Catheter site discomfort

CAMBRIDGE, Mass.--( BUSINESS WIRE )--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the second quarter 2024. “ The second quarter of 2024 represents the most significant achievement in the advancement of medicine for Duchenne since researchers identified that lack of dystrophin was the underlying cause of Duchenne in 1986. Based on a wealth of compelling clinical evidence, the FDA broadened access to our gene therapy ELEVIDYS for all patients at least 4 years of age, with a traditional approval for ambulatory patients and accelerated approval for all non-ambulatory patients. We look forward to reviewing the comprehensive data supporting the safety and efficacy of ELEVIDYS at the 29 th Annual Congress of the World Muscle Society taking place in October, including muscle and cardiac MRI data and other biomarker results showing improvement in muscle health of treated patients,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “ Sarepta is second to no other organization in the world in its ability to launch Duchenne therapies and support the community with education, access and reimbursement. We were well prepared for all aspects of this broadened launch and all signals thus far exceed even our optimistic expectations. To that point, second quarter total net product revenues across our four approved therapies were $360.5 million, a 51% increase over the same quarter last year. Our PMO products - EXONDYS 51, VYONDYS 53 and AMONDYS 45 - continue to perform, contributing $238.8 million in net product revenues. ELEVIDYS achieved $121.7 million in net product revenue, which positions us well for significant growth in the latter half of 2024 as families complete the 3-to-5-month process from enrollment form to infusion. Tracking our launch beyond 2024, based on our early launch signals and continuing performance of our PMOs, we anticipate that net product revenues in 2025 will be in the range of $2.9 to $3.1 billion.” Second Quarter 2024 and Recent Developments: U.S. FDA expands labeled indication for ELEVIDYS : On June 20, 2024, Sarepta announced U.S. Food and Drug Administration (FDA) approval of an expansion to the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age. Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients. The FDA granted accelerated approval for non-ambulatory patients. Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial. Consistent with the accelerated approval pathway, Sarepta has committed to conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of ELEVIDYS in patients with Duchenne who are non-ambulatory. ENVISION (Study SRP-9001-303), a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne, is underway and intended to serve as this postmarketing requirement. Sarepta’s partner Roche announced that the European Medicines Agency (EMA) has initiated review of the ELEVIDYS marketing authorization application (MAA) for the treatment of Duchenne: On June 24, 2024, Roche announced that the EMA has initiated the review of the MAA for ELEVIDYS for the treatment of ambulatory patients ages 3 to 7 years and is expecting approval in 2025. Roche is responsible for commercialization of ELEVIDYS outside of the United States. Sarepta to present data from its SRP-9001 program at the 29 th Annual Congress of the World Muscle Society (WMS 2024): In October, the Company will share data from its SRP-9001 program at WMS 2024, taking place Oct. 8-12, 2024, in Prague, Czechia. The full program is available at: https://www.wms2024.com/page/programme . This list does not include Sarepta's encore or pipeline presentations nor does it include late-breaker submissions. The following have been accepted as regular submissions for SRP-9001: Presentation # Title Date, Time 19O Muscle MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 Oct. 12, 2024 1:45-2:00 AM EDT 7:45-8:00 AM CET 428P Cardiac MRI outcomes in patients with Duchenne Muscular Dystrophy treated with delandistrogene moxeparvovec: Findings from EMBARK Part 1 Oct. 9, 2024 11:15 AM-12:15 PM EDT 5:15-6:15 PM CET 424P Micro-dystrophin expression and safety with delandistrogene moxeparvovec gene therapy for DMD in a broad population: Phase 1b trial (ENDEAVOR) 425P Five-year outcomes with delandistrogene moxeparvovec in patients with Duchenne Muscular Dystrophy (DMD): a phase 1/2a study Execution on the ELEVIDYS long-term follow-up studies: The long-term follow-up studies for ELEVIDYS include ENDURE, a Phase 4 observational study that will follow individuals treated with ELEVIDYS for up to 10 years, and EXPEDITION, a Phase 3 study enrolling approximately 400 patients who were previously enrolled in ELEVIDYS clinical trials and followed for consistent safety and efficacy measures for up to 5 years. U.S. FDA grants Fast Track designation to SRP-9003 (bidridistrogene xeboparvovec): The Company announced that they received Fast Track designation for SRP-9003 an investigational gene therapy being developed for the treatment of limb-girdle muscular dystrophy Type 2E (LGMD2E/R4), or beta sarcoglycanopathy. SRP-9003 is intended to deliver a full-length beta-sarcoglycan transgene and uses the MHCK7 promoter, chosen for its ability to robustly express in the heart, which is critically important for patients with LGMD2E/R4, many of whom die from pulmonary or cardiac complications. The Fast Track designation is a process designed to facilitate the development and expedited review of drugs that treat serious conditions and fill unmet medical needs. Jerry R. Mendell, M.D., named to TIME100 Health inaugural list of 2024 most influential people in global health: TIME named renown neuromuscular researcher, physician and gene therapy pioneer Jerry R. Mendell, M.D., to the inaugural 2024 TIME100 Health, a list of 100 individuals who most influenced global health this year. Dr. Mendell was recognized for his lifetime commitment to neuromuscular disease and achievements in genetic medicine that propelled a new age of treatment for several genetic diseases, including Duchenne muscular dystrophy. The TIME100 Health list recognizes the impact, innovation, and achievement of the world’s most influential individuals in health. TIME selected a community of leaders across industries dedicated to creating tangible and credible change for a healthier population, including pioneering scientists who are steering the evolution of global health in 2024. 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. Q2 2024 Financial Highlights 1 For the Three Months Ended June 30, 2024 2023 QTD Change (in millions, except for per share amounts) $ % Total Revenues $ 362.9 $ 261.2 101.7 39 % Operating (loss) income: GAAP $ (0.7 ) $ (133.5 ) 132.8 (99 %) Non-GAAP $ 57.9 $ (75.5 ) 133.4 NM* Net income (loss): GAAP $ 6.5 $ (23.9 ) 30.4 NM* Non-GAAP $ 46.7 $ (89.9 ) 136.6 NM* Diluted earnings (loss) per share: GAAP $ 0.07 $ (0.27 ) 0.34 NM* Non-GAAP $ 0.44 $ (1.01 ) 1.45 NM* For the Six Months Ended June 30, 2024 2023 YTD Change (in millions, except for per share amounts) $ % Total Revenues $ 776.4 $ 514.7 261.7 51 % Operating income (loss): GAAP $ 34.2 $ (271.6 ) 305.8 NM* Non-GAAP $ 141.6 $ (161.2 ) 302.8 NM* Net income (loss): GAAP $ 42.6 $ (540.7 ) 583.3 NM* Non-GAAP $ 124.9 $ (177.6 ) 302.5 NM* Diluted earnings (loss) per share: GAAP $ 0.44 $ (6.11 ) 6.55 NM* Non-GAAP $ 1.16 $ (2.01 ) 3.17 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. As of June 30, 2024 As of December 31, 2023 (in millions) Cash, cash equivalents, restricted cash and investments $ 1,476.1 $ 1,691.8 Revenues Total revenues increased by $101.7 million for the three months ended June 30, 2024, compared to the same period of 2023. The increase primarily