On track to deliver key regulatory updates for del-brax and topline data from dose escalation cohorts in the FORTITUDE™ trial in the second quarter

Positive topline del-zota data further supports first BLA submission at year end 2025 – continues to highlight reproducibility and consistency across three late-stage clinical trials for DMD44, DM1 and FSHD

 Executing on global commercial infrastructure development and on track with preparations for first potential commercial launch in U.S. in 2026

SAN DIEGO, May 6, 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 Avidity management will participate in a fireside chat during the following investor conference:

SAN DIEGO, April 21, 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 on April 20, 2025, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 263,000 shares of its common stock and 131,450 restricted stock units ("RSUs") to thirty-three (33) new non-executive employees under the Avidity Biosciences, Inc. 2022 Employment Inducement Incentive Award Plan (the "2022 Inducement Plan"). The awards were granted as inducements material to the employees entering into employment with Avidity in accordance with Nasdaq Listing Rule 5635(c)(4).

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).

SAN DIEGO, April 1, 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 Avidity management will participate in a fireside chat during the following investor conference:

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.

SAN DIEGO, March 21, 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 on March 20, 2025, the Human Capital Management Committee of Avidity's Board of Directors granted non-qualified stock option awards to purchase an aggregate of 101,200 shares of its common stock and 50,900 restricted stock units ("RSUs") to sixteen (16) new non-executive employees under the Avidity Biosciences, Inc. 2022 Employment Inducement Incentive Award Plan (the "2022 Inducement Plan"). The awards were granted as inducements material to the employees entering into employment with Avidity in accordance with Nasdaq Listing Rule 5635(c)(4).

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.

Aravindhan Veerapandiyan, MD, Associate Professor of Pediatrics, University of Arkansas for Medical Sciences and Arkansas Children's Hospital and EXPLORE44® trial investigator, will present topline del-zota data from Phase 1/2 EXPLORE44 trial at 2025 MDA Clinical & Scientific Conference in Dallas, Texas

Avidity to host investor and analyst webcast event March 17, 2025, at 8:00 a.m. ET

Key opinion leaders to participate in Avidity sponsored industry forum event "Advancing RNA Therapeutics", March 17, 2025, at 7:00 a.m. CT/8:00 a.m. ET

Building on success across its three clinical programs, Avidity is leading in rare neuromuscular diseases with a strong balance sheet to execute on a transformational 2025

Major milestones anticipated for each rare neuromuscular program in 2025, including preparing for Avidity's first BLA submission 

Commercial preparations well underway in anticipation of three potential successive product launches for DMD, DM1 and FSHD starting in 2026

Phase 1/2 EXPLORE44® top-line del-zota data to be presented at the 2025 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in Dallas, Texas