- ELEVIDYS net product revenues for the quarter totaled $69.1
million
- Total revenues, which consist of net product revenues and
collaboration revenues, for the third quarter 2023 totaled $331.8
million
- Net product revenues for the third quarter 2023 totaled
$309.3 million, a 49% increase over the same quarter of prior
year
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in
precision genetic medicine for rare diseases, today reported
financial results for the third quarter 2023.
“The third quarter was a defining moment for Sarepta. We
launched ELEVIDYS, our fourth therapy and the first gene therapy
for boys with Duchenne muscular dystrophy, we continued to drive
great performance of our three PMOs and importantly, on a non-GAAP
basis we have achieved profitability, placing us in ever more
rarified territory in biotech,” said Doug Ingram, president and
CEO, Sarepta Therapeutics. “Reflecting a superb launch, ELEVIDYS
net product revenue came in at $69.1 million. Total net product
revenue stands at $309.3 million, growing 49 percent over the same
quarter last year. And non-GAAP earnings stood at approximately
$38.0 million in the quarter, a major milestone for Sarepta.”
Third Quarter 2023 and Recent Development:
- Announced topline results from EMBARK, a global pivotal
study of ELEVIDYS gene therapy for Duchenne muscular dystrophy:
The topline results from EMBARK support the conclusion that
ELEVIDYS modifies the course of the disease in patients with
Duchenne. ELEVIDYS-treated patients showed an increase on the North
Star Ambulatory Assessment, a measure of motor function, compared
to placebo-treated patients at 52 weeks, although the primary
endpoint was not met. Robust, statistically significant results on
all key functional pre-specified secondary endpoints, including
time to rise (p=0.0025), and 10-meter walk test (p=0.0048),
demonstrated evidence of a clinically meaningful treatment benefit
that was similar in magnitude and statistical significance across
all age groups. No new safety signals were observed. Importantly,
we have shared the EMBARK topline results with Food and Drug
Administration (FDA) leadership and they have confirmed that, based
on the totality of the evidence, they are open to such label
expansion if supported by review of the data, and that they intend
to proceed rapidly with consideration of the submission.
Conference Call
The event will be webcast live under the investor relations
section of Sarepta's website at
https://investorrelations.sarepta.com/events-presentations and
following the event a replay will be archived there for one year.
Interested parties participating by phone will need to register
using this online form. After registering for dial-in
details, all phone participants will receive an auto-generated
e-mail containing a link to the dial-in number along with a
personal PIN number to use to access the event by phone.
Financial Results
For the three months ended September 30, 2023, the Company
reported a GAAP net loss of $40.9 million, or $0.46 per basic and
diluted share, compared to a GAAP net loss of $257.7 million
reported for the same period of 2022, or $2.94 per basic and
diluted share. The non-GAAP net income for the three months ended
September 30, 2023 was $37.7 million, or $0.37 per diluted share,
compared to a non-GAAP net loss of $70.0 million, or $0.80 per
diluted share for the same period of 2022.
For the nine months ended September 30, 2023, the Company
reported a GAAP net loss of $581.6 million, or $6.56 per basic and
diluted share, compared to a GAAP net loss of $594.2 million
reported for the same period of 2022, or $6.79 per basic and
diluted share. The non-GAAP net loss for the nine months ended
September 30, 2023 was $123.0 million, or $1.39 per diluted share,
compared to a non-GAAP net loss of $221.7 million, or $2.53 per
diluted share for the same period of 2022.
Revenues
For the three months ended September 30, 2023, the Company
recorded total revenues of $331.8 million, which consist of net
product revenues and collaboration revenues, compared to total
revenues of $230.3 million for the same period of 2022, an increase
of $101.5 million. For the nine months ended September 30, 2023,
the Company recorded total revenues of $846.6 million, compared to
total revenues of $674.6 million for the same period of 2022, an
increase of $172.0 million. The increase primarily reflects
increasing demand for EXONDYS 51, AMONDYS 45 and VYONDYS 53
(collectively, the “PMO Products”), as well as $69.1 million of net
product revenues associated with sales of ELEVIDYS during the three
and nine months ended September 30, 2023.
