Latest news with #Avidity
Yahoo
8 hours ago
- Business
- Yahoo
Avidity Biosciences Touts Encouraging Functional Gains And Biomarker Reductions In Rare Muscular Weakness Disease Trial
Avidity Biosciences, Inc. (NASDAQ:RNA) revealed topline data from the dose escalation cohorts of the delpacibart braxlosiran (del-brax) Phase 1/2 FORTITUDE program in Facioscapulohumeral Muscular Dystrophy (FSHD). FSHD is a genetically acquired disease that leads to progressive muscle weakness and severely decreased functional capacity. The data will be presented at the 32nd Annual FSHD Society International Research is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the disease-causing gene, double homeobox 4 (DUX4). FSHD affects approximately 45,000 to 87,000 people in the United States and Europe. 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. 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 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. In a recent research note, Chardan Capital analyst Keay Nakae set a 12-month price target of $75 per share for Avidity Biosciences. The valuation stems from a Net Present Value (NPV) model of the company's projected revenues from its RNA therapeutics pipeline, forecasted out to the year 2034. Chardan maintained its Buy rating and increased its price forecast from $65 to $75. According to Nakae's analysis, the valuation incorporates specific 'probability of success' rates for each of Avidity's drug candidates to account for clinical and regulatory risks. His model assigns a 60% chance of success for the company's FSHD candidate, 50% for its DM1 and DMD exon 44 candidates, and lower probabilities for other assets. The analyst applied a 14% Weighted Average Cost of Capital (WACC) as the discount rate to reflect the high-risk profile of the development-stage company. Nakae also highlighted several significant risks that could impede Avidity from reaching the $75 price target. He noted that the company, which currently has no revenue-generating products, will likely require additional financing, potentially leading to shareholder dilution. The analyst also pointed to the risks of intellectual property challenges, the critical nature of upcoming clinical trial outcomes, and the intense competition from larger, better-resourced companies in the biotechnology sector. Price Action: RNA stock is trading lower by 11.6% to $32.03 at last check Monday. Read Next:Photo by Gorodenkoff via Shutterstock UNLOCKED: 5 NEW TRADES EVERY WEEK. Click now to get top trade ideas daily, plus unlimited access to cutting-edge tools and strategies to gain an edge in the markets. Get the latest stock analysis from Benzinga? AVIDITY BIOSCIENCES (RNA): Free Stock Analysis Report This article Avidity Biosciences Touts Encouraging Functional Gains And Biomarker Reductions In Rare Muscular Weakness Disease Trial originally appeared on © 2025 Benzinga does not provide investment advice. All rights reserved.
Yahoo
14 hours ago
- Business
- Yahoo
Avidity Biosciences Announces the Accelerated Approval Regulatory Pathway in the U.S. is Open for Del-Brax and Initiates the Global, Confirmatory Phase 3 FORWARD™ Study in FSHD
-- Planning accelerated approval BLA submission in H2 2026, following topline data fromFORTITUDE™ biomarker cohort in Q2 2026 -- -- Initiated global, confirmatory Phase 3 FORWARD™ study of del-brax 2mg/kg every six weeks -- -- Investor and analyst webcast event today, Monday, June 9, 2025 at 8 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 the accelerated approval regulatory pathway in the United States is open for delpacibart braxlosiran (del-brax) in the treatment of facioscapulohumeral muscular dystrophy (FSHD). Additionally, the Company announced that it has initiated its global, confirmatory, Phase 3 FORWARD™ study intended to support a subsequent full approval package for del-brax for people living with FSHD in the U.S. and additional countries around the world. 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. "Our regulatory and clinical development progress announced today reflect our continued leadership in rare neuromuscular diseases and bring us a step closer to providing a treatment option to the FSHD community that could meaningfully impact their disease," said Sarah Boyce, president and chief executive officer at Avidity. "We have confirmed with the FDA that the accelerated approval pathway is open for del-brax. In addition, we have initiated our global confirmatory Phase 3 study intended to support our global approval strategy for del-brax." In addition to the standard guidance for the accelerated approval pathway, the company has been given detailed direction by FDA on the necessary validation steps for the surrogate biomarker. FDA's Accelerated Approval Program was instituted to allow for earlier approval of drugs that treat serious conditions, and fill an unmet medical need based on a surrogate endpoint. A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval. Drug companies are still required to conduct studies to confirm the anticipated clinical benefit. To support full approval Avidity