DMPK

SAN DIEGO, April 23, 2025 (GLOBE NEWSWIRE) -- Bionano Genomics, Inc. (Nasdaq: BNGO) today announced a peer-reviewed publication in Genome Research demonstrating that optical genome Mapping (OGM) can be an accurate, cost-effective method for detecting and sizing large repeat expansions, which are a class of structural variation (SV) linked to as many as 40 genetic disorders. Led by Alexander Hoischen and researchers at Radboud University Medical Center in the Netherlands, the study provides one of the most comprehensive evaluations to date of OGM as a single workflow for characterizing large repeat expansions. Researchers evaluated 85 subjects containing known pathogenic expansions in three genes, DMPK (associated with myotonic dystrophy (DM) type 1), CNBP (associated with DM type 2), and RFC1 (associated with cerebellar ataxia, neuropathy, and vestibular areflexia syndrome (CANVAS)). DM is a genetic condition causing progressive muscle weakness and wasting, affecting both skeletal and smooth muscles and CANVAS is a genetic condition characterized by progressive loss of coordination, sensory nerve damage, and impaired balance reflexes. Key Findings: High detection accuracy: OGM identified 84 out of 85 (98.8%) pathogenic repeat expansions.No apparent upper size limit: OGM measured accurately the repeat sizes, even for extremely long expansions exceeding 7,000 repeat units.Somatic instability revealed: Evidence of somatic instability in 36 out of 85 samples, with variability observed in 30% of DMPK samples, 92% of CNBP samples, and 16% of RFC1 samples. These findings provide new insights into the unpredictable nature of these disorders.Single technique workflow: Unlike traditional methods that require multiple gene-specific assays, OGM delivers a single, comprehensive assay that detects large repeat expansions across multiple loci, potentially reducing turnaround times and lowering costs. This publication appears alongside two other OGM studies in Part II of a series released by Genome Research focusing on long-read methods such as OGM and long-read sequencing (LRS) for genome analysis. In an accompanying editorial, the editors of the series describe how OGM has the potential to address long-standing challenges in genome analysis, from structural variation to paralog resolution and repeat expansion detection, sizing and evaluation of their somatic instability. “Repeat expansions are a clinically relevant class of structural variations (SVs). Their size, stability and variability make them challenging to analyze using conventional methods such as PCR, Southern blotting and next-generation sequencing (NGS). LRS may be useful, but LRS can be too costly for routine use,” commented Erik Holmlin, president and CEO of Bionano. “This study demonstrates how can OGM address a critical limitation in genome analysis and lays the foundation for researchers around the world to broaden their scope of OGM use in research to include this class of SVs, which we believe may pave the way for future development of better diagnostics and therapeutics for genetic diseases like repeat expansion disorders.” The full research publication is available at: https://genome-cshlp-org.libproxy1.nus.edu.sg/content/early/2025/03/19/gr.279491.124.full.pdf+html The editorial accompanying the special issue of Genome Research is available at:https://genome-cshlp-org.libproxy1.nus.edu.sg/content/35/4/xi.full.pdf+html About Bionano Bionano is a provider of genome analysis solutions that can enable researchers and clinicians to reveal answers to challenging questions in biology and medicine. The Company’s mission is to transform the way the world sees the genome through optical genome mapping (OGM) solutions, diagnostic services and software. The Company offers OGM solutions for applications across basic, translational and clinical research. The Company also offers an industry-leading, platform-agnostic genome analysis software solution, and nucleic acid extraction and purification solutions using proprietary isotachophoresis (ITP) technology. Through its Lineagen, Inc. d/b/a Bionano Laboratories business, the Company also offers OGM-based diagnostic testing services. For more information, visit www.bionano.com or www.bionanolaboratories.com. Bionano’s products are for research use only and not for use in diagnostic procedures. Forward-Looking Statements of Bionano Genomics This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “believe,” “can,” “may,” “potential” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) convey uncertainty of future events or outcomes and are intended to identify these forward-looking statements. Forward-looking statements describe future expectations, plans, results, or strategies, among other things, and in this release include, but are not limited to, statements OGM’s ability to be an accurate, cost-effective method for detecting and sizing large repeat expansions; OGM’s ability to identify pathogenic repeat expansions; OGM’s ability to measure accurately expansion sizes, even for extremely long expansions; OGM’s ability to show evidence of somatic instability; OGM’s ability to deliver a single, comprehensive assay that detects large repeat expansions across multiple loci, reducing turnaround times and lowering costs; the ability of OGM to address critical limitations in genomics analysis; the ability of OGM to pave the way for future development of better diagnostics and therapeutics for genetic diseases like repeat expansion disorders; and other statements not of historical fact. Such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements. Each of these forward-looking statements involves risks and uncertainties. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include the impact of adverse geopolitical and macroeconomic events, such as bank failures, the ongoing conflicts between Ukraine and Russia and in the Middle East and related sanctions, regional or global pandemics, uncertain market conditions, including tariffs and inflation, and supply chain disruptions on our business and the global economy; the failure of OGM to be an accurate, cost-effective method for detecting and sizing large repeat expansions; the failure of OGM to identify pathogenic repeat expansions; the failure of OGM to measure accurately expansion sizes, even for extremely long expansions; the failure of OGM to show evidence of somatic instability; the failure of OGM to deliver a single, comprehensive assay that detects large repeat expansions across multiple loci, reducing turnaround times and lowering costs; the failure of OGM to address critical limitations in genomics analysis; the failure of OGM to pave the way for future development of better diagnostics and therapeutics for genetic diseases like repeat expansion disorders; the inability to achieve results similar to those referenced in this press release using the methods described therein; the failure of the publication referenced in this press release to prove to be an important advancement in repeat expansion research; the failure of the method described in this press release to further open researchers’ ability to expand their OGM menu; future publications that contradict the findings of the publication referenced in this press release; the failure of our ability to drive adoption and utilization of optical genome mapping as a replacement to traditional cytogenetic techniques; challenges inherent in developing, manufacturing and commercializing products; our ability to further deploy new products and applications for our technology platforms; our expectations and beliefs regarding future growth of the business and the markets in which we operate; changes in our strategic and commercial plans; our ability to continue as a “going concern,” which requires us to manage costs and obtain significant additional financing to fund our strategic plans and commercialization efforts; our ability to consummate any strategic alternatives; the risk that if we fail to obtain additional financing we may seek relief under applicable insolvency laws; and other risks and uncertainties including those described in our filings with the Securities and Exchange Commission (“SEC”), including, without limitation, our Annual Report on Form 10-K for the year ended December 31, 2024 and in other filings subsequently made by us with the SEC. All forward-looking statements contained in this report speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. We are under no duty to update any of these forward-looking statements after the date they are made to conform these statements to actual results or revised expectations, except as required by law. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date the statements are made. Moreover, except as required by law, neither we nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements contained in this press release. CONTACTSCompany Contact:Erik Holmlin, CEOBionano Genomics, Inc.+1 (858) 888-7610eholmlin@bionano.com Investor Relations:David HolmesGilmartin Group+1 (858) 888-7625IR@bionano.com

Del-desiran first-ever investigational treatment for DM1 to receive Orphan Drug designation in Japan SAN DIEGO, April 8, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation (ODD) to delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1), an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Del-desiran is the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). "This decision by MHLW further reinforces the significant potential of del-desiran to address the root cause of DM1 and the urgent need to bring an approved therapy to the many people impacted by this devastating rare disease in Japan and around the world," said Steve Hughes, M.D., chief medical officer at Avidity. "We are very encouraged by del-desiran data reported from the MARINA and MARINA-OLE studies thus far, demonstrating favorable long-term safety and tolerability, reversal of disease progression, and consistent and durable improvements in multiple clinical endpoints. Looking ahead, there are multiple key milestones this year as we complete enrollment in the phase 3 HARBOR trial and continue to advance our global commercialization preparations to potentially deliver the first globally approved drug for people living with DM1." Avidity has aligned with global regulators on the registrational path for del-desiran for the treatment of DM1, which informed the design of the ongoing Phase 3 HARBOR™ study. Avidity expects to complete participant enrollment