Immune-mediated necrotising myopathy

$374 million in fourth quarter and $1.2 billion in full year global net product sales sBLA for VYVGART® Hytrulo for CIDP accepted for priority review by FDA with PDUFA target action date of June 21, 2024 On track to report data from six Phase 2 proof-of-concept trials by end of 2024 Management to host conference call today at 2:30 pm CET (8:30 am ET) February 29, 2024, 7:00 am CET Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today reported financial results for the full year 2023 and provided a fourth quarter business update. “argenx reached thousands of new patients and their families in 2023 by delivering on our commitment to make VYVGART available to the global MG community,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx. “This expansion demonstrates that VYVGART has the potential to address the high unmet need for innovation in patients suffering from MG, and moves us closer to sustainability as we build an integrated immunology company. Clinically, we generated significant data through multiple study readouts, achieving key milestones for both the CIDP and MMN patient communities and importantly advancing our second molecule, empasiprubart. Looking forward to 2024, we will act with a continued sense of purpose to expand our patient reach. We will use the learnings and momentum from our gMG launch to strategically lay the groundwork for a potential CIDP approval, leveraging our current infrastructure and deep relationships in the neurology community to position VYVGART SC for success. CIDP patients have been waiting for innovation, and we are eager to translate the transformative ADHERE data into potential benefit for patients as quickly as possible.” FOURTH QUARTER 2023 AND RECENT BUSINESS UPDATE Reaching More Patients with VYVGART VYVGART® (efgartigimod alfa-fcab) is a first-in-class antibody fragment targeting the neonatal Fc receptor (FcRn), and is now approved in more than 30 countries globally for the treatment of generalized myasthenia gravis (gMG). VYVGART subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) is approved in the U.S. (as VYVGART Hytrulo), Japan (as VYVDURA®) and Europe, making VYVGART the only gMG treatment available as both an IV and simple SC injection. argenx is planning to reach more patients commercially through its multi-dimensional expansion efforts, including patients earlier in the MG treatment paradigm and new patient populations through global regulatory approvals for MG and the expansion of use to treat additional autoimmune indications. Generated global net product revenues (inclusive of both VYVGART and VYVGART SC) of $374 million in the fourth quarter and $1.2 billion in the full year of 2023Medicines and Healthcare products Regulatory Agency (MHRA) approved VYVGART SC in the United Kingdom for the treatment of adult patients with gMG on February 6, 2024, with self-administrationMinistry of Health, Labour and Welfare (MHLW) approved VYVDURA in Japan for the treatment of adult patients with gMG, inclusive of seronegative patients, on January 18, 2024, with self-administrationDecisions on regulatory approvals of VYVGART for gMG expected in Switzerland, Australia, Saudi Arabia and South Korea by end of 2024Decision on approval of VYVGART SC for gMG in China through Zai Lab expected by end of 2024Decision on approval of VYVGART for primary immune thrombocytopenia (ITP) in Japan expected in first quarter of 2024Supplemental Biologics License Application (sBLA) for VYVGART Hytrulo accepted for priority review by FDA for chronic inflammatory demyelinating polyneuropathy (CIDP); Prescription Drug User Fee Act (PDUFA) target action date of June 21, 2024Regulatory submissions of VYVGART SC for CIDP in Japan, Europe, China and Canada expected in 2024Registrational studies to expand VYVGART label into broader MG populations, including in seronegative patients, expected to start in 2024Update on pre-filled syringe development expected in first half of 2024; ongoing clinical studies expected to support potential approval in gMG and CIDP in 2024 Advancing Current Pipeline argenx continues to demonstrate breadth and depth within its immunology pipeline and is advancing multiple pipeline-in-a-product candidates. With efgartigimod, argenx is solidifying its leadership in FcRn and is on track to be approved or in development in 15 autoimmune indications by 2025. Beyond efgartigimod, argenx is advancing its earlier stage pipeline programs, including empasiprubart (C2 inhibitor) with Phase 2 studies ongoing in multifocal motor neuropathy (MMN), delayed graft function (DGF) and dermatomyositis (DM). In addition, argenx is evaluating ARGX-119, a muscle-specific kinase (MuSK) agonist in both congenital myasthenic syndrome (CMS) and amyotrophic lateral sclerosis (ALS). Evaluation ongoing to determine path forward in BALLAD study evaluating efgartigimod in bullous pemphigoid (BP), with an update expected in 2024Topline data from Phase 2 RHO study evaluating efgartigimod in primary Sjogren’s syndrome expected in first half of 2024Topline data from Phase 2 ALPHA study evaluating efgartigimod in post-COVID-19 postural orthostatic tachycardia syndrome (PC-POTS) expected in first half of 2024Topline data from seamless Phase 2/3 ALKIVIA study evaluating efgartigimod across three myositis subsets (immune-mediated necrotizing myopathy (IMNM), anti-synthetase syndrome (ASyS), and DM) expected in second half of 2024Full Phase 2 topline data from ARDA study evaluating empasiprubart in MMN expected to be shared in 2024; cohort 2 is ongoing to determine dose response ahead of Phase 3 study startPhase 1 study of ARGX-119 ongoing in healthy volunteers; subsequent Phase 1b/2a trials planned to assess early signal detection in patients with CMS and ALS in 2024 Leveraging Repeatable Innovation Playbook to Drive Long-Term Pipeline Growth argenx continues to invest in its discovery engine, the Immunology Innovation Program (IIP), to drive long-term sustainable pipeline growth. Through the IIP, four new pipeline candidates have been nominated, including: ARGX-213 targeting FcRn and further solidifying argenx’s leadership in this new class of medicine; ARGX-121 and ARGX-220, which are first-in-class targets broadening argenx’s focus across the immune system; and ARGX-109, targeting IL-6, which plays an important role in inflammation. On track to file four investigational new drug (IND) applications by end of 2025Received $30M milestone from AbbVie for advancement of ABBV-151 (ARGX-115) to Phase 2 FOURTH QUARTER AND FULL YEAR 2023 FINANCIAL RESULTS argenx SE UNAUDITED CONDENSED CONSOLIDATED STATEMENTS OF PROFIT OR LOSS Three Months Ended December 31, Twelve Months Ended December 31, (in thousands of $ except for shares and EPS) 2023 2022 2023 2022Product net sales $374,351 $173,396 $1,190,783 $400,720Collaboration revenue 32,486 764 35,533 10,026Other operating income 11,003 7,956 42,278 34,520Total operating income $417,840 $182,116 $1,268,594 $445,267 Cost of sales $(39,477) $(12,786) $(117,835) $(29,431)Research and development expenses (306,373) (147,798) (859,492) (663,366)Selling, general and administrative expenses (208,826) (135,287) (711,905) (472,132)Loss from investment in joint venture (1,788) (677) (4,411) (677)Total operating expenses (556,464) (296,548) (1,693,643) (1,165,607) Operating loss $(138,624) $(114,432) $(425,049) $(720,341) Financial income $40,308 $13,925 $107,386 $27,665Financial expense (280) (990) (906) (3,906)Exchange gains/(losses) 37,418 60,259 14,073 (32,732) Loss for the period before taxes $(61,178) $(41,238) $(304,496) $(729,314)Income tax benefit / (expense) $(37,994) $2,625 $9,443 $19,720Loss for the period $(99,172) $(38,613) $(295,053) $(709,594)Loss for the year attributable to: Owners of the parent $(99,172) $(38,613) $(295,053) $(709,594)Weighted average number of shares outstanding 59,118,827 55,364,124 57,169,253 54,381,371Basis