- Net product revenues for the first quarter 2024 totaled
$359.5 million, a 55% increase over the same quarter of the prior
year
- ELEVIDYS net revenues for the quarter totaled $133.9
million
- Achieved GAAP Earnings of $36.1 million for the first
quarter 2024 and non-GAAP Earnings of $78.2 million for the same
period
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in
precision genetic medicine for rare diseases, today reported
financial results for the first quarter 2024.
“We are pleased to announce another strong quarter of growth
from our four approved therapies, posting net product revenue of
$359.5 million, a 55% increase over the same quarter of the prior
year, and achieving profitability on a GAAP and non-GAAP basis,”
said Doug Ingram, president and chief executive officer, Sarepta
Therapeutics. “In particular, our recently approved gene therapy,
ELEVIDYS, achieved nearly $134.0 million in net product revenue in
the quarter. Although its initial label is quite narrow, ELEVIDYS
has posted cumulative sales of over $334.0 million since its
approval in June of last year, far exceeding performance of all
other gene therapies approved in the last few years combined.
Working with the FDA, we continue to productively prosecute our BLA
supplement to expand the ELEVIDYS addressable population, with a
target action date of June 21, 2024. If successful, 2024 could be
the most profound year yet in our fight against the effects of
Duchenne muscular dystrophy and a bellwether for the transformative
potential of gene therapy for rare disease.”
First Quarter 2024 and Recent Developments:
- U.S. FDA review of efficacy supplement to expand the
ELEVIDYS indication is ongoing: The Company is currently
awaiting U.S. Food and Drug Administration (FDA) Priority Review of
its efficacy supplement to the Biologics License Application (BLA)
for ELEVIDYS (delandistrogene moxeparvovec-rokl) where the review
goal date is June 21, 2024. At acceptance of the BLA in February,
the Agency also confirmed they are not planning to hold an advisory
committee to discuss the supplement. The goals of the efficacy
supplement are twofold:
- To expand the labeled indication for ELEVIDYS as follows:
“[ELEVIDYS is indicated for] the treatment of Duchenne muscular
dystrophy (DMD) patients with a confirmed mutation in the DMD
gene.”
- To convert the ELEVIDYS accelerated approval to a traditional
approval.
- Real world evidence published in Muscle & Nerve (March
2024) finds eteplirsen treatment associated with longer survival
compared to natural history: The publication details real-world
evidence showing that treatment with eteplirsen resulted in
statistically significant survival benefits compared to a
controlled natural history comparator group of Duchenne patients.
In the analysis the survival age of the eteplirsen treated patients
(n=579) was 32.8 years, 5.4 years longer than the median survival
age of 27.4 years in the Duchenne natural history studies
(n=1,224). Additionally, earlier initiation of eteplirsen treatment
and longer duration of treatment were associated with increases in
the survival benefit.
Other findings from the analysis include:
- Overall, eteplirsen-treated patients had a 66% reduction in
risk of death compared with natural history control group.
- Longer duration of treatment with eteplirsen was associated
with greater survival benefit, with patients treated with
eteplirsen for longer than four years demonstrating an 85% lower
risk of death compared to patients who had less than two years of
treatment with eteplirsen.
- Patients receiving eteplirsen treatment for more than four
years survived 32.8 years (95% confidence interval), and patients
receiving eteplirsen between two to four years survived 29.4 years
(95% confidence interval).
- Patients treated with eteplirsen for less than two years had a
median survival of 28.1 years, compared to natural history of 27.4
years.
- The median survival age has not yet been reached in patients
taking eteplirsen for longer than two years.
- In patients aged 10 to 28 years, for whom deaths are most
likely to be observed, eteplirsen treatment had a 42% lower risk of
death than age-matched natural history patients.
These findings add to the large and growing body of real-world
evidence generated for eteplirsen, where treatment is associated
with delays in time to loss of ambulation, improvements in lung
function and various quality of life measures when compared to
standard of care or natural history controls.
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.
Q1 2024 Financial Highlights1
For the Three Months Ended
March 31,
2024
2023
Change
Change
(in millions, except for per
share amounts)
$
%
Total Revenues
$
413.5
$
253.5
$
160.0
63
%
Operating income (loss):
GAAP
$
34.9
$
(138.1
)
$
173.0
NM*
Non-GAAP
$
83.7
$
(85.7
)
$
169.4
NM*
Net income (loss):
GAAP
$
36.1
$
(516.8
)
$
552.9
NM*
Non-GAAP
$
78.2
$
(87.7
)
$
165.9
NM*
Diluted earnings (loss) per share
GAAP
$
0.37
$
(5.86
)
$
6.23
NM*
Non-GAAP
$
0.73
$
(0.99
)
$
1.72
NM*
*NM: not meaningful
[1] For an explanation of our use of
non-GAAP financial measures, please refer to the "Use of Non-GAAP
Financial Measures" section later in this press release and for a
reconciliation of each non-GAAP financial measure to the most
comparable GAAP measures, see the table at the end of this press
release.
