SRPT Sarepta Therapeutics Inc New

 

UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549

 

FORM 8-K

 

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): February 26, 2025

 

 

Sarepta Therapeutics, Inc.

(Exact name of Registrant as Specified in Its Charter)

 

 

Delaware			001-14895	93-0797222
(State or Other Jurisdiction of Incorporation)			(Commission File Number)	(IRS Employer Identification No.)
215 First Street
Cambridge, Massachusetts				02142
(Address of Principal Executive Offices)				(Zip Code)

 

Registrant’s Telephone Number, Including Area Code: (617) 274-4000

 

(Former Name or Former Address, if Changed Since Last Report)

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

Securities registered pursuant to Section 12(b) of the Act:

Title of each class		Trading Symbol(s)		Name of each exchange on which registered
Common Stock, $0.0001 par value per share		SRPT		Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

 

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Item 2.02. Results of Operations and Financial Condition.

 

On February 26, 2025, Sarepta Therapeutics, Inc. issued a press release announcing its financial results for the quarter and year ended December 31, 2024. The full text of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K.

The information in Item 2.02 of this Form 8-K (including Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.

 

 

Item 9.01 Financial Statements and Exhibits.

 

(d) Exhibits

Exhibit Number		Description
99.1		Press Release dated February 26, 2025
104		The cover page from this Current Report on Form 8-K of Sarepta Therapeutics, Inc., formatted in Inline XBRL and included as Exhibit 101

 

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SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

			Sarepta Therapeutics, Inc.
Date:	February 26, 2025	By:	/s/ Douglas S. Ingram
			Douglas S. Ingram President and Chief Executive Officer

 

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Exhibit 99.1

Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2024 Financial Results and Recent Corporate Developments

CAMBRIDGE, Mass., Feb. 26, 2025 -- (BUSINESS WIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2024.

“2024 performance represented the fruition of our multi-year strategy to become a self-sustaining profitable biotech dedicated to improving the lives of patients with rare genetic disease. After obtaining a broad label for our gene therapy ELEVIDYS covering the vast majority of Duchenne patients, we had the most successful gene therapy launch in history, even as we continued to serve the community with our PMOs, EXONDYS 51, VYONDYS 53 and AMONDYS 45. And as we advanced our internal gene therapy pipeline, we also continued our diversification and secured our future by in-licensing a broad platform of siRNA programs, with potential blockbuster opportunities that could reach the market in 2028 and 2029,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “In 2025, we intend to capitalize on our 2024 achievements. In addition to 2025 net product revenue guidance of $2.9 billion to $3.1 billion, representing 70% year-over-year growth and 162% yearly growth for ELEVIDYS, we expect to reach multiple important milestones this year, including the proof of biology readouts in our myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy type 1 (FSHD) programs and the Biologics License Application (BLA) submission for SRP-9003 which, if successful, would lead to our first approval in our LGMD pipeline.”

Fourth Quarter 2024 and Recent Developments:

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At two years the Part 1 patients showed clinically meaningful and statistically significant functional benefit in NSAA, TTR and 10MWR compared with EC. Patients treated in Part 1 of the study had biopsies taken at 64 weeks after dosing and showed consistent and sustained expression of ELEVIDYS micro-dystrophin compared to week 12 biopsies, as measured by western blot, and provide biological support for observed functional outcomes. Additionally, skeletal MRI conducted on Part 1 patients indicated minimal muscle pathology progression, aligning with observed functional benefits.

These results contribute to the growing body of clinical evidence supporting both the durability of ELEVIDYS treatment and the importance of intervening early to preserve muscle. The safety profile of ELEVIDYS remained consistent with previous findings.

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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.

