to continuing
global expansion; and
- $3.6 million
decrease in compensation and other personnel expenses primarily due
to a decrease in bonus expense.
Selling general and administrative expenses were
$317.9 million for the twelve months ended December 31, 2020,
compared to $284.8 million for the same period of 2019, an increase
of $33.1 million. The increase in selling, general and
administrative expenses primarily reflects the following:
- $15.5 million increase in stock-based compensation primarily
due to increases in headcount and stock price; and
- $15.2 million and $1.4 million increases in professional
services and facility- and technology-related expense,
respectively, both of which primarily due to continuing global
expansion.
Non-GAAP selling, general and administrative
expenses were $65.2 million and $65.8 million for the three months
ended December 31, 2020 and 2019, respectively, a decrease of $0.6
million. Non-GAAP selling, general and administrative expenses were
$232.0 million and $225.5 million for the twelve months ended
December 31, 2020 and 2019, respectively, an increase of $6.5
million.
Acquired in-process research and development
As a result of the Myonexus acquisition, the
Company recorded acquired in-process research and development
expense of approximately $173.2 million during the second quarter
of 2019. There was no such transaction during 2020.
Settlement and license charges
In December 2019, the Company recognized a $10.0
million settlement charge related to contingent settlement payments
to BioMarin as a result of the approval of VYONDYS 53. This was a
result of a settlement and license agreement with BioMarin in July
2017. There was no such expense recognized during 2020.
Amortization of in-licensed rights
For each of the three months ended December 31,
2020 and 2019, the Company recorded amortization of in-licensed
rights of approximately $0.2 million. For the twelve months ended
December 31, 2020 and 2019, the Company recorded amortization of
in-licensed rights of approximately $0.7 million and $0.8 million,
respectively. This is related to the amortization of the
in-licensed right assets recognized as a result of agreements the
Company entered into with BioMarin and UWA.
Gain from sale of Priority Review Voucher
In February 2020, the Company entered into an
agreement with Vifor (International) Ltd. to sell the rare
pediatric disease Priority Review Voucher (“PRV”) it received from
the FDA in connection with the approval of VYONDYS 53. Following
the early termination of the applicable waiting period under the
Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended,
in March 2020, the Company completed its sale of the PRV and
received proceeds of $108.1 million, net of commission, which was
recorded as a gain from sale of the PRV as it did not have a
carrying value at the time of the sale. There was no similar
activity during 2019.
Loss on contingent consideration
The loss on contingent consideration relates to
the fair value adjustment of the Company’s contingent consideration
liability related to regulatory-related contingent payments to
Myonexus selling shareholders as well as to an academic institution
under separate license agreements that meet the definition of a
derivative. For the year ended December 31, 2020, the Company
recognized $45.0 million of expense to adjust the fair value of the
contingent consideration liabilities based on the most current
assumptions relating to the achievement of the milestones. There
was no similar activity during 2019.
Other expense, net
For the three months and twelve months ended
December 31, 2020, other expense, net was approximately $17.8
million and $52.0 million, respectively. For the three and twelve
months ended December 31, 2019, other expense, net was
approximately $4.8 million and $8.3 million, respectively. The
increases for both periods primarily reflect the interest expense
on the Company’s debt facilities as well as a decrease in interest
income and the amortization of investment discounts due to the
investment mix of the Company’s investment portfolio.
Cash, Cash Equivalents, Investments and
Restricted Cash and Investments
The Company had approximately $1.9 billion in
cash, cash equivalents and investments as of December 31, 2020,
compared to $1.1 billion as of December 31, 2019. The increase is
primarily driven by the $1.2 billion of up-front payments received
from the Roche as a result of the execution of the collaboration
and equity investment agreements offset by cash used to fund the
Company’s ongoing operations during 2020.
Use of Non-GAAP Measures
In addition to the GAAP financial measures set
forth in this press release, the Company has included certain
non-GAAP measurements. The non-GAAP loss is defined by the Company
as GAAP net loss excluding interest expense, net, income tax
expense, depreciation and amortization expense, stock-based
compensation expense and other items. Non-GAAP research and
development expenses are defined by the Company as GAAP research
and development expenses excluding depreciation and amortization
expense, stock-based compensation expense and other items. Non-GAAP
selling, general and administrative expenses are defined by the
Company as GAAP selling, general and administrative expenses
excluding depreciation and amortization expense, stock-based
compensation expense and other items.