As of March 31, 2024
As of December 31,
2023
(in millions)
Cash, cash equivalents, and
investments
$
1,440.3
$
1,691.8
Revenues
Total revenues increased by $160.0 million for the three months
ended March 31, 2024 compared to the same period of 2023. The
increase primarily reflects the increasing demand for ELEVIDYS as
well as the $48.0 million of collaboration revenue recognized
related to F. Hoffman-La Roche Ltd.'s ("Roche") declined option to
acquire the ex-US rights to a certain external, early-stage
Duchenne development program. Additionally, for the three months
ended March 31, 2024, we recognized $5.8 million of contract
manufacturing collaboration revenue associated with commercial
ELEVIDYS supply delivered to Roche, with no similar activity for
the same period of 2023.
Cost of sales (excluding amortization of in-licensed
rights)
Cost of sales (excluding amortization of in-license rights)
increased by $15.5 million for the three months ended March 31,
2024, compared with the same period of 2023, which primarily
reflects continued increasing demand for our products, an increase
in royalty payments related to ELEVIDYS sales, as well as an
increase in the write-offs of certain batches of our products not
meeting our quality specifications. The majority of inventory costs
for ELEVIDYS sold during the three months ended March 31, 2024 were
previously expensed as research and development expense as they
were incurred prior to the regulatory approval of ELEVIDYS in June
2023.
Operating expenses and others
For the three months ended March 31, 2024, research and
development expenses decreased by $45.3 million compared with the
same period of 2023. The changes in research and development
expenses primarily reflect capitalization of commercial batches of
ELEVIDYS manufactured after its approval in June 2023, partially
offset by a ramp-up of our late-stage clinical trials. For the
three months ended March 31, 2024, non-GAAP research and
development expenses decreased by $42.6 million compared with the
same period of 2023.
Selling, general and administrative expenses increased $16.3
million for the three months ended March 31, 2024 compared with the
same period of 2023. The increase is primarily driven by
professional services used for the launch of ELEVIDYS and ongoing
litigation matters, as well as the timing of charitable
contributions. Non-GAAP selling, general and administrative
expenses increased $17.2 million for the three months ended March
31, 2024 compared with the same period of 2023.
For the three months ended March 31, 2024, other income, net
decreased by $6.2 million compared with the same period of 2023,
which primarily reflects an increase in the fair value of our
contingent consideration liability.
Income tax expense for the three months ended March 31, 2024 and
2023, was approximately $5.3 million and $4.0 million,
respectively. Income tax expense for all periods presented
primarily relates to federal and state income taxes.
Use of Non-GAAP Measures
In addition to the GAAP financial measures set forth in this
press release, we have included certain non-GAAP measurements. The
non-GAAP income (loss) is defined by us as GAAP net income (loss)
excluding interest income, net, depreciation and amortization
expense, stock-based compensation expense, the estimated income tax
impact of each pre-tax non-GAAP adjustment and other items. The
non-GAAP operating income (loss) is defined as GAAP operating
income (loss) excluding depreciation and amortization expense,
stock-based compensation expense and other items. Non-GAAP research
and development expenses are defined by us 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 us as
GAAP selling, general and administrative expenses excluding
depreciation expense, stock-based compensation expense and other
items.
1. Interest, depreciation and amortization
Interest income, 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 our operations.
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 our
operating performance. Amortization expense primarily associated
with patent costs are amortized over a period of several years
after acquisition or patent application or renewal.
2. Stock-based compensation expenses
Stock-based compensation expenses represent non-cash charges
related to equity awards we have granted. Although these are
recurring charges to operations, we believe 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 our control. Therefore, we
believe that excluding these charges facilitates comparisons of our
operational performance in different periods.
3. Other items
We evaluate other items of expense and income on an individual
basis. We take into consideration quantitative and qualitative
characteristics of each item, including (a) nature, (b) whether the
items relate to our ongoing business operations, and (c) whether we
expect the items to continue or occur on a regular basis. These
other items include impairment of strategic investments, change in
fair value of contingent consideration, net, gain from sale of the
PRV and loss on debt extinguishment and may include other items
that fit the above characteristics in the future.
- We exclude from our non-GAAP results the impairment of any
strategic 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.
- We exclude from our non-GAAP results the loss on debt
extinguishment, which is considered to be an infrequent and
non-cash event as it is associated with a distinct financing
decision and is not indicative of the performance of our core
operations, which accordingly, would make it difficult to compare
our results to peer companies that also provide non-GAAP
disclosures.
