Latest news with #FSHD
Malaysian Reserve
17-07-2025
- Health
- Malaysian Reserve
Facioscapulohumeral Muscular Dystrophy Market Gearing Up for Outstanding Expansion Across the 7MM During the Forecast Period (2025-2034)
According to DelveInsight's analysis, the market for facioscapulohumeral muscular dystrophy is anticipated to increase during the forecast period (2025–2034), owing to improved diagnosis, treatment advancements, aging demographics, increased awareness, the launch of emerging therapies, and healthcare spending globally. LAS VEGAS, July 17, 2025 /PRNewswire/ — Facioscapulohumeral muscular dystrophy (FSHD) often starts with muscle weakness in the face, shoulders, and upper arms, and gradually progresses to affect the abdominal muscles, legs, and pelvic area as the disease advances. Symptoms commonly emerge during childhood or adolescence, though they can begin at any stage of life, from infancy to old age. There is considerable variation in symptom onset and severity, even among relatives with the condition. Roughly 80% of individuals carrying the FSHD gene mutation experience symptoms, while the remaining 20% are asymptomatic, either showing no signs or only very mild symptoms that may go unnoticed until later in life. For FSHD1, children of an affected parent have a 50% likelihood of inheriting the disorder. In FSHD2, the inheritance risk depends on the genetic background of both parents, typically ranging from 25% to 50%. In 2024, the United States represented about 45% of all FSHD cases across the 7MM, with numbers projected to rise by 2034. Most US patients in 2024 scored between 7 and 10 on the RICCI scale, indicating moderate to severe levels of muscle dysfunction. Download the report to understand which factors are driving FSHD epidemiology trends @ Facioscapulohumeral Muscular Dystrophy Treatment Algorithm At present, there are no available therapies that can slow, halt, or reverse the progression of muscle weakness in facioscapulohumeral muscular dystrophy. While physical therapy can be beneficial in some cases, it is generally recommended that individuals engage in regular low-resistance and aerobic exercise. Collaborating with a physical therapist is important to create a personalized and safe exercise regimen. Some patients may benefit from surgical stabilization of the scapula to enhance their ability to raise their arms above shoulder level. Scapular fixation involves anchoring the shoulder blades to the ribs and may be performed on one or both sides. This is a complex procedure that should only be undertaken by skilled surgeons after thorough evaluation and discussion with both the surgeon and the neurologist. Additionally, medications such as NSAIDs, opioids, and antidepressants are commonly prescribed to manage FSHD-related pain. Current care practices are centered around managing complications and preserving physical function. Rehabilitation consultations are advised for individuals facing mobility or functional issues. However, there remains a significant unmet need for effective and affordable approved therapies, as existing supportive treatments are often costly and limited in efficacy. Learn more about the FSHD treatment @ New Treatment for Facioscapulohumeral Muscular Dystrophy There remains a significant unmet need in the treatment of FSHD, as current approaches primarily involve off-label therapies. Management strategies are largely aimed at alleviating symptoms and preserving physical function. Although FSHD is a progressive disorder with potentially serious long-term effects, treatment options are still limited to symptomatic care. Several investigational therapies are in development, including GYM329/RO7204239/RG6237 by Roche and Chugai Pharmaceutical, Delpacibart braxlosiran (del-brax) by Avidity Biosciences, EPI-321 by Epicrispr Biotechnologies, among others. Discover which therapies are expected to grab major FSHD market share @ Facioscapulohumeral Muscular Dystrophy Market Report GYM329 (also known as RO7204239 or RG6237) is an experimental anti-myostatin antibody aimed at enhancing skeletal muscle mass and growth. It has been specifically engineered as a 'recycling' and 'sweeping' antibody, meaning it may clear myostatin from the bloodstream more effectively than traditional antibodies. This targeted approach is expected to help address conditions marked by muscle wasting and reduced muscle strength. In November 2022, Roche began a Phase II clinical trial (NCT05548556) in patients with facioscapulohumeral muscular dystrophy, and according to the company, regulatory filing is anticipated after 2028. As per Chugai Pharmaceutical's Q1 2025 update, results