1. Interest, tax, depreciation and
amortization
Interest expense, net amounts can vary
substantially from period to period due to changes in cash and debt
balances and interest rates driven by market conditions outside of
the Company’s operations. Tax amounts can vary substantially from
period to period due to tax adjustments that are not directly
related to underlying operating performance. Depreciation expense
can vary substantially from period to period as the purchases of
property and equipment may vary significantly from period to period
and without any direct correlation to the Company’s operating
performance. Amortization expense primarily associated with
in-licensed rights as well as patent costs are amortized over a
period of several years after acquisition or patent application or
renewal and generally cannot be changed or influenced by
management.
2. Stock-based compensation expenses
Stock-based compensation expenses represent
non-cash charges related to equity awards granted by the Company.
Although these are recurring charges to operations, the Company
believes the measurement of these amounts can vary substantially
from period to period and depend significantly on factors that are
not a direct consequence of operating performance that is within
the Company’s control. Therefore, the Company believes that
excluding these charges facilitates comparisons of the Company’s
operational performance in different periods.
3. Other items
The Company evaluates other items of expense and
income on an individual basis. It takes into consideration
quantitative and qualitative characteristics of each item,
including (a) nature, (b) whether the items relate to the Company’s
ongoing business operations, and (c) whether the Company expects
the items to continue on a regular basis. These other items include
collaboration revenue and transaction cost related to the Roche
transaction, up-front and milestone payments, acquired in-process
research and development expense, gain from sale of PRV and loss on
contingent consideration.
The Company excludes collaboration revenue and
transaction cost associated with the Roche transaction from its
non-GAAP results. While collaboration revenue is recurring, as the
Company’s ordinary activities do not include contracting with third
parties to provide them with research and development services,
collaboration revenue is treated as a non-GAAP adjustment item.
Additionally, the transaction fee related to the Roche transaction
is non-recurring and is excluded from its non-GAAP results.
However, the Company does not exclude reimbursement of costs by
Roche from its non-GAAP results.
The Company excludes up-front, milestone, and
acquired in-process research and development expenses associated
with its license and collaboration agreements from its non-GAAP
results and research and development expenses because the Company
does not consider them to be normal operating expenses due to their
nature, variability of amounts, and lack of predictability as to
occurrence and/or timing. Up-front payments are made at the
commencement of a collaborative relationship or a license agreement
anticipated to continue for a multi-year period and provide the
Company with intellectual property rights, option rights and other
rights with respect to particular programs. Milestone payments are
made when certain development, regulatory and sales milestone
events are achieved. The variability of amounts and lack of
predictability of collaboration- and license-related up-front and
milestone payment makes the identification of trends in the
Company’s ongoing research and development activities more
difficult.
As a result of the Myonexus acquisition, the
Company recorded acquired in-process research and development
expense, which represents a non-recurring expense and, therefore,
was treated as a non-GAAP adjustment item. The Company believes the
presentation of adjusted research and development, which does not
include license- and collaboration-related up-front and milestone
expenses, provides useful and meaningful information about its
ongoing research and development activities by enhancing investors’
understanding of the Company’s normal, recurring operating research
and development expenses and facilitates comparisons between
periods and with respect to projected performance.
The sale of the PRV obtained as a result of the
FDA approval of VYONDYS 53 in December 2019 is a non-recurring
event and excluded from the Company’s non-GAAP results.
The Company excludes from its non-GAAP results
loss on contingent consideration related to the Company’s
acquisition of Myonexus in 2019 as it is a non-cash item and is not
considered to be normal operating expenses due to its variability
of amounts and lack of predictability as to occurrence and/or
timing.