in the Phase 3 HARBOR study in mid-2025 and submit marketing applications starting 2026 in the U.S., European Union and Japan. Japan's MHLW grants Orphan Drug designation to drugs in development for the treatment of diseases that affect fewer than 50,000 patients in Japan and for which there is a high unmet medical need. An investigational therapy is eligible to qualify for Orphan Drug designation if there is no approved alternative treatment option or if there is high efficacy or safety expected compared to existing treatment options. Orphan Drug designation provides certain benefits, including prioritized consultation regarding clinical development, reduced consultation fees, tax incentives, priority review of applications, reduced application fees, and extended registration validity period. About the Phase 3 HARBOR™ Trial The global Phase 3 HARBOR™ trial is a randomized, placebo-controlled, double blind pivotal study designed to evaluate del-desiran in approximately 150 people (age 16 and older) living with DM1. The trial will be conducted at approximately 40 sites globally. Patients will be administered either del-desiran or placebo (1:1) every eight weeks. HARBOR is designed to assess multiple key functional aspects of DM1. The primary endpoint is video hand opening time (vHOT), a measurement of myotonia, which is a hallmark symptom of DM1. Key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. All study participants, regardless of whether they receive active treatment or placebo, will have the option to enroll into an open-label extension trial. For more information about the HARBOR trial, visit the HARBOR study website or visit and search for NCT06411288. About the Phase 2 MARINA-OLE™ Study MARINA-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of del-desiran in participants with DM1 who were previously enrolled in the MARINA® Phase 1/2 trial. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-desiran in participants enrolled in the randomized, placebo-controlled, Phase 1/2 MARINA clinical trial. Participants enrolled in the MARINA-OLE study receive quarterly doses of del-desiran regardless of whether they received active treatment or placebo in the MARINA study. The total duration of active treatment with del-desiran in the MARINA-OLE study is approximately 24 months. Once patients have completed active treatment, there will be a nine-month safety follow-up period. Avidity may extend active treatment beyond 24 months at a future timepoint. For more information on this study click here or visit and search for NCT05479981. About Del-desiran Del-desiran, Avidity's lead product candidate utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. Del-desiran consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. Del-desiran is currently being assessed in the global Phase 3 HARBOR™ trial and in the ongoing MARINA-OLE™ trial in people with DM1. Long-term data from the MARINA-OLE trial showed reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT) as a measure of hand function and myotonia, muscle strength and activities of daily living when compared to END-DM1 natural history data. Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). About Myotonic Dystrophy Type 1 Myotonic dystrophy type 1 (DM1) is an underrecognized, autosomal dominantly inherited, progressive and often fatal disease caused by a triplet-repeat in the DMPK gene, resulting in a toxic gain of function mRNA. The disease is highly variable with respect to severity, presentation and age of onset, however all forms of DM1 are associated with high levels of disease burden and may cause premature mortality. DM1 primarily affects skeletal and cardiac muscle, however patients can suffer from a constellation of manifestations including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment. Currently, there are no approved treatments for people living with DM1. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare neuromuscular diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit and engage with us on LinkedIn and X. Forward-Looking Statements Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company's current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: planned marketing applications for del-desiran in the U.S., European Union and Japan, and the timing thereof; Avidity's plans to become a global commercial organization and the status of its commercialization efforts; the characterization of data associated with del-desiran in the MARINA and MARINA-OLE studies, the conclusions drawn therefrom, and the impact of such data on the advancement of del-desiran and its ability to treat DM1; the designs, goals, statuses and enrollment levels of, and plans for, the MARINA-OLE and HARBOR studies; Avidity's platform, planned operations and programs; and Avidity's cash position and runway. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: Avidity may not realize any benefit from Orphan Drug designation of del-desiran by global health authorities; the data and results produced in Avidity's ongoing studies of del-desiran as of the most recent respective cutoff dates may not be indicative of final results, may not support a BLA submission, may not be satisfactory to the MHLW and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; even if approved, Avidity may not be able to execute any successful product launches; Avidity's efforts to build a global commercial organization may be unsuccessful; unexpected adverse side effects to, or inadequate efficacy of, del-desiran that may delay or limit its development, regulatory approval and/or commercialization; later developments with the MHLW and other global regulators that could be inconsistent with the feedback received to date; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of the MARINA-OLE and HARBOR studies; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; legislative, judicial and regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2024. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact: Kat Lange (619) 837-5014 [email protected] Media Contact: (619) 837-5016 [email protected] SOURCE Avidity Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

« Back 02/26/25 4:05 PM EST – Net product revenues for the fourth quarter 2024 totaled $638.2 million , a 75% increase over the same quarter of the prior year – ELEVIDYS net product revenue for the quarter totaled $384.2 million ; Royalty revenue from the sales of ELEVIDYS by Roche for the quarter totaled $4.9 million – Achieved GAAP and non-GAAP net income of $159.0 million and $206.0 million for the fourth quarter of 2024, respectively CAMBRIDGE, Mass. --(BUSINESS WIRE)--Feb. 26, 2025-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2024. “2024 performance represented the fruition of our multi-year strategy to become a self-sustaining profitable biotech dedicated to improving the lives of patients with rare genetic disease. After obtaining a broad label for our gene therapy ELEVIDYS covering the vast majority of Duchenne patients, we had the most successful gene therapy launch in history, even as we continued to serve the community with our PMOs, EXONDYS 51, VYONDYS 53 and AMONDYS 45. And as we advanced our internal gene therapy pipeline, we also continued our diversification and secured our future by in-licensing a broad platform of siRNA programs, with potential blockbuster opportunities that could reach the market in 2028 and 2029,” said Doug Ingram , president and chief executive officer, Sarepta Therapeutics . “In 2025, we intend to capitalize on our 2024 achievements. In addition to 2025 net product revenue guidance of $2.9 billion to $3.1 billion , representing 70% year-over-year growth and 162% yearly growth for ELEVIDYS, we expect to reach multiple important milestones this year, including the proof of biology readouts in our myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy type 1 (FSHD) programs and the Biologics License Application (BLA) submission for SRP-9003 which, if successful, would lead to our first approval in our LGMD pipeline.” Fourth Quarter 2024 and Recent Developments: Established inaugural $600 million senior secured revolving credit facility: This instrument, available to Sarepta because of the Company’s financial strength and positive business outlook, provides flexibility to use non-dilutive financing to supplement a strong balance sheet and offer contingent liquidity in execution of the Company’s strategic plan. Announced positive results from Part 2 of the EMBARK study: In February, Sarepta reported topline results from Part 2 of EMBARK (Study SRP-9001-301), a Phase 3 clinical study of ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for Duchenne muscular dystrophy. The study showed that crossover-treated patients, who received ELEVIDYS after initially receiving a placebo, improved 2.34 points from baseline compared to matched external controls on the North Star Ambulatory Assessment (NSAA) 52 weeks after treatment (P<0.0001), during which time the study remained blinded. Despite being one year older (average age 7.18 years) than those treated in Part 1 (average age 5.98 years), crossover-treated patients showed clinically meaningful and statistically significant functional benefit for NSAA, Time to Rise (TTR), and 10-meter walk/run (10MWR) function tests compared with a pre-specified, propensity-weighted external control group (EC). At two years the Part 1 patients showed clinically meaningful and statistically significant functional benefit in NSAA, TTR and 10MWR compared with EC. Patients treated in Part 1 of the study had biopsies taken at 64 weeks after dosing and showed consistent and sustained expression of ELEVIDYS micro-dystrophin compared to week 12 biopsies, as measured by western blot, and provide biological support for observed functional outcomes. Additionally, skeletal MRI conducted on Part 1 patients indicated minimal muscle pathology progression, aligning with observed functional benefits. These results contribute to the growing body of clinical evidence supporting both the durability of ELEVIDYS treatment and the importance of intervening early to preserve muscle. The safety profile of ELEVIDYS remained consistent with previous findings. Closed global licensing and collaboration agreement with Arrowhead Pharmaceuticals: Sarepta has obtained exclusive global rights to four clinical-stage and three preclinical-stage programs in muscle, central nervous system, and rare pulmonary disorders, including potential