from the ongoing MANOEUVRE Phase II study for FSHD are expected in 2025. With proof of concept established, Chugai plans to advance to Phase III trials, set to begin in 2026. Delpacibart braxlosiran (del-brax, formerly AOC 1020) is currently being assessed in the Phase I/II FORTITUDE trial (NCT06547216) in both adult and adolescent participants with FSHD. This study is focused on evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenously administered del-brax. In June 2025, Avidity Biosciences announced that del-brax may qualify for an accelerated approval pathway in the U.S. for FSHD treatment. The company also launched the global Phase III FORWARD trial to serve as a confirmatory study for full regulatory approval. Additionally, Avidity reported positive topline results from the dose-escalation cohorts of the FORTITUDE trial, which will be shared at the 32nd Annual FSHD Society International Research Congress. EPI-321 is an investigational, one-time gene-silencing therapy targeting abnormal DUX4 expression. Administered systemically via a validated AAV vector, EPI-321 has shown strong preclinical results, including effective suppression of DUX4 and preservation of muscle tissue. It has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA. The company is preparing to launch a global Phase I/II trial in 2025. Discover more about drugs for FSHD in development @ Facioscapulohumeral Muscular Dystrophy Clinical Trials The anticipated launch of these emerging therapies for FSHD are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the FSHD market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for FSHD is expected to grow at a significant CAGR by 2034. This growth is mainly driven by increased awareness, improved diagnostic techniques, and ongoing research into genetic therapies. Rising investment from pharmaceutical companies and the emergence of targeted therapies are expanding treatment options. Regulatory support for orphan diseases is also accelerating drug development. DelveInsight's latest published market report, titled as Facioscapulohumeral Muscular Dystrophy Market Insight, Epidemiology, and Market Forecast – 2034, will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the FSHD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The FSHD market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of FSHD Total Diagnosed Prevalent Cases of FSHD Type-specific Diagnosed Prevalent Cases of FSHD Gender-specific Diagnosed Prevalent Cases of FSHD Age-specific Diagnosed Prevalent Cases of FSHD Severity-specific Diagnosed Prevalent Cases of FSHD Total Treated Cases of FSHD The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM FSHD market. Highlights include: 10-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this FSHD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the FSHD market. Also, stay abreast of the mitigating factors to improve your market position in the FSHD therapeutic space. Related Reports Facioscapulohumeral Muscular Dystrophy Pipeline Facioscapulohumeral Muscular Dystrophy Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key FSHD companies, including Fulcrum Therapeutics, Dyne Therapeutics, Hoffmann-La Roche, aTyr Pharma, Inc., Avidity Biosciences, Inc., among others. Facioscapulohumeral Muscular Dystrophy Epidemiology Forecast Facioscapulohumeral Muscular Dystrophy Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted FSHD epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Market Duchenne Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key DMD companies, including Sarepta Therapeutics, PTC Therapeutics, Nippon Shinyaku, Santhera Pharmaceuticals, ReveraGen BioPharma, Taiho Pharmaceutical, FibroGen, Capricor, Daiichi Sankyo, Italfarmaco, Antisense Therapeutics, Solid Biosciences, among others. Spinal Muscular Atrophy Market Spinal Muscular Atrophy Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key SMA companies, including Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Therapeutics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact Us Shruti Thakur info@ +14699457679 Logo: View original content:
Cision Canada
16-07-2025
- Health
- Cision Canada
Springbok Analytics Publishes Predictive Disease Progression Model for Muscular Dystrophy