The Company uses these non-GAAP measures as key
performance measures for the purpose of evaluating operational
performance and cash requirements internally. The Company also
believes these non-GAAP measures increase comparability of
period-to-period results and are useful to investors as they
provide a similar basis for evaluating the Company’s performance as
is applied by management. These non-GAAP measures are not intended
to be considered in isolation or to replace the presentation of the
Company’s financial results in accordance with GAAP. Use of the
terms non-GAAP research and development expenses, non-GAAP selling,
general and administrative expenses, non-GAAP other income and loss
adjustments, non-GAAP income tax expense, non-GAAP net loss, and
non-GAAP basic and diluted net loss per share may differ from
similar measures reported by other companies, which may limit
comparability, and are not based on any comprehensive set of
accounting rules or principles. All relevant non-GAAP measures are
reconciled from their respective GAAP measures in the attached
table "Reconciliation of GAAP Financial Measures to Non-GAAP
Financial Measures.”
About EXONDYS 51
EXONDYS 51 uses Sarepta’s proprietary
phosphorodiamidate morpholino oligomer (PMO) chemistry and
exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA,
resulting in “skipping” of this exon during mRNA processing in
patients with genetic mutations that are amenable to exon 51
skipping. Exon skipping is intended to allow for production of an
internally truncated dystrophin protein.
EXONDYS 51 is indicated for the treatment of
Duchenne muscular dystrophy in patients who have a confirmed
mutation of the DMD gene that is amenable to exon 51 skipping.
This indication is approved under accelerated
approval based on an increase in dystrophin production in skeletal
muscle observed in some patients treated with EXONDYS 51. Continued
approval may be contingent upon verification of a clinical benefit
in confirmatory trials.
EXONDYS 51 has met the full statutory standards
for safety and effectiveness and as such is not considered
investigational or experimental.
Important Safety Information About
EXONDYS 51
Hypersensitivity reactions, including rash and
urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and
hypotension, have occurred in patients who were treated with
EXONDYS 51. If a hypersensitivity reaction occurs, institute
appropriate medical treatment and consider slowing the infusion or
interrupting the EXONDYS 51 therapy.
Adverse reactions in DMD patients (N=8) treated
with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion
with an incidence of at least 25% more than placebo (N=4) (Study 1,
24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%),
vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most
common adverse reactions were balance disorder and vomiting.
Because of the small numbers of patients, these represent crude
frequencies that may not reflect the frequencies observed in
practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is
not recommended.
In the 88 patients who received ≥30 mg/kg/week
of EXONDYS 51 for up to 208 weeks in clinical studies, the
following events were reported in ≥10% of patients and occurred
more frequently than on the same dose in Study 1: vomiting,
contusion, excoriation, arthralgia, rash, catheter site pain, and
upper respiratory tract infection.
For further information, please see the full
Prescribing Information.
About VYONDYS 53 VYONDYS 53
uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer
(PMO) chemistry and exon-skipping technology to bind to exon 53 of
dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this
exon during mRNA processing in patients with genetic mutations that
are amenable to exon 53 skipping. Exon skipping is intended to
allow for production of an internally truncated dystrophin
protein.
VYONDYS 53 is indicated for the treatment of
Duchenne muscular dystrophy in patients who have a confirmed
mutation of the DMD gene that is amenable to exon 53 skipping.
This indication is approved under accelerated
approval based on an increase in dystrophin production in skeletal
muscle observed in patients treated with VYONDYS 53. Continued
approval may be contingent upon verification of a clinical benefit
in confirmatory trials.
VYONDYS 53 has met the full statutory standards
for safety and effectiveness and as such is not considered
investigational or experimental.
Important Safety Information for VYONDYS
53Hypersensitivity reactions, including rash, pyrexia,
pruritus, urticaria, dermatitis, and skin exfoliation have occurred
in VYONDYS 53-treated patients, some requiring treatment. If a
hypersensitivity reaction occurs, institute appropriate medical
treatment and consider slowing the infusion or interrupting the
VYONDYS 53 therapy.
Kidney toxicity was observed in animals who
received golodirsen. Although kidney toxicity was not observed in
the clinical studies with VYONDYS 53, the clinical experience with
VYONDYS 53 is limited, and kidney toxicity, including potentially
fatal glomerulonephritis, has been observed after administration of
some antisense oligonucleotides. Kidney function should be
monitored in patients taking VYONDYS 53. Because of the effect of
reduced skeletal muscle mass on creatinine measurements, creatinine
may not be a reliable measure of kidney function in DMD patients.
