US Market News
3月前
When Science Becomes an Asset: How Advancing Drug Pipelines Are Driving Real-Time Valuation in BiotechApril 20, 2026 9:00 AM
InvestorsHub NewsWireWhen Science Becomes an Asset: How Advancing Drug Pipelines Are Driving Real-Time Valuation in BiotechBioMedWire Editorial Coverage: Biotechnology is undergoing a quiet but profound transformation, one that is reshaping how investors understand value in a sector historically defined by long timelines and uncertain outcomes. As drug candidates advance closer to commercialization, scientific progress is no longer viewed solely as research expenditure but increasingly as a measurable financial asset. This shift is being reinforced by fair-value accounting under U.S. GAAP, which allows life sciences companies to reflect clinical progress, probability of success and commercialization timing as measurable balance sheet value. Leading companies, such as Oncotelic Therapeutics Inc. (OTCQB: OTLC) (Profile), are keenly aware of this evolution and are leveraging their expertise as pioneers in this space. Through its diversified pipeline and strategic holdings, including a 45% ownership stake in GMP Bio, which was recently measured at more than $1 billion enterprise value, the company exemplifies how advancing science can directly influence financial positioning. As the biotech sector increasingly aligns valuation with progress rather than revenue alone, Oncotelic provides a case study in how innovation is becoming a recognized asset class. Oncotelic joins an elite group of other key companies focused on advanced biotech platforms that target disease at the genetic or molecular level, including Sarepta Therapeutics Inc. (NASDAQ: SRPT), Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY), Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) and Denali Therapeutics Inc. (NASDAQ: DNLI).The biotechnology sector is evolving from a model centered on long-term R&D investment into one where scientific advancement can be reflected as measurable financial value.As drug candidates advance through clinical development, their value increases significantly due to rising probabilities of success and proximity to commercialization.The rise of fair-value accounting in biotechnology is enabling companies to provide more transparent and timely representations of asset value.Institutional investors are increasingly recognizing clinical-stage biotech assets as investable opportunities, even in the absence of current revenue.Oncotelic Therapeutics operates directly within this convergence, combining oncology-focused drug development with AI-enabled platforms and strategic manufacturing exposure.Click here to view the custom infographic of the Oncotelic Therapeutics editorial.Scientific Progress Is Becoming Financial ValueThe biotechnology sector is evolving from a model centered on long-term R&D investment into one where scientific advancement can be reflected as measurable financial value. Historically, investors relied heavily on discounted cash-flow projections tied to eventual commercialization, often leaving a gap between innovation and valuation. However, the adoption of fair-value frameworks under U.S. GAAP is beginning to bridge that gap by allowing companies to reassess asset value as clinical milestones are achieved.Under Financial Accounting Standards Board guidance, particularly ASC 820, companies can measure the fair value of assets, including illiquid or early-stage investments, based on observable and unobservable inputs. This includes Level 3 assets, which are commonly used in biotech to value pipeline-stage programs and strategic investments. These frameworks allow companies to incorporate scientific progress, regulatory advancement and probability of success into financial reporting.Advisory firms such as Deloitte and PwC note that in life sciences, valuation can reflects forward-looking indicators such as clinical progress and expected commercialization potential, as GAAP fair-value frameworks require assets to be measured based on market participant assumptions about future economic benefits. This approach aligns financial reporting more closely with operational reality, particularly in sectors where revenue may lag innovation by years.This shift is particularly relevant for Oncotelic Therapeutics, which operates in a space where pipeline advancement is a primary driver of value. By aligning its financial reporting with the progression of its therapeutic programs and strategic investments, the company reflects the broader industry movement toward recognizing science itself as an asset.Pipeline Proximity Drives Valuation InflectionAs drug candidates advance through clinical development, their value increases significantly due to rising probabilities of success and proximity to commercialization. This phenomenon — often referred to as valuation inflection — is well documented across the biotech industry. Late-stage assets, particularly those approaching regulatory approval, tend to command a disproportionate share of enterprise value.According to McKinsey & Company, value creation in biopharma is