– Preliminary total net product revenue is expected to total
$1.145 billion for full-year 2023
– Preliminary ELEVIDYS net product revenue is expected to be
$131.3 million for the fourth quarter and $200.4 million for
full-year 2023, significantly exceeding consensus
– Preliminary RNA-based PMO net product revenue for the
fourth quarter and full-year of 2023 are expected to total $234.3
million and $945.0 million, respectively, exceeding 2023 full-year
guidance of $925 million
– Preliminary year-end 2023 cash, cash equivalents,
restricted cash and investments balance of approximately $1.7
billion
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in
precision genetic medicine for rare diseases, today reported
preliminary* fourth quarter and full-year 2023 net product revenue
and cash on hand as of December 31, 2023, as part of its
presentation today at the 42nd Annual J.P. Morgan Healthcare
Conference in San Francisco, Calif.
Financial Update* (preliminary and unaudited):
- Fourth quarter and full-year 2023 net product revenue for
ELEVIDYS are expected to be approximately $131.3 million and $200.4
million, respectively. Sarepta’s expected net product revenue does
not include collaboration revenue.
- Fourth quarter and full-year 2023 net product revenue for
Sarepta’s RNA-based PMOs are expected to be approximately $234.3
million and $945.0 million, respectively. Sarepta’s expected net
product revenue does not include collaboration revenue.
- As of December 31, 2023, the Company had preliminary cash, cash
equivalents, restricted cash and investments of approximately $1.7
billion.
“2023 was a consequential year for Sarepta and the patients we
serve, highlighted by the approval, launch and performance of
ELEVIDYS, the first gene therapy to treat Duchenne, and the
continued performance of our PMOs, EXONDYS 51, VYONDYS 53, and
AMONDYS 45,” said Doug Ingram, president and chief executive
officer, Sarepta Therapeutics. “Our preliminary financial results,
totaling over $1.145 billion in net product revenue, including over
$200 million in the first two quarters alone for ELEVIDYS, reflect
our team’s ability to execute and deliver on our commitment to
patients.”
*These preliminary selected financial results are unaudited and
subject to adjustment. Sarepta will report its final and complete
fourth quarter and full-year 2023 financial results in late
February 2024. The Company has not completed its financial closing
procedures for the quarter or year-ended December 31, 2023, and its
actual results could be materially different from these preliminary
financial results.
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 (DMD) – mutations or changes
in the dystrophin gene that result in the lack of dystrophin
protein – through the delivery of a shortened 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 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.
In addition to EMBARK, Study SRP-9001-301, which serves as the
postmarketing confirmatory study, ELEVIDYS has been evaluated in
three clinical studies: SRP-9001-101, SRP-9001-102 and
SRP-9001-103. Accelerated approval was primarily based on data from
SRP-9001-102 and SRP-9001-103.
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.
For further information, please see the full Prescribing
Information.
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.
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 Twitter, LinkedIn, Instagram and Facebook.
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.
Forward-Looking Statements
This press release contains “forward-looking statements.” Any
statements that are not statements of historical fact may be deemed
to be forward-looking statements. Words such as “believe,”
“anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,”
“look,” “potential,” “possible” and similar expressions are
intended to identify forward-looking statements. These
forward-looking statements include statements relating to our
expected financial results.
These forward-looking statements involve risks and
uncertainties, many of which are beyond Sarepta’s control. Actual
results could materially differ from those stated or implied by
these forward-looking statements as a result of such risks and
uncertainties. Known risk factors include the following: the
estimates and judgments we make, or the assumptions on which we
rely, in preparing our consolidated financial statements could
prove inaccurate; our revenues and operating results could
fluctuate significantly, which may adversely affect our stock
price; and those risks identified under the heading “Risk Factors”
in our Quarterly Report on Form 10-Q for the quarter ended
September 30, 2023 as well as other SEC filings made by the Company
which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect
the Company’s business, results of operations and the trading price
of Sarepta’s common stock. For a detailed description of risks and
uncertainties Sarepta faces, you are encouraged to review the SEC
filings made by Sarepta. We caution investors not to place
considerable reliance on the forward-looking statements contained
in this press release. Sarepta does not undertake any obligation to
publicly update its forward-looking statements based on events or
circumstances after the date hereof, except as required by law.
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version on businesswire.com: https://www.businesswire.com/news/home/20240108854944/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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