Serum cystatin C, urine dipstick, and urine protein-to-creatinine
ratio should be measured before starting VYONDYS 53. Consider also
measuring glomerular filtration rate using an exogenous filtration
marker before starting VYONDYS 53. During treatment, monitor urine
dipstick every month, and serum cystatin C and urine
protein-to-creatinine ratio every three months. Only urine expected
to be free of excreted VYONDYS 53 should be used for monitoring of
urine protein. Urine obtained on the day of VYONDYS 53 infusion
prior to the infusion, or urine obtained at least 48 hours after
the most recent infusion, may be used. Alternatively, use a
laboratory test that does not use the reagent pyrogallol red, as
this reagent has the potential to cross react with any VYONDYS 53
that is excreted in the urine and thus lead to a false positive
result for urine protein.
If a persistent increase in serum cystatin C or
proteinuria is detected, refer to a pediatric nephrologist for
further evaluation.
Adverse reactions observed in at least 20% of
treated patients and greater than placebo were (VYONDYS 53,
placebo): headache (41%, 10%), pyrexia (41%, 14%), fall (29%, 19%),
abdominal pain (27%, 10%), nasopharyngitis (27%, 14%), cough (27%,
19%), vomiting (27%, 19%), and nausea (20%, 10%).
Other adverse reactions that occurred at a
frequency greater than 5% of VYONDYS 53-treated patients and at a
greater frequency than placebo were: administration site pain, back
pain, pain, diarrhea, dizziness, ligament sprain, contusion,
influenza, oropharyngeal pain, rhinitis, skin abrasion, ear
infection, seasonal allergy, tachycardia, catheter site related
reaction, constipation, and fracture.
For further information, please see the full
Prescribing Information.
About AMONDYS 45AMONDYS 45
(casimersen) is an antisense oligonucleotide indicated for the
treatment of Duchenne muscular dystrophy in patients who have a
confirmed mutation of the DMD gene that is amenable to exon 45
skipping. AMONDYS 45 uses Sarepta’s proprietary phosphorodiamidate
morpholino oligomer (PMO) chemistry and exon-skipping technology to
bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or
“skipping,” of this exon during mRNA processing in patients with
genetic mutations that are amenable to exon 45 skipping. Exon
skipping is intended to allow for production of an internally
truncated dystrophin protein.
AMONDYS 45 is approved under accelerated review
based on an increase in dystrophin production in skeletal muscle of
patients amenable to exon 45 skipping. Continued approval may be
contingent upon verification of a clinical benefit in confirmatory
trials.
AMONDYS 45 has met the full statutory standards
for safety and effectiveness and as such is not considered
investigational or experimental.
Important Safety Information for AMONDYS
45Kidney toxicity was observed in animals who received
casimersen. Although kidney toxicity was not observed in the
clinical studies with AMONDYS 45, kidney toxicity, including
potentially fatal glomerulonephritis, has been observed after
administration of some antisense oligonucleotides. Kidney function
should be monitored in patients taking AMONDYS 45. Because of the
effect of reduced skeletal muscle mass on creatinine measurements,
creatinine may not be a reliable measure of kidney function in DMD
patients. Serum cystatin C, urine dipstick, and urine
protein-to-creatinine ratio should be measured before starting
AMONDYS 45. Consider also measuring glomerular filtration rate
using an exogenous filtration marker before starting AMONDYS 45.
During treatment, monitor urine dipstick every month, and serum
cystatin C and urine protein-to-creatinine ratio (UPCR) every three
months. Only urine expected to be free of excreted AMONDYS 45
should be used for monitoring of urine protein. Urine obtained on
the day of AMONDYS 45 infusion prior to the infusion, or urine
obtained at least 48 hours after the most recent infusion, may be
used. Alternatively, use a laboratory test that does not use the
reagent pyrogallol red, as this reagent has the potential to cross
react with any AMONDYS 45 that is excreted in the urine and thus
lead to a false positive result for urine protein.
If a persistent increase in serum cystatin C or
proteinuria is detected, refer to a pediatric nephrologist for
further evaluation.