For the three months ended September 30, 2023, the Company
recorded net product revenues of $309.3 million, compared to net
product revenues of $207.8 million for the same period of 2022, an
increase of $101.5 million. For the nine months ended September 30,
2023, the Company recorded net product revenues of $779.8 million,
compared to net product revenues of $607.8 million for the same
period of 2022, an increase of $172.0 million. The increase
primarily reflects increasing demand for the PMO Products, as well
as $69.1 million of net product revenues associated with sales of
ELEVIDYS during the three and nine months ended September 30,
2023.
For both the three and nine months ended September 30, 2023 and
2022, the Company recognized $22.5 million and $66.8 million of
collaboration revenue, respectively. For all periods presented,
collaboration revenue primarily relates to the F. Hoffman-La Roche
Ltd. (Roche) collaboration arrangement.
Cost and Expenses
Cost of sales (excluding amortization of
in-licensed rights)
For the three months ended September 30, 2023, cost of sales
(excluding amortization of in-licensed rights) was $37.0 million,
compared to $40.0 million for the same period of 2022, a decrease
of $3.0 million. For the nine months ended September 30, 2023, cost
of sales (excluding amortization of in-licensed rights) was $106.2
million, compared to $109.2 million for the same period of 2022, a
decrease of $3.0 million. The decrease in the three months ended
September 30, 2023 primarily reflects write-offs of certain batches
of the Company's products not meeting the Company's quality
specifications in the three months ended September 30, 2022, with
no similar activity for the same period of 2023, partially offset
by an increasing demand for the Company's PMO Products. The change
for the nine months ended September 30, 2023 primarily reflects a
decrease in royalty payments during the nine months ended September
30, 2023 due to changes in the BioMarin Pharmaceuticals, Inc.
(BioMarin) royalty terms and a decrease in write-offs of certain
batches of the Company's products not meeting the Company's quality
specifications for the nine months ended September 30, 2023, as
compared to the same period of 2022, partially offset by an
increasing demand for the Company's PMO Products.
Research and development
Research and development expenses were $194.3 million for the
three months ended September 30, 2023, compared to $216.7 million
for the same period of 2022, a decrease of $22.4 million. The
decrease in research and development expenses primarily reflects
the following:
- $50.5 million decrease in manufacturing expenses primarily due
to lower purchases of raw material consumables in the three months
ended September 30, 2023, compared to the same period of 2022, as
well as the capitalization of commercial batches of ELEVIDYS
manufactured after its approval in June 2023;
- $1.7 million decrease in up-front, milestone and other expenses
primarily due to $5.0 million of up-front payments as a result of
the execution of certain research and license agreements and $0.5
million of expense incurred as a result of milestone achievements
in certain research and license agreements in the three months
ended September 30, 2022, partially offset by $3.8 million of
expense incurred as a result of the milestone achievements in
certain research and license agreements in the same period of
2023;
- $1.2 million increase in pre-clinical expenses primarily due to
an increase in toxicology study activity across multiple gene
therapy and RNA platforms;
- $1.9 million increase in research and other expenses primarily
driven by an increase in sponsored research with academic
institutions during the three months ended September 30, 2023 and
an increase in lab-related expenses as a result of changes in
headcount;
- $2.4 million increase in professional service expenses
primarily due to an increase in reliance on third-party research
and development contractors for clinical programs;
- $2.4 million increase in collaboration cost-sharing expenses
primarily due to the timing of expense incurred related to
Genethon's micro-dystrophin drug candidate;
- $5.7 million increase in facility- and technology-related
expenses primarily due to the Company’s continuing expansion
efforts;
- $6.3 million increase in compensation and other personnel
expenses primarily due to changes in headcount;
- $7.5 million increase in stock-based compensation expense
primarily due to the achievement of performance conditions related
to certain shares with performance conditions (PSUs), as well as
changes in headcount and the value of stock awards;
- $15.3 million increase in clinical trial expenses primarily due
to an increased patient enrollment and site activation for the
Company's MIS51ON, MOMENTUM and ENVISION programs; and
- $12.9 million increase in the offset to expense associated with
a collaboration reimbursement from Roche due to continuing
development of the Company’s SRP-9001 gene therapy programs.
