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Avidity Biosciences to Present New AOC 1001 Long-term Efficacy and Safety Data from MARINA-OLE™ Trial in People Living with Myotonic Dystrophy Type 1 (DM1) at 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Key Takeaway: Avidity Biosciences announced it will present long-term efficacy and safety data for AOC 1001, an RNA therapeutic targeted at myotonic dystrophy type 1 (DM1), at the 2024 MDA Clinical & Scientific Conference. The event is set to take place in Orlando, Florida, from March 3-6, 2024, where the company will also highlight findings from various clinical development programs addressing rare muscle diseases. This presentation will be led by John W. Day, MD, PhD, a notable researcher in the field. A live webcast will accompany the presentations, promoting broader access to the data.
Price reaction · baseline $13.51 (2024-02-14 close) · hit after-hours · 2 other RNA headline(s) in the window, move may be shared
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Market Sentiment Analysis

POSITIVE FACTORS

  • Presentation of new long-term efficacy and safety data for AOC 1001.
  • Involvement of prominent investigator John W. Day shows credibility.
  • The event highlights Avidity's commitment to addressing rare muscle diseases.
  • Avidity is advancing its proprietary RNA therapeutic platform.

BiopharmaWatch Analysis

From our catalyst data and publicly available data · not financial advice
Cash runway
~201 mo
Minimal dilution risk
Lead asset
Durvalumab
Phase 2 · Lung Cancer Stage II

Full Press Release Details

John W. Day, MD, PhD, Professor of Neurology and Pediatrics, and Director, Division of Neuromuscular Medicine, Stanford University School of Medicine and a principal investigator in END-DM1, ongoing natural history study will join Avidity management to present data from MARINA-OLE™
Avidity to host Volume 8 of investor and analyst event series via webcast March 4, 2024, at 8:00 a.m. ET
SAN DIEGO, Feb. 15, 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 that the company will be presenting multiple posters from all three clinical development programs in rare muscle diseases, including new AOC 1001 long-term efficacy and safety data from the MARINA open-label extension (MARINA-OLE™) trial in people living with myotonic dystrophy type 1 (DM1) at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 3-6, 2024, in Orlando, Florida.
(PRNewsfoto/Avidity Biosciences, Inc.)
In addition, Avidity management will be joined by John W. Day, MD, PhD, Professor of Neurology and Pediatrics, and Director, Division of Neuromuscular Medicine, Stanford University School of Medicine on March 4, 2024, at 8:00 a.m. ET for a live video webcast event, which will be available on the company's website.
Dr. Day is one of the principal investigators in END-DM1, an ongoing natural history study being run by the Myotonic Dystrophy Clinical Research Network (DMCRN) and is an investigator in the AOC 1001 Phase 1/2 MARINA® trial and the MARINA-OLE trial.
POSTER PRESENTATIONS
March 4, 2024: 6:00 p.m. – 8:00 p.m. ET
AOC 1044 as a Novel Therapeutic Approach for DMD Patients Amenable to Exon 44 Skipping: EXPLORE44 Phase 1/2 Healthy Volunteer Data
March 5, 2024: 6:00 p.m. – 8:00 p.m. ET
Initial results of the Phase 2 Open-Label Extension Study of AOC 1001 in Adults with Myotonic Dystrophy Type 1: MARINA-OLE™
AOC 1001-mediated Reduction of DMPK Leads to Increase in Functional MBNL Levels, Improving Muscle Function in Patients with DM1
Targeting DUX4 for Silencing with AOC for the Treatment of FSHD
Increasing Diversity in Clinical Trial Participation: An Exploration of Clinical Trial Site Engagement
All posters will be viewable on the MDA Clinical & Scientific Conference virtual platform March 3-5, 2024, 6:00 p.m. – 8:00 p.m. ET and will also be available on the publications page of Avidity's website at https://www.aviditybiosciences.com.
Video Webcast Information
The company is hosting Volume 8 of its investor and analyst event series on March 4, 2024, beginning at 8:00 a.m. ET to discuss new AOC 1001 long-term efficacy and safety data from the MARINA-OLE™ trial in people living with DM1. 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.
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 muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through internal discovery efforts and key partnerships. Avidity is headquartered in San Diego, CA. For more information about our AOC platform, clinical development pipeline and people, please visit www.aviditybiosciences.com 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 topics of poster presentations Avidity plans to present at the 2024 MDA Clinical and Scientific Conference, including long-term efficacy and safety data from the MARINA-OLE™ trial. 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 risks described in Avidity's Annual Report on Form 10-K for the fiscal year ended December 31, 2022, and in subsequent SEC filings and press releases. 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.
Geoffrey Grande, CFA

Frequently Asked Questions

What is the MARINA-OLE™ trial about?

The MARINA-OLE™ trial studies long-term efficacy and safety of AOC 1001 in myotonic dystrophy type 1 (DM1) patients.

When will Avidity's webcast event take place?

Avidity's webcast event is scheduled for March 4, 2024, at 8:00 a.m. ET.

Who is presenting at the Avidity event?

Dr. John W. Day, a neurology and pediatrics professor, will present at the event.

What diseases are targeted by Avidity's AOCs?

Avidity's AOCs are aimed at myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral muscular dystrophy.

Where will the poster presentations be available?

Poster presentations will be accessible on the MDA conference platform and Avidity's website.

Last updated: Feb 15, 2024