has initiated FORWARD, a global, confirmatory, Phase 3, randomized, placebo-controlled, double-blind, 18-month study designed to evaluate 2mg/kg of del-brax every six weeks in approximately 200 people (ages 16-70) living with FSHD in North America, Europe and Japan. FORWARD is designed to assess key FSHD-related endpoints that measure functional mobility (10MWRT and TUG), strength (QMT) and patient-reported outcomes. The flexible design of FORWARD allows the Company to gather additional data from the ongoing FORTITUDE™ biomarker cohort and elevate any of these measures to the primary endpoint. Currently, QMT is assigned as the primary endpoint. "People living with FSHD experience severe and unpredictable muscle loss, along with the constant uncertainty of how the disease will progress," said Mark Stone, CEO of FSHD Society. "We are grateful to Avidity for their innovative approach that takes into account feedback from our community, specifically in the development of a biomarker that provides insights into the real, whole-body burden of FSHD. This research enables the pursuit of a regulatory pathway that may make del-brax available to the FSHD community more quickly. This progress, along with the initiation of the FORWARD Phase 3 study and the stellar del-brax data from FORTITUDE, offers real hope for those urgently needing treatment options." 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 3 FORWARD™ TrialFORWARD™ is a global, confirmatory Phase 3, randomized, placebo-controlled, double-blind, 18-month study designed to evaluate delpacibart braxlosiran (del-brax) in approximately 200 people (ages 16-70) living with FSHD. The trial will be conducted at approximately 45 global sites including in the U.S., Canada, Europe and Japan. Patients will be administered either 2 mg/kg of del-brax or placebo (1:1) every six weeks. Following regulatory alignment, FORWARD is designed to be a confirmatory study to support potential full approval of del-brax. FORWARD is assessing the impact of del-brax on key FSHD-related endpoints that measure functional mobility (10-Meter Walk-Run test, or 10 MWRT, and Timed Up and Go, or TUG), strength (quantitative muscle testing, or QMT, total score), patient-reported outcomes (PROs) and decrease in KHDC1L, a novel, circulating biomarker. All study participants, regardless of whether they receive active treatment or placebo, will have the option to enroll into an open-label extension trial. For more information about the FORWARD trial, visit the FORWARD study website. 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 approval from the FDA; the status of accelerated approval as a regulatory pathway for del-brax; the use of KHDC1L as the primary endpoint in the FORTITUDE™ biomarker cohort; topline data from the biomarker cohort of the FORTITUDE trial and the timing thereof; characterization of data from the FORTITUDE trial; 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. 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
Los Angeles Times
18-05-2025
- Business
- Los Angeles Times
Bioscience Firms in Southern California Focus on Innovation & Collaboration
Southern California is one of the nation's leading life science hubs, led by the vast cluster of companies in San Diego County that focus on research and development. However, the sector is spread throughout the region with employers such as Amgen in Thousand Oaks and Edwards Lifesciences in Irvine featured among the top companies. New drug developments and delivery systems are some of the leading recent breakthroughs from local companies. Treatments have been created for a wide range of ailments, such as neuromuscular diseases, diabetes, eye disease and cancer, among others. Over the past few months, San Diego-based Avidity Biosciences Inc. reported multiple positive datasets from its three clinical-stage programs in rare muscle diseases. It is preparing for commercialization in anticipation of three potential products that could launch in close succession next year. 'Avidity's mission is to revolutionize the delivery of RNA therapeutics to make a profound difference in the lives of people living with serious rare diseases, many of whom have no or limited treatment options,' said Sarah Boyce, Avidity's chief executive. 'We plan to file our first biologics license application for our DMD44 drug candidate and accelerate commercial preparations for three potential product launches in rapid succession in all three rare neuromuscular diseases.' In Northridge, Medtronic Diabetes received approval from the Food and Drug Administration in April for its Simplera Sync sensor for use with the insulin delivery system MiniMed 780G. The new sensor is a disposable all-in-one that requires no fingerpricks. It plans a limited launch of the product this fall. 