Research and development expenses were $681.9 million for the
nine months ended September 30, 2023, compared to $663.3 million
for the same period of 2022, an increase of $18.6 million. The
increase in research and development expenses primarily reflects
the following:
- $39.4 million increase in clinical trial expenses primarily due
to an increased patient enrollment and site activation for the
Company's MIS51ON, MOMENTUM, ENVISION programs, as well as
additional PPMO and LGMD clinical trials;
- $31.0 million increase in compensation and other personnel
expenses primarily due to changes in headcount;
- $18.0 million increase in stock-based compensation expense
primarily due to changes in headcount and the value of stock
awards, as well as the achievement of performance conditions
related to certain PSUs as of the nine months ended September 30,
2023;
- $13.5 million increase in facility- and technology-related
expenses primarily due to the Company’s continuing expansion
efforts;
- $12.0 million increase in research and other expenses primarily
driven by an increase in sponsored research with academic
institutions during the nine months ended September 30, 2023 and an
increase in lab-related expenses as a result of changes in
headcount;
- $7.8 million increase in professional service expenses
primarily due to an increase in reliance on third-party research
and development contractors for the launch of ELEVIDYS prior to the
ELEVIDYS approval in June 2023 and for clinical programs;
- $3.5 million increase in collaboration cost-sharing expenses
primarily due to the timing of expense incurred related to
Genethon's micro-dystrophin drug candidate;
- $1.5 million increase in pre-clinical expenses primarily due to
an increase in toxicology study activity across multiple gene
therapy and PPMO platforms;
- $4.9 million decrease in up-front, milestone and other expenses
primarily due to $7.8 million of up-front payments as a result of
the execution of certain research and license agreements, $4.5
million of expense incurred as a result of milestone achievements
in certain research and license agreements and $4.5 million of
option and termination expenses in the nine months ended September
30, 2022, partially offset by $7.8 million of up-front payments as
a result of the execution of certain research and license
agreements and $3.9 million of expense incurred as a result of the
milestone achievements in certain research and license agreements
in the same period of 2023;
- $86.2 million decrease in manufacturing expenses primarily due
to lower purchases of raw material consumables in the nine months
ended September 30, 2023, compared to the same period of 2022, as
well as the capitalization of commercial batches of ELEVIDYS, the
$53.0 million shortfall payment accrual related to the gene therapy
manufacturing and supply agreement with Thermo Fisher Scientific,
Inc. and the $17.1 million termination charge related to the
manufacturing and supply agreement with Henogen SA, both of which
occurred in the nine months ended September 30, 2022, with no
similar activity in the same period of 2023. These amounts were
partially offset by a continuing ramp-up of SRP-9001 manufacturing
prior to the ELEVIDYS approval in June 2023 and continued
manufacturing of clinical batches of SRP-9001; and
- $17.0 million increase in the offset to expense associated with
a collaboration reimbursement from Roche due to continuing
development of the Company’s SRP-9001 gene therapy programs.
Non-GAAP research and development expenses were $163.9 million
and $193.7 million for the three months ended September 30, 2023
and 2022, respectively, a decrease of $29.8 million. Non-GAAP
research and development expenses were $596.8 million and $597.3
million for the nine months ended September 30, 2023 and 2022,
respectively, a decrease of $0.5 million.
Selling, general and
administrative
Selling, general and administrative expenses were $120.9 million
for the three months ended September 30, 2023, compared to $104.8
million for the same period in 2022, an increase of $16.1 million.
The increase in selling, general and administrative expenses
primarily reflects the following:
- $12.8 million increase in professional service expenses
primarily due to an increase in reliance on third-party selling,
general and administrative contractors for the launch of ELEVIDYS
and ongoing litigation matters;
- $6.6 million increase in compensation and other personnel
expenses primarily due to changes in headcount;
- $3.4 million increase in facility- and technology-related
expenses primarily due to the Company’s continuing expansion
efforts;
- $3.2 million increase in other expenses primarily due to
changes in charitable contribution activity; and
- $9.9 million decrease in stock-based compensation expense
primarily related to the Chief Executive Officer grant modification
agreement executed in 2022, partially offset by the achievement of
performance conditions related to certain PSUs, as well as changes
in headcount and the value of stock awards.
