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Avidity Biosciences Announces Positive AOC 1001 Long-term Data Showing Reversal of Disease Progression in People Living with Myotonic Dystrophy Type 1 Across Multiple Endpoints; Same Key Endpoints Agreed for Phase 3 HARBOR™ Trial | ||
By: PR Newswire Association LLC. - 04 Mar 2024 | Back to overview list |
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Avidity accelerates global Phase 3 HARBOR™ study initiation to Q2 2024 following regulatory agreement on study design; primary endpoint is video hand opening time (vHOT) and secondary endpoints include muscle strength and activities of daily living Delpacibart etedesiran (AOC 1001) demonstrated consistent and durable improvements in myotonia, muscle strength and activities of daily living in people with DM1 in long-term data from MARINA-OLE™ Delpacibart etedesiran (AOC 1001) data from MARINA-OLE showed reversal of disease progression in multiple functional measures in people living with DM1 compared to END-DM1 natural history data Avidity to host Volume 8 of investor and analyst event series via webcast March 4, 2024, at 8:00 a.m. ET SAN DIEGO, March 4, 2024 /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 new positive long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE™) trial showing reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data. These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT), and 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. Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024. Avidity also announced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran. Del-desiran (AOC 1001) is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. "The long-term data from the MARINA-OLE study demonstrating that del-desiran improved measures of disease progression in DM1 patients compared to natural history data is remarkable," said John W. Day, MD, PhD, Professor of Neurology and Pediatrics, and Director, Division of Neuromuscular Medicine, Stanford University School of Medicine, an investigator of the MARINA® and MARINA-OLE trials. "The favorable long-term safety data and consistent, durable improvement in myotonia, muscle strength and patient-reported outcomes measures show the potential of del-desiran to make a meaningful difference in the lives of DM1 patients. I am very encouraged by the prospect of del-desiran as a potential treatment for DM1." "Initiating our global pivotal study for del-desiran in the coming months is a significant step forward in bringing a much-needed treatment to people living with DM1 as quickly as possible. We are pleased that the agreed upon functional endpoints in the Phase 3 HARBOR study are the same endpoints in which del-desiran has demonstrated consistent and durable improvements in the MARINA studies," said Sarah Boyce, president and chief executive officer at Avidity. "Thank you to the DM1 community, in particular - the participants, their families, the investigators and their teams - for their support, dedication and commitment to the MARINA program. The depth and breadth of del-desiran data that we have gathered from these studies offer hope for people living with DM1, a devastating rare disease for which there are no treatment options available." Data Presented at 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 3-6 Long-term efficacy data presented were assessed from 12 participants on 4 mg/kg del-desiran (AOC 1001) in the MARINA-OLE study. The endpoints used in the MARINA-OLE measure important aspects of the disease and correspond to those utilized in the ongoing END-DM1 natural history study. The long-term data from the MARINA-OLE trial are being presented in a poster session at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 3-6, 2024, in Orlando, Florida and can also be found on Avidity's website on the Publications page. MARINA-OLE data compared to END-DM1 natural history data Avidity is grateful to the END-DM1 team for their partnership over the years and for their approval and permission to share this analysis from the study. Del-desiran long-term efficacy data from MARINA-OLE
Del-desiran safety and tolerability data from MARINA-OLE
Video Webcast Information About the Phase 1/2 MARINA® Trial About the Phase 2 MARINA-OLE™ Study About Del-desiran (AOC 1001) About Myotonic Dystrophy Type 1 About Avidity Forward-Looking Statements 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, including, without limitation: Avidity may not be able to resolve the partial clinical hold related to the serious adverse event which occurred in the Phase 1/2 MARINA trial, which may result in delays in the clinical development of del-desiran; additional participant data related to del-desiran that continues to become available may be inconsistent with the data produced as of the date hereof, and further analysis of existing data and analysis of new data may lead to conclusions different from those established as of the date hereof; 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, or may result in additional clinical holds which may not be timely lifted, recalls or product liability claims; Avidity is early in its development efforts; Avidity's approach to the discovery and development of product candidates based on its AOC platform is unproven, and the company does not know whether it will be able to develop any products of commercial value; potential delays in the commencement, enrollment, data readouts and completion of preclinical studies or clinical trials; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; regulatory developments in the United States and foreign countries; and other risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, filed with the Securities and Exchange Commission (SEC) on February 28, 2024, and in 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: Media Contact: View original content to download multimedia:https://www.prnewswire.com/news-releases/avidity-biosciences-announces-positive-aoc-1001-long-term-data-showing-reversal-of-disease-progression-in-people-living-with-myotonic-dystrophy-type-1-across-multiple-endpoints-same-key-endpoints-agreed-for-phase-3-harbor-trial-302078020.html SOURCE Avidity Biosciences, Inc. |
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