'We're committed to driving innovation that makes life easier for those living with diabetes so they can forget about their diabetes as much as possible throughout the day,' said Que Dallara, president of Medtronic Diabetes, in a statement. Aliso Viejo-based Glaukos Corp. reported positive clinical updates in January for its iDose TR sustained-release procedural pharmaceutical platform. The company focuses on treatments of chronic eye diseases such as glaucoma, cataracts and diseases that impact the cornea and retina. In February, it followed up on their previous announcement with FDA acceptance of its new drug application for Epioxa, a therapy for keratoconus, which affects the cornea, and set an October goal for the FDA to review the treatment. In January, Agoura Hills-based A2 Biotherapeutics closed an $80-million Series C funding round that will be used for three clinical development programs of its precision cell therapies. Investors include The Column Group and Samsara BioCapital. 'We are excited by the initial clinical data from our lead programs, which we believe validates our proprietary logic-gate technology approach to solid tumor cancers,' said Jim Robinson, CEO of A2 Bio, in a statement. Other companies are expanding through collaboration. San Diego-based Amprion announced a collaboration with Mayo Clinic Laboratories to expand access to Amprion's SAAmplify test across the United States. The partnership is expected to improve diagnostic accuracy for neurodegenerative diseases. 'Amprion has developed early and accurate diagnostic tools for a range of neurodegenerative diseases, including Parkinson's, Lewy Body Dementia, and Multiple System Atrophy. Through this partnership, Amprion and Mayo are able to provide patients, physicians and families with one of the most comprehensive diagnostic offerings available,' said Russ Lebovitz, Amprion chief executive.
Yahoo
21-04-2025
- Business
- Yahoo
Avidity Biosciences Receives Orphan Drug Designation in Japan for Delpacibart Etedesiran (del-desiran) for Treatment of Myotonic Dystrophy Type 1
Del-desiran first-ever investigational treatment for DM1 to receive Orphan Drug designation in Japan SAN DIEGO, April 8, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation (ODD) to delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1), an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Del-desiran is the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). "This decision by MHLW further reinforces the significant potential of del-desiran to address the root cause of DM1 and the urgent need to bring an approved therapy to the many people impacted by this devastating rare disease in Japan and around the world," said Steve Hughes, M.D., chief medical officer at Avidity. "We are very encouraged by del-desiran data reported from the MARINA and MARINA-OLE studies thus far, demonstrating favorable long-term safety and tolerability, reversal of disease progression, and consistent and durable improvements in multiple clinical endpoints. Looking ahead, there are multiple key milestones this year as we complete enrollment in the phase 3 HARBOR trial and continue to advance our global commercialization preparations to potentially deliver the first globally approved drug for people living with DM1." Avidity has aligned with global regulators on the registrational path for del-desiran for the treatment of DM1, which informed the design of the ongoing Phase 3 HARBOR™ study. Avidity expects to complete participant enrollment in the Phase 3 HARBOR study in mid-2025 and submit marketing applications starting 2026 in the U.S., European Union and Japan. Japan's MHLW grants Orphan Drug designation to drugs in development for the treatment of diseases that affect fewer than 50,000 patients in Japan and for which there is a high unmet medical need. An investigational therapy is eligible to qualify for Orphan Drug designation if there is no approved alternative treatment option or if there is high efficacy or safety expected compared to existing treatment options. Orphan Drug designation provides certain benefits, including prioritized consultation regarding clinical development, reduced consultation fees, tax incentives, priority review of applications, reduced application fees, and extended registration validity period. About the Phase 3 HARBOR™ TrialThe global Phase 3 HARBOR™ trial is a randomized, placebo-controlled, double blind pivotal study designed to evaluate del-desiran in approximately 150 people (age 16 and older) living with DM1. The trial will be conducted at approximately 40 sites globally. Patients will be administered either del-desiran or placebo (1:1) every eight weeks. HARBOR is designed to assess multiple key functional aspects of DM1. The primary endpoint is video hand opening time (vHOT), a measurement of myotonia, which is a hallmark symptom of DM1. Key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. All study participants, regardless of whether they receive active treatment or placebo, will have the option to enroll into an open-label extension trial. For more information about the HARBOR trial, visit the HARBOR study website or visit and search for NCT06411288. About the Phase 2 MARINA-OLE™ StudyMARINA-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of del-desiran in participants with DM1 who were previously enrolled in the MARINA® Phase 1/2 trial. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-desiran in participants enrolled in the randomized, placebo-controlled, Phase 1/2 MARINA clinical trial. Participants enrolled in the MARINA-OLE study receive quarterly doses of del-desiran regardless of whether they received active treatment or placebo in the MARINA study. The total duration of active treatment with del-desiran in the MARINA-OLE study is approximately 24 months. Once patients have completed active treatment, there will be a nine-month safety follow-up period. Avidity may extend active treatment beyond 24 months at a future timepoint. For more information on this study click here or visit and search for NCT05479981. About Del-desiranDel-desiran, Avidity's lead product candidate utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of a disease-related mRNA called DMPK. Del-desiran consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA. Del-desiran is currently being assessed in the global Phase 3 HARBOR™ trial and in the ongoing MARINA-OLE™ trial in people with DM1. Long-term data from the MARINA-OLE trial showed reversal of disease progression in people living with DM1 across multiple endpoints including video hand opening time (vHOT) as a measure of hand function and myotonia, muscle strength and activities of daily living when compared to END-DM1 natural history data. Del-desiran has received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). About Myotonic Dystrophy Type 1Myotonic dystrophy type 1 (DM1) is an underrecognized, autosomal dominantly inherited, progressive and often fatal disease caused by a triplet-repeat in the DMPK gene, resulting in a toxic gain of function mRNA. The disease is highly variable with respect to severity, presentation and age of onset, however all forms of DM1 are associated with high levels of disease burden and may cause premature mortality. DM1 primarily affects skeletal and cardiac muscle, however patients can suffer from a constellation of manifestations including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment. Currently, there are no approved treatments for people living with DM1. About Avidity Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity is revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare neuromuscular diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is also advancing two wholly-owned precision cardiology development candidates addressing rare genetic cardiomyopathies. In addition, Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit and engage with us on LinkedIn and X. Forward-Looking 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: planned marketing applications for del-desiran in the U.S., European Union and Japan, and the timing thereof; Avidity's plans to become a global commercial organization and the status of its commercialization efforts; the characterization of data associated with del-desiran in the MARINA and MARINA-OLE studies, the conclusions drawn therefrom, and the impact of such data on the advancement of del-desiran and its ability to treat DM1; the designs, goals, statuses and enrollment levels of, and plans for, the MARINA-OLE and HARBOR studies; Avidity's platform, planned operations and programs; and Avidity's cash position and runway. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: Avidity may not realize any benefit from Orphan Drug designation of del-desiran by global health authorities; the data and results produced in Avidity's ongoing studies of del-desiran as of the most recent respective cutoff dates may not be indicative of final results, may not support a BLA submission, may not be satisfactory to the MHLW and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; even if approved, Avidity may not be able to execute any successful product launches; Avidity's efforts to build a global commercial organization may be unsuccessful; unexpected adverse side effects to, or inadequate efficacy of, del-desiran that may delay or limit its development, regulatory approval and/or commercialization; later developments with the MHLW and other global regulators that could be inconsistent with the feedback received to date; Avidity's approach to the discovery and development of product candidates based on its AOC™ platform is unproven and may not produce any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of the MARINA-OLE and HARBOR studies; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; legislative, judicial and regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2024. Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that arise after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Investor Contact:Kat Lange(619) 837-5014investors@ Media Contact:(619) 837-5016media@ View original content to download multimedia: SOURCE Avidity Biosciences, Inc. Sign in to access your portfolio
Yahoo
21-04-2025
- Business
- Yahoo
Avidity Biosciences Completes Enrollment in Biomarker Cohort in Phase 1/2 FORTITUDE™ Trial for Delpacibart Braxlosiran (del-brax) in People Living with Facioscapulohumeral Muscular Dystrophy