heavily concentrated in late-stage assets, with companies generating the majority of returns as products progress toward approval and commercialization, reflecting reduced risk and clearer revenue visibility. Additional industry analysis shows that assets approaching commercialization drive significant increases in company valuation due to higher probability of success and expected market entry.This dynamic highlights a critical change in how investors assess biotech companies. Rather than focusing solely on current revenue or earnings, market participants increasingly evaluate the maturity and trajectory of pipeline assets. As a result, companies with strong late-stage programs can experience significant valuation increases even before generating commercial sales.For Oncotelic Therapeutics, this trend is particularly relevant, given the company's focus on advancing oncology therapeutics and its strategic positioning within high-value development pathways. As its programs progress and move closer to commercialization, the company's valuation profile reflects the broader industry shift toward pipeline-driven value creation.Fair-Value Accounting Gains Ground in BiotechThe rise of fair-value accounting in biotechnology is enabling companies to provide more transparent and timely representations of asset value. Under GAAP, Level 3 valuations are used to measure assets without observable market prices, relying on models that incorporate forward-looking assumptions such as expected cash flows and probability-weighted outcomes.The ASC 820 framework defines fair value as the exit price that would be received to sell an asset in an orderly transaction between market participants at the measurement date. It provides a structured hierarchy for the subsequent measurement of complex, illiquid assets using market-participant assumptions and the best available information, aligning financial reporting with a current, market-based view of an asset's value.Oncotelic Therapeutics exemplifies this approach through the remeasurement of its stake in GMP Bio, which reflects an approximate $1 billion-plus enterprise value. This demonstrates how clinical and strategic progress can translate directly into balance sheet strength, reinforcing the concept of science as a financial asset.Institutional Capital Embraces Prerevenue BiotechInstitutional investors are increasingly recognizing clinical-stage biotech assets as investable opportunities, even in the absence of current revenue. This shift reflects a growing confidence in structured valuation methodologies that incorporate scientific progress, rather than relying solely on traditional financial metrics.Many biotechnology companies are valued primarily on the strength of their pipelines rather than their existing revenue streams, reflecting the long development timelines and lack of early-stage earnings typical of the sector. Instead, valuation is closely tied to scientific progress, with factors such as clinical development stage, probability of success and regulatory milestones serving as key drivers of equity value. This approach underscores that value creation in biotech often occurs well before commercialization, as investors increasingly rely on milestone-based and probability-adjusted models to assess future potential.Within this context, Oncotelic Therapeutics is positioned to benefit from growing institutional interest in pipeline-driven valuation. Its diversified portfolio and strategic investments align with the types of assets that are increasingly attracting capital in the biotech sector.AI, GMP and Commercial Readiness ConvergeAdvancements in AI-driven drug development and GMP-compliant manufacturing are accelerating timelines and improving scalability across the biotech industry. These technologies enable more efficient clinical development, better data analysis and streamlined production processes, reducing both time and cost to market.AI is increasingly being used to identify drug targets, optimize trial design and analyze complex datasets, improving the probability of success in clinical programs. At the same time, GMP-compliant manufacturing platforms are enabling more reliable and scalable production, which is critical as therapies move toward commercialization.Research indicates that data-driven approaches are becoming central to biopharma value creation, with AI and advanced analytics playing a key role in accelerating development timelines. These advancements enhance the reliability of valuation models by reducing uncertainty and improving execution. This convergence of AI, manufacturing and clinical development is reshaping how value is created and measured in biotech. Companies that can integrate these capabilities are better positioned to advance therapies efficiently while maintaining regulatory compliance.Oncotelic Therapeutics operates directly within this convergence, combining oncology-focused drug development with AI-enabled platforms and strategic manufacturing exposure. This integrated approach supports both scientific advancement and financial valuation, positioning the company within one of