and diluted (loss) per share (in $) (1.68) (0.70) (5.16) (13.05)Net increase/(decrease) in cash, cash equivalents and current financial assets compared to year-end 2022 and 2021 $987,296 $(144,180)Cash and cash equivalents and current financial assets at the end of the period $3,179,844 $2,192,548 DETAILS OF THE FINANCIAL RESULTS Total operating income for the fourth quarter and full year in 2023 was $418 million and $1,269 million, respectively, compared to $182 million and $445 million for the same periods in 2022, and mainly consists of: Product net sales of VYVGART and VYVGART SC for the fourth quarter and full year in 2023, were $374 million and $1,191 million, respectively, compared to $173 million and $401 million for the same periods in 2022.Collaboration revenue for the fourth quarter and full year in 2023 was $32 million and $36 million, respectively, compared to $1 million and $10 million for the same periods in 2022. The increase is mainly related to the clinical development milestone argenx achieved with AbbVie following the dosing of the first patient in the Phase 2 trial for ABBV-151. Collaboration revenue for full year in 2023 also includes $1 million in royalty revenue from VYVGART sales in China.Other operating income for the fourth quarter and full year in 2023 was $11 million and $42 million, respectively, compared to $8 million and $35 million for the same periods in 2022. The other operating income for the fourth quarter and full year in 2023, primarily relates to research and development tax incentives and payroll tax rebates. Total operating expenses for the fourth quarter and full year in 2023 were $556 million and $1,694 million, respectively, compared to $297 million and $1,166 million for the same periods in 2022, and mainly consists of: Cost of sales for the fourth quarter and full year in 2023 was $39 million and $118 million, respectively, compared to $13 million and $29 million for the same periods in 2022. The cost of sales was recognized with respect to the sale of VYVGART and VYVGART SC.Research and development expenses for the fourth quarter and full year in 2023 were $306 million and $859 million, respectively, compared to $148 million and $663 million for the same periods in 2022. The research and development expenses mainly relate to external research and development expenses and personnel expenses incurred in the clinical development of efgartigimod in various indications and the expansion of other clinical and preclinical pipeline candidates. The research and development expenses for the fourth quarter and the full year in 2023, includes the amortization of the priority review voucher submitted with the sBLA filling for VYVGART Hytrulo for the treatment of CIDP, which resulted in an expense of $102 million.Selling, general and administrative expenses for the fourth quarter and full year in 2023 were $209 million and $712 million, respectively, compared to $135 million and $472 million for the same periods in 2022. The selling, general and administrative expenses mainly relate to professional and marketing fees linked to the commercialization of VYVGART and VYVGART SC, and personnel expenses. Financial income for the fourth quarter and full year in 2023 was $40 million and $107 million, respectively, compared to $14 million and $28 million for the same periods in 2022. The increase in financial income is mainly due to an increase in interest income which results from higher interest rates and a higher amount of current financial assets, cash and cash equivalents as a result of the financing round in July 2023. Exchange gains for the fourth quarter and full year in 2023 were $37 million and $14 million respectively, compared to $60 million of exchange gains and $33 million of exchange losses for the same periods in 2022. Exchange gains/losses are mainly attributable to unrealized exchange rate gains or losses on the cash, cash equivalents and current financial assets denominated in Euro. Income tax for the fourth quarter and full year in 2023 was $38 million of tax expense and $9 million of tax benefit, respectively, compared to $3 million and $20 million of tax benefit for the same periods in 2022. Tax expense for the fourth quarter in 2023, consists of $12 million of income tax benefit and $50 million of deferred tax expense, compared to $12 million of income tax expense and $15 million of deferred tax benefit for the comparable prior period. Net loss for the fourth quarter and full year in 2023, was $99 million and $295 million, respectively, compared to $39 million and $710 million over the prior year periods. On a per weighted average share basis, the net loss was $5.16 and $13.05 for the twelve months ended December 31, 2023 and 2022, respectively. Cash, cash equivalents and current financial assets totalled $3.2 billion as of December 31, 2023, compared to $2.2 billion as of December 31, 2022. The increase in cash and cash equivalents and current financial assets resulted primarily from the closing of a global offering of shares, including a U.S. offering, which resulted in the receipt of $1.2 billion in net proceeds in July 2023, partially offset by net cash flows used in operating activities. FINANCIAL GUIDANCE Based on its current operating plans, argenx expects its combined Research and development and Selling, general and administrative expenses in 2024 to be less than $2 billion. argenx expects to utilize up to $500 million of net cash in 2024 on these anticipated operating expenses as well as working capital and capital expenditures. EXPECTED 2024 FINANCIAL CALENDAR May 9, 2024: Q1 2024 financial results and business updateJuly 25, 2024: Q2 2024 financial results and business updateOctober 31, 2024: Q3 2024 financial results and business update CONFERENCE CALL DETAILS The full year 2023 financial results and fourth quarter business update will be discussed during a conference call and webcast presentation today at 2:30 pm CET/8:30 am ET. A webcast of the live call may be accessed on the Investors section of the argenx website at argenx.com/investors. A replay of the webcast will be available on the argenx website. Dial-in numbers: Please dial in 15 minutes prior to the live call. Belgium 32 800 50 201France 33 800 943355Netherlands 31 20 795 1090United Kingdom 44 800 358 0970United States 1 888 415 4250Japan 81 3 4578 9081Switzerland 41 43 210 11 32 About argenx argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker, globally in the U.S., Japan, Israel, the EU, the UK, China and Canada. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, Twitter, and Instagram. For further information, please contact: Media:Ben Petokbpetok@argenx.com Investors:Alexandra Roy (US)aroy@argenx.com Lynn Elton (EU)lelton@argenx.com Forward-looking Statements The contents of this announcement include statements that are, or may be deemed to be, “forward-looking statements.” These forward-looking statements can be identified by the use of forward-looking terminology, including the terms “plans,” “aims,” “continues,” “anticipates,” “expects,” “will,” or “commitment” and include statements argenx makes concerning its utilization of its learnings and momentum from its gMG launch for a potential CIDP approval and to position VYVGART SC for success; its plans to expand its patient reach, including through its multidimensional expansion efforts aimed at including patients earlier in the MG treatment paradigm and pursuing global regulatory approvals for MG as well as additional autoimmune indications; our goal to translate the ADHERE data into potential benefit for patients; the advancement of, and anticipated clinical development, data readouts and regulatory milestones and plans, including: (1) expected decisions on regulatory approvals of VYVGART for gMG in Switzerland, Australia, Saudi Arabia and South Korea by end of 2024, (2) expected decisions on approval of VYVGART SC for gMG in