Avidity Biosciences today announced positive AOC 1044 data in healthy volunteers from the Phase 1/2 EXPLORE44™ clinical trial for the treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44).
AOC 1044 delivered up to 50-times greater concentrations of PMO in skeletal muscle following a single dose compared to peptide conjugated PMOs in healthy volunteers AOC 1044 was well tolerated, demonstrated statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg and increased exon skipping in all participants Avidity plans to provide first look at AOC 1044 data in people living with DMD44 in 2H 2024 SAN DIEGO, Dec. 13, 2023 /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 AOC 1044 data in healthy volunteers from the Phase 1/2 EXPLORE44™ clinical trial for the treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44). AOC 1044 delivered unprecedented concentrations of phosphorodiamidate morpholino oligomers (PMO) in skeletal muscle with up to 50-times greater concentrations of PMO in skeletal muscle following a single dose compared to peptide conjugated PMOs in healthy volunteers. AOC 1044 was well tolerated, demonstrated statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg AOC 1044 and increased exon skipping in all participants. Avidity plans to provide a first look at AOC 1044 data in people living with DMD44 in 2H 2024. AOC 1044 is designed to deliver PMO to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production. AOC 1044 has been granted Orphan Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and Fast Track Designation by the FDA. AOC 1044 is the first of multiple AOCs the company is developing for DMD. “We are excited with the early data set of AOC 1044 demonstrating unprecedented delivery of therapeutic oligonucleotide in skeletal muscle and consistent exon skipping in healthy volunteers,” said Sarah Boyce, president and chief executive officer. “We are rapidly advancing AOC 1044 and look forward to sharing data in people living with DMD44 in 2024. Data from our clinical programs continue to reinforce the broad and disruptive potential of our AOC platform for the treatment of high burden muscle diseases like DMD, DM1 and FSHD.” Phase 1/2 EXPLORE44 Healthy Volunteer Data
In addition to AOC 1044, Avidity is also advancing AOC 1001 in the MARINA open-label extension (MARINA-OLE™) study for people living with myotonic dystrophy type 1 (DM1) and AOC 1020 in the Phase 1/2 FORTITUDE™ trial for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The EXPLORE44™ Phase 1/2 Trial of AOC 1044 About Duchenne muscular dystrophy (DMD) About AOC 1044 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 and beyond its control, including, without limitation: additional healthy volunteer data related to AOC 1044 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; AOC 1044 data in people living with DMD44 may not meet Avidity’s expectations; 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 clinical holds which may not be timely lifted (if at all), 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; the success of its preclinical studies and clinical trials for the company’s product candidates; Avidity’s dependence on third parties in connection with preclinical and clinical testing and product manufacturing; Avidity may not realize the expected benefits of its collaborations; regulatory developments in the United States and foreign countries; Avidity could exhaust its available capital resources sooner than it currently expects and fail to raise additional needed funds; and other risks described in Avidity’s Annual Report on Form 10-K for the fiscal year ended December 31, 2022, filed with the Securities and Exchange Commission (SEC) on February 28, 2023, 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-reports-positive-data-demonstrating-aoc-1044-delivers-unprecedented-concentrations-of-pmo-in-muscle-following-a-single-dose-in-healthy-volunteers-from-phase-12-explore44-trial-for-duchenne-muscular-dystrophy-302013456.html SOURCE Avidity Biosciences, Inc. | ||
Company Codes: NASDAQ-NMS:RNA |