reflects the product launch of ELEVIDYS in June 2023, partially offset by a $22.3 million decrease in the amortization of the single, combined performance obligation under our collaboration agreement with F. Hoffman-La Roche Ltd.'s (“Roche”), which was fully amortized as of December 31, 2023. Total revenues increased by $261.7 million for the six months ended June 30, 2024, compared to the same period of 2023. The increase primarily reflects the product launch of ELEVIDYS in June 2023, as well as the $48.0 million of collaboration revenue recognized related to an option declined by Roche to acquire the ex-US rights to a certain external, early stage Duchenne-specific program. Additionally, for the three and six months ended June 30, 2024, we recognized $2.4 million and $8.4 million, respectively, of contract manufacturing and other revenues associated with commercial ELEVIDYS supply delivered to Roche and royalty revenue received from Roche, with no similar activity for the same periods of 2023. Cost of sales (excluding amortization of in-licensed rights) Cost of sales (excluding amortization of in-license rights) increased by $10.4 million and $26.0 million for the three and six months ended June 30, 2024, compared with the same periods of 2023. The increase in both periods primarily reflects the product launch of ELEVIDYS in June 2023. Operating expenses and others Research and development expenses decreased by $62.2 million and $107.5 million for the three and six months ended June 30, 2024, compared with the same periods of 2023. The decreases in research and development expenses for both periods primarily reflect capitalization of commercial batches of ELEVIDYS manufactured after its approval in June 2023. Non-GAAP research and development expenses decreased by $58.3 million and $100.9 million for the three and six months ended June 30, 2024, compared with the same periods of 2023. Selling, general and administrative expenses increased by $20.2 million and $36.5 million for the three and six months ended June 30, 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 for the launch of ELEVIDYS and ongoing litigation matters, the timing of charitable contributions, as well as the achievement of performance conditions related to certain Performance Stock Units. Non-GAAP selling, general and administrative expenses increased by $15.7 million and $32.9 million for the three and six months ended June 30, 2024, compared with the same periods of 2023. For the three months ended June 30, 2024, other income, net decreased by $104.6 million, compared with the same period of 2023, which primarily reflects a $102.0 million decrease in the gain on sale of a Priority Review Voucher (“PRV”) period over period. For the six months ended June 30, 2024, other income (loss), net increased by $276.5 million compared with the same period of 2023, which primarily reflects a $387.3 million loss on debt extinguishment that occurred during the six months ended June 30, 2023, offset by a $102.0 million gain on sale of a PRV, with no similar activities in 2024. Income tax expense for the three months ended June 30, 2024 and 2023, was approximately $7.1 million and $9.4 million, respectively. Income tax expense for the six months ended June 30, 2024 and 2023, was approximately $12.4 million and $13.4 million, respectively. Income tax expense for all periods presented primarily relates to state, federal and foreign income taxes. Use of Non-GAAP Measures In addition to the GAAP financial measures set forth in this press release, we have included certain non-GAAP measurements. The non-GAAP income (loss) is defined by us as GAAP net income (loss) excluding interest income, net, depreciation and amortization expense, stock-based compensation expense, the estimated income tax impact of each pre-tax non-GAAP adjustment and other items. The non-GAAP earnings (loss) per share is defined by us as non-GAAP income (loss), as defined previously, divided by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding. The non-GAAP earnings per share is calculated using diluted shares whereas the non-GAAP net loss per share is calculated using basic shares as all other instruments are anti-dilutive. The non-GAAP operating income (loss) is defined by us as GAAP operating income (loss) excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP research and development expenses are defined by us as GAAP research and development expenses excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP selling, general and administrative expenses are defined by us as GAAP selling, general and administrative expenses excluding depreciation expense, stock-based compensation expense and other items. 1. Interest, depreciation and amortization Interest income, net amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of our operations. Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to our operating performance. Amortization expense primarily associated with patent costs are amortized over a period of several years after acquisition or patent application or renewal. 2. Stock-based compensation expenses Stock-based compensation expenses represent non-cash charges related to equity awards we have granted. Although these are recurring charges to operations, we believe the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within our control. Therefore, we believe that excluding these charges facilitates comparisons of our operational performance in different periods. 3. Other items We evaluate other items of expense and income on an individual basis. We take into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relate to our ongoing business operations, and (c) whether we expect the items to continue or occur on a regular basis. These other items include impairment of strategic investments, change in fair value of contingent consideration, net, gain from sale of the PRV and loss on debt extinguishment and may include other items that fit the above characteristics in the future. We exclude from our non-GAAP results the impairment of any strategic investments as it is a non-cash item and is not considered to be a normal operating expense due to the variability of amount and lack of predictability as to the occurrence and/or timing of such impairments. We exclude from our non-GAAP results the loss on debt extinguishment, which is considered to be an infrequent and non-cash event as it is associated with a distinct financing decision and is not indicative of the performance of our core operations, which accordingly, would make it difficult to compare our results to peer companies that also provide non-GAAP disclosures. We exclude from our non-GAAP results the gain from sale of the PRV obtained as a result of the FDA accelerated approval of ELEVIDYS in June 2023 as it is a non-recurring event. We exclude from our non-GAAP results the change in fair value of contingent consideration, net related to regulatory-related contingent payments meeting the definition of a derivative to Myonexus selling shareholders as well as to academic institutions under separate license agreements as it is a non-cash item and is not considered to be normal operating expenses due to its variability of amounts and lack of predictability as to occurrence and/or timing. Beginning in the fourth quarter of 2023, amortization of in-licensed rights (formerly included within depreciation and amortization expense) and income tax (benefit) expense are no longer excluded from the non-GAAP results. We now include the income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the for the three and six months ended June 30, 2023 have been updated to reflect this change for comparability. We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We also believe these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating our performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of our financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income and loss adjustments, non-GAAP operating (loss) income, non-GAAP net income (loss), and non-GAAP diluted net earnings (loss) per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “ Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.” About EXONDYS 51 EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion, or “skipping”, of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin 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. 