China through Zai Lab by end of 2024, (3) expected decisions on approval of VYVGART for ITP in Japan in the first quarter of 2024, (4) expected regulatory submissions of VYVGART SC of CIDP in Japan, Europe, China and Canada in 2024, (5) the expansion of our VYVGART registrational studies into broader MG populations, including in seronegative patients, expected to start in 2024, (6) the update on pre-filled syringe development expected in the first half of 2024, (7) clinical studies expected to support potential approval in gMG and CIDP in 2024, (8) expected update on the path forward for BALLAD study in 2024, (9) expected topline data from Phase 2 RHO in the first half of 2024, (10) expected topline data from Phase 2 ALPHA study in the first half of 2024, (11) expected topline data from Phase 2/3 ALKIVIA in the second half of 2024, (12) the full Phase 2 topline data from ARDA study expected in 2024, (13) planned Phase 1b/2a clinical trials of ARGX-119 in 2024, (14) four IND applications expected to be filed by end of 2025, (15) expected data from six Phase 2 proof-of-concept trials by the end of 2024, and (16) the expected approval or development in 15 autoimmune indications by 2025; the potential of its continued investment in its IIP to drive long-term sustainable pipeline growth; its future financial and operating performance, including its anticipated operating expenses and utilization of net cash for 2024; and our goal of translating immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. argenx’s actual results may differ materially from those predicted by the forward-looking statements as a result of various important factors, including but not limited to, the results of argenx’s clinical trials, expectations regarding the inherent uncertainties associated with development of novel drug therapies, preclinical and clinical trial and product development activities and regulatory approval requirements, the acceptance of our products and product candidates by our patients as safe, effective and cost-effective, and the impact of governmental laws and regulations on our business. A further list and description of these risks, uncertainties and other risks can be found in argenx’s U.S. Securities and Exchange Commission (SEC) filings and reports, including in argenx’s most recent annual report on Form 20-F filed with the SEC as well as subsequent filings and reports filed by argenx with the SEC. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements. These forward-looking statements speak only as of the date of publication of this document. argenx undertakes no obligation to publicly update or revise the information in this press release, including any forward-looking statements, except as may be required by law.

SHANGHAI and NANJING, China and SAN JOSE, Calif., Feb. 29, 2024 /PRNewswire/ -- On February 26, 2024, the impactful international academic journal, EMBO Molecular Medicine, published the clinical data of the fully human BCMA targeting autologous CAR-T cell injection (Equecabtagene Autoleucel, Eque-cel, CT103A) for the treatment of two refractory myasthenia gravis (MG) subjects. B cell lineage reconstitution underlies CAR-T cell therapeutic efficacy in patients with refractory myasthenia gravis, initially demonstrated the good tolerability and safety of Eque-cel in the treatment of MG, as well as the durable clinical efficacy. MG is an autoimmune disease with acquired neuromuscular junction (NMJ) transmission disorders mediated by antibodies. Antibodies to the Acetylcholine Receptor (AChR) are the most common pathogenic antibodies; in addition, antibodies such as Muscle-Specific receptor tyrosine Kinase (MuSK), low-density Lipoprotein Receptor-related Protein 4 (LRP4), and Ryanodine Receptor (RyR), have also been found to be involved in the pathogenesis of MG. Currently, the treatment of MG is still based on cholinesterase inhibitors, glucocorticoids, immunosuppressants, intravenous immunoglobulin, plasma exchange, and thymectomy. The main causes of death in MG patients include respiratory failure and pulmonary infections. This is an investigator-initiated open-label study to evaluate the safety and efficacy of Eque-cel for the treatment of relapsed/refractory antibody-mediated idiopathic inflammatory disorders of the nervous system (NCT04561557). It was conducted by Prof. Wei Wang's team at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology. Two subjects with refractory MG were enrolled in this study. One was a 33-year-old female, AChR-IgG and Titin-IgG positive, who had been treated with thymectomy 21 months prior to enrollment and had not achieved clinical remission after treatment with cholinesterase inhibitors, glucocorticoids, immunosuppressants, and anti-CD20 monoclonal antibody. The other subject was a 60-year-old female, MuSK- IgG4 positive, with a 20-year history of the disease and failed the previous treatment with hormones, immunosuppressants, and anti-CD20 monoclonal antibody. The 2 subjects were treated with a single infusion of Eque-cel at the doses of 1.01×106 CAR-T/Kg and 0.96×106 CAR-T/Kg， respectively. Safety: Of the 2 subjects, only 1 subject developed grade 1 Cytokine Release Syndrome (CRS), and no Immune effector Cell-Associated Neurotoxicity Syndrome (ICANS). All grade ≥3 hemocytopenia recovered within 4 weeks post infusion. No new safety risk was identified, and the safety profile was superior compared to that in multiple myeloma indication. Effectiveness: Clinical symptoms continued to improve over 18 months in the 2 subjects. From 3 months after the infusion, patients showed significant improvement in limb strength and vital capacity, and sustained improvement in Myasthenia Gravis-Activities of Daily Living Score (MG-ADL), Quantitative Myasthenia Gravis Score (QMG), Myasthenia Gravis-Quality of Life Score (MG-QOL), and Modified Rankin Score (mRS). No immunomodulatory therapy other than low dose pyridostigmine (90 mg/day and 60 mg/day, respectively) was used during the follow-up period. PK/PD: Eque-cel expanded well after infusion and persisted for a shorter period than that in multiple myeloma patients. Anti-AChR antibodies, anti-Titin antibodies, and anti-MuSK antibodies decreased rapidly and maintained at very low levels in both subjects after infusion. B cells and plasma cells decreased to undetectable levels in both subjects within 2 months after infusion and then recovered gradually. At 18 months post-infusion, the B cells of both subjects had returned to normal levels, with approximately 80% of them being naïve B cells, while plasma cells remained at low levels. This result suggests that the long-term efficacy of CAR-T cell therapy may be related to the reconstitution of B cells with a predominantly naïve phenotype and the continued clearance of plasma cells. The principal investigator of this study, Prof. Wei Wang of Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, said "Myasthenia Gravis has a long course, is difficult to cure, and is prone to recurrence. Current treatment with traditional medications, although is able to improve symptoms of Myasthenia Gravis to a certain extent, is deficient in terms of disease control and long-term safety, and there is an urgent need for patients to have a better clinical outcome and treatment modality. In our study, it is gratifying to see that BCMA CAR-T cell therapy can prevent MG disease progression and show signs of reversal of the disease, which is expected to change the MG treatment landscape and bring hope for a cure to patients." In addition to the results of this published study, IASO Bio and a team of investigators continue to explore the safety and efficacy of Equecabtagene Autoleucel in the treatment of other antibody-mediated autoimmune diseases, including optic neuromyelitis optica spectrum disorders (NMOSD), Immune-Mediated Necrotizing Myelopathy (IMNM), and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), with an aim to change the landscape of treatment for autoimmune diseases. About IASO Bio IASO Bio is a biopharmaceutical company engaged in the discovery and development of novel cell therapies and biologics for oncology and autoimmune diseases. IASO Bio possesses comprehensive capabilities spanning the entire drug development process, from early discovery to clinical development, regulatory approval, and commercial production. The pipeline in the company includes a diversified portfolio of over 10 novel products, including Equecabtagene Autoleucel (a fully human BCMA CAR-T injection). Equecabtagene Autoleucel received New Drug Application (NDA) approval from China's National Medical Products Administration (NMPA) and U.S. FDA IND approval for the treatment of RRMM. Leveraging its strong management team, innovative product pipeline, GMP production, as well as integrated manufactural and clinical capabilities, IASO aims to deliver transformative, curable, and affordable therapies that fulfil unmet medical needs to patients in China as well as around the world. For more information, please visit or . SOURCE IASO Bio