the most significant growth trends in the biotechnology sector.The biotechnology sector is entering a new phase in which scientific progress is no longer viewed solely as a cost center but as a measurable financial asset. As fair-value accounting, pipeline maturity and AI-driven development reshape the industry, the traditional gap between innovation and valuation is narrowing.Companies such as Oncotelic Therapeutics illustrate how this transformation is unfolding in real time, demonstrating that clinical advancement can materially strengthen financial positioning even before revenue generation begins. As investors increasingly focus on pipeline-driven value, the ability to translate science into measurable economic impact is becoming one of the defining characteristics of modern biotech.From Research Spend to Revenue-Ready AssetsA new wave of biotechnology innovation is increasingly blurring the line between scientific progress and financial value creation. As drug candidates targeting disease at the genetic and molecular level advance through clinical development in 2026, progress is no longer viewed solely as research expenditure but as a measurable and evolving asset. Recent updates across the sector highlight how advancements in RNA interference, gene therapy and targeted delivery platforms are not only improving clinical outcomes but also enhancing the underlying value of biotech pipelines.Sarepta Therapeutics Inc. (NASDAQ: SRPT) announced approval of its clinical trial application for SRP-1005, its investigational treatment for Huntington's disease. Sarepta expects to initiate this first-in-human clinical trial of SRP-1005 (formerly ARO-HTT) in the second quarter of 2026. SRP-1005 is an investigational small interfering RNA ("siRNA") therapeutic. INSIGHTT is a phase 1, multicenter, dose-escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants.Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY) reported new clinical and real-world data from its cardiovascular ("CV") portfolio. The data was presented at the American College of Cardiology's Annual Scientific Session and Expo ("ACC.26"). The new data continues to support the benefits of vutrisiran in transthyretin amyloid cardiomyopathy (ATTR-CM), as well as the potential of zilebesiran for hypertension, underscoring the versatility of RNAi therapeutics across cardiovascular diseases.Arcturus Therapeutics Holdings Inc. (NASDAQ: ARCT) is advancing a suite of messenger RNA ("mRNA")–based therapies. The company's Q4 update highlights progress in both rare diseases and vaccine platforms, including its lead candidate, ARCT-032, an inhaled mRNA therapy for cystic fibrosis, which has demonstrated favorable safety and tolerability and is advancing into a phase 2 clinical study expected to begin in 2026. In addition, the update noted advancement with its ARCT-810 program for ornithine transcarbamylase ("OTC") deficiency, a rare genetic disorder, with ongoing regulatory interactions aimed at supporting future pivotal studies.Denali Therapeutics Inc. (NASDAQ: DNLI) is making significant progress in 2026 with its TransportVehicle(TM) platform. The platform is designed to enable the delivery of biologics across the blood-brain barrier, one of the most persistent challenges in treating neurological diseases. The company has outlined 2026 as a pivotal year, with plans to deliver its first TransportVehicle-enabled medicine to patients and advance two TV-enabled programs into clinical studies for Alzheimer's disease and a program for Pompe disease.These latest developments underscore a broader industry transition from discovery to execution, where scientific innovation is increasingly quantified in financial terms. As genetic and molecular therapies move closer to commercialization, the ability to demonstrate clinical efficacy, scalability and delivery precision is translating into tangible asset value within company pipelines. This convergence of science and finance is reshaping how biotechnology is evaluated, positioning advanced therapeutic platforms not just as research initiatives, but as structured, value-generating assets.For further information about Oncotelic Therapeutics Inc., visit the Oncotelic Therapeutics profile.About BioMedWireBioMedWire ("BMW") is a specialized communications platform with a focus on the latest developments in the Biotechnology (BioTech), Biomedical Sciences (BioMed) and Life Sciences sectors. 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Original: When Science Becomes an Asset: How Advancing Drug Pipelines Are Driving Real-Time Valuation in Biotech
US Market News
4月前
Sarepta Announces First Clinical Data from siRNA Pipeline Targeting FSHD1 and DM1March 25, 2026 8:05 AM
Business Wire
In early clinical results, Sarepta’s avß6 integrin-targeted siRNA approach achieves high muscle concentrations without dose limiting toxicity for FSHD1 and DM1
Company to host investor call on March 25, 2026, at 8:30 a.m. Eastern time
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today shared the first clinical results from two of its siRNA programs for neuromuscular diseases.
