Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Recent Corporate Developments
- Net product revenues for Q4 2023 totaled $365.1 million, a 55% increase YoY.
- Full-year 2023 net product revenues reached $1.1 billion, a 36% increase over the prior year.
- Sarepta achieved GAAP profitability in Q4 2023 after non-GAAP profitability in Q3 2023.
- FDA accepted an efficacy supplement for ELEVIDYS for the treatment of Duchenne muscular dystrophy patients with a confirmed mutation in the DMD gene.
- Positive data from Part B of the MOMENTUM study on SRP-5051 showed mean dystrophin expression of 5.17% and mean exon skipping of 11.11% at 28 weeks.
- Initiated screening in EMERGENE, a phase 3 clinical study of SRP-9003 for limb-girdle muscular dystrophy type 2E/R4.
- None.
Insights
The reported financial results from Sarepta Therapeutics indicate a substantial increase in net product revenues, both quarterly and annually, which is a clear sign of the company's growth trajectory. The 55% quarterly and 36% annual revenue growth are significant, outpacing many competitors in the biotech sector. This performance is largely driven by the successful launch of ELEVIDYS and sustained demand for the PMO Products. The profitability milestone achieved in Q4 2023, transitioning from a non-GAAP profitability in Q3 to GAAP profitability, is noteworthy as it reflects improved operational efficiency and cost management. Investors should consider the implications of these results on future earnings potential and the company's ability to reinvest in R&D without compromising profitability.
The acceptance of an efficacy supplement for ELEVIDYS by the FDA, with a Priority Review designation, is a pivotal development for Sarepta. This could potentially broaden the treatment's indication, enhancing its market potential. The conversion from accelerated to traditional approval would also solidify the drug's standing in the market. The clinical data supporting this supplement, from EMBARK and ENDEAVOR studies, demonstrate Sarepta's commitment to robust research and development. Furthermore, the positive data from the MOMENTUM study of SRP-5051, a next-generation treatment for Duchenne muscular dystrophy, indicates a strong pipeline that could sustain long-term growth. These developments are likely to have a positive ripple effect on the company's valuation, as they suggest a trajectory of continued innovation and market expansion.
From a market perspective, Sarepta's performance and strategic regulatory milestones position the company favorably among its peers in the biotechnology industry. The growth in product revenues, particularly from ELEVIDYS, suggests effective market penetration and brand strength. The company's ability to increase sales amidst a competitive landscape indicates strong execution of its commercial strategy. It is also essential to recognize the potential market expansion from the broader label for ELEVIDYS and the implications of a successful transition to traditional approval. Such regulatory advancements could significantly enhance market confidence and investor sentiment. However, it's important to monitor the competitive landscape for emerging therapies that could impact Sarepta's market share.
– Net product revenues for the fourth quarter 2023 totaled
– ELEVIDYS revenues for the quarter totaled
“We are pleased to report another strong quarter and year of performance serving the patient community. With our three approved PMO therapies, EXONDYS 51, VYONDYS 53 and AMONDYS 45, and supported by the successful launch of our gene therapy, ELEVIDYS, Sarepta’s net product revenue grew
Fourth Quarter 2023 and Recent Developments:
-
Announced
U.S. FDA acceptance of an efficacy supplement to expand the ELEVIDYS indication: TheU.S. Food and Drug Administration (FDA) has accepted and filed Sarepta’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl). 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.
The FDA has granted the efficacy supplement a Priority Review with a review goal date of June 21, 2024. The Agency has also confirmed they are not planning to hold an advisory committee meeting to discuss the supplement.
-
Submitted efficacy supplement to expand the ELEVIDYS label to include Duchenne muscular dystrophy patients without restriction to age or ambulatory status: Submitted an efficacy supplement to the BLA for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows "[ELEVIDYS is indicated for] the treatment of DMD patients with a confirmed mutation in the DMD gene.” The efficacy supplement is supported by results from EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study in patients with Duchenne between the ages of 4 through 7 years and data from ENDEAVOR (Study SRP-9001-103), an open label clinical study in patients with Duchenne, that is enrolling patients ages 2 years and older. The supplement was submitted to the
U.S. Food and Drug Administration (FDA) with a request for Priority Review. Sarepta has also completed the EMBARK postmarketing requirement (PMR) and submitted the PMR to FDA requesting conversion from accelerated approval to traditional approval.
