Latest news with #SarahBoyce
Yahoo
2 days 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.
Associated Press
17-03-2025
- Business
- Associated Press
Avidity Biosciences Announces Positive Topline Del-zota Data Demonstrating Consistent, Statistically Significant Improvements in Dystrophin, Exon Skipping and Creatine Kinase in People Living with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping in Phase 1/2 EXPLORE44® Trial
On track for year end 2025 BLA submission for accelerated approval of 5 mg/kg every six weeks of del-zota in DMD44 Consistent favorable safety and tolerability across del-zota dose cohorts Plan to present functional data in fourth quarter of 2025 Investor and analyst webcast event today at 8:00 a.m. ET SAN DIEGO, March 17, 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 positive del-zota topline data from the Phase 1/2 EXPLORE44® trial in people living with Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44) demonstrating consistent, statistically significant improvements in dystrophin, exon skipping and creatine kinase as well as favorable safety and tolerability across the dose cohorts. The data will be highlighted in an oral and poster presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held March 16-19, 2025, in Dallas, Texas. 'Del-zota has shown remarkable improvements across multiple measures, including a substantial increase in dystrophin production and a significant reduction in serum creatine kinase levels to near normal after just three doses. The consistency of these results in such a short time frame, coupled with favorable safety and tolerability, underscores the potential of del-zota to become a groundbreaking treatment for individuals living with DMD with genetic variants amenable to exon 44 skipping,' said Aravindhan Veerapandiyan, M.D., Associate Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital. 'These results bring new hope for patients and families affected by DMD who are in urgent need of targeted therapies that can preserve muscle integrity and possibly prevent or delay the progression of muscle weakness and loss of function associated with this disease.' Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal and cardiac muscle tissue to specifically skip exon 44 of the dystrophin gene and enable production of near-full length dystrophin. Del-zota has been granted Orphan designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA has also granted del-zota Rare Pediatric Disease and Fast Track designations. Del-zota is the first of multiple AOCs currently in development at Avidity for the treatment of DMD. 'These data are a true testament to the power of our AOC platform and the potential of del-zota to change the course of this relentless and devastating rare disease,' said Sarah Boyce, president and chief executive officer at Avidity. 'We are particularly encouraged that these data demonstrate the consistent effects of del-zota across a variety of participants, including broad age range, genotypes and ambulatory and non-ambulatory participants. The early and durable effects observed with del-zota further strengthen our commitment to rapidly bring this potentially transformative therapy to people living with DMD44. Based on our interactions with FDA on accelerated approval, we believe dystrophin data from EXPLORE44 combined with the safety data from our fully enrolled EXPLORE44-OLE trial will support our planned BLA submission at the end of this year. We are immensely grateful for the continued dedication of our team, the investigators and, most importantly, the participants in our clinical trials and their families.' The topline data from the randomized, double-blind, placebo-controlled Phase 1/2 EXPLORE44 trial demonstrated consistent, statistically significant improvements across key biomarkers as well as safety and tolerability of del-zota across two dose levels, 5 mg/kg and 10 mg/kg. Participants received three doses of either 5 mg/kg del-zota or placebo every six weeks, or 10 mg/kg del-zota or placebo every eight weeks. Data on muscle delivery, exon skipping, dystrophin production and creatine kinase levels were assessed from seven (7) participants in the 5 mg/kg cohort, 10 participants in the 10 mg/kg cohort, and six (6) placebo participants, 28 days after the third dose. Safety and tolerability data were assessed from 26 participants in the completed Phase 1/2 EXPLORE44 trial and 38 participants in the ongoing EXPLORE44 Open-Label Extension (OLE) trial, as of January 22, 2025. The data presented at MDA highlight the consistent data across all parameters in both the 5 mg/kg and 10 mg/kg cohorts of del-zota, including: Targeted delivery of PMOs resulting in tissue concentrations of approximately 200nM in skeletal muscle; Statistically significant increases of approximately 40% in exon 44 skipping; Statistically significant increase of approximately 25% of normal in dystrophin production and restored total dystrophin up to 58% of normal; Reduction in creatine kinase levels to near normal with greater than 80% reductions compared to baseline: Similarly, placebo participants demonstrated a reduction in creatine kinase levels to near normal upon treatment with del-zota; Significant reductions in creatine kinase