Early results from Phase 1/2 ascending dose studies of SRP-1001 for facioscapulohumeral muscular dystrophy type 1 (FSHD1) and SRP-1003 for myotonic dystrophy type 1 (DM1) demonstrated dose- dependent muscle exposure, early biomarker effects, and favorable tolerability, reinforcing scientific confidence in the potential for differentiated benefits of the avß6 integrin-targeted delivery platform. In addition, the Company has generated proof-of-concept data which found that after a single dose, both SRP-1001 and SRP-1003 support reduction, or knockdown, of the target protein or mRNA. In both studies, the majority of adverse events were mild to moderate and were not dose dependent.
For rare, genetic diseases, such as FSHD and DM1, which are caused by overexpression of mutant proteins or toxic mRNA, RNA-targeted therapies hold significant promise but have been limited by rapid degradation of drug before it reaches the intended cells. SRP-1001 and SRP-1003 are each designed with an optimized siRNA chemistry and a proprietary, avß6 integrin-targeted ligand, intended to enable the siRNA to enter the cell and penetrate muscle tissue. Through this targeted delivery approach, these investigational treatments aim to overcome some of the delivery and safety challenges of other approaches.
“We are pleased that these early clinical results showed high levels of siRNA delivery to muscle, with no saturation of muscle siRNA uptake or dose-limiting safety signals to date. We believe this supports the differentiated potential of this siRNA platform and strengthens our belief that this approach could meaningfully change the treatment landscape for patients with FSHD and DM1,” said Louise Rodino-Klapac, Ph.D., President, Research & Development and Technical Operations. “These preliminary clinical data show consistent dose-dependent increases in plasma and muscle drug exposures across clinical and nonclinical studies and suggest that the avß6 integrin-targeting ligand mediates robust siRNA muscle delivery, which we hypothesize will ultimately enable higher dosing and translate into clinical efficacy for patients with FSHD1 and DM1.”
Investor Webcast Information
The Company will host an investor call on March 25, 2026, at 8:30 a.m. Eastern time to discuss the data in greater detail. 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.
About FSHD and SRP-1001
Facioscapulohumeral muscular dystrophy (FSHD) is a rare genetic disease that causes progressive weakness of skeletal muscles. The disease typically affects muscles of the face, shoulder girdle, upper limbs, abdomen, pelvis, and legs, although the pattern and severity of muscle involvement vary widely among individuals. FSHD is caused by abnormal activation of the DUX4 gene on chromosome 4, leading to production of the DUX4 protein, which is toxic to muscle cells and drives muscle degeneration. There is currently no cure for FSHD, and no disease modifying treatments are available. Approximately 16,000 individuals in the United States are diagnosed with FSHD.
SRP-1001 is an investigational siRNA treatment designed to reduce, or knock down, the production of DUX4 protein in skeletal muscle in patients living with FSHD1. Study 1001-101 is combined Phase 1/2, single ascending dose (SAD)/multiple ascending dose (MAD), randomized, placebo-controlled trial in participants with FSHD1 aged 16 through 70.
About DM1 and SRP-1003
Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy and is caused by a repeat expansion in the DMPK gene. DM1 is a progressive, multisystem disorder that affects skeletal and smooth muscle, as well as the heart, respiratory system, eye, endocrine, gastrointestinal, and central nervous systems. The disease is commonly associated with muscle weakness, daytime sleepiness, breathing difficulties, and cardiac conduction abnormalities. There is currently no cure for DM1, and no disease modifying treatments are available. Approximately 40,000 individuals in the United States are diagnosed with DM1.
SRP-1003 is an investigational siRNA treatment for DM1 and is designed to target and knockdown, or silence, the DMPK mRNA in target cells. Study SRP-1003-101 is a first-in-human, Phase 1/2, SAD/MAD, randomized, placebo-controlled clinical trial being conducted in individuals with DM1 aged 18 to 65.
About Sarepta’s siRNA Platform
Sarepta’s next-generation siRNA platform is focused on chronically administered therapies for neurodegenerative and pulmonary diseases and includes the following investigational treatments:
SRP-1001 for Facioscapulohumeral muscular dystrophy (FSHD)
SRP-1002 for Idiopathic Pulmonary Fibrosis (IPF)
SRP-1003 for Myotonic dystrophy type 1 (DM1)
SRP-1004 for Spinocerebellar ataxia type 2 (SCA2)
SRP-1005 for Huntington’s disease (HD)
Sarepta is advancing these programs, as well as preclinical programs for Spinocerebellar ataxia type 1 (SCA1) and Spinocerebellar ataxia type 3 (SCA3) and additional discovery targets in muscle or central nervous system disorders, under an exclusive license with Arrowhead Pharmaceuticals.