-
Announced positive data from Part B of MOMENTUM, a phase 2 study of SRP-5051 in patients with Duchenne muscular dystrophy amenable to skipping exon 51: Announced positive data from Part B of the MOMENTUM study (Study SRP-5051-201), a global, Phase 2, multi-ascending dose clinical trial of SRP-5051 (vesleteplirsen) that enrolled patients aged 8 to 21 years. SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. Data from Part B of MOMENTUM found that at the higher, target dose, approximately 30 mg/kg dosed every four weeks, SRP-5051 resulted in mean dystrophin expression of
5.17% , and mean exon skipping of11.11% at 28 weeks (n=20). Consistent dystrophin expression was seen in ambulatory (4.76% , n=11) and non-ambulatory (5.67% , n=9) participants at 28 weeks. Hypomagnesemia has previously been identified in patients taking SRP-5051 and was managed and monitored through prophylactic magnesium supplementation as part of the study protocol.
- Initiated screening in EMERGENE, a phase 3 clinical study of SRP-9003 for the treatment of limb-girdle muscular dystrophy type 2E/R4: Announced that screening is underway in Study SRP-9003-301, also known as EMERGENE. Study 9003-301 is a Phase 3, multi-national, open-label study of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy Type 2E (LGMD2E/R4), or beta sarcoglycanopathy. EMERGENE will enroll 15 participants (ambulatory and non-ambulatory), aged 4 and older, and uses commercially representative process SRP-9003 material. The EMERGENE study design incorporates a six-month natural history lead-in. The primary endpoint is expression of beta-sarcoglycan 60 days after dosing. Other endpoints include functional measures through month 60 and safety.
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 December 31, 2023, the Company reported a GAAP net income of
For the twelve months ended December 31, 2023, the Company reported a GAAP net loss of
Revenues
For the three months ended December 31, 2023, the Company recorded total revenues of
For the three months ended December 31, 2023, the Company recorded net product revenues of
For the three and twelve months ended December 31, 2023, the Company recognized
Cost and Expenses
Cost of sales (excluding amortization of in-licensed rights)
For the three months ended December 31, 2023, cost of sales (excluding amortization of in-licensed rights) was
For the twelve months ended December 31, 2023, cost of sales (excluding amortization of in-licensed rights) was
Research and development
Research and development expenses were
-
$57.6 million -
$16.9 million -
$3.5 million -
$1.6 million -
$1.9 million -
$3.2 million -
$4.8 million -
$7.9 million -
$12.0 million -
$28.2 million
Research and development expenses were
-
$51.5 million -
$38.8 million -
$21.2 million -
$18.3 million -
$12.0 million -
$9.7 million -
$3.1 million -
$21.9 million -
$143.7 million $54.0 million -
$11.3 million
Non-GAAP research and development expenses were
Selling, general and administrative
Selling, general and administrative expenses were
-
$18.4 million -
$7.9 million -
$3.6 million -
$7.9 million -
$10.7 million
Selling, general and administrative expenses were
-
$60.9 million -
$35.2 million -
$10.9 million -
$4.6 million -
$71.7 million
Non-GAAP selling, general and administrative expenses were
Amortization of in-licensed rights
For the three and twelve months ended December 31, 2023, the Company recorded amortization of in-licensed rights of approximately
Loss on debt extinguishment
On November 14, 2017, the Company issued
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
Other income (expense), net
For the three months ended December 31, 2023 and 2022, other income, net was
Income tax (benefit) expense
Income tax benefit for the three months ended December 31, 2023 was approximately
Cash, Cash Equivalents, Restricted Cash and Investments
The Company had approximately
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 income (loss) excluding interest (income) expense, net, depreciation and amortization expense, stock-based compensation expense, the estimated income tax impact of each pre-tax non-GAAP adjustment 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 expense, stock-based compensation expense and other items.
1. Interest, 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. 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 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 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 or occur on a regular basis. These other items include impairment of strategic investments, loss (gain) on 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.
- The Company excludes from its 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.
- The Company excludes from its 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 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 (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 an infrequent 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.
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. The Company now includes 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 fourth quarter and full-year 2022 have been updated to reflect this change for comparability.
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 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.
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
The most common adverse reactions from observational clinical studies (N=163) seen in greater than
For further information, please see the full Prescribing Information.