levels were sustained in the EXPLORE44-OLE trial with continued treatment up to one year; and, Del-zota demonstrated favorable safety and tolerability at both doses, with most treatment emergent adverse events (TEAEs) mild or moderate. Based on the consistent data between the 5 mg/kg every six weeks and the 10mg/kg every eight weeks groups across all parameters, Avidity has selected the dose of 5 mg/kg every six weeks of del-zota for the Biologics License Application (BLA) submission and future clinical studies. Participants currently receiving the 10 mg/kg dose in the EXPLORE44-OLE trial are in the process of being transitioned to 5 mg/kg every six weeks. Following alignment with FDA on the accelerated approval path late last year, including dose selection, Avidity's commercial preparations for a potential U.S. launch of del-zota in DMD44 are well underway. Del-zota's anticipated launch sets the foundation for sequential launches of Avidity's three neuromuscular programs, including del-desiran for myotonic dystrophy type 1 (DM1) and del-brax for facioscapulohumeral muscular dystrophy (FSHD). Video Webcast Information The company is hosting an investor and analyst event today at 8:00 am ET and will be joined by Kevin M. Flanigan, MD, Director, Center for Gene Therapy and Robert F. & Edgar T. Wolfe Foundation Endowed Chair in Neuromuscular Research at Nationwide Children's Hospital, and Professor of Pediatrics and Neurology at Ohio State University, to discuss del-zota topline data from the EXPLORE44 trial. The 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 EXPLORE44® Phase 1/2 Trial The EXPLORE44® trial was a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial that enrolled 26 participants with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44). The study was designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of del-zota (formerly AOC 1044) administered intravenously in healthy volunteers and participants living with DMD44. The EXPLORE44 trial assessed exon skipping and dystrophin protein levels in participants with DMD44. Participants with DMD44 had the option to enroll into EXPLORE44-OLE™, an open-label extension study, at the end of the post-treatment period. For more information about the EXPLORE44 trial, visit the EXPLORE44 study website or visit and search for NCT05670730. About the Phase 2 EXPLORE44-OLE™ Study EXPLORE44-OLE™ is an open-label, multi-center trial designed to evaluate the long-term safety, tolerability, pharmacokinetics, pharmacodynamic effects and efficacy of del-zota in participants with DMD44. Enrollment has been completed in the EXPLORE44-OLE study, with 23 participants who were previously enrolled in the Phase 1/2 EXPLORE44® trial and 16 participants who directly enrolled in the EXPLORE44-OLE study. Participants in the EXPLORE44-OLE study will receive 5 mg/kg of del-zota every six weeks. The total duration of active treatment with del-zota in the EXPLORE44-OLE study is approximately 24 months. Once participants have completed active treatment, there will be a three-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 NCT06244082. About Duchenne muscular dystrophy (DMD) Duchenne muscular dystrophy (DMD) causes a lack of functional dystrophin that leads to stress and tears of muscle cell membranes, resulting in muscle cell death and the progressive loss of muscle function. The dystrophin protein maintains the integrity of muscle fibers and acts as a shock absorber through its role as the foundation of a group of proteins that connects the inner and outer elements of muscle cells. People living with DMD suffer from progressive muscle weakness that typically starts at a very young age. Over time, people with Duchenne will develop problems walking and breathing, and eventually, the heart and respiratory muscles will stop working. Those living with the condition often require special aid and assistance throughout their lives and have significantly shortened life expectancy. While there are treatments approved to treat people with DMD, there remains a very high unmet need. DMD is a monogenic, X-linked, recessive disease that primarily affects males, with one in 3,500 to 5,000 boys born worldwide having Duchenne. About Del-zota Del-zota is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production in people living with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44). DMD is characterized by progressive muscle degeneration and weakness due to alterations of a protein called dystrophin that protects muscle cells from injury during contraction. Del-zota consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a PMO targeting exon 44. The Phase 1/2 EXPLORE44® trial of del-zota has been completed, and the EXPLORE44 Open-Label Extension trial (OLE™) of del-zota is currently ongoing. Topline data from the completed del-zota Phase 1/2 EXPLORE44 trial demonstrated unsurpassed delivery of PMOs to skeletal muscle, robust increases in dystrophin production, significant increases in exon 44 skipping, and significant and sustained decreases of creatine kinase levels to near normal in people living with DMD44. Del-zota has received Rare Pediatric Disease, 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 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: the characterization of data associated with del-zota and the EXPLORE44® trial; the safety and tolerability of del-zota; the dose selection and frequency of administration of del-zota in the EXPLORE44-OLE study; the active treatment and safety follow-up periods in EXPLORE44-OLE; Avidity's plans for a BLA submission for del-zota and the timing thereof; the design and goals of the EXPLORE44 and EXPLORE44-OLE trials; the ability for del-zota to achieve accelerated approval; the status of Avidity's commercialization efforts; the ability for del-zota to treat DMD44 and Avidity's product candidates to treat their intended indications; and the potential of the AOC platform. 