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 a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.
Internet Posting of Information
We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
Forward-Looking Statements
This presentation contains forward-looking statements. Any statements contained in this presentation that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements related to our priorities, technologies and research and development programs; early and limited results from our Phase 1/2 clinical trials of SRP-1001 and SRP-1003; and the potential benefits of SRP-1001, and SRP-1003, and Arrowhead Pharmaceuticals’ TRiM™ platform.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: 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; differences in trial design between early-stage clinical trials and later-stage clinical trials make it difficult to extrapolate the results of earlier clinical trials to later clinical trials; interim, initial, “topline” and preliminary data from our clinical trials that we announce or publish from time to time may differ materially from final data as more patient data become available, including data and analyses of additional cohorts; pre-clinical and clinical data are often susceptible to varying interpretations and analyses, and the different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results; even if we believe data collected from clinical trials of our product candidates are promising, these data may not be sufficient to support approval, including that FDA or foreign regulatory authorities could interpret these data in different ways from us; we rely on third parties, including in some cases our strategic partners, to conduct some aspects of our early-stage research and pre-clinical and clinical development, and accordingly, the inadequate performance by or loss of any of these third parties could affect the development and commercialization of our product candidate development, including delayed timelines; 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; we face intense competition and rapid technological change, which may result in other companies discovering, developing or commercializing competitive products before our candidates; our product candidates may cause undesirable side effects, result in new safety signals or have other properties that could delay or prevent regulatory approval of product candidates; we may not be able to execute on our business plans, including meeting expected or planned regulatory milestones and timelines, clinical development plans, and bringing products to markets for various reasons including possible limitations of 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 Sarepta’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this presentation. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.
Source: Sarepta Therapeutics, Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260325621936/en/
Investor Contacts:
Ian Estepan, 617-274-4052, iestepan@sarepta.com
Ryan Wong, 617-800-4112, rwong@sarepta.com
Tam Thornton, 617-803-3825, tthornton@sarepta.com
Media Contact:
Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com
Original: Sarepta Announces First Clinical Data from siRNA Pipeline Targeting FSHD1 and DM1
US Market News
4月前
Sarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with DuchenneMarch 16, 2026 8:33 AM
Business Wire
- Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the ENDEAVOR study
- The enhanced immunosuppressive regimen is designed to mitigate the risk of acute liver injury (ALI) and acute liver failure (ALF) associated with AAV gene therapy in non-ambulatory patients
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced screening and enrollment are underway in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to assess prophylactic sirolimus treatment as part of an enhanced safety protocol during treatment with ELEVIDYS (delandistrogene moxeparvovec-rokl) in non-ambulant individuals with Duchenne muscular dystrophy.
Data from Cohort 8 will be used to determine whether administering sirolimus prior to and after ELEVIDYS infusion can help reduce acute liver injury (ALI), a known risk associated with AAV gene therapy. The cohort is enrolling approximately 25 participants in the U.S. who are non-ambulatory. The immunosuppression regimen will include 14 days of peri-infusion sirolimus dosing (prior to ELEVIDYS administration) and will continue for 12 weeks after ELEVIDYS administration. Primary endpoints include incidence of ALI and ELEVIDYS-dystrophin expression at 12 weeks. The approach is based on preclinical data and shaped by real-world clinical experience, including guidance from independent specialists in Duchenne and liver health.
“We are proud to announce that clinical trial sites are now open and actively recruiting non-ambulatory individuals with Duchenne to participate in ENDEAVOR Cohort 8,” said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta. “Individuals with non-ambulatory Duchenne face profound unmet need and fewer treatment options. Cohort 8 of ENDEAVOR is expected to build on the dystrophin expression data generated with ELEVIDYS to-date while deepening our understanding of its safety profile in older patients with more advanced disease so we can urgently advance this treatment option for them.”
Additional information can be found on clinicaltrials.gov (NCT04626674). ELEVIDYS is the only approved gene therapy for Duchenne. To date, ELEVIDYS has been administered to over 1,200 patients globally in clinical and real-world settings.