Adverse reactions observed in at least 20% of
patients treated with AMONDYS 45 and at least 5% more frequently
than in the placebo group were (AMONDYS 45, placebo): upper
respiratory tract infections (65%, 55%), cough (33%, 26%), pyrexia
(33%, 23%), headache (32%, 19%), arthralgia (21%, 10%), and
oropharyngeal pain (21%, 7%).
Other adverse reactions that occurred in at
least 10% of patients treated with AMONDYS 45 and at least 5% more
frequently in the placebo group, were: ear pain, nausea, ear
infection, post-traumatic pain, and dizziness and
light-headedness.
For further information, please see the full
Prescribing Information.
About Sarepta Therapeutics
At Sarepta, we are leading a revolution in
precision genetic medicine and every day is an opportunity to
change the lives of people living with rare disease. The Company
has built an impressive position in Duchenne muscular dystrophy
(DMD) and in gene therapies for limb-girdle muscular dystrophies
(LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth
(CMT), and other CNS-related disorders, with more than 40 programs
in various stages of development. The Company’s programs and
research focus span several therapeutic modalities, including RNA,
gene therapy and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Forward-Looking Statements In
order to provide Sarepta’s investors with an understanding of its
current results and future prospects, this press release contains
statements that are forward-looking. Any statements contained in
this press release that are not statements of historical fact may
be deemed to be forward-looking statements. Words such as
“believes,” “anticipates,” “plans,” “expects,” “will,” “may,”
“intends,” “prepares,” “looks,” “potential,” “possible” and similar
expressions are intended to identify forward-looking statements.
These forward-looking statements include statements relating to our
future operations, financial performance and projections, business
plans, market opportunities, priorities and research and
development programs including the potential of AMONDYS 45 to treat
8% of Duchenne patients with a confirmed exon 45 amendable
mutation; our goal of treating the greatest percentage of the
Duchenne community as possible; the potential of our three
therapies together to treat nearly 30% of Duchenne patients in the
U.S.; the potential of our RNA technology to build constructs to
treat over 80% of Duchenne mutations; the potentially
transformative benefits of SRP-9001; the potential benefits of our
collaboration with Genevant and the obligations, rights,
responsibilities, potential payments and fees under the
collaboration agreement; and expected plans and milestones,
including reporting data from Study SRP-9001-103 in Q2 2021,
reporting data from the 30 mg/kg arm of the MOMENTUM trial in Q2
2021, our plan to present data at the 2021 MDA Clinical and
Scientific Conference from SRP-9001-102 Part 1 and new long-term
functional data from study SRP-9003-101, and concluding our ESSENCE
trial in 2024.
These forward-looking statements involve risks
and uncertainties, many of which are beyond Sarepta’s control.
Actual results could materially differ from those stated or implied
by these forward-looking statements as a result of such risks and
uncertainties. Known risk factors include the following: the
commercial launch for AMONDYS 45 in the U.S. may not be successful
for various reasons, including the actual market size and drug
supply needed may not be consistent with the company’s
expectations, the degree to which AMONDYS 45 is accepted by
patients and prescribed by physicians, manufacturing limitations,
and competitive, reimbursement and regulatory conditions that could
negatively impact the commercial launch of AMONDYS 45; we may not
be able to comply with all FDA post-approval commitments and
requirements with respect to our products in a timely manner or at
all; our dependence on certain manufacturers to produce our
products and product candidates, including any inability on our
part to accurately anticipate product demand and timely secure
manufacturing capacity to meet product demand, may impair the
availability of product to successfully support various programs;
success in preclinical testing and early clinical trials,
especially if based on a small patient sample, does not ensure that
later clinical trials will be successful, and the results of future
research may not be consistent with past positive results or may
fail to meet regulatory approval requirements for the safety and
efficacy of product candidates; the expected benefits and
opportunities related to our agreements with our strategic partners
may not be realized or may take longer to realize than expected due
to a variety of reasons, including any inability of the parties to
perform their commitments and obligations under the agreements,
challenges and uncertainties inherent in product research and
development and manufacturing limitations; if the actual number of
patients suffering from the diseases we aim to treat is smaller
than estimated, our revenue and ability to achieve profitability
may be adversely affected; we may not be able to execute on our
business plans, including meeting our expected or planned
regulatory milestones and timelines, research and clinical
development plans, and bringing our product candidates to market,
for various reasons, some of which may be outside of our control,
including possible limitations of company financial and other
resources, manufacturing limitations that may not be anticipated or
resolved for in a timely manner, the COVID-19 pandemic and
regulatory, court or agency decisions, such as decisions by the
United States Patent and Trademark Office with respect to patents
that cover our product candidates; and those risks identified under
the heading “Risk Factors” in our most recent Annual Report on Form
10-K for the year ended December 31, 2020 filed with
the Securities and Exchange Commission (SEC) as well as
other SEC filings made by the Company which you are
encouraged to review.