 

Q4 2024 Financial Highlights1

		For the Three Months Ended December 31,
		2024				2023				QTD Change
		(in millions, except for per share amounts)								$				%
Total Revenues		$	658.4			$	396.8				261.6				66	%
Operating income:
GAAP		$	161.7			$	24.6				137.1			NM*
Non-GAAP		$	221.2			$	81.1				140.1				173	%
Net income:
GAAP		$	159.0			$	45.7				113.3			NM*
Non-GAAP		$	206.0			$	86.6				119.4				138	%
Diluted earnings per share:
GAAP		$	1.50			$	0.47				1.03			NM*
Non-GAAP		$	1.90			$	0.82				1.08				132	%

 

		For the Twelve Months Ended December 31,
		2024				2023				YTD Change
		(in millions, except for per share amounts)								$				%
Total Revenues		$	1,902.0			$	1,243.3				658.7				53	%
Operating income (loss):
GAAP		$	218.1			$	(267.8	)			485.9			NM*
Non-GAAP		$	437.7			$	(42.5	)			480.2			NM*
Net income (loss):
GAAP		$	235.2			$	(536.0	)			771.2			NM*
Non-GAAP		$	397.9			$	(59.5	)			457.4			NM*
Diluted earnings (loss) per share:
GAAP		$	2.34			$	(5.80	)			8.14			NM*
Non-GAAP		$	3.69			$	(0.64	)			4.33			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 tables at the end of this press release.

 

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		As of December 31, 2024				As of December 31, 2023
		(in millions)
Cash, cash equivalents, restricted cash and investments		$	1,503.5			$	1,691.8

 

Revenues

Total revenues increased by $261.6 million and $658.7 million for the three and twelve months ended December 31, 2024, respectively, compared to the same periods of 2023. The increases primarily reflect the initial product launch of ELEVIDYS in June 2023 and subsequent expanded label approval in June 2024.

 

Additionally, included in total revenues for the three and twelve months ended December 31, 2024, is $20.3 million and $66.0 million, respectively, of contract manufacturing and other revenues associated with commercial ELEVIDYS supply delivered to Roche and royalty revenue received from Roche, as compared to $9.2 million of contract manufacturing and other revenues for the three and twelve months ended December 31, 2023.

 

Cost of sales (excluding amortization of in-licensed rights)

Cost of sales (excluding amortization of in-license rights) increased by $88.1 million and $168.8 million for the three and twelve months ended December 31, 2024, respectively, compared with the same periods of 2023. The increases in both periods primarily reflect the initial product launch of ELEVIDYS in June 2023 and subsequent expanded label approval in June 2024, as well as cost of sales related to products sold to Roche under our collaboration agreement increasing by $5.1 million and $20.4 million for the three and twelve months ended December 31, 2024, respectively, compared with the same periods of 2023.

 

Operating expenses and others

Research and development expenses increased by $4.4 million for the three months ended December 31, 2024, compared with the same period of 2023, primarily as a result of an increase in manufacturing expense related to a ramp up of batches produced for our Limb-girdle muscular dystrophy (“LGMD”) programs, partially offset by a decrease in clinical trial expenses primarily due to our decision to discontinue our PPMO program during 2024.

 

Research and development expenses decreased by $72.9 million for the twelve months ended December 31, 2024, compared with the same period of 2023. The decrease in research and development expense primarily reflects capitalization of commercial batches of ELEVIDYS manufactured after its approval in June 2023, partially offset by costs associated with the termination of the development, commercial manufacturing and supply agreement (the “Thermo Agreement”) related to Brammer Bio MA, LLC, an affiliate of Thermo Fisher Scientific, Inc. in August 2024, net of the reimbursable termination costs by Roche.

 

Non-GAAP research and development expenses increased by $7.6 million for the three months ended December 31, 2024, compared with the same period of 2023. Non-GAAP research and development expenses decreased by $57.4 million for the twelve months ended December 31, 2024, compared with the same period of 2023.

 

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Selling, general and administrative expenses increased by $32.2 million and $76.0 million for the three and twelve months ended December 31, 2024, compared with the same periods of 2023. The increase in selling, general and administrative expenses for both periods is primarily driven by professional services used to support the continued efforts to commercialize ELEVIDYS and ongoing litigation matters, the timing of charitable contributions, and compensation-related expenses, including stock-based compensation, partially due to changes in headcount. Non-GAAP selling, general and administrative expenses increased by $25.9 million and $66.3 million for the three and twelve months ended December 31, 2024, respectively, compared with the same periods of 2023.