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 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. 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) 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. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. 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: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as 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, 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 in exons 1 to 17 and/or 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. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. 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. Preexisting 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 patients developed anti-AAVrh74 antibodies. Perform baseline testing for 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 injury, pyrexia, and thrombocytopenia. 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, products and technologies; the potential benefits of our technologies and scientific approaches; our expectation that net product revenues in 2025 will be in the range of $2.9 to $3.1 billion; the potential for our partner Roche to receive approval from the EMA in 2025 for ELEVIDYS for the treatment of ambulatory patients ages 3 to 7 years; and expected milestones and plans, including reviewing the comprehensive data supporting the safety and efficacy of ELEVIDYS at the 29 th Annual Congress of the World Muscle Society taking place in October. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; if the actual number of patients suffering from the diseases we aim to treat is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, changes in coverage and reimbursement policies of health plans and health insurers, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2023, and our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Internet Posting of Information We routinely post information that may be important to investors in the '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 Income (Loss) (unaudited, in thousands, except per share amounts) For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 Revenues: Products, net $ 360,548 $ 238,988 $ 720,032 $ 470,483 Collaboration and other 2,383 22,250 56,363 44,255 Total revenues 362,931 261,238 776,395 514,738 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 44,545 34,124 95,104 69,141 Research and development 179,690 241,890 380,086 487,569 Selling, general and administrative 138,796 118,564 265,799 229,278 Amortization of in-licensed rights 601 179 1,202 357 Total cost and expenses 363,632 394,757 742,191 786,345 Operating (loss) income (701 ) (133,519 ) 34,204 (271,607 ) Other income (loss), net: Other income, net 14,278 16,934 20,821 29,641 Gain from sale of Priority Review Voucher — 102,000 — 102,000 Loss on debt extinguishment — — — (387,329 ) Total other income (loss), net 14,278 118,934 20,821 (255,688 ) Income (loss) before income tax expense 13,577 (14,585 ) 55,025 (527,295 ) Income tax expense 7,117 9,355 12,446 13,400 Net income (loss) $ 6,460 $ (23,940 ) $ 42,579 $ (540,695 ) Earnings (loss) per share: Basic $ 0.07 $ (0.27 ) $ 0.45 $ (6.11 ) Diluted $ 0.07 $ (0.27 ) $ 0.44 $ (6.11 ) Weighted average number of shares of common stock used in computing earnings (loss) per share: Basic 94,618 88,743 94,305 88,466 Diluted 99,144 88,743 99,129 88,466 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 June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 GAAP net income (loss) $ 6,460 $ (23,940 ) $ 42,579 $ (540,695 ) Interest income, net (14,010 ) (15,980 ) (29,741 ) (28,972 ) Depreciation and amortization expense* 8,118 10,613 16,261 21,740 Stock-based compensation expense 50,482 47,377 91,174 88,627 Change in fair value on contingent consideration — (800 ) 10,100 (800 ) Gain from sale of Priority Review Voucher — (102,000 ) — (102,000 ) Loss on debt extinguishment — — — 387,329 Impairment of strategic investments — — — 321 Income tax effect of adjustments** (4,389 ) (5,172 ) (5,472 ) (3,189 ) Non-GAAP net income (loss)** $ 46,661 $ (89,902 ) $ 124,901 $ (177,639 ) GAAP net earnings (loss) per share - diluted: $ 0.07 $ (0.27 ) $ 0.44 $ (6.11 ) Add: impact of GAAP to Non-GAAP adjustments $ 0.37 $ (0.74 ) $ 0.73 $ 4.10 Non-GAAP net earnings (loss) per share - diluted*** $ 0.44 $ (1.01 ) $ 1.16 $ (2.01 ) Weighted average number of shares of common stock used in computing diluted earnings (loss) per share:**** GAAP 99,144 88,743 99,129 88,466 Non-GAAP 107,245 88,743 107,230 88,466 *Beginning in the fourth quarter of 2023, depreciation and amortization excludes amortization of in-licensed rights. Non-GAAP financial results for the three and six months ended June 30, 2023, have been updated to reflect this change for comparability. **Beginning in the fourth quarter of 2023, income tax (benefit) expense is no longer excluded from the non-GAAP results. We have replaced this metric with income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable statutory income tax rate. Refer below for a reconciliation of effective tax rates. Non-GAAP financial results for the three and six months ended June 30, 2023, have been updated to reflect this change for comparability. ***Non-GAAP earnings per share is calculated using diluted shares whereas non-GAAP net loss per share is calculated using basic shares as all other instruments are anti-dilutive. ****The difference between the weighted average number of shares of common stock used in computing diluted GAAP and non-GAAP earnings per share for the three and six months ended June 30, 2024, is a result of the exclusion of the potential share settlement of the 2027 Notes from the GAAP earnings per share as the inclusion of such shares was anti-dilutive. For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 Total effective tax rate, GAAP 8.4 % (1.7 )% 22.6 % (2.5 )% Less: impact of GAAP to Non-GAAP adjustments (4.6 ) (6.2 ) (10.0 ) (7.8 ) Total effective tax rate, Non-GAAP 3.8 % (7.9 )% 12.6 % (10.3 )% Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands) For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 GAAP research and development expenses $ 179,690 $ 241,890 $ 380,086 $ 487,569 Stock-based compensation expense (19,806 ) (21,577 ) (36,079 ) (37,990 ) Depreciation and amortization expense (5,982 ) (8,134 ) (12,028 ) (16,685 ) Non-GAAP research and development expenses $ 153,902 $ 212,179 $ 331,979 $ 432,894 For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 GAAP selling, general and administrative expenses $ 138,796 $ 118,564 $ 265,799 $ 229,278 Stock-based compensation expense (30,676 ) (25,800 ) (55,095 ) (50,637 ) Depreciation expense (2,136 ) (2,479 ) (4,233 ) (5,055 ) Non-GAAP selling, general and administrative expenses $ 105,984 $ 90,285 $ 206,471 $ 173,586 For the Three Months Ended June 30, For the Six Months Ended June 30, 2024 2023 2024 2023 GAAP operating (loss) income $ (701 ) $ (133,519 ) $ 34,204 $ (271,607 ) Stock-based compensation expense 50,482 47,377 91,174 88,627 Depreciation and amortization expense 8,118 10,613 16,261 21,740 Non-GAAP operating income (loss) $ 57,899 $ (75,529 ) $ 141,639 $ (161,240 ) Sarepta Therapeutics, Inc. Condensed Consolidated Balance Sheets (unaudited, in thousands, except share and per share data) As of June 30, 2024 As of December 31, 2023 Assets Current assets: Cash and cash equivalents $ 383,622 $ 428,430 Short-term investments 1,076,852 1,247,820 Accounts receivable, net 359,997 400,327 Inventory 485,795 322,859 Manufacturing-related deposits and prepaids 302,627 102,181 Other current assets 74,743 77,714 Total current assets 2,683,636 2,579,331 Property and equipment, net 276,200 227,154 Right of use assets 124,001 129,952 Non-current inventory 204,691 191,368 Other non-current assets 135,729 136,771 Total assets $ 3,424,257 $ 3,264,576 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 107,417 $ 164,918 Accrued expenses 350,404 314,997 Deferred revenue, current portion 122,036 50,416 Current portion of long-term debt 91,505 105,483 Other current liabilities 17,128 17,845 Total current liabilities 688,490 653,659 Long-term debt 1,134,810 1,132,515 Lease liabilities, net of current portion 143,601 140,965 Deferred revenue, net of current portion 325,000 437,000 Contingent consideration 48,200 38,100 Other non-current liabilities 7,087 3,000 Total liabilities 2,347,188 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; 95,282,600 and 93,731,831 issued and outstanding at June 30, 2024, and December 31, 2023, respectively 10 9 Additional paid-in capital 5,481,723 5,304,623 Accumulated other comprehensive (loss) income, net of tax (1,030 ) 918 Accumulated deficit (4,403,634 ) (4,446,213 ) Total stockholders’ equity 1,077,069 859,337 Total liabilities and stockholders’ equity $ 3,424,257 $ 3,264,576

Net product revenues for the first quarter 2024 totaled $359.5 million, a 55% increase over the same quarter of the prior year ELEVIDYS net revenues for the quarter totaled $133.9 million Achieved GAAP Earnings of $36.1 million for the first quarter 2024 and non-GAAP Earnings of $78.2 million for the same period CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the first quarter 2024. “We are pleased to announce another strong quarter of growth from our four approved therapies, posting net product revenue of $359.5 million, a 55% increase over the same quarter of the prior year, and achieving profitability on a GAAP and non-GAAP basis,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “In particular, our recently approved gene therapy, ELEVIDYS, achieved nearly $134.0 million in net product revenue in the quarter. Although its initial label is quite narrow, ELEVIDYS has posted cumulative sales of over $334.0 million since its approval in June of last year, far exceeding performance of all other gene therapies approved in the last few years combined. Working with the FDA, we continue to productively prosecute our BLA supplement to expand the ELEVIDYS addressable population, with a target action date of June 21, 2024. If successful, 2024 could be the most profound year yet in our fight against the effects of Duchenne muscular dystrophy and a bellwether for the transformative potential of gene therapy for rare disease.” First Quarter 2024 and Recent Developments: U.S. FDA review of efficacy supplement to expand the ELEVIDYS indication is ongoing: The Company is currently awaiting U.S. Food and Drug Administration (FDA) Priority Review of its efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl) where the review goal date is June 21, 2024. At acceptance of the BLA in February, the Agency also confirmed they are not planning to hold an advisory committee to discuss the supplement. The goals of the efficacy supplement are twofold: To expand the labeled indication for ELEVIDYS as follows: “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene.” To convert the ELEVIDYS accelerated approval to a traditional approval. Real world evidence published in Muscle & Nerve (March 2024) finds eteplirsen treatment associated with longer survival compared to natural history: The publication details real-world evidence showing that treatment with eteplirsen resulted in statistically significant survival benefits compared to a controlled natural history comparator group of Duchenne patients. In the analysis the survival age of the eteplirsen treated patients (n=579) was 32.8 years, 5.4 years longer than the median survival age of 27.4 years in the Duchenne natural history studies (n=1,224). Additionally, earlier initiation of eteplirsen treatment and longer duration of treatment were associated with increases in the survival benefit. Other findings from the analysis include: Overall, eteplirsen-treated patients had a 66% reduction in risk of death compared with natural history control group. Longer duration of treatment with eteplirsen was associated with greater survival benefit, with patients treated with eteplirsen for longer than four years demonstrating an 85% lower risk of death compared to patients who had less than two years of treatment with eteplirsen. Patients receiving eteplirsen treatment for more than four years survived 32.8 years (95% confidence interval), and patients receiving eteplirsen between two to four years survived 29.4 years (95% confidence interval). Patients treated with eteplirsen for less than two years had a median survival of 28.1 years, compared to natural history of 27.4 years. The median survival age has not yet been reached in patients taking eteplirsen for longer than two years. In patients aged 10 to 28 years, for whom deaths are most likely to be observed, eteplirsen treatment had a 42% lower risk of death than age-matched natural history patients. These findings add to the large and growing body of real-world evidence generated for eteplirsen, where treatment is associated with delays in time to loss of ambulation, improvements in lung function and various quality of life measures when compared to standard of care or natural history controls. Conference Call The event will be webcast live under the investor relations section of Sarepta's website at and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. Q1 2024 Financial Highlights1 For the Three Months Ended March 31, 2024 2023 Change Change (in millions, except for per share amounts) $ % Total Revenues $ 413.5 $ 253.5 $ 160.0 63 % Operating income (loss): GAAP $ 34.9 $ (138.1 ) $ 173.0 NM* Non-GAAP $ 83.7 $ (85.7 ) $ 169.4 NM* Net income (loss): GAAP $ 36.1 $ (516.8 ) $ 552.9 NM* Non-GAAP $ 78.2 $ (87.7 ) $ 165.9 NM* Diluted earnings (loss) per share GAAP $ 0.37 $ (5.86 ) $ 6.23 NM* Non-GAAP $ 0.73 $ (0.99 ) $ 1.72 NM* *NM: not meaningful [1] For an explanation of our use of non-GAAP financial measures, please refer to the "Use of Non-GAAP Financial Measures" section later in this press release and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measures, see the table at the end of this press release. As of March 31, 2024 As of December 31, 2023 (in millions) Cash, cash equivalents, and investments $ 1,440.3 $ 1,691.8 Revenues Total revenues increased by $160.0 million for the three months ended March 31, 2024 compared to the same period of 2023. The increase primarily reflects the increasing demand for ELEVIDYS as well as the $48.0 million of collaboration revenue recognized related to F. Hoffman-La Roche Ltd.'s ("Roche") declined option to acquire the ex-US rights to a certain external, early-stage Duchenne development program. Additionally, for the three months ended March 31, 2024, we recognized $5.8 million of contract manufacturing