Internet Posting of
Information
We routinely post information that may be important to investors
in the 'For Investors' section of our website
at www.sarepta.com. We encourage investors and potential
investors to consult our website regularly for important
information about us.
|
|
|
|
Sarepta Therapeutics, Inc.Condensed Consolidated Statements of
Operations(unaudited, in thousands, except per share amounts) |
|
|
|
|
|
For the Three Months EndedDecember
31, |
|
|
For the Twelve Months EndedDecember
31, |
|
|
|
2020 |
|
|
2019 |
|
|
2020 |
|
|
2019 |
|
Revenues: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Products, net |
|
$ |
122,644 |
|
|
$ |
100,113 |
|
|
$ |
455,865 |
|
|
$ |
380,833 |
|
Collaboration |
|
|
22,494 |
|
|
|
— |
|
|
|
84,234 |
|
|
|
— |
|
Total revenues |
|
|
145,138 |
|
|
|
100,113 |
|
|
|
540,099 |
|
|
|
380,833 |
|
Cost and expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cost of sales (excluding amortization of in-licensed rights) |
|
|
22,404 |
|
|
|
15,567 |
|
|
|
63,382 |
|
|
|
56,586 |
|
Research and development |
|
|
207,239 |
|
|
|
223,141 |
|
|
|
722,343 |
|
|
|
560,909 |
|
Selling, general and administrative |
|
|
86,046 |
|
|
|
81,424 |
|
|
|
317,875 |
|
|
|
284,812 |
|
Acquired in-process research and development |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
173,240 |
|
Settlement and license charges |
|
|
— |
|
|
|
10,000 |
|
|
|
— |
|
|
|
10,000 |
|
Amortization of in-licensed rights |
|
|
165 |
|
|
|
200 |
|
|
|
662 |
|
|
|
849 |
|
Total cost and expenses |
|
|
315,854 |
|
|
|
330,332 |
|
|
|
1,104,262 |
|
|
|
1,086,396 |
|
Operating loss |
|
|
(170,716 |
) |
|
|
(230,219 |
) |
|
|
(564,163 |
) |
|
|
(705,563 |
) |
Other (loss) income: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Gain from sale of Priority Review Voucher |
|
|
— |
|
|
|
— |
|
|
|
108,069 |
|
|
|
— |
|
Loss on contingent consideration |
|
|
— |
|
|
|
— |
|
|
|
(45,000 |
) |
|
|
— |
|
Other expense, net |
|
|
(17,769 |
) |
|
|
(4,773 |
) |
|
|
(51,971 |
) |
|
|
(8,317 |
) |
Total other (loss) income |
|
|
(17,769 |
) |
|
|
(4,773 |
) |
|
|
11,098 |
|
|
|
(8,317 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss before income tax
expense |
|
|
(188,485 |
) |
|
|
(234,992 |
) |
|
|
(553,065 |
) |
|
|
(713,880 |
) |
Income tax expense |
|
|
832 |
|
|
|
711 |
|
|
|
1,063 |
|
|
|
1,195 |
|
Net loss |
|
$ |
(189,317 |
) |
|
$ |
(235,703 |
) |
|
$ |
(554,128 |
) |
|
$ |
(715,075 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share - basic and
diluted |
|
$ |
(2.40 |
) |
|
$ |
(3.16 |
) |
|
$ |
(7.11 |
) |
|
$ |
(9.71 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average number of
shares of common stock |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
used in computing basic and diluted net loss per share |
|
|
78,905 |
|
|
|
74,557 |
|
|
|
77,956 |
|
|
|
73,615 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Sarepta Therapeutics, Inc. |
|
Reconciliation of GAAP Financial Measures to Non-GAAP Financial
Measures |