best-in-class siRNA-based treatments for DM1 and FSHD. The agreement also encompasses a discovery collaboration for up to six additional muscle, cardiac and or CNS targets, using Arrowhead’s novel delivery technologies. The agreement adds meaningfully to Sarepta’s mid- and early-stage pipeline, complementing the Company’s existing leadership in Duchenne muscular dystrophy and limb-girdle muscular dystrophies and gene therapy, while adding new indications and expanding into adjacent therapeutic areas. In addition, Doug Ingram , president and chief executive officer, Sarepta, has been appointed to Arrowhead’s Board of Directors. The clinical-stage programs covered under the agreement are: SRP-1001 (formerly ARO-DUX4): designed to reduce the production of human double homeobox 4 (DUX4) protein in skeletal muscle; currently in a Phase 1/2 clinical study for the treatment of facioscapulohumeral muscular dystrophy (FSHD) SRP-1003 (formerly ARO-DM1): designed to target and suppress myotonic dystrophy protein kinase (DMPK) in skeletal muscle; Phase 1/2 clinical study for myotonic dystrophy type 1 (DM1) SRP-1002 (formerly ARO-MMP7): designed to reduce expression of matrix metalloproteinase 7 (MMP7) in pulmonary epithelial cells; Phase 1/2 clinical study for idiopathic pulmonary fibrosis (IPF) SRP-1004 (formerly ARO-ATXN2): designed to target the ataxin-2 protein (ATXN2) in the CNS; Phase 1/2 clinical study for spinocerebellar ataxia 2 (SCA2) commenced at the end of 2024 SRP-1001 (formerly ARO-DUX4): designed to reduce the production of human double homeobox 4 (DUX4) protein in skeletal muscle; currently in a Phase 1/2 clinical study for the treatment of facioscapulohumeral muscular dystrophy (FSHD) SRP-1003 (formerly ARO-DM1): designed to target and suppress myotonic dystrophy protein kinase (DMPK) in skeletal muscle; Phase 1/2 clinical study for myotonic dystrophy type 1 (DM1) SRP-1002 (formerly ARO-MMP7): designed to reduce expression of matrix metalloproteinase 7 (MMP7) in pulmonary epithelial cells; Phase 1/2 clinical study for idiopathic pulmonary fibrosis (IPF) SRP-1004 (formerly ARO-ATXN2): designed to target the ataxin-2 protein (ATXN2) in the CNS; Phase 1/2 clinical study for spinocerebellar ataxia 2 (SCA2) commenced at the end of 2024 Enrollment and dosing completed in EMERGENE (Study SRP-9003-301) for LGMD2E/R4: EMERGENE is a Phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec), an investigational gene therapy for the treatment of limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E/R4), or beta-sarcoglycanopathy. EMERGENE is a global study, and the primary endpoint is the biomarker expression of beta-sarcoglycan protein, the absence of which is the sole cause of LGMD2E/R4. The design of the trial is notable as it sets an important precedent informing development plans for Sarepta’s other LGMD pipeline programs, including LGMD2D and LGMD2C for which clinical trials are underway. Data from EMERGENE are expected in the first half of 2025. Following a positive pre-BLA meeting with FDA, the Company remains on track to submit a BLA filing later this year seeking accelerated approval for SRP-9003. Conference Call The event will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone. Q4 2024 Financial Highlights1 For the Three Months Ended December 31 , 2024 2023 QTD Change (in millions, except for per share amounts) $ % Total Revenues $ 658.4 $ 396.8 261.6 66 % Operating income: GAAP $ 161.7 $ 24.6 137.1 NM* Non-GAAP $ 221.2 $ 81.1 140.1 173 % Net income: GAAP $ 159.0 $ 45.7 113.3 NM* Non-GAAP $ 206.0 $ 86.6 119.4 138 % Diluted earnings per share: GAAP $ 1.50 $ 0.47 1.03 NM* Non-GAAP $ 1.90 $ 0.82 1.08 132 % For the Twelve Months Ended December 31 , 2024 2023 YTD Change (in millions, except for per share amounts) $ % Total Revenues $ 1,902.0 $ 1,243.3 658.7 53 % Operating income (loss): GAAP $ 218.1 $ (267.8 ) 485.9 NM* Non-GAAP $ 437.7 $ (42.5 ) 480.2 NM* Net income (loss): GAAP $ 235.2 $ (536.0 ) 771.2 NM* Non-GAAP $ 397.9 $ (59.5 ) 457.4 NM* Diluted earnings (loss) per share: GAAP $ 2.34 $ (5.80 ) 8.14 NM* Non-GAAP $ 3.69 $ (0.64 ) 4.33 NM* *NM: not meaningful [1] For an explanation of our use of non-GAAP financial measures, please refer to the “Use of Non-GAAP Financial Measures” section later in this press release, and for a reconciliation of each non-GAAP financial measure to the most comparable GAAP measures, see the tables at the end of this press release. As of December 31, 2024 As of December 31, 2023 (in millions) Cash, cash equivalents, restricted cash and investments $ 1,503.5 $ 1,691.8 Revenues Total revenues increased by $261.6 million and $658.7 million for the three and twelve months ended December 31, 2024 , respectively, compared to the same periods of 2023. The increases primarily reflect the initial product launch of ELEVIDYS in June 2023 and subsequent expanded label approval in June 2024 . Additionally, included in total revenues for the three and twelve months ended December 31, 2024 , is $20.3 million and $66.0 million , respectively, of contract manufacturing and other revenues associated with commercial ELEVIDYS supply delivered to Roche and royalty revenue received from Roche, as compared to $9.2 million of contract manufacturing and other revenues for the