– Net product revenues for the fourth quarter 2023 totaled $365.1 million, a 55% increase over the same quarter of the prior year; 2023 net product revenues for the full-year 2023 totaled $1.1 billion, an increase of approximately 36% over the prior year – ELEVIDYS revenues for the quarter totaled $131.2 million and for full-year totaled $200.4 million CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc., (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2023. “We are pleased to report another strong quarter and year of performance serving the patient community. With our three approved PMO therapies, EXONDYS 51, VYONDYS 53 and AMONDYS 45, and supported by the successful launch of our gene therapy, ELEVIDYS, Sarepta’s net product revenue grew 55% in the fourth quarter and 36% for the full year when compared to 2022, as we posted $1.14 billion in full-year net product revenue and we achieved GAAP profitability in the fourth quarter 2023 after achieving profitability on a non-GAAP basis in the third quarter 2023,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “2023 was arguably Sarepta’s most significant year in service of our mission to improve the lives of Duchenne patients. With the acceptance for review of our BLA supplement to expand the ELEVIDYS label, continued performance of our four approved therapies, and as we continue to advance our pipeline, 2024 offers the potential of being the most important year yet for families living with Duchenne and those invested in the improvement of patients’ lives.” Fourth Quarter 2023 and Recent Developments: Announced U.S. FDA acceptance of an efficacy supplement to expand the ELEVIDYS indication: The U.S. Food and Drug Administration (FDA) has accepted and filed Sarepta’s efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS (delandistrogene moxeparvovec-rokl). The goals of the efficacy supplement are twofold: To expand the labeled indication for ELEVIDYS as follows: “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with a confirmed mutation in the DMD gene.” To convert the ELEVIDYS accelerated approval to a traditional approval. The FDA has granted the efficacy supplement a Priority Review with a review goal date of June 21, 2024. The Agency has also confirmed they are not planning to hold an advisory committee meeting to discuss the supplement. Submitted efficacy supplement to expand the ELEVIDYS label to include Duchenne muscular dystrophy patients without restriction to age or ambulatory status: Submitted an efficacy supplement to the BLA for ELEVIDYS (delandistrogene moxeparvovec-rokl) to expand its labeled indication as follows "[ELEVIDYS is indicated for] the treatment of DMD patients with a confirmed mutation in the DMD gene.” The efficacy supplement is supported by results from EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study in patients with Duchenne between the ages of 4 through 7 years and data from ENDEAVOR (Study SRP-9001-103), an open label clinical study in patients with Duchenne, that is enrolling patients ages 2 years and older. The supplement was submitted to the U.S. Food and Drug Administration (FDA) with a request for Priority Review. Sarepta has also completed the EMBARK postmarketing requirement (PMR) and submitted the PMR to FDA requesting conversion from accelerated approval to traditional approval. Announced positive data from Part B of MOMENTUM, a phase 2 study of SRP-5051 in patients with Duchenne muscular dystrophy amenable to skipping exon 51: Announced positive data from Part B of the MOMENTUM study (Study SRP-5051-201), a global, Phase 2, multi-ascending dose clinical trial of SRP-5051 (vesleteplirsen) that enrolled patients aged 8 to 21 years. SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. Data from Part B of MOMENTUM found that at the higher, target dose, approximately 30 mg/kg dosed every four weeks, SRP-5051 resulted in mean dystrophin expression of 5.17%, and mean exon skipping of 11.11% at 28 weeks (n=20). Consistent dystrophin expression was seen in ambulatory (4.76%, n=11) and non-ambulatory (5.67%, n=9) participants at 28 weeks. Hypomagnesemia has previously been identified in patients taking SRP-5051 and was managed and monitored through prophylactic magnesium supplementation as part of the study protocol. Initiated screening in EMERGENE, a phase 3 clinical study of SRP-9003 for the treatment of limb-girdle muscular dystrophy type 2E/R4: Announced that screening is underway in Study SRP-9003-301, also known as EMERGENE. Study 9003-301 is a Phase 3, multi-national, open-label study of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of limb-girdle muscular dystrophy Type 2E (LGMD2E/R4), or beta sarcoglycanopathy. EMERGENE will enroll 15 participants (ambulatory and non-ambulatory), aged 4 and older, and uses commercially representative process SRP-9003 material. The EMERGENE study design incorporates a six-month natural history lead-in. The primary endpoint is expression of beta-sarcoglycan 60 days after dosing. Other endpoints include functional measures through month 60 and safety. Conference Call The event will be webcast live under the investor relations section of Sarepta's website at and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. Financial Results For the three months ended December 31, 2023, the Company reported a GAAP net income of $45.7 million, or $0.49 per basic and $0.47 per diluted share, compared to a GAAP net loss of $109.2 million reported for the same period of 2022, or $1.24 per basic and diluted share. The non-GAAP net income for the three months ended December 31, 2023 was $86.6 million, or $0.82 per diluted share, compared to a non-GAAP net loss of $53.6 million, or $0.61 per diluted share for the same period of 2022. For the twelve months ended December 31, 2023, the Company reported a GAAP net loss of $536.0 million, or $5.80 per basic and diluted share, compared to a GAAP net loss of $703.5 million reported for the same period of 2022, or $8.03 per basic and diluted share. The non-GAAP net loss for the twelve months ended December 31, 2023 was $59.5 million, or $0.64 per diluted share, compared to a non-GAAP net loss of $290.4 million, or $3.32 per diluted share for the same period of 2022. Revenues For the three months ended December 31, 2023, the Company recorded total revenues of $396.8 million, which consist of net product revenues and collaboration and other revenues, compared to total revenues of $258.4 million for the same period of 2022, an increase of $138.4 million. For the twelve months ended December 31, 2023, the Company recorded total revenues of $1,243.3 million, compared to total revenues of $933.0 million for the same period of 2022, an increase of $310.3 million. The increases primarily reflect increasing demand for EXONDYS 