Selling, general and administrative expenses were $350.2 million
for the nine months ended September 30, 2023, compared to $330.9
million for the same period in 2022, an increase of $19.3 million.
The increase in selling, general and administrative expenses
primarily reflects the following:
- $42.6 million increase in professional service expenses
primarily due to an increase in reliance on third-party selling,
general and administrative contractors for the launch of ELEVIDYS
and ongoing litigation matters;
- $27.3 million increase in compensation and other personnel
expenses primarily due to changes in headcount;
- $7.3 million increase in facility- and technology-related
expenses primarily due to the Company’s continuing expansion
efforts;
- $6.1 million increase in other expenses primarily due to
changes in charitable contribution activity; and
- $63.8 million decrease in stock-based compensation expense
primarily related to the Chief Executive Officer grant modification
agreement executed in 2022, partially offset by the achievement of
performance conditions related to certain PSUs as of the nine
months ended September 30, 2023, as well as changes in headcount
and the value of stock awards.
Non-GAAP selling, general and administrative expenses were $92.8
million and $66.8 million for the three months ended September 30,
2023 and 2022, respectively, an increase of $26.0 million. Non-GAAP
selling, general and administrative expenses were $266.4 million
and $183.7 million for the nine months ended September 30, 2023 and
2022, respectively, an increase of $82.7 million.
Amortization of in-licensed
rights
For the three and nine months ended September 30, 2023, the
Company recorded amortization of in-licensed rights of
approximately $0.4 million and $0.8 million, respectively. For the
three and nine months ended September 30, 2022, the Company
recorded amortization of in-licensed rights of approximately $0.2
million and $0.5 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 the University of
Western Australia, Nationwide Children's Hospital, BioMarin and
Parent Project Muscular Dystrophy in April 2013, December 2016,
July 2017 and May 2018, respectively.
Gain from Sale of Priority Review
Voucher
In June 2023, the Company entered into an agreement to sell the
rare pediatric disease Priority Review Voucher (ELEVIDYS PRV) it
received from the FDA in connection with the approval of ELEVIDYS
for consideration of $102.0 million, with no commission costs. The
closing of the transaction was not subject to the conditions set
forth under the Hart-Scott-Rodino Antitrust Improvements Act of
1976, as amended, and closed during June 2023. The net proceeds
were recorded as a gain from sale of the ELEVIDYS PRV during the
nine months ended September 30, 2023 as it did not have a carrying
value at the time of the sale.
Loss on debt extinguishment
On November 14, 2017, the Company issued $570.0 million
aggregate principal amount of senior convertible notes due on
November 15, 2024 (2024 Notes). On March 2, 2023, the Company
entered into separate, privately negotiated exchange agreements
with certain holders of the outstanding 2024 Notes (Exchange
Agreements). The Exchange Agreements resulted in a conversion of
$313.5 million in aggregate principal value of the 2024 Notes held
by the holders (2024 Notes Conversion). In connection with the 2024
Notes Conversion, the Company issued approximately 4.5 million
shares of its common stock representing the agreed upon contractual
conversion rate under the Exchange Agreements. The conversion was
not pursuant to the conversion privileges included in the terms of
the debt at issuance and therefore was accounted for as a debt
extinguishment. The Company accounted for the debt extinguishment
by recognizing the difference between the fair value of the shares
of common stock transferred on the conversion date and the net
carrying amount of the extinguished debt as a loss on debt
extinguishment. The loss incurred on the extinguishment for the
nine months ended September 30, 2023 was $387.3 million, inclusive
of $6.9 million in third-party debt conversion costs.