About ENDEAVOR (Study 9001-103)
Study SRP-9001-103, also known as ENDEAVOR, is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS (delandistrogene moxeparvovec) in multiple cohorts of male patients with Duchenne. The study has enrolled 55 participants across 7 cohorts and has dosed younger ambulatory individuals aged 2-7 at time of treatment, older ambulant individuals and non-ambulant individuals.
The primary endpoint in ENDEAVOR is the change from baseline in the quantity of ELEVIDYS micro-dystrophin protein expression measured by western blot at 12 weeks. Secondary outcome measures include change from baseline in micro-dystrophin expression measured by percent dystrophin positive fibers at 12 weeks.
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 ambulatory patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.
Limitations of Use
ELEVIDYS is not recommended in patients with:
Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limit of normal or total bilirubin > the upper limit of normal not due to Gilbert’s syndrome) or active hepatic viral infection due to the high risk of acute serious liver injury and acute liver failure.
Recent vaccination (within 4 weeks of treatment) due to immunogenicity and potential safety concerns.
Active or recent (within 4 weeks) infections due to safety concerns.
IMPORTANT SAFETY INFORMATION
BOXED WARNING: Acute Serious Liver Injury and Acute Liver Failure
Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred. Patients with preexisting liver impairment may be at higher risk.
Prior to infusion, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroids before and after ELEVIDYS infusion. Continue to monitor liver function weekly for the first 3 months after infusion and continue until results are unremarkable.
Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion.
Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected.
CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene.
WARNINGS AND PRECAUTIONS:
Acute Serious Liver Injury and Acute Liver Failure
See Boxed Warning.
Acute serious liver injury marked by elevations of liver enzymes (e.g., GGT, ALT) and total bilirubin and acute liver failure has occurred with ELEVIDYS. Onset of the liver injury typically begins within 8 weeks of ELEVIDYS administration. In non-ambulatory patients treated with ELEVIDYS, acute liver failure with fatal outcome has occurred in the clinical and post-marketing settings.
Life-threatening mesenteric vein thrombosis, complicated by bowel ischemia and necrosis, and portal hypertension have been reported following acute liver injury associated with ELEVIDYS in a non-ambulatory patient.
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 or acute liver failure. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.
Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated.
Serious Infections
Increased susceptibility to serious infections may occur due to concomitant administration of corticosteroid regimen and additional immunosuppressants, and ELEVIDYS. Serious respiratory infections, including with fatal outcomes, have occurred in patients taking immunosuppressant corticosteroids required for ELEVIDYS administration.
Monitor patients for signs and symptoms of infection before and after ELEVIDYS administration and treat appropriately.
Administer immunizations according to best clinical practices and immunization guidelines prior to initiation of the corticosteroid regimen required before ELEVIDYS infusion.
Avoid administration of ELEVIDYS to patients with active infections.
Myocarditis
Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed within 24 hours to more than 1 year following ELEVIDYS infusion.
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, until results return to near baseline levels or stabilize.
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.
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 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.
Immune-mediated Myositis
Immune-mediated myositis, including serious and life-threatening events, has occurred approximately 1 month following ELEVIDYS infusion. Signs and symptoms include severe muscle weakness, including dysphagia, dyspnea, dysphonia, and hypophonia.
Severe to life-threatening immune-mediated myositis has been reported in patients with deletions including portions of exons 1-17 and/or exons 59-71 of the DMD gene.
Regardless of genetic mutation, advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, dysphonia, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment based on patient’s clinical presentation and medical history if these symptoms occur.
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 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 ≥1:400.
ADVERSE REACTIONS
The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased.
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).
Please see the full Prescribing Information for ELEVIDYS, including Boxed Warning and Medication Guide.
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 a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.
Forward-Looking Statements
This press release contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our technologies, strategies, priorities, and future operations; ELEVIDYS, including the potential benefits of sirolimus in connection with ELEVIDYS; and our clinical trials, including Study 9001-103.