collaboration revenue associated with commercial ELEVIDYS supply delivered to Roche, with no similar activity for the same period of 2023. Cost of sales (excluding amortization of in-licensed rights) Cost of sales (excluding amortization of in-license rights) increased by $15.5 million for the three months ended March 31, 2024, compared with the same period of 2023, which primarily reflects continued increasing demand for our products, an increase in royalty payments related to ELEVIDYS sales, as well as an increase in the write-offs of certain batches of our products not meeting our quality specifications. The majority of inventory costs for ELEVIDYS sold during the three months ended March 31, 2024 were previously expensed as research and development expense as they were incurred prior to the regulatory approval of ELEVIDYS in June 2023. Operating expenses and others For the three months ended March 31, 2024, research and development expenses decreased by $45.3 million compared with the same period of 2023. The changes in research and development expenses primarily reflect capitalization of commercial batches of ELEVIDYS manufactured after its approval in June 2023, partially offset by a ramp-up of our late-stage clinical trials. For the three months ended March 31, 2024, non-GAAP research and development expenses decreased by $42.6 million compared with the same period of 2023. Selling, general and administrative expenses increased $16.3 million for the three months ended March 31, 2024 compared with the same period of 2023. The increase is primarily driven by professional services used for the launch of ELEVIDYS and ongoing litigation matters, as well as the timing of charitable contributions. Non-GAAP selling, general and administrative expenses increased $17.2 million for the three months ended March 31, 2024 compared with the same period of 2023. For the three months ended March 31, 2024, other income, net decreased by $6.2 million compared with the same period of 2023, which primarily reflects an increase in the fair value of our contingent consideration liability. Income tax expense for the three months ended March 31, 2024 and 2023, was approximately $5.3 million and $4.0 million, respectively. Income tax expense for all periods presented primarily relates to federal and state income taxes. Use of Non-GAAP Measures In addition to the GAAP financial measures set forth in this press release, we have included certain non-GAAP measurements. The non-GAAP income (loss) is defined by us as GAAP net income (loss) excluding interest income, net, depreciation and amortization expense, stock-based compensation expense, the estimated income tax impact of each pre-tax non-GAAP adjustment and other items. The non-GAAP operating income (loss) is defined as GAAP operating income (loss) excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP research and development expenses are defined by us as GAAP research and development expenses excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP selling, general and administrative expenses are defined by us as GAAP selling, general and administrative expenses excluding depreciation expense, stock-based compensation expense and other items. 1. Interest, depreciation and amortization Interest income, net amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of our operations. Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to our operating performance. Amortization expense primarily associated with patent costs are amortized over a period of several years after acquisition or patent application or renewal. 2. Stock-based compensation expenses Stock-based compensation expenses represent non-cash charges related to equity awards we have granted. Although these are recurring charges to operations, we believe the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within our control. Therefore, we believe that excluding these charges facilitates comparisons of our operational performance in different periods. 3. Other items We evaluate other items of expense and income on an individual basis. We take into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relate to our ongoing business operations, and (c) whether we expect the items to continue or occur on a regular basis. These other items include impairment of strategic investments, change in fair value of contingent consideration, net, gain from sale of the PRV and loss on debt extinguishment and may include other items that fit the above characteristics in the future. We exclude from our non-GAAP results the impairment of any strategic investments as it is a non-cash item and is not considered to be a normal operating expense due to the variability of amount and lack of predictability as to the occurrence and/or timing of such impairments. We exclude from our non-GAAP results the loss on debt extinguishment, which is considered to be an infrequent and non-cash event as it is associated with a distinct financing decision and is not indicative of the performance of our core operations, which accordingly, would make it difficult to compare our results to peer companies that also provide non-GAAP disclosures. We exclude from our non-GAAP results the change in fair value of contingent consideration, net related to regulatory-related contingent payments meeting the definition of a derivative to Myonexus selling shareholders as well as to academic institutions under separate license agreements as it is a non-cash item and is not considered to be normal operating expenses due to its variability of amounts and lack of predictability as to occurrence and/or timing. Beginning in the fourth quarter of 2023, amortization of in-licensed rights (formerly included within depreciation and amortization expense) and income tax (benefit) expense are no longer excluded from the non-GAAP results. We now include the income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the first quarter of 2023 have been updated to reflect this change for comparability. We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We also believe these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating our performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of our financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income and loss adjustments, non-GAAP net income (loss), and non-GAAP diluted net earnings (loss) per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.” About EXONDYS 51 EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion, or “skipping”, of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. EXONDYS 51 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information About EXONDYS 51 Hypersensitivity reactions, bronchospasm, chest pain, cough, tachycardia, and urticaria have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended. The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting. For further information, please see the full Prescribing Information. About VYONDYS 53 VYONDYS 53 (golodirsen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. VYONDYS 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information for VYONDYS 53 Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the VYONDYS 53 therapy. Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. Adverse reactions observed in at least 20% of treated patients and greater than placebo were (VYONDYS 53, placebo): headache (41%, 10%), pyrexia (41%, 14%), fall (29%, 19%), abdominal pain (27%, 10%), nasopharyngitis (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%). Other adverse reactions that occurred at a frequency greater than 5% of VYONDYS 53-treated patients and at a greater frequency than placebo were: administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture. For further information, please see the full Prescribing Information. About AMONDYS 45 AMONDYS 45 (casimersen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 45 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. AMONDYS 45 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. AMONDYS 45 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information for AMONDYS 45 CONTRAINDICATION: Known hypersensitivity to casimersen or any of the inactive ingredients. Instances of hypersensitivity including angioedema and anaphylaxis have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity: Hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. If a hypersensitivity reaction occurs, institute appropriate medical treatment, and consider slowing the infusion, interrupting, or discontinuing the AMONDYS 45 infusion and monitor until the condition resolves. Kidney Toxicity: Kidney toxicity was observed in animals who received casimersen. Although kidney toxicity was not observed in the clinical studies with AMONDYS 45, kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking AMONDYS 45. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting AMONDYS 45. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting AMONDYS 45. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio (UPCR) every three months. Only urine expected to be free of excreted AMONDYS 45 should be used for monitoring of urine protein. Urine obtained on the day of AMONDYS 45 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any AMONDYS 45 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. Adverse Reactions: Adverse reactions occurring in at least 20% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were (AMONDYS 45, placebo): upper respiratory infections (65%, 55%), cough (33%, 26%), pyrexia (33%, 23%), headache (32%, 19%), arthralgia (21%, 10%), and oropharyngeal pain (21%, 7%). Other adverse reactions that occurred in at least 10% of patients treated with AMONDYS 45 and at least 5% more frequently than in the placebo group were: ear pain, nausea, ear infection, post-traumatic pain, and dizziness and light-headedness. For further information, please see the full Prescribing Information. About ELEVIDYS (delandistrogene moxeparvovec-rokl) ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV) based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the dystrophin gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is a one-time infusion indicated for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene and is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin in skeletal muscle observed in patients treated with ELEVIDYS. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. ELEVIDYS has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. ELEVIDYS has been evaluated in four clinical studies: SRP-9001-101, SRP-9001-102, SRP-9001-103 (ENDEAVOR) and SRP-9001-301 (EMBARK). Accelerated approval of ELEVIDYS was primarily based on data from SRP-9001-102 and SRP-9001-103. The EMBARK study serves as the postmarketing confirmatory trial. Important Safety Information for ELEVIDYS CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS. Administration of ELEVIDYS may result in elevations of liver enzymes (e.g., GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, a consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness including dysphagia, dyspnea and hypophonia were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene between exons 1 to 17 and exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea or hypophonia as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Pre-existing Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all subjects developed anti-AAVrh74 antibodies. Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥ 5%) reported in clinical studies were vomiting, nausea, liver function test increased, pyrexia, and thrombocytopenia. Sarepta is responsible for global development and manufacturing for ELEVIDYS, and distribution within the U.S. will commence immediately. In December 2019, Sarepta partnered with Roche to accelerate access to ELEVIDYS for patients outside the United States. ELEVIDYS is approved under accelerated review based on expression of ELEVIDYS micro-dystrophin in skeletal muscle. Continued approval for this indication in this and other age groups will be contingent upon verification of a clinical benefit in confirmatory trials. ELEVIDYS has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. For further information, please see the full Prescribing Information. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit or follow us on LinkedIn, X (formerly Twitter), Instagram and Facebook. Forward-Looking Statements In order to provide Sarepta’s investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,” “looks,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our future operations, financial performance and projections, business plans, market opportunities, priorities and research and development programs and technologies; the potential benefits of our technologies and scientific approaches; the potential to bring a better life to the patient communities we serve and for 2024 to be the most profound year yet in the fight against the effects of Duchenne muscular dystrophy and a bellwether for the transformative potential of gene therapy for rare disease; our understanding that FDA does not plan to hold an advisory committee regarding the ELEVIDYS expansion; the potential for our comprehensive approach of measuring total antibodies to help improve the safety and efficacy of AAV-based gene transfer therapies; and milestones and plans, including working with FDA to expand the ELEVIDYS addressable population, with a target action date of June 21, 2024. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; if the actual number of patients suffering from the diseases we aim to treat is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2023 and our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at . We encourage investors and potential investors to consult our website regularly for important information about us. Sarepta Therapeutics, Inc. Condensed Consolidated Statements of Operations (unaudited, in thousands, except per share amounts) For the Three Months Ended March 31, 2024 2023 Revenues: Products, net $ 359,484 $ 231,495 Collaboration and other 53,980 22,005 Total revenues 413,464 253,500 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 50,559 35,017 Research and development 200,396 245,679 Selling, general and administrative 127,003 110,714 Amortization of in-licensed rights 601 178 Total cost and expenses 378,559 391,588 Operating income (loss) 34,905 (138,088 ) Other income (loss), net: Loss on debt extinguishment — (387,329 ) Other income, net 6,543 12,707 Total other income (loss), net 6,543 (374,622 ) Income (loss) before income tax expense 41,448 (512,710 ) Income tax expense 5,329 4,045 Net income (loss) $ 36,119 $ (516,755 ) Net earnings (loss) per share: Basic $ 0.38 $ (5.86 ) Diluted $ 0.37 $ (5.86 ) Weighted average number of shares of common stock used in computing net earnings (loss) per share: Basic 93,991 88,186 Diluted 99,114 88,186 Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands, except per share amounts) For the Three Months Ended March 31, 2024 2023 GAAP net income (loss) $ 36,119 $ (516,755 ) Interest income, net (15,731 ) (12,992 ) Depreciation and amortization expense* 8,143 11,127 Stock-based compensation expense 40,692 41,250 Change in fair value on contingent consideration 10,100 — Loss on debt extinguishment — 387,329 Impairment of strategic