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 EXPLORE44 trial as of the most recent cutoff date may not be indicative of final results, may not support a 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; the FDA and other regulators may change their positions on the status of del-zota and its clinical development; 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, Avidity's product candidates that may delay or limit their development, regulatory approval and/or commercialization; Avidity's approach to the discovery and development of product candidates based on its AOC platform is unproven; potential delays in the commencement, enrollment, data readouts and completion of Avidity's ongoing 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. Kat Lange Media Contact:
Yahoo
28-02-2025
- Business
- Yahoo
Avidity Biosciences Honors Rare Disease Day® and Global Efforts to Support People and Families Impacted by Rare Neuromuscular Diseases
SAN DIEGO, Feb. 28, 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 it is joining the global community of patients, caregivers, families and healthcare providers to build broader awareness of rare diseases, including rare neuromuscular diseases, and the urgent need for more approved treatments for many of these conditions in support of Rare Disease Day®. "Rare Disease Day represents an important opportunity for all of us, including people living with rare diseases, their friends, family and caregivers, and advocacy organizations around the world, to spotlight the many challenges that rare diseases present in daily living," said Sarah Boyce, President and Chief Executive Officer at Avidity. "We remain dedicated to building broader awareness of the devastating impact of rare, progressive neuromuscular disorders including DMD, DM1 and FSHD as well as rare cardiomyopathies and to hearing from the many patients and caregivers who inform and inspire our efforts to help. Their optimism, courage and insights fuel the work that we do every day." Rare Disease Day takes place on the last day of February each year with the goal to raise awareness of the impact of rare diseases worldwide. EURORDIS established Rare Disease Day in 2008 and coordinates with more than 70 national alliance patient organizations each year to honor those living with rare diseases as well as their families and caregivers. Avidity joins with members of the global rare disease community including patients, caregivers, clinicians, friends and family in support of Rare Disease Day. To learn how you can get involved, visit the EURORDIS Rare Disease Day website here: In recognition of Rare Disease Day, Avidity is supporting a range of activities in efforts to build broader awareness of rare diseases including DMD, DM1 and FSHD, such as: Supported the EveryLife Foundation for Rare Diseases "Rare Disease Week on Capitol Hill", that took place February 24-26, 2025 in Washington, D.C. Sponsoring the Jett Foundation's webinar "Thriving with Duchenne," being held today, February 28, 2025, including a panel discussion with people impacted by DMD who will share their experiences. Participating in EURORDIS "More Than You Can Imagine" campaign, a global effort to support people living with rare diseases and shine a spotlight on the power of resilience and connection. Sharing perspectives from the Avidity team about our commitment to the rare disease community and how we are inspired by members of the patient and treatment communities. "On Rare Disease Day, let us recognize the commitment and work necessary to bring support to those impacted by rare diseases around the world," said Luisa Leal, Founder and CEO of the Akari Foundation. "Alone we are rare, but the partnership among rare disease advocacy organizations, industry, researchers, and healthcare providers makes a profound difference in fueling innovation and providing hope to those who are most in need. At Akari, we work to highlight the importance of creating a world where people impacted by Duchenne muscular dystrophy and other rare diseases have access to new and promising treatments as well as education and support, regardless of their culture or background. We are proud to partner with industry leaders such as Avidity in efforts to foster awareness and unite the global rare disease community to achieve a better, brighter future." 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. Investor Contact:Kat Lange(619) 837-5014investors@ Media Contact:(619) 837-5016media@ View original content to download multimedia: SOURCE Avidity Biosciences, Inc.