three and twelve months ended December 31, 2023 . Cost of sales (excluding amortization of in-licensed rights) Cost of sales (excluding amortization of in-license rights) increased by $88.1 million and $168.8 million for the three and twelve months ended December 31, 2024 , respectively, compared with the same periods of 2023. The increases in both periods primarily reflect the initial product launch of ELEVIDYS in June 2023 and subsequent expanded label approval in June 2024 , as well as cost of sales related to products sold to Roche under our collaboration agreement increasing by $5.1 million and $20.4 million for the three and twelve months ended December 31, 2024 , respectively, compared with the same periods of 2023. Operating expenses and others Research and development expenses increased by $4.4 million for the three months ended December 31, 2024 , compared with the same period of 2023, primarily as a result of an increase in manufacturing expense related to a ramp up of batches produced for our Limb-girdle muscular dystrophy (“LGMD”) programs, partially offset by a decrease in clinical trial expenses primarily due to our decision to discontinue our PPMO program during 2024. Research and development expenses decreased by $72.9 million for the twelve months ended December 31, 2024 , compared with the same period of 2023. The decrease in research and development expense primarily reflects capitalization of commercial batches of ELEVIDYS manufactured after its approval in June 2023 , partially offset by costs associated with the termination of the development, commercial manufacturing and supply agreement (the “Thermo Agreement”) related to Brammer Bio MA, LLC , an affiliate of Thermo Fisher Scientific, Inc. in August 2024 , net of the reimbursable termination costs by Roche. Non-GAAP research and development expenses increased by $7.6 million for the three months ended December 31, 2024 , compared with the same period of 2023. Non-GAAP research and development expenses decreased by $57.4 million for the twelve months ended December 31, 2024 , compared with the same period of 2023. Selling, general and administrative expenses increased by $32.2 million and $76.0 million for the three and twelve months ended December 31, 2024 , compared with the same periods of 2023. The increase in selling, general and administrative expenses for both periods is primarily driven by professional services used to support the continued efforts to commercialize ELEVIDYS and ongoing litigation matters, the timing of charitable contributions, and compensation-related expenses, including stock-based compensation, partially due to changes in headcount. Non-GAAP selling, general and administrative expenses increased by $25.9 million and $66.3 million for the three and twelve months ended December 31, 2024 , respectively, compared with the same periods of 2023. For the three months ended December 31, 2024 , other income, net decreased by $5.7 million , compared with the same period of 2023, which primarily reflects a decrease in interest income and accretion of investment discount, net as a result of lower interest rates and the investment mix of our investment portfolio during the three months ended December 31, 2024 . For the twelve months ended December 31, 2024 , other income (loss), net increased by $295.0 million compared with the same period of 2023, which primarily reflects a $387.3 million loss on debt extinguishment, partially offset by a $102.0 million gain on the sale of a Priority Review Voucher (“PRV”) during the twelve months ended December 31, 2023 , with no similar activities in 2024. Income tax expense for the three months ended December 31, 2024 , was approximately $12.7 million . Income tax benefit for the three months ended December 31, 2023 , was approximately $5.3 million . Income tax expense for the twelve months ended December 31, 2024 and 2023, was approximately $25.5 million and $15.9 million , respectively. Income tax expense (benefit) for all periods presented primarily relates to state, federal and foreign income taxes for which available tax losses or credits were not available to offset. Use of Non-GAAP Measures In addition to the GAAP financial measures set forth in this press release, we have included the following non-GAAP measurements: The following components are used to adjust our GAAP financial measures into the previously defined non-GAAP measurements: a. 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. b. 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. c. The gain from sale of the PRV obtained as a result of the Food and Drug Administration's (“FDA”) accelerated approval of ELEVIDYS in June 2023 as it is a non-recurring event and is not indicative of our core operations. d. The change in fair value of derivatives related to regulatory-related contingent payments meeting the definition of a derivative to Myonexus selling shareholders as well as to an academic institution under a separate license agreement as these are non-cash items and are not considered to be normal operating expenses due to the variability of amounts and lack of predictability as to occurrence and/or timing. We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We also believe these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating our performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of our financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP other income and loss adjustments, non-GAAP operating income (loss), non-GAAP net income (loss), and non-GAAP diluted earnings (loss) per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.” About EXONDYS 51 EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion, or “skipping”, of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. EXONDYS 51 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information About EXONDYS 51 Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy. Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended. The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting. Other adverse events may occur. To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. For further information, please see the full Prescribing Information. About VYONDYS 53 VYONDYS 53 (golodirsen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. VYONDYS 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental. Important Safety Information for VYONDYS 53 CONTRAINDICATIONS: VYONDYS 53 is contraindicated in patients with a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Anaphylaxis has occurred in patients receiving VYONDYS 53. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion, interrupting, or discontinuing the VYONDYS 53 therapy and monitor until the condition resolves. VYONDYS 53 is contraindicated in patients with a history of a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Kidney Toxicity: Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 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 and technologies; the potential benefits of our technologies, scientific approaches and strategic partnerships; and expected milestones and plans, including the potential for siRNA programs to reach the market in 2028 and 2029, our expectation to disclose proof of biology readouts in our myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy type 1 (FSHD) programs in 2025, and announcing data from EMERGENE in the first half of 2025 with a potential Biologics License Application (BLA) submission for SRP-9003 in 2025. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: success in preclinical and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; the expected benefits and opportunities related to our agreements with strategic partners may not be realized or may take longer to realize than expected due to a variety of reasons, including any inability of the parties to perform their commitments and obligations, challenges and uncertainties inherent in product research and development and manufacturing limitations; we may not be able to comply with all FDA post-approval commitments and requirements with respect to our products in a timely manner or at all; certain programs may never advance in the clinic or may be discontinued for a number of reasons, including regulators imposing a clinical hold and us suspending or terminating clinical research or trials; if the actual number of patients suffering from the diseases we aim to treat is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and timelines, research and clinical development plans, and bringing our product candidates to market, for various reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in our most recent Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review. Internet Posting of Information We routinely post information that may be important to investors in the 'Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us. Sarepta Therapeutics, Inc. Condensed Consolidated Statements of Income (Loss) (unaudited, in thousands, except per share amounts) For the Three Months Ended December 31 , For the Twelve Months Ended December 31 , 2024 2023 2024 2023 Revenues: Products, net $ 638,157 $ 365,071 $ 1,787,960 $ 1,144,876 Collaboration and other 20,255 31,710 114,019 98,460 Total revenues 658,412 396,781 1,901,979 1,243,336 Cost and expenses: Cost of sales (excluding amortization of in-licensed rights) 132,304 44,176 319,099 150,343 Research and development 199,953 195,517 804,522 877,387 Selling, general and administrative 163,873 131,700 557,872 481,871 Amortization of in-licensed rights 601 763 2,405 1,559 Total cost and expenses 496,731 372,156 1,683,898 1,511,160 Operating income (loss) 161,681 24,625 218,081 (267,824 ) Other income (loss), net: Other income, net 10,062 15,746 42,693 33,055 Gain from sale of Priority Review Voucher — — — 102,000 Loss on debt extinguishment — — — (387,329 ) Total other income (loss), net 10,062 15,746 42,693 (252,274 ) Income (loss) before income tax expense (benefit) 171,743 40,371 260,774 (520,098 ) Income tax expense (benefit) 12,694 (5,284 ) 25,535 15,879 Net income (loss) $ 159,049 $ 45,655 $ 235,239 $ (535,977 ) Earnings (loss) per share: Basic $ 1.65 $ 0.49 $ 2.47 $ (5.80 ) Diluted $ 1.50 $ 0.47 $ 2.34 $ (5.80 ) Weighted average number of shares of common stock used in computing earnings (loss) per share: Basic 96,283 93,617 95,075 92,398 Diluted 108,474 97,494 107,875 92,398 Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands, except per share amounts) For the Three Months