About VYONDYS 53
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.
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
Other adverse reactions that occurred at a frequency greater than
For further information, please see the full Prescribing Information.
About AMONDYS 45
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.
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
Other adverse reactions that occurred in at least
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).
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.
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 X (formerly 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, product candidates and scientific approaches; the potential for the ELEVIDYS efficacy supplement to expand the approved label for ELEVIDYS and convert the ELEVIDYS accelerated approval to a traditional approval; the review goal date of June 21, 2024 for the ELEVIDYS efficacy supplement; our understanding that FDA does not plan to hold an advisory committee to discuss the ELEVIDYS supplement; the potential for 2024 being the most important year yet for families living with Duchenne and those invested in the improvement of patients’ lives; 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; 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; 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 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
|
|
|
For the Twelve Months Ended
|
|
||||||||||
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
||||
Revenues: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Products, net |
|
$ |
365,071 |
|
|
$ |
235,933 |
|
|
$ |
1,144,876 |
|
|
$ |
843,769 |
|
Collaboration and other |
|
|
31,710 |
|
|
|
22,494 |
|
|
|
98,460 |
|
|
|
89,244 |
|
Total revenues |
|
|
396,781 |
|
|
|
258,427 |
|
|
|
1,243,336 |
|
|
|
933,013 |
|
Cost and expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Cost of sales (excluding amortization of in-licensed rights) |
|
|
44,176 |
|
|
|
30,799 |
|
|
|
150,343 |
|
|
|
139,989 |
|
Research and development |
|
|
195,517 |
|
|
|
213,804 |
|
|
|
877,387 |
|
|
|
877,090 |
|
Selling, general and administrative |
|
|
131,700 |
|
|
|
120,478 |
|
|
|
481,871 |
|
|
|
451,421 |
|
Amortization of in-licensed rights |
|
|
763 |
|
|
|
179 |
|
|
|
1,559 |
|
|
|
714 |
|
Total cost and expenses |
|
|
372,156 |
|
|
|
365,260 |
|
|
|
1,511,160 |
|
|
|
1,469,214 |
|
Operating income (loss) |
|
|
24,625 |
|
|
|
(106,833 |
) |
|
|
(267,824 |
) |
|
|
(536,201 |
) |
Other income (loss), net: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Other income (expense), net |
|
|
15,746 |
|
|
|
5,527 |
|
|
|
33,055 |
|
|
|
(28,321 |
) |
Loss on debt extinguishment |
|
|
— |
|
|
|
— |
|
|
|
(387,329 |
) |
|
|
(125,441 |
) |
Gain from sale of Priority Review Voucher |
|
|
— |
|
|
|
— |
|
|
|
102,000 |
|
|
|
— |
|
Total other income (loss), net |
|
|
15,746 |
|
|
|
5,527 |
|
|
|
(252,274 |
) |
|
|
(153,762 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
Income (loss) before income tax (benefit) expense |
|
|
40,371 |
|
|
|
(101,306 |
) |
|
|
(520,098 |
) |
|
|
(689,963 |
) |
Income tax (benefit) expense |
|
|
(5,284 |
) |
|
|
7,938 |
|
|
|
15,879 |
|
|
|
13,525 |
|
Net income (loss) |
|
$ |
45,655 |
|
|
$ |
(109,244 |
) |
|
$ |
(535,977 |
) |
|
$ |
(703,488 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
Net earnings (loss) per share: |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Basic |