|
(unaudited, in thousands, except per share amounts) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
For the Three Months EndedDecember
31, |
|
|
For the Twelve Months EndedDecember
31, |
|
|
|
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
GAAP net loss |
|
$ |
(189,317 |
) |
|
$ |
(235,703 |
) |
|
$ |
(554,128 |
) |
|
$ |
(715,075 |
) |
Interest expense, net |
|
|
18,446 |
|
|
|
4,562 |
|
|
|
52,488 |
|
|
|
8,081 |
|
Income tax expense |
|
|
832 |
|
|
|
711 |
|
|
|
1,063 |
|
|
|
1,195 |
|
Gain from sale of Priority Review Voucher |
|
|
— |
|
|
|
— |
|
|
|
(108,069 |
) |
|
|
— |
|
Loss on contingent consideration |
|
|
— |
|
|
|
— |
|
|
|
45,000 |
|
|
|
— |
|
Collaboration revenue |
|
|
(22,494 |
) |
|
|
— |
|
|
|
(84,234 |
) |
|
|
— |
|
Depreciation and amortization expense |
|
|
7,288 |
|
|
|
6,646 |
|
|
|
26,911 |
|
|
|
24,500 |
|
Stock-based compensation expense |
|
|
29,527 |
|
|
|
22,064 |
|
|
|
108,070 |
|
|
|
78,602 |
|
Roche transaction costs |
|
|
— |
|
|
|
— |
|
|
|
11,292 |
|
|
|
— |
|
Up-front, milestone, and other expenses |
|
|
10,622 |
|
|
|
74,816 |
|
|
|
47,280 |
|
|
|
103,162 |
|
Settlement and license charges |
|
|
— |
|
|
|
10,000 |
|
|
|
— |
|
|
|
10,000 |
|
Acquired in-process research and development |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
173,240 |
|
Non-GAAP net loss |
|
$ |
(145,096 |
) |
|
$ |
(116,904 |
) |
|
$ |
(454,327 |
) |
|
$ |
(316,295 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Non-GAAP net loss per
share: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted |
|
$ |
(1.84 |
) |
|
$ |
(1.57 |
) |
|
$ |
(5.83 |
) |
|
$ |
(4.30 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average number of
shares of common stock |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
used in computing basic and
diluted net loss per share |
|
|
78,905 |
|
|
|
74,557 |
|
|
|
77,956 |
|
|
|
73,615 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
For the Three Months EndedDecember
31, |
|
|
For the Twelve Months EndedDecember
31, |
|
|
|
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
GAAP research and development
expenses |
|
$ |
207,239 |
|
|
$ |
223,141 |
|
|
$ |
722,343 |
|
|
$ |
560,909 |
|
Up-front, milestone, and other expenses |
|
|
(10,622 |
) |
|
|
(74,816 |
) |
|
|
(47,280 |
) |
|
|
(103,162 |
) |
Stock-based compensation expense |
|
|
(10,637 |
) |
|
|
(8,699 |
) |
|
|
(41,671 |
) |
|
|
(27,681 |
) |
Depreciation and amortization expense |
|
|
(5,162 |
) |
|
|
(4,188 |
) |
|
|
(18,054 |
) |
|
|
(15,240 |
) |
Non-GAAP research and
development expenses |
|
$ |
180,818 |
|
|
$ |
135,438 |
|
|
$ |
615,338 |
|
|
$ |
414,826 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
For the Three Months EndedDecember
31, |
|
|
For the Twelve Months EndedDecember
31, |
|
|
|
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
GAAP selling, general and
administrative expenses |
|
$ |
86,046 |
|
|
$ |
81,424 |
|
|
$ |
317,875 |
|
|
$ |
284,812 |
|
Stock-based compensation expense |
|
|
(18,890 |