 

For the three months ended December 31, 2024, other income, net decreased by $5.7 million, compared with the same period of 2023, which primarily reflects a decrease in interest income and accretion of investment discount, net as a result of lower interest rates and the investment mix of our investment portfolio during the three months ended December 31, 2024. For the twelve months ended December 31, 2024, other income (loss), net increased by $295.0 million compared with the same period of 2023, which primarily reflects a $387.3 million loss on debt extinguishment, partially offset by a $102.0 million gain on the sale of a Priority Review Voucher (“PRV”) during the twelve months ended December 31, 2023, with no similar activities in 2024.

Income tax expense for the three months ended December 31, 2024, was approximately $12.7 million. Income tax benefit for the three months ended December 31, 2023, was approximately $5.3 million. Income tax expense for the twelve months ended December 31, 2024 and 2023, was approximately $25.5 million and $15.9 million, respectively. Income tax expense (benefit) for all periods presented primarily relates to state, federal and foreign income taxes for which available tax losses or credits were not available to offset.

 

Use of Non-GAAP Measures

In addition to the GAAP financial measures set forth in this press release, we have included the following non-GAAP measurements:

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The following components are used to adjust our GAAP financial measures into the previously defined non-GAAP measurements:

 

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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 operating income (loss), non-GAAP net income (loss), and non-GAAP diluted 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 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, including 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.

 

Other adverse events may occur.

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To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1- 888-

SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

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

CONTRAINDICATIONS: VYONDYS 53 is contraindicated in patients with a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Anaphylaxis has occurred in patients receiving VYONDYS 53.

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis, 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, interrupting, or discontinuing the VYONDYS 53 therapy and monitor until the condition resolves. VYONDYS 53 is contraindicated in patients with a history of a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53.

Kidney Toxicity: 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

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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: 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.

Other adverse events may occur.

To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-

888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

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.

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Important Safety Information for AMONDYS 45

CONTRAINDICATIONS: AMONDYS 45 is contraindicated in patients with a known serious hypersensitivity to casimersen or any of the inactive ingredients in AMONDYS 45. 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. AMONDYS 45 is contraindicated in patients with known serious hypersensitivity to casimersen or to any of the inactive ingredients in AMONDYS 45.

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.

 

Other adverse events may occur.

To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-

888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

For further information, please see the full Prescribing Information.

About ELEVIDYS (delandistrogene moxeparvovec-rokl)

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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 DMD 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 indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.

 

The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

 

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:

Infusion-related Reactions:

 

Acute Serious Liver Injury:

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Immune-mediated Myositis:

 

Myocarditis:

 

Preexisting Immunity against AAVrh74:

 

Adverse Reactions:

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Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

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, scientific approaches and strategic partnerships; and expected milestones and plans, including the potential for siRNA programs to reach the market in 2028 and 2029, our expectation to disclose proof of biology readouts in our myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy type 1 (FSHD) programs in 2025, and announcing data from EMERGENE in the first half of 2025 with a potential Biologics License Application (BLA) submission for SRP-9003 in 2025.

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: 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; the expected benefits and opportunities related to our agreements with strategic partners may not be realized or may take longer to realize than expected due to a variety of reasons, including any inability of the parties to perform their commitments and obligations, challenges and uncertainties inherent in product research and development and manufacturing limitations; 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; 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

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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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 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 '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.

 

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Sarepta Therapeutics, Inc.