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: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials are positive, 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 our products; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; we may observe adverse reactions in our clinical trials or in patients who receive our approved products; our products may not be widely adopted by patients, payors or healthcare providers, which would adversely impact our business; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business; and those risks identified under the heading “Risk Factors” in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.
Internet Posting of Information
We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260316238001/en/
Investor Contacts:
Ian Estepan, 617-274-4052, iestepan@sarepta.com
Ryan Wong, 617-800-4112, rwong@sarepta.com
Tam Thornton, 617-803-3825, tthornton@sarepta.com
Media Contacts:
Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com
Kara Hoeger, 617-710-3898, khoeger@sarepta.com
Original: Sarepta Announces that Screening and Enrollment are Underway in ENDEAVOR Cohort 8 to Evaluate Enhanced Immunosuppression Regimen as Part of ELEVIDYS Gene Therapy for Non-Ambulant Individuals with Duchenne
US Market News
5月前
Sarepta Therapeutics Announces Commercial Launch of ELEVIDYS in JapanFebruary 24, 2026 9:07 AM
Business Wire
- Japan now offers ELEVIDYS gene therapy to children with Duchenne muscular dystrophy aged 3 years to less than 8 years
- Company is eligible to receive a $40 million milestone payment upon first commercial sale in Japan
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced the commercial launch of ELEVIDYS (delandistrogene moxeparvovec) in Japan by Chugai Pharmaceutical Co., Ltd., following its reimbursement listing on Japan’s National Health Insurance (NHI) price list.
Elevidys is the first gene therapy to be launched in Japan for Duchenne muscular dystrophy (DMD). In Japan, ELEVIDYS is available for ambulatory individuals with Duchenne ages 3-to less than 8-years-old, a deletion of any portion or the entirety of exon 8 and/or exon 9 in the DMD gene, and who are negative for anti-AAVrh74 antibodies.
“The commercial launch of ELEVIDYS in Japan marks an important step in expanding access to this therapy, where early intervention may offer the greatest opportunity to preserve muscle function,” said Louise Rodino-Klapac-, Ph.D., president of research & development and technical operations, Sarepta. “We are delighted that ELEVIDYS is now available to eligible patients in Japan and remain focused on advancing rigorous science and generating long-term clinical and real-world evidence to help inform care for the Duchenne community worldwide.”
Chugai announced that ELEVIDYS has been launched in Japan following reimbursement listing, enabling access for eligible patients under the conditional and time limited approval granted by Japan’s Ministry of Health, Labour and Welfare (MHLW) in May 2025. Chugai will be responsible for postmarketing clinical studies and all case postmarketing surveillance in Japan as part of the Roche Group collaboration to further evaluate long-term efficacy and safety.
The approval in Japan was based on efficacy and safety data from the ELEVIDYS clinical development program, including results from the global Phase 3 EMBARK study. EMBARK evaluated ELEVIDYS in ambulatory boys with DMD and demonstrated clinically meaningful improvements in key motor function measures. Longer term follow-up data have shown sustained functional efficacy over time and slower disease progression compared to external control, with no new treatment-related safety signals observed in ambulatory patients. Two-year EMBARK results were published in Neurology & Therapy in January, and three-year results will be shared at an upcoming medical congress. To date, ELEVIDYS has been administered to over 1,200 patients globally in clinical and real-world settings.
As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to transform the future for the Duchenne community, with the goal of enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and commercialization in territories outside the U.S. Commercialization of ELEVIDYS in Japan is through Chugai Pharmaceuticals, a member of the Roche Group.
Under Sarepta’s collaboration agreement with Roche, the first commercial sale of ELEVIDYS in Japan will trigger a $40 million milestone payment to Sarepta.
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.
In the United States, ELEVIDYS is indicated for the treatment of ambulatory patients 4 years of age and older with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.
The following is excerpted from the United States Prescribing Information (USPI).
Limitations of Use
ELEVIDYS is not recommended in patients with:
Preexisting liver impairment (defined as gamma-glutamyl transferase [GGT] > 2 x upper limit of normal or total bilirubin > the upper limit of normal not due to Gilbert’s syndrome) or active hepatic viral infection due to the high risk of acute serious liver injury and acute liver failure.
Recent vaccination (within 4 weeks of treatment) due to immunogenicity and potential safety concerns.