) |
|
|
(13,365 |
) |
|
|
(66,399 |
) |
|
|
(50,921 |
) |
Depreciation and amortization expense |
|
|
(1,961 |
) |
|
|
(2,258 |
) |
|
|
(8,195 |
) |
|
|
(8,411 |
) |
Roche transaction costs |
|
|
— |
|
|
|
— |
|
|
|
(11,292 |
) |
|
|
— |
|
Non-GAAP selling, general and
administrative expenses |
|
$ |
65,195 |
|
|
$ |
65,801 |
|
|
$ |
231,989 |
|
|
$ |
225,480 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Sarepta Therapeutics, Inc.Condensed Consolidated Balance
Sheets(unaudited, in thousands, except share and per share
data) |
|
|
|
As of December 31, |
|
|
|
2020 |
|
|
2019 |
|
Assets |
|
|
|
|
|
|
|
|
Current assets: |
|
|
|
|
|
|
|
|
Cash and cash equivalents |
|
$ |
1,502,648 |
|
|
$ |
835,080 |
|
Short-term investments |
|
|
435,923 |
|
|
|
289,668 |
|
Accounts receivable |
|
|
101,340 |
|
|
|
90,879 |
|
Inventory |
|
|
231,961 |
|
|
|
171,379 |
|
Other current assets |
|
|
213,324 |
|
|
|
81,907 |
|
Total current assets |
|
|
2,485,196 |
|
|
|
1,468,913 |
|
Property and equipment,
net |
|
|
190,430 |
|
|
|
129,620 |
|
Intangible assets, net |
|
|
13,628 |
|
|
|
12,497 |
|
Right of use assets |
|
|
91,761 |
|
|
|
37,933 |
|
Other non-current assets |
|
|
203,703 |
|
|
|
173,859 |
|
Total assets |
|
$ |
2,984,718 |
|
|
$ |
1,822,822 |
|
|
|
|
|
|
|
|
|
|
Liabilities and
Stockholders’ Equity |
|
|
|
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
|
|
|
Accounts payable |
|
$ |
111,090 |
|
|
$ |
68,094 |
|
Accrued expenses |
|
|
193,553 |
|
|
|
185,527 |
|
Deferred revenue, current portion |
|
|
89,244 |
|
|
|
3,303 |
|
Other current liabilities |
|
|
22,139 |
|
|
|
7,843 |
|
Total current liabilities |
|
|
416,026 |
|
|
|
264,767 |
|
Long-term debt |
|
|
992,493 |
|
|
|
681,900 |
|
Lease liabilities, net of
current portion |
|
|
80,367 |
|
|
|
47,720 |
|
Deferred revenue, net of
current portion |
|
|
663,488 |
|
|
|
— |
|
Contingent consideration |
|
|
50,800 |
|
|
|
5,200 |
|
Other non-current
liabilities |
|
|
19,785 |
|
|
|
5,048 |
|
Total liabilities |
|
|
2,222,959 |
|
|
|
1,004,635 |
|
Stockholders’ equity: |
|
|
|
|
|
|
|
|
Preferred stock, $0.0001 par
value, 3,333,333 shares authorized; none issued |
|
|
|
|
|
|
|
|
and outstanding |
|
|
— |
|
|
|
— |
|
Common stock, $0.0001 par
value, 198,000,000 shares authorized; |
|
|
|
|
|
|
|
|
79,374,247 and 75,184,863 issued and outstanding at December 31,
2020 and 2019, respectively |
|
|
8 |
|
|
|
8 |
|
Additional paid-in
capital |
|
|
3,609,877 |
|
|
|
3,112,130 |
|
Accumulated other
comprehensive income, net of tax |
|
|
3 |
|
|
|
50 |
|
Accumulated deficit |
|
|
(2,848,129 |
) |
|
|
(2,294,001 |
) |
Total stockholders’ equity |
|
|
761,759 |
|
|
|
818,187 |
|
Total liabilities and stockholders’ equity |
|
$ |
2,984,718 |
|
|
$ |
1,822,822 |
|
|
|
|
|
|
|
|
|
|
Source: Sarepta Therapeutics, Inc.
Sarepta Therapeutics, Inc.Investors:Ian Estepan, 617-274-4052,
iestepan@sarepta.com
Media:Tracy Sorrentino, 617-301-8566,
tsorrentino@sarepta.com
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