51, AMONDYS 45 and VYONDYS 53 (collectively, the “PMO Products”), as well as $131.2 million and $200.4 million of net product revenues associated with sales of ELEVIDYS during the three and twelve months ended December 31, 2023, respectively, after its approval in June 2023. For the three months ended December 31, 2023, the Company recorded net product revenues of $365.1 million, compared to net product revenues of $235.9 million for the same period of 2022, an increase of $129.2 million. For the twelve months ended December 31, 2023, the Company recorded net product revenues of $1,144.9 million, compared to net product revenues of $843.8 million for the same period of 2022, an increase of $301.1 million. The increases primarily reflect increasing demand for PMO Products, as well as $131.2 million and $200.4 million of net product revenues associated with sales of ELEVIDYS during the three and twelve months ended December 31, 2023, respectively. For the three and twelve months ended December 31, 2023, the Company recognized $31.7 million and $98.5 million of collaboration and other revenues, respectively. For the three and twelve months ended December 31, 2022, the Company recognized $22.5 million and $89.2 million of collaboration revenue, respectively. For all periods presented, collaboration and other revenues primarily relate to the F. Hoffman-La Roche Ltd. (Roche) collaboration arrangement. For the three and twelve months ended December 31, 2023, the Company recognized $9.2 million of contract manufacturing collaboration revenue associated with multiple batches of commercial ELEVIDYS supply delivered to Roche, with no similar activity for the three and twelve months ended December 31, 2022. Cost and Expenses Cost of sales (excluding amortization of in-licensed rights) For the three months ended December 31, 2023, cost of sales (excluding amortization of in-licensed rights) was $44.2 million, compared to $30.8 million for the same period of 2022, an increase of $13.4 million. The increase in the three months ended December 31, 2023 primarily reflects increasing demand for the Company's PMO Products, an increase in royalty payments due to ELEVIDYS sales in 2023 with no similar activity in 2022 and write-offs of certain batches of the Company's products not meeting the Company's quality specifications for the three months ended December 31, 2023, with no similar activity for the three months ended December 31, 2022. For the twelve months ended December 31, 2023, cost of sales (excluding amortization of in-licensed rights) was $150.3 million, compared to $140.0 million for the same period of 2022, an increase of $10.3 million. The change for the twelve months ended December 31, 2023 primarily reflects increasing demand for the Company's PMO Products, partially offset by a decrease in write-offs of certain batches of the Company's products not meeting the Company's quality specifications for the twelve months ended December 31, 2023, as compared to the same period of 2022, as well as a decrease in royalty payments due to changes in the BioMarin Pharmaceuticals, Inc. (BioMarin) royalty terms. Research and development Research and development expenses were $195.5 million for the three months ended December 31, 2023, compared to $213.8 million for the same period of 2022, a decrease of $18.3 million. The decrease in research and development expenses primarily reflects the following: $57.6 million decrease in manufacturing expenses primarily due to an increase in capitalization of commercial batches of ELEVIDYS manufactured after its approval in June 2023; $16.9 million decrease in up-front, milestone and other expenses primarily due to timing and costs related to the execution of certain research and license agreements and achievement of certain milestones year over year; $3.5 million decrease in research and other expenses primarily driven by a decrease in collaboration cost-sharing expenses related to Genethon's micro-dystrophin drug candidate; $1.6 million increase in pre-clinical expenses primarily due to an increase in toxicology study activity across multiple gene therapy and PPMO platforms; $1.9 million increase in professional service expenses primarily due to clinical consulting fees for various clinical trials sponsored by the Company; $3.2 million increase in stock-based compensation expense primarily due to the achievement of performance conditions related to certain shares with performance conditions (PSUs) during the twelve months ended December 31, 2023 with continuing vesting requirements related to a service condition, as well as changes in headcount and the value of stock awards; $4.8 million increase in facility- and technology-related expenses primarily due to the Company’s continuing expansion efforts; $7.9 million increase in compensation and other personnel expenses primarily due to changes in headcount; $12.0 million increase in clinical trial expenses primarily due to an increased patient enrollment and site activation for the Company's MOMENTUM and EXPEDITION programs, as well as additional PPMO clinical trials; and $28.2 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to a decrease in reimbursed cost related to the minimum purchase requirements under the gene therapy manufacturing and supply agreement (the Thermo Agreement) with Thermo Fisher Scientific, Inc. (Thermo) for the three months ended December 31, 2022, with no similar activity in 2023, partially offset by the continuing development of the Company's SRP-9001 gene therapy programs. Research and development expenses were $877.4 million for the twelve months ended December 31, 2023, compared to $877.1 million for the same period of 2022, a slight increase of $0.3 million. The increase in research and development expenses primarily reflects the following: $51.5 million increase in clinical trial expenses primarily due to an increased patient enrollment and site activation for the Company's MIS51ON, MOMENTUM, ENVISION, EMERGENE and EXPEDITION programs, as well as additional PPMO clinical trials; $38.8 million increase in compensation and other personnel expenses primarily due to changes in headcount; $21.2 million increase in stock-based compensation expense primarily due to changes in headcount and the value of stock awards, as well as the achievement of performance conditions related to certain PSUs during the twelve months ended December 31, 2023 with continuing vesting requirements related to a service condition; $18.3 million increase in facility- and technology-related expenses primarily due to the Company’s continuing expansion efforts; $12.0 million increase in research and other expenses primarily driven by an increase in sponsored research with academic institutions during the twelve months ended December 31, 2023 and an increase in collaboration cost-sharing expenses related to Genethon's micro-dystrophin drug candidate; $9.7 million increase in professional service expenses primarily related to the launch of ELEVIDYS prior to its regulatory approval in June 2023; $3.1 million increase in pre-clinical expenses primarily due to an increase in toxicology study activity across multiple gene therapy and PPMO platforms; $21.9 million decrease in up-front, milestone and other expenses, primarily due to timing and costs related to the execution of certain research and license agreements and achievement of certain milestones year over year; $143.7 million decrease in manufacturing expenses primarily due to the capitalization of commercial batches of ELEVIDYS manufactured after its approval in June 2023 and a decrease of $54.0 million related to the minimum purchase requirements under the Thermo Agreement for the twelve months ended December 31, 2022, with no similar activity in the same period of 2023; and $11.3 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to a decrease in reimbursed cost related to the minimum purchase requirements