Other income (expense), net
For the three months ended September 30, 2023 and 2022, other
expense, net was $12.3 million and other income, net was $0.4
million, respectively. The change was primarily due to the
impairment of equity investment and a loss on contingent
consideration, net, partially offset by increases in accretion of
investment discount, net and increases in interest income due to
the investment mix of the Company's investment portfolio, as well
as a reductions of interest expense incurred as a result of the
repayment of the Company's December 2019 Term Loan in 2022. For the
nine months ended September 30, 2023 and 2022, other income, net
was $17.3 million and other expense, net was $33.8 million,
respectively. The change was primarily due to increases in
accretion of investment discount, net and interest income due to
the investment mix of the Company's investment portfolio, as well
as a reductions of interest expense incurred as a result of the
repayment of the Company's December 2019 Term Loan in 2022,
partially offset by the impairment of equity investment and a loss
on contingent consideration, net incurred in the nine months ended
September 30, 2023.
Income tax expense
Income tax expense for the three and nine months ended September
30, 2023 was approximately $7.8 million and $21.2 million,
respectively. Income tax expense for the three and nine months
ended September 30, 2022 was $1.3 million and $5.6 million,
respectively. Income tax expense for the three and nine months
ended September 30, 2023 relates to state, foreign and federal
income taxes, while income tax expense for the three and nine
months ended September 30, 2022 relates to state and foreign income
taxes.
Cash, Cash Equivalents, Restricted Cash and
Investments
The Company had approximately $1.8 billion in cash, cash
equivalents, investments and long-term restricted cash as of
September 30, 2023, compared to $2.0 billion as of December 31,
2022. This decrease is driven by cash used to fund the Company’s
ongoing operations during 2023.
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 income (loss) is defined by the Company
as GAAP net loss excluding interest (income) 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 (income) 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 impairment
of equity investments, loss (gain) on contingent consideration,
net, gain from sale of the PRV and loss on debt extinguishment.
- The Company excludes from its non-GAAP results the impairment
of any equity investments as it is a non-cash item and is not
considered to be a normal operating expense due to the variability
of amount and lack of predictability as to the occurrence and/or
timing of such impairments.
- The Company excludes from its non-GAAP results the loss (gain)
on contingent consideration, net related to regulatory-related
contingent payments meeting the definition of a derivative to
Myonexus selling shareholders as well as to academic institutions
under separate license agreements 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 excludes from its non-GAAP results the gain from
sale of the PRV obtained as a result of the FDA approval of
ELEVIDYS in June 2023 as it is a non-recurring event and is not
indicative of the performance of the Company’s core operations,
which accordingly would make it difficult to compare the Company’s
results to peer companies that also provide non-GAAP
disclosures.
- The Company excludes from its non-GAAP results the loss on debt
extinguishment, which is considered to be a non-recurring event as
it is associated with a distinct financing decision and is not
indicative of the performance of the Company’s core operations,
which accordingly would make it difficult to compare the Company’s
results to peer companies that also provide non-GAAP
disclosures.
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 net income (loss), and non-GAAP diluted net
earnings (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 (eteplirsen) uses Sarepta’s proprietary
phosphorodiamidate morpholino oligomer (PMO) chemistry and
exon-skipping technology to bind to exon 51 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 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
dystrophin 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, bronchospasm, chest pain, cough,
tachycardia, and urticaria 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 Duchenne 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.
The following adverse reactions have been identified during
observational studies that were conducted as part of the clinical
development program and continued post approval.
In open-label observational studies, 163 patients received at
least one intravenous dose of EXONDYS 51, with doses ranging
between 0.5 mg/kg (0.017 times the recommended dosage) and 50 mg/kg
(1.7 times the recommended dosage). All patients were male and had
genetically confirmed Duchenne muscular dystrophy. Age at study
entry was 6 months to 19 years. Most (85%) patients were
Caucasian.
The most common adverse reactions from observational clinical
studies (N=163) seen in greater than 10% of the study population
were headache, cough, rash, and vomiting.
For further information, please see the full Prescribing
Information.
About VYONDYS 53
VYONDYS 53 (golodirsen) 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
dystrophin 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 53
Hypersensitivity 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 45
AMONDYS 45 (casimersen) 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 indicated for the treatment of Duchenne muscular
dystrophy in patients who have a confirmed mutation of the
dystrophin gene that is amenable to exon 45 skipping.
This indication is approved under accelerated approval based on
an increase in dystrophin production in skeletal muscle observed in
patients treated with AMONDYS 45. 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 45
CONTRAINDICATION:
Known hypersensitivity to casimersen or any of the inactive
ingredients. Instances of hypersensitivity including angioedema and
anaphylaxis have occurred.