- We exclude from our non-GAAP results the change in fair value
of 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.
Beginning in the fourth quarter of 2023, amortization of
in-licensed rights (formerly included within depreciation and
amortization expense) and income tax (benefit) expense are no
longer excluded from the non-GAAP results. We now include the
income tax effect of adjustments, which represents the estimated
income tax impact of each pre-tax non-GAAP adjustment based on the
applicable effective income tax rate. Non-GAAP financial results
for the first quarter of 2023 have been updated to reflect this
change for comparability.
We use these non-GAAP measures as key performance measures for
the purpose of evaluating operational performance and cash
requirements internally. We also believe these non-GAAP measures
increase comparability of period-to-period results and are useful
to investors as they provide a similar basis for evaluating our
performance as is applied by management. These non-GAAP measures
are not intended to be considered in isolation or to replace the
presentation of our 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 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 (DMD) 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 for this indication 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 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.
The most common adverse reactions from observational clinical
studies (N=163) seen in greater than 10% of patients 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 (DMD) 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 for this indication 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 (DMD) in patients who have a confirmed mutation of the
DMD 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 for this indication 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 (delandistrogene moxeparvovec-rokl) is a single-dose,
adeno-associated virus (AAV) based gene transfer therapy for
intravenous infusion designed to address the underlying genetic
cause of Duchenne muscular dystrophy – mutations or changes in the
dystrophin gene that result in the lack of dystrophin protein –
through the delivery of a transgene that codes for the targeted
production of ELEVIDYS micro-dystrophin in skeletal muscle.
ELEVIDYS is a one-time infusion 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
and is approved under accelerated approval based on expression of
ELEVIDYS micro-dystrophin in skeletal muscle observed in patients
treated with ELEVIDYS. Continued approval for this indication may
be contingent upon verification of a clinical benefit in
confirmatory trials. ELEVIDYS has met the full statutory standards
for safety and effectiveness and as such is not considered
investigational or experimental.
ELEVIDYS has been evaluated in four clinical studies:
SRP-9001-101, SRP-9001-102, SRP-9001-103 (ENDEAVOR) and
SRP-9001-301 (EMBARK). Accelerated approval of ELEVIDYS was
primarily based on data from SRP-9001-102 and SRP-9001-103. The
EMBARK study serves as the postmarketing confirmatory trial.
Important Safety Information for ELEVIDYS
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.
ELEVIDYS is approved under accelerated review based on
expression of ELEVIDYS micro-dystrophin in skeletal muscle.
Continued approval for this indication in this and other age groups
will be contingent upon verification of a clinical benefit in
confirmatory trials. ELEVIDYS has met the full statutory standards
for safety and effectiveness and as such is not considered
investigational or experimental.
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
(DMD) 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 LinkedIn, X (formerly Twitter), 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 to bring a better life to the patient
communities we serve and for 2024 to be the most profound year yet
in the fight against the effects of Duchenne muscular dystrophy and
a bellwether for the transformative potential of gene therapy for
rare disease; our understanding that FDA does not plan to hold an
advisory committee regarding the ELEVIDYS expansion; the potential
for our comprehensive approach of measuring total antibodies to
help improve the safety and efficacy of AAV-based gene transfer
therapies; and milestones and plans, including working with FDA to
expand the ELEVIDYS addressable population, with a target action
date of June 21, 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: 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; 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, 2023 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
March 31,
2024
2023
Revenues:
Products, net
$
359,484
$
231,495
Collaboration and other
53,980
22,005
Total revenues
413,464
253,500
Cost and expenses:
Cost of sales (excluding amortization of
in-licensed rights)
50,559
35,017
Research and development
200,396
245,679
Selling, general and administrative
127,003
110,714
Amortization of in-licensed rights
601
178
Total cost and expenses
378,559
391,588
Operating income (loss)
34,905
(138,088
)
Other income (loss), net:
Loss on debt extinguishment
—
(387,329
)
Other income, net
6,543
12,707
Total other income (loss), net
6,543
(374,622
)
Income (loss) before income tax
expense
41,448
(512,710
)
Income tax expense
5,329
4,045
Net income (loss)
$
36,119
$
(516,755
)
Net earnings (loss) per share:
Basic
$
0.38
$
(5.86
)
Diluted
$
0.37
$
(5.86
)
Weighted average number of shares of
common stock used in computing net earnings (loss) per share:
Basic
93,991
88,186
Diluted
99,114
88,186
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
March 31,
2024
2023
GAAP net income (loss)
$
36,119
$
(516,755
)
Interest income, net
(15,731
)
(12,992
)
Depreciation and amortization expense*
8,143
11,127
Stock-based compensation expense
40,692
41,250
Change in fair value on contingent
consideration
10,100
—
Loss on debt extinguishment
—
387,329
Impairment of strategic investments
—
321
Income tax effect of adjustments**
(1,083
)
1,983
Non-GAAP net income (loss)**
$
78,240
$
(87,737
)
GAAP net earnings (loss) per share -
diluted:
$
0.37
$
(5.86
)
Add: impact of GAAP to Non-GAAP
adjustments
0.36
4.87
Non-GAAP net earnings (loss) per share -
diluted***
$
0.73
$
(0.99
)
Weighted average number of shares of
common stock used in computing diluted earnings (loss) per
share:****
GAAP
99,114
88,186
Non-GAAP
107,215
88,186
*Beginning in the fourth quarter of 2023,
depreciation and amortization excludes amortization of in-licensed
rights. Non-GAAP financial results for the first quarter 2023 have
been updated to reflect this change for comparability.