Condensed Consolidated Statements of Income (Loss)

(unaudited, in thousands, except per share amounts)

		For the Three Months Ended December 31,								For the Twelve Months Ended December 31,
		2024				2023				2024				2023
Revenues:
Products, net		$	638,157			$	365,071			$	1,787,960			$	1,144,876
Collaboration and other			20,255				31,710				114,019				98,460
Total revenues			658,412				396,781				1,901,979				1,243,336
Cost and expenses:
Cost of sales (excluding amortization of in-licensed rights)			132,304				44,176				319,099				150,343
Research and development			199,953				195,517				804,522				877,387
Selling, general and administrative			163,873				131,700				557,872				481,871
Amortization of in-licensed rights			601				763				2,405				1,559
Total cost and expenses			496,731				372,156				1,683,898				1,511,160
Operating income (loss)			161,681				24,625				218,081				(267,824	)
Other income (loss), net:
Other income, net			10,062				15,746				42,693				33,055
Gain from sale of Priority Review Voucher			—				—				—				102,000
Loss on debt extinguishment			—				—				—				(387,329	)
Total other income (loss), net			10,062				15,746				42,693				(252,274	)
Income (loss) before income tax expense (benefit)			171,743				40,371				260,774				(520,098	)
Income tax expense (benefit)			12,694				(5,284	)			25,535				15,879
Net income (loss)		$	159,049			$	45,655			$	235,239			$	(535,977	)
Earnings (loss) per share:
Basic		$	1.65			$	0.49			$	2.47			$	(5.80	)
Diluted		$	1.50			$	0.47			$	2.34			$	(5.80	)
Weighted average number of shares of common stock used in computing earnings (loss) per share:
Basic			96,283				93,617				95,075				92,398
Diluted			108,474				97,494				107,875				92,398

 

 

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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 December 31,								For the Twelve Months Ended December 31,
		2024				2023				2024				2023
GAAP net income (loss)		$	159,049			$	45,655			$	235,239			$	(535,977	)
Interest income, net			(10,753	)			(17,469	)			(53,909	)			(64,034	)
Depreciation and amortization expense			9,854				10,609				35,319				42,838
Stock-based compensation expense			49,676				45,826				184,300				182,514
Change in fair value of derivatives			(727	)			—				7,838				1,200
Gain from sale of Priority Review Voucher			—				—				—				(102,000	)
Loss on debt extinguishment			—				—				—				387,329
Impairment of strategic investments			—				2,500				—				30,321
Income tax effect of adjustments			(1,092	)			(541	)			(10,864	)			(1,738	)
Non-GAAP net income (loss)		$	206,007			$	86,580			$	397,923			$	(59,547	)
GAAP earnings (loss) per share - diluted:		$	1.50			$	0.47			$	2.34			$	(5.80	)
Add: impact of GAAP to Non-GAAP adjustments		$	0.40			$	0.35			$	1.35			$	5.16
Non-GAAP earnings (loss) per share - diluted*		$	1.90			$	0.82			$	3.69			$	(0.64	)
Weighted average number of shares of common stock used in computing diluted earnings (loss) per share:**
GAAP			108,474				97,494				107,875				92,398
Non-GAAP			108,474				105,594				107,875				92,398
*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 December 31, 2023, is a result of the exclusion of the potential share settlement of the 2027 Convertible Notes from the GAAP earnings per share as the inclusion of such shares was anti-dilutive.
		For the Three Months Ended December 31,								For the Twelve Months Ended December 31,
		2024				2023				2024				2023
Total effective tax rate, GAAP			7.4		%		(12.0	)	%		9.8		%		(3.1	)	%
Less: impact of GAAP to Non-GAAP adjustments			(1.1	)			(0.7	)			(1.4	)			(39.0	)
Total effective tax rate, Non-GAAP			6.3		%		(12.7	)	%		8.4		%		(42.1	)	%

 

 

 

 

 

 

 

 

 

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Sarepta Therapeutics, Inc.

Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures

(unaudited, in thousands)

 

		For the Three Months Ended December 31,								For the Twelve Months Ended December 31,
		2024				2023				2024				2023
GAAP research and development expenses		$	199,953			$	195,517			$	804,522			$	877,387
Stock-based compensation expense			(19,897	)			(22,174	)			(74,010	)			(82,489	)
Depreciation and amortization expense			(7,356	)			(8,217	)			(26,048	)			(33,011	)
Non-GAAP research and development expenses		$	172,700			$	165,126			$	704,464			$	761,887
		For the Three Months Ended December 31,								For the Twelve Months Ended December 31,
		2024				2023				2024				2023
GAAP selling, general and administrative expenses		$	163,873			$	131,700			$	557,872			$	481,871
Stock-based compensation expense			(29,779	)			(23,652	)			(110,290	)			(100,025	)
Depreciation expense			(2,498	)			(2,392	)			(9,271	)			(9,827	)
Non-GAAP selling, general and administrative expenses		$	131,596			$	105,656			$	438,311			$	372,019
		For the Three Months Ended December 31,								For the Twelve Months Ended December 31,
		2024				2023				2024				2023
GAAP operating income (loss)		$	161,681			$	24,625			$	218,081			$	(267,824	)
Stock-based compensation expense			49,676				45,826				184,300				182,514
Depreciation and amortization expense			9,854				10,609				35,319				42,838
Non-GAAP operating income (loss)		$	221,211			$	81,060			$	437,700			$	(42,472	)

 

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Sarepta Therapeutics, Inc.

Condensed Consolidated Balance Sheets

(unaudited, in thousands, except share and per share data)

		As of December 31,
		2024				2023
Assets
Current assets:
Cash and cash equivalents		$	1,103,010			$	428,430
Short-term investments			251,782				1,247,820
Accounts receivable, net			601,988				400,327
Inventory			749,960				322,859
Manufacturing-related deposits and prepaids			276,262				102,181
Other current assets			90,461				77,714
Total current assets			3,073,463				2,579,331
Property and equipment, net			340,336				227,154
Right of use assets			148,310				129,952
Non-current inventory			187,986				191,368
Non-current investments			133,163				—
Other non-current assets			79,915				136,771
Total assets		$	3,963,173			$	3,264,576
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable		$	214,442			$	164,918
Accrued expenses			373,513				314,997
Deferred revenue, current portion			130,256				50,416
Current portion of long-term debt			—				105,483
Other current liabilities			13,473				17,845
Total current liabilities			731,684				653,659
Long-term debt			1,137,124				1,132,515
Lease liabilities, net of current portion			192,473				140,965
Deferred revenue, net of current portion			325,000				437,000
Contingent consideration			47,400				38,100
Other non-current liabilities			1,750				3,000
Total liabilities			2,435,431				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;    96,900,496 and 93,731,831 issued and outstanding at December 31, 2024    and 2023, respectively			10				9
Additional paid-in capital			5,738,924				5,304,623
Accumulated other comprehensive (loss) income, net of tax			(218	)			918
Accumulated deficit			(4,210,974	)			(4,446,213	)
Total stockholders’ equity			1,527,742				859,337
Total liabilities and stockholders’ equity		$	3,963,173			$	3,264,576

 

 

 

 

 

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Source: Sarepta Therapeutics, Inc.

 

Investor Contact:

Ian Estepan, 617-274-4052

iestepan@sarepta.com

 

Media Contact:

Tracy Sorrentino, 617-301-8566

tsorrentino@sarepta.com

 

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v3.25.0.1

Document And Entity Information	Feb. 26, 2025
Cover [Abstract]
Document Type	8-K
Amendment Flag	false
Document Period End Date	Feb. 26, 2025
Entity Registrant Name	Sarepta Therapeutics, Inc.
Entity Central Index Key	0000873303
Entity Emerging Growth Company	false
Entity File Number	001-14895
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	93-0797222
Entity Address, Address Line One	215 First Street
Entity Address, City or Town	Cambridge
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02142
City Area Code	(617)
Local Phone Number	274-4000
Written Communications	false
Soliciting Material	false
Pre-commencement Tender Offer	false
Pre-commencement Issuer Tender Offer	false
Title of 12(b) Security	Common Stock, $0.0001 par value per share
Trading Symbol	SRPT
Security Exchange Name	NASDAQ

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