|
$ |
0.49 |
|
|
$ |
(1.24 |
) |
|
$ |
(5.80 |
) |
|
$ |
(8.03 |
) |
Diluted |
|
$ |
0.47 |
|
|
$ |
(1.24 |
) |
|
$ |
(5.80 |
) |
|
$ |
(8.03 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
Weighted-average number of shares of common stock used in computing basic net earnings (loss) per share |
|
|
93,617 |
|
|
|
87,838 |
|
|
|
92,398 |
|
|
|
87,559 |
|
Weighted-average number of shares of common stock used in computing diluted net earnings (loss) per share |
|
|
105,594 |
|
|
|
87,838 |
|
|
|
92,398 |
|
|
|
87,559 |
|
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
|
|
For the Twelve Months Ended
|
|
||||||||
|
2023 |
|
2022 |
|
2023 |
|
2022 |
|
||||
|
|
|
|
|
|
|
|
|
||||
GAAP net income (loss) |
$ |
45,655 |
|
$ |
(109,244 |
) |
$ |
(535,977 |
) |
$ |
(703,488 |
) |
Interest (income) expense, net |
|
(17,469 |
) |
|
(8,865 |
) |
|
(64,034 |
) |
|
25,525 |
|
Depreciation and amortization expense* |
|
10,609 |
|
|
10,374 |
|
|
42,838 |
|
|
41,150 |
|
Stock-based compensation expense |
|
45,826 |
|
|
50,510 |
|
|
182,514 |
|
|
233,018 |
|
Impairment of strategic investments |
|
2,500 |
|
|
2,575 |
|
|
30,321 |
|
|
2,575 |
|
Loss on debt extinguishment |
|
— |
|
|
— |
|
|
387,329 |
|
|
125,441 |
|
Loss (gain) on contingent consideration, net |
|
— |
|
|
— |
|
|
1,200 |
|
|
(6,700 |
) |
Gain from sale of Priority Review Voucher |
|
— |
|
|
— |
|
|
(102,000 |
) |
|
— |
|
Income tax effect of adjustments** |
|
(541 |
) |
|
1,075 |
|
|
(1,738 |
) |
|
(7,915 |
) |
Non-GAAP net income (loss)** |
$ |
86,580 |
|
$ |
(53,575 |
) |
$ |
(59,547 |
) |
$ |
(290,394 |
) |
|
|
|
|
|
|
|
|
|
||||
Non-GAAP net earnings (loss) per share: |
|
|
|
|
|
|
|
|
||||
Diluted*** |
$ |
0.82 |
|
$ |
(0.61 |
) |
$ |
(0.64 |
) |
$ |
(3.32 |
) |
|
|
|
|
|
|
|
|
|
||||
Weighted-average number of shares of common stock
|
|
|
|
|
|
|
|
|
||||
Diluted |
|
105,594 |
|
|
87,838 |
|
|
92,398 |
|
|
87,559 |
|
|
|
|
|
|
|
|
|
|
||||
*Beginning in the fourth quarter of 2023, depreciation and amortization excludes amortization of in-licensed rights. Non-GAAP financial results for the fourth quarter and full-year 2022 have been updated to reflect this change for comparability.
|
||||||||||||
|
||||||||||||
|
For the Three Months Ended
|
|
For the Twelve Months Ended
|
|
||||||||
|
2023 |
|
2022 |
|
2023 |
|
2022 |
|
||||
Total effective tax rate, GAAP |
|
(12.0 |
)% |
|
5.5 |
% |
|
(3.1 |
)% |
|
(2.0 |
)% |
Less: impact of GAAP to Non-GAAP adjustments |
|
(0.7 |
) |
|
(1.8 |
) |
|
(39.0 |
) |
|
(6.0 |
) |
Total effective tax rate, Non-GAAP |
|
(12.7 |
)% |
|
3.7 |
% |
|
(42.1 |
)% |
|
(8.0 |
)% |
Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands) |
||||||||||||||||
|
|
For the Three Months Ended
|
|
|
For the Twelve Months Ended
|
|
||||||||||
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
||||
GAAP research and development expenses |
|
$ |
195,517 |
|
|
$ |
213,804 |
|
|
$ |
877,387 |
|
|
$ |
877,090 |
|
Stock-based compensation expense |
|
|
(22,174 |
) |
|
|
(18,963 |
) |
|
|
(82,489 |
) |
|
|
(61,293 |
) |
Depreciation and amortization expense |
|
|
(8,217 |
) |
|
|
(8,013 |
) |
|
|
(33,011 |
) |
|
|
(31,713 |
) |
Non-GAAP research and development expenses |
|
$ |
165,126 |
|
|
$ |
186,828 |
|
|
$ |
761,887 |
|
|
$ |
784,084 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
||||
|
|
For the Three Months Ended
|
|
|
For the Twelve Months Ended