**Beginning in the fourth quarter of 2023,
income tax (benefit) expense is no longer excluded from the
non-GAAP results. We have replaced this metric with income tax
effect of adjustments, which represents the estimated income tax
impact of each pre-tax non-GAAP adjustment based on the applicable
statutory income tax rate. Refer below for a reconciliation of
effective tax rates. Non-GAAP financial results for the first
quarter 2023 have been updated to reflect this change for
comparability.
***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.
****The difference between the weighted
average number of shares of common stock used in computing diluted
GAAP and non-GAAP earnings per share for the three months ended
March 31, 2024, is a result of the exclusion of the potential share
settlement of the 2027 Notes from the GAAP earnings per share as
the inclusion of such shares was anti-dilutive.
For the Three Months Ended
March 31,
2024
2023
Total effective tax rate, GAAP
14.2
%
(0.8
)%
Less: impact of GAAP to Non-GAAP
adjustments
(5.4
)
(1.6
)
Total effective tax rate, Non-GAAP
8.8
%
(2.4
)%
Sarepta Therapeutics, Inc.
Reconciliation of GAAP Financial
Measures to Non-GAAP Financial Measures
(unaudited, in thousands)
For the Three Months Ended
March 31,
2024
2023
GAAP research and development expenses
$
200,396
$
245,679
Stock-based compensation expense
(16,273
)
(16,413
)
Depreciation and amortization expense
(6,046
)
(8,551
)
Non-GAAP research and development
expenses
$
178,077
$
220,715
For the Three Months Ended
March 31,
2024
2023
GAAP selling, general and administrative
expenses
$
127,003
$
110,714
Stock-based compensation expense
(24,419
)
(24,837
)
Depreciation expense
(2,097
)
(2,576
)
Non-GAAP selling, general and
administrative expenses
$
100,487
$
83,301
Sarepta Therapeutics, Inc.
Condensed Consolidated Balance
Sheets
(unaudited, in thousands, except
share and per share data)
As of March 31, 2024
As of December 31,
2023
Assets
Current assets:
Cash and cash equivalents
$
427,290
$
428,430
Short-term investments
963,453
1,247,820
Accounts receivable, net
378,806
400,327
Inventory
373,530
322,859
Manufacturing-related deposits and
prepaids
238,821
102,181
Other current assets
82,965
77,714
Total current assets
2,464,865
2,579,331
Property and equipment, net
249,302
227,154
Right of use assets
126,269
129,952
Non-current inventory
207,542
191,368
Other non-current assets
176,407
136,771
Total assets
$
3,224,385
$
3,264,576
Liabilities and Stockholders’
Equity
Current liabilities:
Accounts payable
$
91,536
$
164,918
Accrued expenses
283,317
314,997
Deferred revenue, current portion
112,000
50,416
Current portion of long-term debt
105,586
105,483
Other current liabilities
16,270
17,845
Total current liabilities
608,709
653,659
Long-term debt
1,133,660
1,132,515
Lease liabilities, net of current
portion
140,102
140,965
Deferred revenue, net of current
portion
325,000
437,000
Contingent consideration
48,200
38,100
Other non-current liabilities
7,522
3,000
Total liabilities
2,263,193
2,405,239
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; 94,490,157 and 93,731,831 issued and
outstanding at March 31, 2024 and December 31, 2023,
respectively
9
9
Additional paid-in capital
5,371,968
5,304,623
Accumulated other comprehensive (loss)
income, net of tax
(691
)
918
Accumulated deficit
(4,410,094
)
(4,446,213
)
Total stockholders’ equity
961,192
859,337
Total liabilities and stockholders’
equity
$
3,224,385
$
3,264,576
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240501857942/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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