WARNINGS AND PRECAUTIONS
Hypersensitivity: Hypersensitivity reactions, including
angioedema and anaphylaxis, have occurred in patients who were
treated with AMONDYS 45. If a hypersensitivity reaction occurs,
institute appropriate medical treatment, and consider slowing the
infusion, interrupting, or discontinuing the AMONDYS 45 infusion
and monitor until the condition resolves.
Kidney Toxicity: Kidney 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: Adverse reactions occurring 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 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
than 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 ELEVIDYS (delandistrogene moxeparvovec-rokl)
ELEVIDYS is indicated for the treatment of ambulatory pediatric
patients aged 4 through 5 years with Duchenne muscular dystrophy
(DMD) with a confirmed mutation in the DMD gene. This indication is
approved under accelerated approval based on expression of ELEVIDYS
micro-dystrophin observed in patients treated with ELEVIDYS.
Continued approval for this indication may be contingent upon
verification and description of clinical benefit in a confirmatory
trial(s).
ELEVIDYS has met the full statutory standards for safety and
effectiveness and as such is not considered investigational or
experimental.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATION:
ELEVIDYS is contraindicated in patients with any deletion in
exon 8 and/or exon 9 in the DMD gene.
WARNINGS AND PRECAUTIONS:
Acute Serious Liver Injury:
- Acute serious liver injury has been observed with ELEVIDYS.
Administration of ELEVIDYS may result in elevations of liver
enzymes (e.g., GGT, GLDH, ALT, AST) or total bilirubin, typically
seen within 8 weeks.
- Patients with preexisting liver impairment, chronic hepatic
condition, or acute liver disease (e.g., acute hepatic viral
infection) may be at higher risk of acute serious liver injury.
Postpone ELEVIDYS administration in patients with acute liver
disease until resolved or controlled.
- Prior to ELEVIDYS administration, perform liver enzyme test and
monitor liver function (clinical exam, GGT, and total bilirubin)
weekly for the first 3 months following ELEVIDYS infusion. Continue
monitoring if clinically indicated, until results are unremarkable
(normal clinical exam, GGT and total bilirubin levels return to
near baseline levels).
- Systemic corticosteroid treatment is recommended for patients
before and after ELEVIDYS infusion. Adjust corticosteroid regimen
when indicated. If acute serious liver injury is suspected, a
consultation with a specialist is recommended.
Immune-mediated Myositis:
- In clinical trials, immune-mediated myositis has been observed
approximately 1 month following ELEVIDYS infusion in patients with
deletion mutations involving exon 8 and/or exon 9 in the DMD gene.
Symptoms of severe muscle weakness including dysphagia, dyspnea and
hypophonia were observed.
- Limited data are available for ELEVIDYS treatment in patients
with mutations in the DMD gene between exons 1 to 17 and exons 59
to 71. Patients with deletions in these regions may be at risk for
a severe immune-mediated myositis reaction.
- Advise patients to contact a physician immediately if they
experience any unexplained increased muscle pain, tenderness, or
weakness, including dysphagia, dyspnea or hypophonia as these may
be symptoms of myositis. Consider additional immunomodulatory
treatment (immunosuppressants [e.g., calcineurin-inhibitor] in
addition to corticosteroids) based on patient’s clinical
presentation and medical history if these symptoms occur.
Myocarditis:
- Acute serious myocarditis and troponin-I elevations have been
observed following ELEVIDYS infusion in clinical trials.
- Monitor troponin-I before ELEVIDYS infusion and weekly for the
first month following infusion and continue monitoring if
clinically indicated. More frequent monitoring may be warranted in
the presence of cardiac symptoms, such as chest pain or shortness
of breath.
- Advise patients to contact a physician immediately if they
experience cardiac symptoms.
Pre-existing Immunity against AAVrh74:
- In AAV-vector based gene therapies, preexisting anti-AAV
antibodies may impede transgene expression at desired therapeutic
levels. Following treatment with ELEVIDYS, all subjects developed
anti-AAVrh74 antibodies.