Del-brax FORTITUDE biomarker cohort designed for potential accelerated approval; plan to share regulatory update in Q2 2025 Regulatory alignment on global Phase 3 del-brax trial design and study initiation anticipated in Q2 2025 Plan to present topline data from FORTITUDE dose escalation cohorts in Q2 2025 On track to be first globally approved drug for FSHD SAN DIEGO, March 31, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced the completion of enrollment in the biomarker cohort in the Phase 1/2 FORTITUDE™ clinical trial of delpacibart braxlosiran (del-brax) in people living with facioscapulohumeral muscular dystrophy (FSHD). A total of 51 participants were enrolled in the FORTITUDE biomarker cohort. "Completing enrollment in the FORTITUDE biomarker cohort is an important milestone as we pursue a potential accelerated approval path in the U.S. for del-brax and work to bring the first approved drug to people living with this rare, devastating neuromuscular disease who have no treatment options as quickly as possible," said Steve Hughes, M.D., chief medical officer at Avidity. "We are very encouraged by the del-brax 2 mg/kg data thus far, demonstrating unprecedented and consistent reductions in DUX4-regulated genes, significant decreases in novel circulating biomarker and creatine kinase, trends of functional improvement, and favorable safety and tolerability, and look forward to sharing additional data as well as other key milestones in the second quarter of this year." Avidity is on track to deliver multiple updates from the del-brax program in Q2 including: Regulatory alignment on a potential accelerated approval path in the U.S. for the ongoing FORTITUDE biomarker cohort; Regulatory alignment on the design of the global Phase 3 trial as well as initiation of the trial; and Topline data from the dose escalation cohorts in the FORTITUDE trial. Del-brax is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the mRNA transcript of 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 function, significant pain, fatigue, and progressive disability. FSHD affects approximately 45,000 to 87,000 people in the United States and European Union. About the Phase 1/2 FORTITUDE™ trial The 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 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 is designed for a potential accelerated approval path in the U.S. It is assessing 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 endpoints of the study cohort are changes in DUX4-regulated gene expression and DUX4-regulated circulating biomarker. Enrollment in the biomarker cohort is complete. 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. About the Phase 2 FORTITUDE-OLE™ trial FORTITUDE-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of delpacibart braxlosiran or del-brax in participants with facioscapulohumeral muscular dystrophy (FSHD) who were previously enrolled in the Phase 1/2 FORTITUDE™ trial. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-brax in participants who enrolled in the randomized, placebo-controlled, Phase 1/2 FORTITUDE clinical trial. Participants who enroll in the FORTITUDE-OLE study will receive del-brax regardless of whether they received active treatment or placebo in the FORTITUDE study. The total duration of active treatment with del-brax in the FORTITUDE-OLE is approximately 24 months. Avidity may extend active treatment beyond 24 months at a future timepoint. 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. Avidity reported positive initial del-brax 2 mg/kg data at four months from the Phase 1/2 FORTITUDE™ trial demonstrating unprecedented and consistent reductions of greater than 50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability in people living with FSHD. Del-brax is currently in Phase 1/2 development as part of the FORTITUDE 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 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 FORTITUDE study and the timing thereof; Avidity's plans to pursue an accelerated approval path for del-brax and the anticipation of regulatory alignment with such plans; the status of the FORTITUDE trial and cohorts therein, including but not limited to initiation, enrollment, design and goals; the ability for del-brax to achieve accelerated approval; the characterization of data associated with del-brax and the FORTITUDE trial, the conclusions drawn therefrom, the impact of such data on the advancement of del-brax and its abilities to treat FSHD; and the possibility of del-brax becoming the first globally approved drug for FSHD. 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 in the FORTITUDE trial and FORTITUDE-OLE as of the most recent respective cutoff dates may not be indicative of final results, may not support BLA submissions or accelerated approvals, 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; even if approved, Avidity may not be able to execute a successful product launch for del-brax; 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:(619) 837-5016media@ View original content to download multimedia: SOURCE Avidity Biosciences, Inc. Sign in to access your portfolio