under the Thermo Agreement for the twelve months ended December 31, 2022, with no similar activity in the same period of 2023, partially offset by the continuing development of the Company's SRP-9001 gene therapy programs. Non-GAAP research and development expenses were $165.1 million and $186.8 million for the three months ended December 31, 2023 and 2022, respectively, a decrease of $21.7 million. Non-GAAP research and development expenses were $761.9 million and $784.1 million for the twelve months ended December 31, 2023 and 2022, respectively, a decrease of $22.2 million. Selling, general and administrative Selling, general and administrative expenses were $131.7 million for the three months ended December 31, 2023 compared to $120.5 million for the same period in 2022, an increase of $11.2 million. The increase in selling, general and administrative expenses primarily reflects the following: $18.4 million increase in professional service expenses primarily related to the launch of ELEVIDYS and ongoing litigation matters; $7.9 million increase in compensation and other personnel expenses primarily due to changes in headcount; $3.6 million increase in facility- and technology-related expenses primarily due to the Company’s continuing expansion efforts; $7.9 million decrease in stock-based compensation expense primarily related to the execution of the Chief Executive Officer (CEO) grant modification agreement in 2022, partially offset by the achievement of performance conditions related to certain PSUs during the twelve months ended December 31, 2023 with continuing vesting requirements related to a service condition, as well as changes in headcount and the value of stock awards; and $10.7 million decrease in other expenses primarily due to timing of charitable contributions. Selling, general and administrative expenses were $481.9 million for the twelve months ended December 31, 2023, compared to $451.4 million for the same period in 2022, an increase of $30.5 million. The increase in selling, general and administrative expenses primarily reflects the following: $60.9 million increase in professional service expenses primarily related to the launch of ELEVIDYS and ongoing litigation matters; $35.2 million increase in compensation and other personnel expenses primarily due to changes in headcount; $10.9 million increase in facility- and technology-related expenses primarily due to the Company’s continuing expansion efforts; $4.6 million decrease in other expenses primarily related to timing of charitable contributions; and $71.7 million decrease in stock-based compensation expense primarily related to the CEO grant modification agreement in 2022, partially offset by the achievement of performance conditions related to certain PSUs during the twelve months ended December 31, 2023 with continuing vesting requirements related to a service condition, as well as changes in headcount and the value of stock awards. Non-GAAP selling, general and administrative expenses were $105.7 million and $86.6 million for the three months ended December 31, 2023 and 2022, respectively, an increase of $19.1 million. Non-GAAP selling, general and administrative expenses were $372.0 million and $270.3 million for the twelve months ended December 31, 2023 and 2022, respectively, an increase of $101.7 million. Amortization of in-licensed rights For the three and twelve months ended December 31, 2023, the Company recorded amortization of in-licensed rights of approximately $0.8 million and $1.6 million, respectively. For the three and twelve months ended December 31, 2022, the Company recorded amortization of in-licensed rights of approximately $0.2 million and $0.7 million, respectively. This is related to the amortization of the in-licensed right assets recognized as a result of agreements the Company entered into with the University of Western Australia, Nationwide Children's Hospital, BioMarin and Parent Project Muscular Dystrophy in April 2013, December 2016, July 2017 and May 2018, respectively. Loss on debt extinguishment On November 14, 2017, the Company issued $570.0 million aggregate principal amount of senior convertible notes due on November 15, 2024 (2024 Notes). On March 2, 2023, the Company entered into separate, privately negotiated exchange agreements with certain holders of the outstanding 2024 Notes (Exchange Agreements). The Exchange Agreements resulted in a conversion of $313.5 million in aggregate principal value of the 2024 Notes held by the holders (2024 Notes Conversion). In connection with the 2024 Notes Conversion, the Company issued approximately 4.5 million shares of its common stock representing the agreed upon contractual conversion rate under the Exchange Agreements. The conversion was not pursuant to the conversion privileges included in the terms of the debt at issuance and therefore was accounted for as a debt extinguishment. The Company accounted for the debt extinguishment by recognizing the difference between the fair value of the shares of common stock transferred on the conversion date and the net carrying amount of the extinguished debt as a loss on debt extinguishment. The loss incurred on the extinguishment for the twelve months ended December 31, 2023 was $387.3 million, inclusive of $6.9 million in third-party debt conversion costs. Gain from Sale of Priority Review Voucher In June 2023, the Company entered into an agreement to sell the rare pediatric disease Priority Review Voucher (ELEVIDYS PRV) it received from the FDA in connection with the approval of ELEVIDYS for consideration of $102.0 million, with no commission costs. The transaction was not subject to the conditions set forth under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and closed during June 2023. The net proceeds were recorded as a gain from sale of the ELEVIDYS PRV during the twelve months ended December 31, 2023 as it did not have a carrying value at the time of the sale. Other income (expense), net For the three months ended December 31, 2023 and 2022, other income, net was $15.7 million and $5.5 million, respectively. The increase was primarily due to an increase in accretion of investment discount, net due to an increase in interest rates. For the twelve months ended December 31, 2023 and 2022, other income, net was $33.1 million and other expense, net was $28.3 million, respectively. The change is primarily due to an increase in accretion of investment discount, net and an increase in interest income due to the investment mix of the Company's investment portfolio and an increase in interest rates, as well as a reduction of interest expense incurred as a result of the repayment of the Company's December 2019 Term Loan in 2022, partially offset by the impairment of strategic investments and a decrease in gain (loss) on contingent consideration, net. Income tax (benefit) expense Income tax benefit for the three months ended December 31, 2023 was approximately $5.3 million. Income tax expense for the twelve months ended December 31, 2023 was $15.9 million. Income tax expense for the three and twelve months ended December 31, 2022 was $7.9 million and $13.5 million, respectively. Income tax (benefit) expense for the three and twelve months ended December 31, 2023 relates to state, foreign and federal income taxes, while income tax expense for the three and twelve months ended December 31, 2022 relates to state and foreign income taxes. Cash, Cash Equivalents, Restricted Cash and Investments The Company had approximately $1.7 billion in cash, cash equivalents, investments and long-term restricted cash as of December 31, 2023, compared to $2.0 billion as of December 31, 2022. This decrease is driven by cash used to fund the Company’s ongoing operations during 2023. Use of Non-GAAP Measures In addition to the GAAP financial measures set forth in this press release, the Company has included certain non-GAAP measurements. The non-GAAP income (loss) is defined by the Company as GAAP net income (loss) excluding interest (income) expense, net, depreciation and amortization expense, stock-based compensation expense, the estimated income tax impact of each pre-tax non-GAAP adjustment and other items. Non-GAAP research and development expenses are defined by the Company as GAAP research and development expenses excluding depreciation and amortization expense, stock-based compensation expense and other items. Non-GAAP selling, general and administrative expenses are defined by the Company as GAAP selling, general and administrative expenses excluding depreciation expense, stock-based compensation expense and other items. 