Avidity granted FDA fast track designation for AOC 1020

Post by **Administrator** » 05-Feb-2023, 14:43

The Food and Drug Administration (FDA) has granted Fast Track designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy.
AOC - Antibody Oligonucleotide Conjugate. AOC 1020 is designed to reduce the expression of DUX4 mRNA and DUX4 protein in muscles. The investigational treatment consists of a monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA targeting DUX4 mRNA.
The Company is currently investigating AOC 1020 in the phase 1/2 FORTITUDE study in adults with FSHD. The double-blind, placebo-controlled study is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AOC 1020 administered intravenously. Though the study is not powered to assess functional benefit, assessments of efficacy will be made using key biomarkers, including magnetic resonance imaging measures of muscle volume and composition. Mobility, muscle strength, patient reported outcomes, and quality of life measures will also be analyzed.

https://www.aviditybiosciences.com/pipeline/fshd/
https://musculardystrophynews.com/news/ ... treatment/
https://www.fshdsociety.org/2023/01/20/ ... erapeutic/
https://www.prnewswire.com/news-release ... 24062.html