Active or recent (within 4 weeks) infections due to safety concerns.
IMPORTANT SAFETY INFORMATION
BOXED WARNING: Acute Serious Liver Injury and Acute Liver Failure
Acute serious liver injury, including life-threatening and fatal acute liver failure, has occurred. Patients with preexisting liver impairment may be at higher risk.
Prior to infusion, assess liver function by clinical examination and laboratory testing. Administer systemic corticosteroids before and after ELEVIDYS infusion. Continue to monitor liver function weekly for the first 3 months after infusion and continue until results are unremarkable.
Instruct patients to maintain proximity to an appropriate healthcare facility, as determined by the healthcare provider, for at least 2 months following ELEVIDYS infusion.
Obtain prompt consultation with a specialist (e.g., gastroenterologist or hepatologist) if acute serious liver injury or impending acute liver failure is suspected.
CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9, including a deletion of any portion or the entirety of these exons, in the DMD gene.
WARNINGS AND PRECAUTIONS:
Acute Serious Liver Injury and Acute Liver Failure
See Boxed Warning.
Acute serious liver injury marked by elevations of liver enzymes (e.g., GGT, ALT) and total bilirubin and acute liver failure has occurred with ELEVIDYS. Onset of the liver injury typically begins within 8 weeks of ELEVIDYS administration. In non-ambulatory patients treated with ELEVIDYS, acute liver failure with fatal outcome has occurred in the clinical and post-marketing settings.
Life-threatening mesenteric vein thrombosis, complicated by bowel ischemia and necrosis, and portal hypertension have been reported following acute liver injury associated with ELEVIDYS in a non-ambulatory patient.
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 or acute liver failure. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.
Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated.
Serious Infections
Increased susceptibility to serious infections may occur due to concomitant administration of corticosteroid regimen and additional immunosuppressants, and ELEVIDYS. Serious respiratory infections, including with fatal outcomes, have occurred in patients taking immunosuppressant corticosteroids required for ELEVIDYS administration.
Monitor patients for signs and symptoms of infection before and after ELEVIDYS administration and treat appropriately.
Administer immunizations according to best clinical practices and immunization guidelines prior to initiation of the corticosteroid regimen required before ELEVIDYS infusion.
Avoid administration of ELEVIDYS to patients with active infections.
Myocarditis
Acute, serious, life-threatening myocarditis and troponin-I elevations have been observed within 24 hours to more than 1 year following ELEVIDYS infusion.
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, until results return to near baseline levels or stabilize.
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.
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 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.
Immune-mediated Myositis
Immune-mediated myositis, including serious and life-threatening events, has occurred approximately 1 month following ELEVIDYS infusion. Signs and symptoms include severe muscle weakness, including dysphagia, dyspnea, dysphonia, and hypophonia.
Severe to life-threatening immune-mediated myositis has been reported in patients with deletions including portions of exons 1-17 and/or exons 59-71 of the DMD gene.
Regardless of genetic mutation, advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, dysphonia, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment based on patient’s clinical presentation and medical history if these symptoms occur.
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 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 ≥1:400.
ADVERSE REACTIONS
The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, thrombocytopenia, and troponin-I increased.
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).
Please see the full Prescribing Information for ELEVIDYS, including Boxed Warning and Medication Guide.
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 a leadership position in Duchenne muscular dystrophy (Duchenne) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit www.sarepta.com or follow us on LinkedIn, X, Instagram and Facebook.
Internet Posting of Information
We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
Forward-Looking Statements
This press release contains forward-looking statements. 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," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements.
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: 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; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences following any marketing approval; 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; we may not be able to execute on our business plans, including meeting expected or planned regulatory milestones and timelines, clinical development plans, and bringing products to markets for various reasons including possible limitations of 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 Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2024, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260224762469/en/
Investor Contacts:
Ian Estepan, 617-274-4052
iestepan@sarepta.com
Ryan Wong, 617-800-4112
rwong@sarepta.com
Media Contacts:
Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com
Kara Hoeger, 617-710-3898
khoeger@sarepta.com
Original: Sarepta Therapeutics Announces Commercial Launch of ELEVIDYS in Japan
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