California-based biopharma Avidity Biosciences announced this week that the FDA had granted fast track designation to its facioscapulohumeral muscular dystrophy drug (FSHD).
The drug — called AOC 1020 — is currently being assessed in a Phase 1/2 trial in adults living with FHSD. Patients with FSHD struggle with life-long, progressive muscle mass loss, and there are currently no FDA treatments approved for the hereditary disease.
Established in 2012, Avidity focuses on Antibody Oligonucleotide Conjugates (AOCs), a new RNA therapeutics class combining monoclonal antibodies with oligonucleotides. This combination gives the synthetic biomolecules heightened targeting abilities due to the antibodies biodistribution profiles and the functional and structural roles of the oligonucleotides. AOC 1020 consists of proprietary mAbs that bind to receptors implicated in the abnormal expression of a protein that leads to changes in muscle cells’ gene expression. More than 90% of FSHD cases are caused by mutations that lead to abnormally high activity of the DUX4 gene.
Currently, Avidity has two other rare disease programs in clinical trials; AOC 1001 for the treatment of myotonic dystrophy type 1 (DM1) and AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD).
