Avidity Biosciences to Present Topline Data from Phase 1/2 FORTITUDE™ Trial of Del-brax in People Living with Facioscapulohumeral Muscular Dystrophy at 32nd Annual FSHD Society International Research Congress

Rhea-AI Summary

Avidity Biosciences (NASDAQ: RNA) announced upcoming presentations of topline data from their Phase 1/2 FORTITUDE trial for del-brax (delpacibart braxlosiran) in facioscapulohumeral muscular dystrophy (FSHD) at the 32nd Annual FSHD Society International Research Congress in Amsterdam. The company has secured FDA alignment on both accelerated and full approval pathways for del-brax in FSHD treatment. Dr. Jeffrey M. Statland will present the trial's dose escalation data, while Dr. Stephen Tapscott will discuss findings on a novel DUX4-regulated circulating biomarker. The presentations include two oral sessions and one poster presentation scheduled for June 12-13, 2025.

Positive

• FDA alignment secured for both accelerated and full approval pathways for del-brax
• Company advancing development of novel RNA therapeutics (AOCs) for FSHD treatment
• Identification of a new DUX4-regulated circulating biomarker for FSHD

Negative

• None.

Insights

Biotechnology Research Analyst

Avidity's del-brax shows regulatory progress with FDA alignment on approval pathways for FSHD treatment ahead of data presentation.

The announcement that Avidity Biosciences has secured FDA alignment on both accelerated and full approval pathways for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD) represents a significant regulatory milestone. This alignment substantially de-risks the development pathway and suggests the FDA sees potential merit in the clinical program.

The upcoming presentation of topline data from the Phase 1/2 FORTITUDE trial at the FSHD Society International Research Congress is a pivotal moment for the company's AOC platform technology. Having a respected investigator like Dr. Jeffrey Statland presenting these results adds credibility to the findings. The presentation timing indicates the company has confidence in their data package.

Particularly noteworthy is the presentation on a novel DUX4-regulated circulating biomarker. DUX4 is the key pathogenic protein in FSHD, and developing a reliable circulating biomarker could dramatically improve clinical trial design and potentially serve as a surrogate endpoint. This could accelerate development timelines and reduce clinical trial costs if validated.

FSHD affects approximately 1 in 8,000 individuals worldwide with limited treatment options, representing a substantial unmet medical need. Avidity's AOC platform, which combines antibody tissue-targeting capability with oligonucleotide therapeutic effects, offers a potentially differentiated approach to RNA-targeted therapeutics compared to traditional antisense oligonucleotides or siRNA approaches.

-- FDA alignment on accelerated and full approval pathways for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD) --

-- Jeffrey M. Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE trial investigator, will present topline del-brax data from dose escalation cohorts in oral presentation --

-- Stephen Tapscott, M.D., Ph.D., Professor of Human Biology and Clinical Research, Fred Hutchinson Cancer Center, will highlight results on the characterization of a novel DUX4-regulated circulating biomarker in oral and poster presentations --

SAN DIEGO, June 11, 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 that the company will be presenting two oral and one poster presentations at the 32nd Annual FSHD Society International Research Congress, being held June 12-13, 2025, in Amsterdam, the Netherlands. Earlier this week, Avidity announced FDA alignment on accelerated and full approval pathways for delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD).

32^nd Annual FSHD Society International Research Congress Presentations

• Topline Data from Dose Escalation Cohorts A and B in FORTITUDE™, a Phase 1/2 Trial Evaluating Del-brax (delpacibart braxlosiran) in Adults with Facioscapulohumeral Muscular Dystrophy (FSHD)
  • Oral presentation: Jeffrey M. Statland, M.D., to present June 13, 2025, from 5:20 - 5:40 p.m. Central European Summer Time (CEST) / 11:20 - 11:40 a.m. ET

• Characterization of a promising DUX4-regulated circulating biomarker for facioscapulohumeral dystrophy (FSHD)
• • Oral presentation: Stephen Tapscott, M.D., Ph.D., Professor of Human Biology and Clinical Research, Fred Hutchinson Cancer Center, to present on June 12, 2025, from 6:25 - 6:30 p.m. CEST / 12:25 - 12:30 p.m. ET
    
    
  • Poster presentation (#7.08): June 12, 2025, from 7:15 - 8:00 p.m. CEST / 1:15 - 2:00 p.m. ET

The presentations and poster are available on the publications page of Avidity's website at https://www.aviditybiosciences.com.

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 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: Avidity's plans to present topline data from the dose escalation cohorts of the Phase 1/2 FORTITUDE™ trial; the status and availability of accelerated and full approval pathways for del-brax and Avidity's planned participation at the 32^nd Annual FSHD Society International Research Congress and the contents of its scheduled presentations. 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 clinical study of del-brax may not support BLA submission or accelerated or full approval, and may not be satisfactory to the FDA and other regulators; later developments with the FDA and other regulators may be inconsistent with the feedback received as of the date hereof; and the 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) 837-5014
investors@aviditybio.com

Media Contact:
Kristina Coppola
(619) 837-5016
media@aviditybio.com

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SOURCE Avidity Biosciences, Inc.

FAQ

What is the significance of FDA alignment for Avidity Biosciences' del-brax (RNA)?

The FDA alignment on both accelerated and full approval pathways for del-brax represents a significant regulatory milestone that could potentially expedite the drug's development and approval process for FSHD treatment.

What will be presented at the 2025 FSHD Society International Research Congress for RNA stock?

Avidity will present topline data from the Phase 1/2 FORTITUDE trial's dose escalation cohorts and findings on a novel DUX4-regulated circulating biomarker through two oral presentations and one poster session.

What is del-brax and what is it being developed for by Avidity (RNA)?

Del-brax (delpacibart braxlosiran) is an Antibody Oligonucleotide Conjugate (AOC) therapeutic being developed by Avidity Biosciences for the treatment of facioscapulohumeral muscular dystrophy (FSHD).

When will Avidity Biosciences (RNA) present their FORTITUDE trial results?

The FORTITUDE trial results will be presented on June 13, 2025, from 5:20-5:40 PM CEST by Dr. Jeffrey M. Statland at the FSHD Society International Research Congress in Amsterdam.