AI-based digital twin modeling predicts patient-specific decline, addressing key trial design and biomarker challenges in FSHD CHARLOTTESVILLE, Va, July 16, 2025 /CNW/ - Springbok Analytics, a leader in AI-driven muscle analytics, today announced the publication in Scientific Reports of a new disease progression model for facioscapulohumeral muscular dystrophy (FSHD). Springbok's model employs a machine learning–based approach that predicts patient-specific muscle decline, functional outcomes, and fat infiltration using data derived from whole-body MRI. Click here to read the full paper. Facioscapulohumeral muscular dystrophy (FSHD) is a progressive, genetic neuromuscular disorder affecting approximately 1 in 7,500 individuals. It is driven by the aberrant expression of the DUX4 protein, which leads to skeletal muscle wasting and functional decline. Although conventional clinical endpoints, such as the 6-minute walk test (6MWT), Timed Up and Go (TUG), and reachable workspace (RWS), are commonly used to monitor disease progression, these measures often lack sensitivity in short-duration trials due to high variability and patient heterogeneity. The recent inability of RWS to differentiate outcomes in a Phase III FSHD trial further underscored these limitations. "Traditional functional metrics are simply not the best way to measure disease given the complexity and heterogeneity of FSHD," said Silvia Blemker, Ph.D., Chief Scientific Officer and Co-Founder at Springbok Analytics. "What we've shown in this study is that muscle MRI, combined with advanced machine learning, can capture that complexity and make it actionable, supporting more sensitive, predictive, and patient-specific trial designs." Springbok's advanced modeling combines baseline imaging biomarkers with clinical and functional data to predict how an individual's disease will progress. The result is a patient-specific simulation, or "digital twin," capable of forecasting future declines in muscle structure and performance. This approach outperforms current methods that rely on pooled muscle group metrics or single-slice measurements, which can dilute signal and miss early changes. "FSHD doesn't follow a predictable pattern from person to person, and treating every patient as if they use the same muscles in the same way prevents therapeutic breakthroughs," said Scott Magargee, CEO and Co-Founder at Springbok Analytics. "Our team has built a model that learns from each person's muscle composition, clinical markers, and functional data to simulate how their disease may progress, establishing a foundation for digital twins and synthetic control arms that is exciting for not only the FSHD community but many other populations that could benefit from precision healthcare." Study Highlights: Personalized Prediction: The model accurately forecasted annual changes in fat fraction (a marker of muscle tissue replacement) and lean muscle volume using baseline MRI data. Functional Correlation: Model predictions aligned with changes in TUG time, a standard test of lower-body mobility, linking imaging biomarkers to meaningful, patient-relevant outcomes. Enhanced Sensitivity: The study revealed detailed spatial patterns of muscle degradation not visible in grouped or averaged analyses. "The model doesn't just predict what might happen," added Blemker. "It tells us why, where, and how fast it's happening for each individual. That's a powerful shift in how we approach trial design, and ultimately, how we understand muscle disease biology." Springbok's AI-powered platform, which received FDA 510(k) Clearance last year, rapidly analyzes dozens of individual muscles from a single MRI scan, quantifying volume, asymmetry, and fat infiltration. The technology is already integrated into multiple neuromuscular trials, including a first-in-human study of Epicrispr Biotechnologies' investigational therapy, EPI-321. As interest in MRI biomarkers grows across rare and neuromuscular disease trials, this publication establishes a framework for incorporating high-resolution, AI-powered models into trial design, patient stratification, and biomarker development. To explore how Springbok's platform can support your trial or development program, contact: [email protected]. About Springbok Analytics: Springbok Analytics is a leading muscle health analytics company dedicated to advancing health and performance outcomes through innovative, AI-driven solutions that deliver a clearer, more comprehensive view of muscle health. Built on more than 15 years of research and scientific validation, Springbok's FDA-cleared technology transforms MRI data into personalized, 3D visualizations of muscle health. These detailed analyses provide precise metrics, including individual muscle volume and composition, fat infiltration, asymmetries, scar tissue, edema, and tendon morphology. By offering a more accurate and complete understanding of musculoskeletal health, Springbok enhances diagnostic accuracy, treatment monitoring, research capabilities, and performance optimization.