1. Interest, depreciation and amortization Interest (income) expense, net amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of the Company’s operations. Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to the Company’s operating performance. Amortization expense primarily associated with patent costs are amortized over a period of several years after acquisition or patent application or renewal. 2. Stock-based compensation expenses Stock-based compensation expenses represent non-cash charges related to equity awards granted by the Company. Although these are recurring charges to operations, the Company believes the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within the Company’s control. Therefore, the Company believes that excluding these charges facilitates comparisons of the Company’s operational performance in different periods. 3. Other items The Company evaluates other items of expense and income on an individual basis. It takes into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relate to the Company’s ongoing business operations, and (c) whether the Company expects the items to continue or occur on a regular basis. These other items include impairment of strategic investments, loss (gain) on contingent consideration, net, gain from sale of the PRV and loss on debt extinguishment and may include other items that fit the above characteristics in the future. The Company excludes from its non-GAAP results the impairment of any strategic investments as it is a non-cash item and is not considered to be a normal operating expense due to the variability of amount and lack of predictability as to the occurrence and/or timing of such impairments. The Company excludes from its non-GAAP results the loss on debt extinguishment, which is considered to be an infrequent and non-cash event as it is associated with a distinct financing decision and is not indicative of the performance of the Company’s core operations, which accordingly, would make it difficult to compare the Company’s results to peer companies that also provide non-GAAP disclosures. The Company excludes from its non-GAAP results the loss (gain) on contingent consideration, net related to regulatory-related contingent payments meeting the definition of a derivative to Myonexus selling shareholders as well as to academic institutions under separate license agreements as it is a non-cash item and is not considered to be normal operating expenses due to its variability of amounts and lack of predictability as to occurrence and/or timing. The Company excludes from its non-GAAP results the gain from sale of the PRV obtained as a result of the FDA approval of ELEVIDYS in June 2023 as it is an infrequent event and is not indicative of the performance of the Company’s core operations, which accordingly would make it difficult to compare the Company’s results to peer companies that also provide non-GAAP disclosures. Beginning in the fourth quarter of 2023, amortization of in-licensed rights (formerly included within depreciation and amortization expense) and income tax (benefit) expense are no longer excluded from the non-GAAP results. The Company now includes the income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the fourth quarter and full-year 2022 have been updated to reflect this change for comparability. The Company uses these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. The Company also believes these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating the Company’s performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of the Company’s financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income and loss adjustments, non-GAAP net income (loss), and non-GAAP diluted net earnings (loss) per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.” About EXONDYS 51 EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. Important Safety Information About EXONDYS 51 Hypersensitivity reactions, bronchospasm, chest pain, cough, tachycardia, and urticaria have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 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 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. Important Safety Information for VYONDYS 53 Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the VYONDYS 53 therapy. Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. Adverse reactions observed in at least 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 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. Important Safety Information for AMONDYS 45 CONTRAINDICATION: Known hypersensitivity to casimersen or any of the inactive ingredients. Instances of hypersensitivity including angioedema and anaphylaxis have occurred. WARNINGS AND PRECAUTIONS Hypersensitivity: Hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients who were treated with AMONDYS 45. If a hypersensitivity reaction occurs, institute appropriate medical treatment, and consider slowing the infusion, interrupting, or discontinuing the AMONDYS 45 infusion and monitor until the condition resolves. Kidney Toxicity: Kidney toxicity was observed in animals who received casimersen. Although kidney toxicity was not observed in the clinical studies with AMONDYS 45, kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking AMONDYS 45. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting AMONDYS 45. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting AMONDYS 45. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio (UPCR) every three months. Only urine expected to be free of excreted AMONDYS 45 should be used for monitoring of urine protein. Urine obtained on the day of AMONDYS 45 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any AMONDYS 45 that is excreted in the urine and thus lead to a false positive result for urine protein. If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation. Adverse Reactions: Adverse reactions occurring in at least 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 is indicated for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin observed in patients treated with ELEVIDYS. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). Important Safety Information for ELEVIDYS CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS. Administration of ELEVIDYS may result in elevations of liver enzymes (e.g., GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, a consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness including dysphagia, dyspnea and hypophonia were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene between exons 1 to 17 and exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea or hypophonia as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Pre-existing Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all subjects developed anti-AAVrh74 antibodies. Perform baseline testing for the presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥ 5%) reported in clinical studies were vomiting, nausea, liver function test increased, pyrexia, and thrombocytopenia. For further information, please see the full Prescribing Information. About Sarepta Therapeutics Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit or follow us on X (formerly Twitter), LinkedIn, Instagram and Facebook. Forward-Looking Statements In order to provide Sarepta’s investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “may,” “intends,” “prepares,” “looks,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements relating to our future operations, financial performance and projections, business plans, market opportunities, priorities and research and development programs and technologies; the potential benefits of our technologies, product candidates and scientific approaches; the potential for the ELEVIDYS efficacy supplement to expand the approved label for ELEVIDYS and convert the ELEVIDYS accelerated approval to a traditional approval; the review goal date of June 21, 2024 for the ELEVIDYS efficacy supplement; our understanding that FDA does not plan to hold an advisory committee to discuss the ELEVIDYS supplement; the potential for 2024 being the most important year yet for families living with Duchenne and those invested in the improvement of patients’ lives; and expected plans and milestones. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; because we are developing product candidates for the treatment of certain diseases in which there is little clinical experience and we are using new endpoints or methodologies, there is increased risk that the FDA, the EMA or other regulatory authorities may not consider the endpoints of our clinical trials to provide clinically meaningful results and that these results may be difficult to analyze; success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; if the actual number of patients suffering from the diseases we aim to treat is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K for the year ended December 31, 2023 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Internet Posting of Information We routinely post information that may be important to investors in the 'For Investors' section of our website at . 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 December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 Revenues: Products, net $ 365,071 $ 235,933 $ 1,144,876 $ 843,769 Collaboration and other 31,710 22,494 98,460 89,244 Total revenues 396,781 258,427 1,243,336 933,013 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 44,176 30,799 150,343 139,989 Research and development 195,517 213,804 877,387 877,090 Selling, general and administrative 131,700 120,478 481,871 451,421 Amortization of in-licensed rights 763 179 1,559 714 Total cost and expenses 372,156 365,260 1,511,160 1,469,214 Operating income (loss) 24,625 (106,833 ) (267,824 ) (536,201 ) Other income (loss), net: Other income (expense), net 15,746 5,527 33,055 (28,321 ) Loss on debt extinguishment — — (387,329 ) (125,441 ) Gain from sale of Priority Review Voucher — — 102,000 — Total other income (loss), net 15,746 5,527 (252,274 ) (153,762 ) Income (loss) before income tax (benefit) expense 40,371 (101,306 ) (520,098 ) (689,963 ) Income tax (benefit) expense (5,284 ) 7,938 15,879 13,525 Net income (loss) $ 45,655 $ (109,244 ) $ (535,977 ) $ (703,488 ) Net earnings (loss) per share: Basic $ 0.49 $ (1.24 ) $ (5.80 ) $ (8.03 ) Diluted $ 0.47 $ (1.24 ) $ (5.80 ) $ (8.03 ) Weighted-average number of shares of common stock used in computing basic net earnings (loss) per share 93,617 87,838 92,398 87,559 Weighted-average number of shares of common stock used in computing diluted net earnings (loss) per share 105,594 87,838 92,398 87,559 Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands, except per share amounts) For the Three Months Ended December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 GAAP net income (loss) $ 45,655 $ (109,244 ) $ (535,977 ) $ (703,488 ) Interest (income) expense, net (17,469 ) (8,865 ) (64,034 ) 25,525 Depreciation and amortization expense* 10,609 10,374 42,838 41,150 Stock-based compensation expense 45,826 50,510 182,514 233,018 Impairment of strategic investments 2,500 2,575 30,321 2,575 Loss on debt extinguishment — — 387,329 125,441 Loss (gain) on contingent consideration, net — — 1,200 (6,700 ) Gain from sale of Priority Review Voucher — — (102,000 ) — Income tax effect of adjustments** (541 ) 1,075 (1,738 ) (7,915 ) Non-GAAP net income (loss)** $ 86,580 $ (53,575 ) $ (59,547 ) $ (290,394 ) Non-GAAP net earnings (loss) per share: Diluted*** $ 0.82 $ (0.61 ) $ (0.64 ) $ (3.32 ) Weighted-average number of shares of common stock used in computing earnings per share: Diluted 105,594 87,838 92,398 87,559 *Beginning in the fourth quarter of 2023, depreciation and amortization excludes amortization of in-licensed rights. Non-GAAP financial results for the fourth quarter and full-year 2022 have been updated to reflect this change for comparability. **Beginning in the fourth quarter of 2023, income tax (benefit) expense is no longer excluded from the non-GAAP results. The Company has 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 effective income tax rate. Refer below for a reconciliation of effective tax rates. Non-GAAP financial results for the fourth quarter and full-year 2022 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. For the Three Months Ended December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 Total effective tax rate, GAAP (12.0 )% 5.5 % (3.1 )% (2.0 )% Less: impact of GAAP to Non-GAAP adjustments (0.7 ) (1.8 ) (39.0 ) (6.0 ) Total effective tax rate, Non-GAAP (12.7 )% 3.7 % (42.1 )% (8.0 )% Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands) For the Three Months Ended December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 GAAP research and development expenses $ 195,517 $ 213,804 $ 877,387 $ 877,090 Stock-based compensation expense (22,174 ) (18,963 ) (82,489 ) (61,293 ) Depreciation and amortization expense (8,217 ) (8,013 ) (33,011 ) (31,713 ) Non-GAAP research and development expenses $ 165,126 $ 186,828 $ 761,887 $ 784,084 For the Three Months Ended December 31, For the Twelve Months Ended December 31, 2023 2022 2023 2022 GAAP selling, general and administrative expenses $ 131,700 $ 120,478 $ 481,871 $ 451,421 Stock-based compensation expense (23,652 ) (31,547 ) (100,025 ) (171,725 ) Depreciation expense (2,392 ) (2,361 ) (9,827 ) (9,437 ) Non-GAAP selling, general and administrative expenses $ 105,656 $ 86,570 $ 372,019 $ 270,259 Sarepta Therapeutics, Inc. Condensed Consolidated Balance Sheets (unaudited, in thousands, except share and per share data) As of December 31, 2023 2022 Assets Current assets: Cash and cash equivalents $ 428,430 $ 966,777 Short-term investments 1,247,820 1,022,597 Accounts receivable, net 400,327 214,628 Inventory 322,859 203,968 Other current assets 179,895 149,891 Total current assets 2,579,331 2,557,861 Property and equipment, net 227,154 180,037 Right of use assets 129,952 64,954 Non-current inventory 191,368 162,545 Other non-current assets 136,771 162,969 Total assets $ 3,264,576 $ 3,128,366 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 164,918 $ 95,875 Accrued expenses 314,997 418,996 Deferred revenue, current portion 50,416 89,244 Current portion of long-term debt 105,483 — Current portion of lease liabilities 17,845 15,489 Total current liabilities 653,659 619,604 Long-term debt 1,132,515 1,544,292 Lease liabilities, net of current portion 140,965 57,578 Deferred revenue, net of current portion 437,000 485,000 Contingent consideration 38,100 36,900 Other non-current liabilities 3,000 42 Total liabilities 2,405,239 2,743,416 Stockholders’ equity: Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding — — Common stock, $0.0001 par value, 198,000,000 shares authorized; 93,731,831 and 87,950,117 issued and outstanding at December 31, 2023 and 2022, respectively 9 9 Additional paid-in capital 5,304,623 4,296,841 Accumulated other comprehensive income (loss), net of tax 918 (1,664 ) Accumulated deficit (4,446,213 ) (3,910,236 ) Total stockholders’ equity 859,337 384,950 Total liabilities and stockholders’ equity $ 3,264,576 $ 3,128,366