Ended December 31 , For the Twelve Months Ended December 31 , 2024 2023 2024 2023 GAAP net income (loss) $ 159,049 $ 45,655 $ 235,239 $ (535,977 ) Interest income, net (10,753 ) (17,469 ) (53,909 ) (64,034 ) Depreciation and amortization expense 9,854 10,609 35,319 42,838 Stock-based compensation expense 49,676 45,826 184,300 182,514 Change in fair value of derivatives (727 ) — 7,838 1,200 Gain from sale of Priority Review Voucher — — — (102,000 ) Loss on debt extinguishment — — — 387,329 Impairment of strategic investments — 2,500 — 30,321 Income tax effect of adjustments (1,092 ) (541 ) (10,864 ) (1,738 ) Non-GAAP net income (loss) $ 206,007 $ 86,580 $ 397,923 $ (59,547 ) GAAP earnings (loss) per share - diluted: $ 1.50 $ 0.47 $ 2.34 $ (5.80 ) Add: impact of GAAP to Non-GAAP adjustments $ 0.40 $ 0.35 $ 1.35 $ 5.16 Non-GAAP earnings (loss) per share - diluted* $ 1.90 $ 0.82 $ 3.69 $ (0.64 ) Weighted average number of shares of common stock used in computing diluted earnings (loss) per share:** GAAP 108,474 97,494 107,875 92,398 Non-GAAP 108,474 105,594 107,875 92,398 *Non-GAAP earnings per share is calculated using diluted shares whereas non-GAAP net loss per share is calculated using basic shares as all other instruments are anti-dilutive. **The difference between the weighted average number of shares of common stock used in computing diluted GAAP and non-GAAP earnings per share for the three months ended December 31, 2023 , is a result of the exclusion of the potential share settlement of the 2027 Convertible Notes from the GAAP earnings per share as the inclusion of such shares was anti-dilutive. For the Three Months Ended December 31 , For the Twelve Months Ended December 31 , 2024 2023 2024 2023 Total effective tax rate, GAAP 7.4 % (12.0 ) % 9.8 % (3.1 ) % Less: impact of GAAP to Non-GAAP adjustments (1.1 ) (0.7 ) (1.4 ) (39.0 ) Total effective tax rate, Non-GAAP 6.3 % (12.7 ) % 8.4 % (42.1 ) % Sarepta Therapeutics, Inc. Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures (unaudited, in thousands) For the Three Months Ended December 31 , For the Twelve Months Ended December 31 , 2024 2023 2024 2023 GAAP research and development expenses $ 199,953 $ 195,517 $ 804,522 $ 877,387 Stock-based compensation expense (19,897 ) (22,174 ) (74,010 ) (82,489 ) Depreciation and amortization expense (7,356 ) (8,217 ) (26,048 ) (33,011 ) Non-GAAP research and development expenses $ 172,700 $ 165,126 $ 704,464 $ 761,887 For the Three Months Ended December 31 , For the Twelve Months Ended December 31 , 2024 2023 2024 2023 GAAP selling, general and administrative expenses $ 163,873 $ 131,700 $ 557,872 $ 481,871 Stock-based compensation expense (29,779 ) (23,652 ) (110,290 ) (100,025 ) Depreciation expense (2,498 ) (2,392 ) (9,271 ) (9,827 ) Non-GAAP selling, general and administrative expenses $ 131,596 $ 105,656 $ 438,311 $ 372,019 For the Three Months Ended December 31 , For the Twelve Months Ended December 31 , 2024 2023 2024 2023 GAAP operating income (loss) $ 161,681 $ 24,625 $ 218,081 $ (267,824 ) Stock-based compensation expense 49,676 45,826 184,300 182,514 Depreciation and amortization expense 9,854 10,609 35,319 42,838 Non-GAAP operating income (loss) $ 221,211 $ 81,060 $ 437,700 $ (42,472 ) Sarepta Therapeutics, Inc. Condensed Consolidated Balance Sheets (unaudited, in thousands, except share and per share data) As of December 31 , 2024 2023 Assets Current assets: Cash and cash equivalents $ 1,103,010 $ 428,430 Short-term investments 251,782 1,247,820 Accounts receivable, net 601,988 400,327 Inventory 749,960 322,859 Manufacturing-related deposits and prepaids 276,262 102,181 Other current assets 90,461 77,714 Total current assets 3,073,463 2,579,331 Property and equipment, net 340,336 227,154 Right of use assets 148,310 129,952 Non-current inventory 187,986 191,368 Non-current investments 133,163 — Other non-current assets 79,915 136,771 Total assets $ 3,963,173 $ 3,264,576 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 214,442 $ 164,918 Accrued expenses 373,513 314,997 Deferred revenue, current portion 130,256 50,416 Current portion of long-term debt — 105,483 Other current liabilities 13,473 17,845 Total current liabilities 731,684 653,659 Long-term debt 1,137,124 1,132,515 Lease liabilities, net of current portion 192,473 140,965 Deferred revenue, net of current portion 325,000 437,000 Contingent consideration 47,400 38,100 Other non-current liabilities 1,750 3,000 Total liabilities 2,435,431 2,405,239 Stockholders’ equity: Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding — — Common stock, $0.0001 par value, 198,000,000 shares authorized; 96,900,496 and 93,731,831 issued and outstanding at December 31, 2024 and 2023, respectively 10 9 Additional paid-in capital 5,738,924 5,304,623 Accumulated other comprehensive (loss) income, net of tax (218 ) 918 Accumulated deficit (4,210,974 ) (4,446,213 ) Total stockholders’ equity 1,527,742 859,337 Total liabilities and stockholders’ equity $ 3,963,173 $ 3,264,576 View source version on businesswire.com: https://www.businesswire.com/news/home/20250226046752/en/ Investor Contact: Ian Estepan , 617-274-4052 iestepan@sarepta.com Media Contact: Tracy Sorrentino , 617-301-8566 tsorrentino@sarepta.com Source: Sarepta Therapeutics, Inc.