Yahoo
17-06-2025
- Business
- Yahoo
Avidity Biosciences Announces Positive Topline Phase 1/2 FORTITUDE™ Data Demonstrating Consistent Improvement Across Multiple Functional Measures Compared to Placebo in Del-Brax Treated FSHD Participants
-- Unprecedented data from FORTITUDE™ dose escalation cohorts for del-brax treated participants, compared to placebo, demonstrate improvement in function, strength and PROs as well as rapid and significant reduction in biomarkers -- -- Data support planned accelerated approval BLA submission in H2 2026 -- -- Data being presented at the 32nd Annual FSHD Society International Research Congress (IRC); Investor and analyst webcast event today, Monday, June 9 at 8:00 a.m. ET -- SAN DIEGO, June 9, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™) to profoundly improve people's lives, today announced positive topline data from the dose escalation cohorts of the delpacibart braxlosiran (del-brax) Phase 1/2 FORTITUDE™ program in Facioscapulohumeral Muscular Dystrophy (FSHD). These data as well as research supporting KHDC1L as a novel DUX4 regulated circulating biomarker will be presented in oral and poster presentations at the 32nd Annual FSHD Society International Research Congress (IRC), being held June 12-13, 2025, in Amsterdam, the Netherlands. Del-brax is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the disease-causing gene, double homeobox 4 (DUX4). Currently, there are no approved therapies for the treatment of FSHD, a rare, hereditary disorder marked by life-long, relentless loss of muscle strength and function, significant pain, fatigue, and progressive disability. FSHD affects approximately 45,000 to 87,000 people in the United States and Europe. "The positive topline del-brax results from FORTITUDE being presented at FSHD IRC this week are remarkable and consistent across multiple functional measures as well as biomarkers," said Sarah Boyce, president and chief executive officer at Avidity. "Based on these unprecedented data, we are rapidly advancing del-brax as we pursue accelerated approval and prepare to submit a BLA in the second half of 2026. We are incredibly grateful for the continued trust and support from study participants, their caregivers, investigators and their staff, which are paramount to the success of this program." Avidity today also announced that the accelerated approval regulatory pathway in the U.S. is open for del-brax and that the company has initiated the global, confirmatory Phase 3 FORWARD™ study in FSHD. "Del-brax data from the FORTITUDE study continue to demonstrate consistent reductions in a novel circulating biomarker across two cohorts at 12 months. I am particularly encouraged that del-brax shows favorable safety and tolerability with early and consistent trends towards benefit with del-brax compared to placebo across multiple functional and participant-reported outcome measures," said Jeffrey M. Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE trial investigator. "These data indicate that by directly targeting DUX4, del-brax may be able to improve the lives of patients with FSHD and potentially meaningfully control their disease. I look forward to continued evaluation of del-brax in the FORWARD Phase 3 study and remain hopeful that it is on track to become the potentially first approved drug for FSHD." Topline Data from the Phase 1/2 FORTITUDE™ Dose Escalation Cohorts The FORTITUDE™ clinical development program includes a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate multiple doses of del-brax in participants with FSHD as well as an open-label extension study. The two dose escalation cohorts evaluated 39 participants on either 2 mg/kg or 4 mg/kg of del-brax versus placebo over a period of 12 months. In these cohorts, del-brax was given every six weeks for the first three months and then every 13 weeks thereafter. Topline data from these cohorts for del-brax treated participants, compared to placebo, demonstrated: Consistent improvement of functional mobility and muscle strength as measured by 10-Meter Walk-Run test (10MWRT), Timed Up and Go (TUG) and quantitative muscle testing (QMT) as compared to placebo; Consistent improvement in multiple measures of quality of life as measured by patient reported outcomes and compared to placebo; Rapid and significant reductions in levels of KHDC1L and creatine kinase, a biomarker of muscle damage; and Favorable long-term safety and tolerability with most adverse events (AEs) mild or moderate, with no related serious or severe adverse events and no discontinuations. Topline data from the ongoing, fully enrolled del-brax Phase 1/2 FORTITUDE biomarker cohort are anticipated in Q2 2026. The primary endpoint of the FORTITUDE biomarker cohort is reduction of KHDC1L, a novel DUX4-regulated circulating biomarker. Avidity collaborated with Stephen Tapscott, M.D., Ph.D., Professor of Human Biology and Clinical Research at the Fred Hutchinson Cancer Center around the identification of the KHDC1L circulating biomarker in people living with FSHD. Video Webcast InformationThe Company is hosting an investor and analyst event today, June 9, 2025 at 8:00 a.m. ET. Avidity management will be joined by Jeffrey M. Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE™ trial investigator, to discuss these updates relating to del-brax in FSHD. The virtual event will be available via a live video webcast and can be accessed here or from the "Events and Presentations" page in the "Investors" section of Avidity's website. A replay of the webcast will be archived on Avidity's website following the event. About the Phase 1/2 FORTITUDE™ and Phase 2 FORTITUDE-OLE™ trialsThe FORTITUDE™ trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate single and multiple doses of delpacibart braxlosiran or del-brax in 90 participants with facioscapulohumeral muscular dystrophy (FSHD). FORTITUDE is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of del-brax administered intravenously. Activity of del-brax is being assessed using key biomarkers, including DUX4-regulated muscle and circulating biomarkers and magnetic resonance imaging (MRI) measures of muscle volume and composition. Though the Phase 1/2 trial is not statistically powered to assess functional benefit, it explores the clinical activity of del-brax including measures of functional mobility and muscle strength as well as patient reported outcomes and quality of life measures. The trial has a total of three dose cohorts. The first two dose escalation cohorts evaluated 2 mg/kg or 4 mg/kg of del-brax versus placebo and were designed to assess safety as well as inform the dose and dose regimen of del-brax for additional studies. Avidity has completed enrollment in the dose escalation cohorts and identified 2 mg/kg every six weeks of del-brax as the dose for future clinical trials. The third, ongoing biomarker cohort in the FORTITUDE trial assesses the impact of del-brax 2 mg/kg every six weeks versus placebo for 12 months in people living with FSHD, ages 16-70. The primary endpoint of the biomarker cohort is reduction of KHDC1L, a novel DUX4-regulated circulating biomarker. Enrollment in the biomarker cohort is complete and blinded treatment is ongoing. Participants who complete FORTITUDE have the option to enroll in the ongoing FORTITUDE open-label extension (FORTITUDE-OLE™) study evaluating the long-term safety and tolerability of del-brax in participants living with FSHD. For more information about the FORTITUDE trial, visit the FORTITUDE study website or visit and search for NCT05747924. For more information on the FORTITUDE-OLE study click here or visit and search for NCT06547216. About Del-brax Del-brax is designed to treat the underlying cause of FSHD, which is caused by the abnormal expression of a gene called double homeobox 4 or DUX4. The abnormal expression of DUX4 protein leads to changes in gene expression in muscle cells that are associated with the life-long, progressive loss of muscle function in patients with FSHD. Del-brax aims to reduce the expression of DUX4 mRNA and DUX4 protein in muscles in people with FSHD. Del-brax consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA. Del-brax is currently in registrational-stage studies including FORTITUDE biomarker cohort and the global, confirmatory, Phase 3 FORWARD trial in individuals with FSHD. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Orphan designation for del-brax and the FDA has granted del-brax Fast Track designation. About Facioscapulohumeral Muscular Dystrophy (FSHD)Facioscapulohumeral muscular dystrophy (FSHD) is a rare, progressive, and variable hereditary muscle-weakening condition marked by life-long, relentless loss of muscle function, significant pain, fatigue, and progressive disability. It is characterized by progressive and often asymmetric skeletal muscle loss that initially causes weakness in muscles in the face, shoulders, arms and trunk and progresses to weakness in muscles in the lower body. FSHD is an autosomal dominant disease caused by the aberrant expression of the DUX4 (double homeobox 4) gene in the skeletal muscle, which activates genes that are toxic to muscle cells and leads to a series of downstream events that result in skeletal muscle wasting and compromised muscle function. Skeletal muscle weakness results in physical limitations throughout the whole body, including an inability to lift arms for more than a few seconds, loss of ability to show facial expressions and serious speech impediments. These symptoms cause many people affected by FSHD to become dependent on the use of a wheelchair for mobility. Currently, there are no approved treatments for people living with FSHD. About AvidityAvidity 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 StatementsAvidity 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: Avidity's plans to file a BLA for accelerated approval of del-brax and the timing thereof; the potential for del-brax to achieve accelerated and full approval from the FDA and the timing thereof; the status of accelerated approval as a regulatory pathway for del-brax; biomarker selection and data from the study of del-brax; topline data from the biomarker cohort of the FORTITUDE™ trial and the timing thereof; Avidity's plans to pursue full, global approval for del-brax; the potential for del-brax to improve the lives of people with FSHD; the status of the FORTITUDE trial and the cohorts therein, and the FORWARD™ trial, including without limitation progress, initiation, enrollment, design, goals and dosage levels and frequencies. 