Avidity Biosciences has launched the Phase I/II FORTITUDE clinical trial of AOC 1020 in adult patients with facioscapulohumeral muscular dystrophy (FSHD). 

The latest move comes after the company received clearance for the investigational new drug (IND) application of AOC 1020 for FSHD from the US Food and Drug Administration (FDA).

The double-blind, randomised, placebo-controlled trial will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous AOC 1020 for FSHD.

It will enrol a total of 68 adult patients with FSHD.

The safety and tolerability of AOC 1020 in FSHD patients is the primary objective of the trial. 

The therapy’s activity will also be evaluated using key biomarkers, including magnetic resonance imaging (MRI) measures of muscle volume and composition. 

AOC 1020’s clinical activity, including mobility and muscle strength measures, and subject-reported outcomes and quality of life measures will also be analysed. 

It is a muscle-targeting small interfering ribonucleic acid (siRNA) antibody oligonucleotide conjugate (AOC) and comprises a monoclonal antibody that attaches to the transferrin receptor 1 (TfR1) conjugated with an siRNA targeting DUX4 mRNA. 

Avidity Biosciences president and CEO Sarah Boyce said: “Advancing AOC 1020 into the Phase I/II FORTITUDE study is a significant milestone for the FSHD community and our proprietary AOC platform. 

“People living with FSHD have no approved treatments and experience life-long, progressive loss of muscle function, leading to fatigue and disability. 

“AOC 1020, our second siRNA AOC, is designed to directly target the disease-causing gene, DUX4, with the goal of treating the underlying biological cause of FSHD.”

A hereditary muscle-weakening ailment, FSHD is characterised by gradual muscle function loss. 

The latest development comes after the FDA placed a partial clinical hold on the company’s Phase I/II MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1.

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