|
|
||||||||||
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
||||
GAAP selling, general and administrative expenses |
|
$ |
131,700 |
|
|
$ |
120,478 |
|
|
$ |
481,871 |
|
|
$ |
451,421 |
|
Stock-based compensation expense |
|
|
(23,652 |
) |
|
|
(31,547 |
) |
|
|
(100,025 |
) |
|
|
(171,725 |
) |
Depreciation expense |
|
|
(2,392 |
) |
|
|
(2,361 |
) |
|
|
(9,827 |
) |
|
|
(9,437 |
) |
Non-GAAP selling, general and administrative expenses |
|
$ |
105,656 |
|
|
$ |
86,570 |
|
|
$ |
372,019 |
|
|
$ |
270,259 |
|
Sarepta Therapeutics, Inc. Condensed Consolidated Balance Sheets (unaudited, in thousands, except share and per share data) |
||||||||
|
|
As of December 31, |
|
|||||
|
|
2023 |
|
|
2022 |
|
||
Assets |
|
|
|
|
|
|
||
Current assets: |
|
|
|
|
|
|
||
Cash and cash equivalents |
|
$ |
428,430 |
|
|
$ |
966,777 |
|
Short-term investments |
|
|
1,247,820 |
|
|
|
1,022,597 |
|
Accounts receivable, net |
|
|
400,327 |
|
|
|
214,628 |
|
Inventory |
|
|
322,859 |
|
|
|
203,968 |
|
Other current assets |
|
|
179,895 |
|
|
|
149,891 |
|
Total current assets |
|
|
2,579,331 |
|
|
|
2,557,861 |
|
Property and equipment, net |
|
|
227,154 |
|
|
|
180,037 |
|
Right of use assets |
|
|
129,952 |
|
|
|
64,954 |
|
Non-current inventory |
|
|
191,368 |
|
|
|
162,545 |
|
Other non-current assets |
|
|
136,771 |
|
|
|
162,969 |
|
Total assets |
|
$ |
3,264,576 |
|
|
$ |
3,128,366 |
|
|
|
|
|
|
|
|
||
Liabilities and Stockholders’ Equity |
|
|
|
|
|
|
||
Current liabilities: |
|
|
|
|
|
|
||
Accounts payable |
|
$ |
164,918 |
|
|
$ |
95,875 |
|
Accrued expenses |
|
|
314,997 |
|
|
|
418,996 |
|
Deferred revenue, current portion |
|
|
50,416 |
|
|
|
89,244 |
|
Current portion of long-term debt |
|
|
105,483 |
|
|
|
— |
|
Current portion of lease liabilities |
|
|
17,845 |
|
|
|
15,489 |
|
Total current liabilities |
|
|
653,659 |
|
|
|
619,604 |
|
Long-term debt |
|
|
1,132,515 |
|
|
|
1,544,292 |
|
Lease liabilities, net of current portion |
|
|
140,965 |
|
|
|
57,578 |
|
Deferred revenue, net of current portion |
|
|
437,000 |
|
|
|
485,000 |
|
Contingent consideration |
|
|
38,100 |
|
|
|
36,900 |
|
Other non-current liabilities |
|
|
3,000 |
|
|
|
42 |
|
Total liabilities |
|
|
2,405,239 |
|
|
|
2,743,416 |
|
Stockholders’ equity: |
|
|
|
|
|
|
||
Preferred stock, |
|
|
— |
|
|
|
— |
|
Common stock, |
|
|
9 |
|
|
|
9 |
|
Additional paid-in capital |
|
|
5,304,623 |
|
|
|
4,296,841 |
|
Accumulated other comprehensive income (loss), net of tax |
|
|
918 |
|
|
|
(1,664 |
) |
Accumulated deficit |
|
|
(4,446,213 |
) |
|
|
(3,910,236 |
) |
Total stockholders’ equity |
|
|
859,337 |
|
|
|
384,950 |
|
Total liabilities and stockholders’ equity |
|
$ |
3,264,576 |
|
|
$ |
3,128,366 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20240228046825/en/
Investor Contact:
Ian Estepan, 617-274-4052
iestepan@sarepta.com
Media Contact:
Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com
Source: Sarepta Therapeutics, Inc.
FAQ
What were Sarepta Therapeutics' (SRPT) net product revenues for Q4 2023?
What was the full-year 2023 net product revenues for Sarepta Therapeutics (SRPT)?
Did Sarepta Therapeutics achieve profitability in Q4 2023?
What positive development did the FDA announce regarding ELEVIDYS?
What were the results of Part B of the MOMENTUM study on SRP-5051?
What phase 3 clinical study did Sarepta Therapeutics initiate for limb-girdle muscular dystrophy type 2E/R4?