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: the data and results produced from the FORTITUDE trial as of the most recent cutoff dates may not be indicative of final results, may not support BLA submission or accelerated approval, may not be satisfactory to the FDA and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; data delivered to the FDA may not support accelerated approval pathways or BLA submissions and may not be satisfactory to the FDA, including as a result of our inability to establish that a novel biomarker may serve as a surrogate endpoint reasonably likely to predict a clinical benefit; even if approved, Avidity may not be able to execute any successful product launches; unexpected adverse side effects to, or inadequate efficacy of, del-brax that may delay or limit its development, regulatory approval and/or commercialization; later developments with the FDA 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 clinical trials; Avidity's dependence on third parties in connection with 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 and subsequent filings with the SEC. 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) 837-5014investors@ Media Contact:Kristina Coppola(619) 837-5016media@ View original content to download multimedia: SOURCE Avidity Biosciences, Inc. Sign in to access your portfolio
Associated Press
11-06-2025
- Business
- Associated Press
Avidity Biosciences to Present Topline Data from Phase 1/2 FORTITUDE™ Trial of Del-brax in People Living with Facioscapulohumeral Muscular Dystrophy at 32nd Annual FSHD Society International Research Congress
-- FDA alignment on accelerated and full approval pathways for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD) -- -- Jeffrey M. Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE trial investigator, will present topline del-brax data from dose escalation cohorts in oral presentation -- -- Stephen Tapscott, M.D., Ph.D., Professor of Human Biology and Clinical Research, Fred Hutchinson Cancer Center, will highlight results on the characterization of a novel DUX4-regulated circulating biomarker in oral and poster presentations -- SAN DIEGO, June 11, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™) to profoundly improve people's lives, today announced that the company will be presenting two oral and one poster presentations at the 32nd Annual FSHD Society International Research Congress, being held June 12-13, 2025, in Amsterdam, the Netherlands. Earlier this week, Avidity announced FDA alignment on accelerated and full approval pathways for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD). 32nd Annual FSHD Society International Research Congress Presentations The presentations and poster are available on the publications page of Avidity's website at 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: Avidity's plans to present topline data from the dose escalation cohorts of the Phase 1/2 FORTITUDE™ trial; the status and availability of accelerated and full approval pathways for del-brax and Avidity's planned participation at the 32nd Annual FSHD Society International Research Congress and the contents of its scheduled presentations. 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, the data and results produced from the clinical study of del-brax may not support BLA submission or accelerated or full approval, and may not be satisfactory to the FDA and other regulators; later developments with the FDA and other regulators may be inconsistent with the feedback received as of the date hereof; and the risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and subsequent filings with the SEC. 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: Kristina Coppola (619) 837-5016 [email protected] View original content to download multimedia: SOURCE Avidity Biosciences, Inc.
Yahoo
10-06-2025
- Business
- Yahoo
Avidity Biosciences price target raised to $75 from $65 at Chardan
Chardan analyst Keay Nakae raised the firm's price target on Avidity Biosciences (RNA) to $75 from $65 and keeps a Buy rating on the shares after the company announced topline results from the dose escalation portion of the FORTITUDE trial and that it has received written confirmation from the FDA on an accelerated approval pathway for del-brax in Facioscapulohumeral Muscular Dystrophy using a novel circulating biomarker, cDUX. The firm's new price target reflects an increase in the estimated probability of success to 60% from 50% for del-brax targeting FSHD, the analyst tells investors. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on RNA: Disclaimer & DisclosureReport an Issue Avidity Biosciences Launches Global Phase 3 Study Avidity Biosciences announces results from Phase 1/2 FORTITUDE program Avidity announces accelerated approval regulatory pathway for Del-Brax Avidity Biosciences Reports Q1 2025 Earnings Promising Outlook for Avidity Biosciences: Buy Rating Backed by Strategic Advancements and Product Launch Preparations Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