investments — 321 Income tax effect of adjustments** (1,083 ) 1,983 Non-GAAP net income (loss)** $ 78,240 $ (87,737 ) GAAP net earnings (loss) per share - diluted: $ 0.37 $ (5.86 ) Add: impact of GAAP to Non-GAAP adjustments 0.36 4.87 Non-GAAP net earnings (loss) per share - diluted*** $ 0.73 $ (0.99 ) Weighted average number of shares of common stock used in computing diluted earnings (loss) per share:**** GAAP 99,114 88,186 Non-GAAP 107,215 88,186 *Beginning in the fourth quarter of 2023, depreciation and amortization excludes amortization of in-licensed rights. Non-GAAP financial results for the first quarter 2023 have been updated to reflect this change for comparability. **Beginning in the fourth quarter of 2023, income tax (benefit) expense is no longer excluded from the non-GAAP results. We have replaced this metric with income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable statutory income tax rate. Refer below for a reconciliation of effective tax rates. Non-GAAP financial results for the first quarter 2023 have been updated to reflect this change for comparability. ***Non-GAAP earnings per share is calculated using diluted shares whereas non-GAAP net loss per share is calculated using basic shares as all other instruments are anti-dilutive. ****The difference between the weighted average number of shares of common stock used in computing diluted GAAP and non-GAAP earnings per share for the three months ended March 31, 2024, is a result of the exclusion of the potential share settlement of the 2027 Notes from the GAAP earnings per share as the inclusion of such shares was anti-dilutive. For the Three Months Ended March 31, 2024 2023 Total effective tax rate, GAAP 14.2 % (0.8 )% Less: impact of GAAP to Non-GAAP adjustments (5.4 ) (1.6 ) Total effective tax rate, Non-GAAP 8.8 % (2.4 )% Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands) For the Three Months Ended March 31, 2024 2023 GAAP research and development expenses $ 200,396 $ 245,679 Stock-based compensation expense (16,273 ) (16,413 ) Depreciation and amortization expense (6,046 ) (8,551 ) Non-GAAP research and development expenses $ 178,077 $ 220,715 For the Three Months Ended March 31, 2024 2023 GAAP selling, general and administrative expenses $ 127,003 $ 110,714 Stock-based compensation expense (24,419 ) (24,837 ) Depreciation expense (2,097 ) (2,576 ) Non-GAAP selling, general and administrative expenses $ 100,487 $ 83,301 Sarepta Therapeutics, Inc. Condensed Consolidated Balance Sheets (unaudited, in thousands, except share and per share data) As of March 31, 2024 As of December 31, 2023 Assets Current assets: Cash and cash equivalents $ 427,290 $ 428,430 Short-term investments 963,453 1,247,820 Accounts receivable, net 378,806 400,327 Inventory 373,530 322,859 Manufacturing-related deposits and prepaids 238,821 102,181 Other current assets 82,965 77,714 Total current assets 2,464,865 2,579,331 Property and equipment, net 249,302 227,154 Right of use assets 126,269 129,952 Non-current inventory 207,542 191,368 Other non-current assets 176,407 136,771 Total assets $ 3,224,385 $ 3,264,576 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 91,536 $ 164,918 Accrued expenses 283,317 314,997 Deferred revenue, current portion 112,000 50,416 Current portion of long-term debt 105,586 105,483 Other current liabilities 16,270 17,845 Total current liabilities 608,709 653,659 Long-term debt 1,133,660 1,132,515 Lease liabilities, net of current portion 140,102 140,965 Deferred revenue, net of current portion 325,000 437,000 Contingent consideration 48,200 38,100 Other non-current liabilities 7,522 3,000 Total liabilities 2,263,193 2,405,239 Stockholders’ equity: Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding — — Common stock, $0.0001 par value, 198,000,000 shares authorized; 94,490,157 and 93,731,831 issued and outstanding at March 31, 2024 and December 31, 2023, respectively 9 9 Additional paid-in capital 5,371,968 5,304,623 Accumulated other comprehensive (loss) income, net of tax (691 ) 918 Accumulated deficit (4,410,094 ) (4,446,213 ) Total stockholders’ equity 961,192 859,337 Total liabilities and stockholders’ equity $ 3,224,385 $ 3,264,576

LNZ100 selected as lead candidate Primary endpoint was met with 71% of participants dosed with LNZ100 achieving three-lines or greater improvement at 3 hours Rapid onset and long duration shown with 71% of participants achieving three-lines or greater improvement at 30 minutes and 40% at 10 hours New Drug Application submission anticipated in mid-2024 Company to host a conference call and webcast today at 8:00 a.m. ET SAN DIEGO--(BUSINESS WIRE)-- LENZ Therapeutics, Inc. (Nasdaq: LENZ or “LENZ” or the “Company”), a late clinical-stage biopharmaceutical company focused on developing the first aceclidine-based eye drop to improve near vision in people with presbyopia, today announced positive topline results from its Phase 3 CLARITY study of two investigational formulations of aceclidine, LNZ100 and LNZ101, for the treatment of presbyopia, the inevitable loss of near vision that impacts the daily lives of nearly all people over 45. In Phase 3 safety and efficacy trials (CLARITY 1 and 2), our lead product candidate LNZ100 (1.75% aceclidine) achieved the primary endpoints and key secondary endpoints, with statistically significant three-lines or greater improvement in Best Corrected Distance Visual Acuity (BCDVA) at near, without losing one-line or more in distance visual acuity. In the vehicle-controlled CLARITY 2 trial, the Day 1 results showed (all p<0.0001): Rapid onset: 71% achieved three-lines or greater improvement at 30 minutes. Primary endpoint: 71% achieved three-lines or greater improvement at 3 hours. Long duration: 40% achieved three-lines or greater improvement at 10 hours. Near vision improvement was reproducible and consistent across both CLARITY 1 and 2 throughout the four-week study periods. LNZ100 was well-tolerated with no serious treatment-related adverse events observed in the over 30,000 treatment days across all three CLARITY trials. LNZ101 showed similar results, including achieving primary and secondary endpoints in both CLARITY 1 and 2, but did not show superiority to LNZ100. Based on these results, LENZ selected LNZ100 as its lead product candidate, for which it plans to submit a New Drug Application (NDA) in mid-2024. “We are very pleased with the outcome of the CLARITY trials, and most importantly the strong efficacy and safety pro LNZ100 observed in patients with presbyopia. We would like to thank our investigators, clinical sites, and all participants in our study,” said Eef Schimmelpennink, President and Chief Executive Officer of LENZ Therapeutics. “We believe these data support LNZ100 as a potential best-in-class therapy for the treatment of presbyopia. The high responder rate, rapid onset and long duration across a broad range of presbyopes ranging from 45 to 75 years of age and having a refractive range from -4.0 to +1.0D SE are consistent with features that patients are expecting from an effective treatment option. Based on these highly encouraging data, we will direct our focus towards our NDA submission in mid-2024 for LNZ100, and preparations for commercialization in second half of 2025 upon FDA approval, with the goal of moving closer to helping many of the 128 million people experiencing symptoms of presbyopia in the United States.” “These positive CLARITY study data build on the compelling results from our INSIGHT trials, demonstrating a robust safety and efficacy pro the use of aceclidine to treat presbyopia,” said Marc Odrich, Chief Medical Officer of LENZ Therapeutics. “Presbyopes often experience an