Rare Daily Staff

Avidity Biosciences, a biopharmaceutical company focused on delivering a new class of RNA therapeutics for rare diseases, raised $144 million in a public offering of 8 million shares of its common stock at $18 a share.

Proceeds from the offering will be used to complete a phase 1/2 trial in myotonic dystrophy type 1; to advance its pipeline of experimental candidates for the treatment of Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, muscle atrophy, and Pompe disease with two candidates entering the clinic in 2022, and to further advance its Antibody Oligonucleotide Conjugates (AOC) platform beyond muscle tissues through both internal discovery efforts and key partnerships.

Avidity’s proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity’s lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1 (DM1). The FDA has cleared Avidity to proceed with the phase 1/2 MARINA trial of AOC 1001 in adults with DM1.

DM1 is a monogenic, autosomal dominant, progressive disease that primarily affects skeletal and cardiac muscle. The disease is highly variable with respect to disease severity, presentation, and age of onset. All forms of DM1, except the late-onset form, are associated with high levels of disease burden and premature mortality. DM1 patients can suffer from various manifestations of the disease including myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications, and cognitive and behavioral impairment.

AOC 1001 consists of a proprietary mAb that binds to a transporter protein, transferrin receptor 1 (TfR1), conjugated with an siRNA that is designed to address the underlying cause of DM1 by reducing the levels of mutant DMPK RNA and allowing muscle blind-like protein to function. In preclinical studies, AOC 1001 was observed to deliver siRNAs to muscle cells and reduce levels of mRNA for the DMPK gene, the molecular driver of the disease, in a durable, dose-dependent manner.

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