- Perform baseline testing for the presence of anti-AAVrh74 total
binding antibodies prior to ELEVIDYS administration.
- ELEVIDYS administration is not recommended in patients with
elevated anti-AAVrh74 total binding antibody titers greater than or
equal to 1:400.
Adverse Reactions:
- The most common adverse reactions (incidence ≥ 5%) reported in
clinical studies were vomiting, nausea, liver function test
increased, pyrexia, and thrombocytopenia.
Sarepta is responsible for global development and manufacturing
for ELEVIDYS, and distribution within the U.S. will commence
immediately. In December 2019, Sarepta partnered with Roche to
accelerate access to ELEVIDYS for patients outside the United
States.
For further information, please see the full Prescribing
Information.
About Sarepta Therapeutics
Sarepta is on an urgent mission: engineer precision genetic
medicine for rare diseases that devastate lives and cut futures
short. We hold leadership positions in Duchenne muscular dystrophy
(Duchenne) and limb-girdle muscular dystrophies (LGMDs), and we
currently have more than 40 programs in various stages of
development. Our vast pipeline is driven by our multi-platform
Precision Genetic Medicine Engine in gene therapy, RNA 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 and technologies;
the potential benefits of our technologies and scientific
approaches; the potential benefits of ELEVIDYS, including its
potential to modify the course of Duchenne; our understanding that
FDA, based on the totality of the evidence, is open to a label
expansion if supported by the review of the data and intend to
proceed rapidly with consideration of the submission for ELEVIDYS;
and expected plans and milestones.
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: 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; success in preclinical and 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; certain programs may never advance
in the clinic or may be discontinued for a number of reasons,
including regulators imposing a clinical hold and us suspending or
terminating clinical research or trials; 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; because we are developing product
candidates for the treatment of certain diseases in which there is
little clinical experience and we are using new endpoints or
methodologies, there is increased risk that the FDA, the EMA or
other regulatory authorities may not consider the endpoints of our
clinical trials to provide clinically meaningful results and that
these results may be difficult to analyze; 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, 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, 2022 and our most recent Quarterly Report on
Form 10-Q 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 Ended
September 30,
For the Nine Months Ended
September 30,
2023
2022
2023
2022
Revenues:
Products, net
$
309,322
$
207,774
$
779,805
$
607,836
Collaboration
22,495
22,495
66,750
66,750
Total revenues
331,817
230,269
846,555
674,586
Cost and expenses:
Cost of sales (excluding amortization of
in-licensed rights)
37,026
39,952
106,167
109,190
Research and development
194,301
216,707
681,870
663,286
Selling, general and administrative
120,893
104,787
350,171
330,943
Amortization of in-licensed rights
439
178
796
535
Total cost and expenses
352,659
361,624
1,139,004
1,103,954
Operating loss
(20,842
)
(131,355
)
(292,449
)
(429,368
)
Other loss, net:
Gain from sale of Priority Review
Voucher
—
—
102,000
—
Loss on debt extinguishment
—
(125,441
)
(387,329
)
(125,441
)
Other (expense) income, net
(12,332
)
378
17,309
(33,848
)
Total other loss, net
(12,332
)
(125,063
)
(268,020
)
(159,289
)
Loss before income tax expense
(33,174
)
(256,418
)
(560,469
)
(588,657
)
Income tax expense
7,763
1,320
21,163
5,587
Net loss
$
(40,937
)
$
(257,738
)
$
(581,632
)
$
(594,244
)
Net loss per share — basic and diluted
$
(0.46
)
$
(2.94
)
$
(6.56
)
$
(6.79
)
Weighted average number of shares of
common stock used in computing basic and diluted net loss per
share
88,889
87,628
88,609
87,465
Sarepta Therapeutics, Inc.
Reconciliation of GAAP Financial Measures to Non-GAAP Financial
Measures (unaudited, in thousands, except per share amounts)
For the Three Months Ended
September 30,
For the Nine Months Ended
September 30,
2023
2022
2023
2022
GAAP net loss
$
(40,937
)
$
(257,738
)
$
(581,632
)
$
(594,244
)
Interest (income) expense, net
(17,593
)
6,521
(46,565
)
34,390
Income tax expense
7,763
1,320
21,163
5,587
Impairment of equity investments
27,500
—
27,821
—
Loss (gain) on contingent consideration,
net
2,000
(6,700
)
1,200
(6,700
)
Gain from sale of Priority Review
Voucher
—
—
(102,000
)
—
Loss on debt extinguishment
—
125,441
387,329
125,441
Depreciation and amortization expense
10,928
10,703
33,025
31,311
Stock-based compensation expense
48,061
50,418
136,688
182,508
Non-GAAP net income (loss)
$
37,722
$
(70,035
)
$
(122,971
)
$
(221,707
)
Non-GAAP net earnings (loss) per
share:
Diluted Non-GAAP net earnings (loss) per
share*
$
0.37
$
(0.80
)
$
(1.39
)
$
(2.53
)
Weighted average number of shares of
common stock used in computing earnings per share:
Diluted
101,722
87,628
88,609
87,465
*Non-GAAP earnings per share is calculated
using diluted shares whereas non-GAAP net loss per share is
calculated using basic shares as all other instruments are
anti-dilutive. There was a $0.05 impact to the calculation of
non-GAAP net earnings per share as a result of the inclusion of
diluted shares for the three months ended September 30, 2023.
For the Three Months Ended
September 30,
For the Nine Months Ended
September 30,
2023
2022
2023
2022
GAAP research and development expenses
$
194,301
$
216,707
$
681,870
$
663,286
Stock-based compensation expense
(22,325
)
(14,795
)
(60,315
)
(42,330
)
Depreciation and amortization expense
(8,109
)
(8,166
)
(24,794
)
(23,700
)
Non-GAAP research and development
expenses
$
163,867
$
193,746
$
596,761
$
597,256
For the Three Months Ended
September 30,
For the Nine Months Ended
September 30,
2023
2022
2023
2022
GAAP selling, general and administrative
expenses
$
120,893
$
104,787
$
350,171
$
330,943
Stock-based compensation expense
(25,736
)
(35,623
)
(76,373
)
(140,178
)
Depreciation and amortization expense
(2,380
)
(2,359
)
(7,435
)
(7,076
)
Non-GAAP selling, general and
administrative expenses
$
92,777
$
66,805
$
266,363
$
183,689
Sarepta Therapeutics, Inc.
Condensed Consolidated Balance Sheets (unaudited, in thousands,
except share and per share data)
As of September 30,
2023
As of December 31,
2022
Assets
Current assets:
Cash and cash equivalents
$
541,932
$
966,777
Short-term investments
1,191,610
1,022,597
Accounts receivable
318,855
214,628
Inventory
244,011
203,968
Other current assets
154,441
149,891
Total current assets
2,450,849
2,557,861
Property and equipment, net
212,367
180,037
Right of use assets
133,454
64,954
Non-current inventory
176,112
162,545
Other non-current assets
136,925
162,969
Total assets
$
3,109,707
$
3,128,366
Liabilities and Stockholders’
Equity
Current liabilities:
Accounts payable
$
87,948
$
95,875
Accrued expenses
322,350
418,996
Deferred revenue, current portion
22,494
89,244
Other current liabilities
17,951
15,489
Total current liabilities
450,743
619,604
Long-term debt
1,236,755
1,544,292
Lease liabilities, net of current
portion
134,752
57,578
Deferred revenue, net of current
portion
485,000
485,000
Contingent consideration
38,100
36,900
Other non-current liabilities
—
42
Total liabilities
2,345,350
2,743,416
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; 93,537,355 and 87,950,117 issued and
outstanding at September 30, 2023, and December 31, 2022,
respectively
9
9
Additional paid-in capital
5,256,854
4,296,841
Accumulated other comprehensive loss, net
of tax
(638
)
(1,664
)
Accumulated deficit
(4,491,868
)
(3,910,236
)
Total stockholders’ equity
764,357
384,950
Total liabilities and stockholders’
equity
$
3,109,707
$
3,128,366
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231101211984/en/
Investor Contact: Ian Estepan, 617-274-4052
iestepan@sarepta.com Media Contact: Tracy Sorrentino,
617-301-8566 tsorrentino@sarepta.com
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