abrupt change in their daily life as the symptoms become progressively pronounced starting in their mid-40s, when reading glasses or other corrective aids often become necessary to read, text or conduct close-up work. The statistically significant data and clinically meaningful outcomes observed in the CLARITY trials support the potential paradigm-shifting impact LNZ100 can have as an alternative and convenient therapeutic option to reading glasses.” CLARITY Phase 3 Study Topline Data Highlights: CLARITY is a Phase 3 multi-center, double-masked, randomized, controlled, efficacy and safety study for LNZ100 and LNZ101 for the treatment of presbyopia. It is comprised of two six-week efficacy trials, CLARITY 1 and 2, and a six-month safety trial, CLARITY 3. The trials enrolled a total of 1,059 participants ranging from ages 45 to 75, and a refractive range of -4.0D SE to +1.0D SE, and included users who previously had LASIK surgery or are pseudophakes. The primary efficacy endpoint in both CLARITY 1 and 2 is the percentage of participants who achieve three-lines or greater improvement in BCDVA at near without losing one-line (5 letters or more of distance vision) at 3 hours post-treatment. In both the vehicle-controlled CLARITY 2 trial and the brimonidine-controlled CLARITY 1 trial, our lead product candidate LNZ100 (1.75% aceclidine) achieved all primary and secondary near vision improvement endpoints, including demonstrating (in all cases, p<0.0001): Rapid onset: at 30 minutes, for CLARITY 2, 71% and 91% of participants achieved three- and two-lines or greater improvement, respectively, and for CLARITY 1, 72% and 87% of participants achieved three- and two-lines or greater improvement, respectively; At 3 hours (primary endpoint for three-lines): for CLARITY 2, 71% and 91% of participants achieved three- and two-lines or greater improvement, respectively, and for CLARITY 1, 64% and 83% of participants achieved three- and two-lines or greater improvement, respectively; and Long duration: at 10 hours, for CLARITY 2, 40% and 69% of participants achieved three- and two-lines or greater improvement, respectively, and for CLARITY 1, 27% and 61% of participants achieved three- and two-lines or greater improvement, respectively. Near vision improvement was reproducible and consistent across both CLARITY 1 and 2 throughout the four-week study periods. Additionally, in CLARITY 2, nearly all (95%) participants who received LNZ100 achieved the clinically meaningful two-lines or greater improvement (p<0.0001) at 1 hour post-treatment. LNZ100 also demonstrated statistically significant (p<0.0001) improvement of 2-4 letters on distance vision in normal light and no negative impact to distance vision in low light at all time points. LNZ100 was well-tolerated with no treatment-related serious adverse events observed in the over 30,000 treatment days across all three CLARITY trials. The only reported adverse events with an incidence at 5% or more were installation site irritation, visual impairment and hyperemia which were 100% characterized by participants as mild, and headaches which were characterized as mild by 89% of participants. We believe these adverse events were transient, consistent with those observed in previous trials. On day 28 of the CLARITY 1 and 2 trials, 223 participants who received LNZ100 were surveyed on their experience. 90% indicated they noticed an improvement in their near vision and 75% said they would want to continue to use these eye drops after the study, of which 81% noted they expected to use them 4-7 days a week. We believe these patient survey outcomes further confirm the potential commercial opportunity for LNZ100. Based on these results, LENZ Therapeutics anticipates submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for LNZ100 as a treatment for presbyopia mid-2024. Additional results from the CLARITY study will be presented at future medical meetings. Conference Call Information The Company will host a conference call and webcast today, Wednesday, April 3, 2024, at 8:00 a.m. ET to discuss the topline results. The live webcast and materials from today’s conference call can be accessed here and on the LENZ Therapeutics website at in the Investors & Media section or by calling 877-315-3033 or 215-268-9883. A replay of the webcast will be archived and available for 30 days following the event. About Presbyopia Presbyopia is the inevitable loss of near vision associated with aging and impacts the daily lives of nearly all people over 45. In the United States, the estimated addressable population who suffer from this condition, known as presbyopes, is 128 million, almost four times the number of individuals suffering from dry eye disease and three times the number of individuals suffering from childhood myopia, macular degeneration, diabetic retinopathy, and glaucoma combined. About LENZ Therapeutics LENZ Therapeutics is a late clinical-stage biopharmaceutical company focused on developing the first aceclidine-based eye drop to improve vision in patients diagnosed with presbyopia. LENZ’s product candidate LNZ100 is a preservative-free, single-use, once-daily eye drop containing aceclidine. The Phase 3 CLARITY trials of LNZ100 were completed in March 2024 and NDA submission is planned in mid-2024. LENZ is committed to commercializing a potential best-in-class pharmaceutical presbyopia solution that enhances vision for “all eyes, all day.” LENZ is headquartered in San Diego, California. For more information, visit: LENZ-tx.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of federal securities laws. You can identify forward-looking statements by words such as “may,” “will,” “could,” “can,” “would,” “should,” “expect,” “intend,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “project,” “potential,” “poised,” “continue,” “ongoing” or the negative of these terms or other comparable terminology, but not all forward-looking statements will contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding the potential of LNZ100 to have best-in-class performance; our plans relating to the clinical development of our product candidates, including statements regarding the timing, presentation and reporting of data from our clinical trials and studies and the timing of a potential NDA submission for LNZ100; the size of the addressable population for our product candidates; our expectations regarding the commercial opportunity and beneficial characteristics of our product candidates; and our plans regarding commercialization of LNZ100, if approved. These statements are based on numerous assumptions concerning the development of LENZ’s products and target markets and involve substantial risks, uncertainties and other factors that may cause actual results, levels of activity, performance or achievement to be materially different from the information expressed or implied by these forward-looking statements, including those risk factors described in the final 424B3 proxy statement/prospectus filed with the SEC on February 13, 2024. We cannot assure you that the forward-looking statements in this press release or the assumptions upon which they are based will prove to be accurate. The forward-looking statements in this press release are as of the date of this press release. Except as otherwise required by applicable law, LENZ disclaims any duty to update any forward-looking statements. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release. View source version on businesswire.com: Contacts Dan Chevallard LENZ Therapeutics IR@LENZ-Tx.com Janhavi Mohite Stern Investor Relations, Inc. janhavi.mohite@sternir.com Source: LENZ Therapeutics, Inc. View this news release online at: