Avidity Reports Positive AOC 1020 Data from Phase 1/2 FORTITUDE Trial

June 12, 2024

DENVER, Colo., Jun 12, 2024 (247marketnews.com)- Avidity Biosciences, Inc. (Nasdaq:RNA) reported positive initial AOC 1020 data from the Phase 1/2 FORTITUDE trial, representing reductions of greater than 50% in DUX4 regulated genes, trends of functional improvement, and favorable safety and tolerability in people living with facioscapulohumeral muscular dystrophy (FSHD).

“As the first therapy to directly target DUX4, it is very encouraging to see that the del-brax data demonstrate consistent reductions in DUX4 regulated genes and provided trends of functional improvement in patients with FSHD at the four-month timepoint. These early data would support the notion that del-brax has the potential to change the course of disease for people living with FSHD,” stated Jeffrey M. Statland, M.D., Professor of Neurology, University of Kansas Medical Center, and FORTITUDE™ trial investigator. “Early data showing trends in del-brax to improve muscle strength and function are very encouraging for patients with FSHD who are in need of treatments to prevent the muscle weakness and disability that is associated with this relentlessly, progressive disease.”

The initial AOC 1020 data will be presented at the 31st Annual FSHD Society International Research Congress, June 13-14, 2024, in Denver, Colorado.

“With the unprecedented del-brax data from the FORTITUDE trial, we are now focused on accelerating our registrational plans as we understand the urgency to develop a treatment for people living with FSHD who have no treatment options,” commented Avidity’s president and chief executive officer, Sarah Boyce. “By directly targeting the root cause of FSHD, we believe that del-brax has the potential to be a first-in-class, best-in-class therapy for people living with FSHD. This is our third rare muscle disease program to show delivery to muscle and target engagement and second therapy to provide signs of functional improvement in patients with rare neuromuscular diseases, further reinforcing the promise of our AOC platform to profoundly improve people’s lives by revolutionizing the delivery of RNA therapeutics.”

“Data from the FORTITUDE trial are very promising for people living with FSHD as the progression of the disease can make it increasingly difficult to perform critical day-to-day activities, pursue work and can affect many aspects of life, including family and social life,” said president and chief executive officer of FSHD Society, Mark Stone. “FSHD is one of the most prevalent forms of muscular dystrophy and currently, there are no treatment options. The initial del-brax data offers real hope for those living with the disease, their families, and their caregivers, who are desperately waiting for a treatment.”

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