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A Quick Look At Avidity (RNA)

Avidity Biosciences Inc. (RNA) will be moving its first product candidate, AOC 1001, into clinic this year.

AOC 1001, which belongs to a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs), is being developed for the treatment of Myotonic Dystrophy Type 1 (DM1).

Myotonic Dystrophy Type 1 is a form of muscular dystrophy that is caused by a mutation in the dystrophia myotonica protein kinase, or DMPK gene, found on chromosome 19. This rare progressive genetic disease begins in adolescence or young adulthood, often with weakness in the muscles of the face, neck, fingers, and ankles.
Over 40,000 people in the U.S. and more than 74,000 people in Europe are affected by DMI. Currently, there are no approved disease modifying therapies for DM1.

Avidity Biosciences will be initiating a phase I/II study of AOC 1001 in Myotonic Dystrophy Type 1 in the second half of this year.

In addition to AOC 1001, the company has a several compounds targeting indications like Facioscapulohumeral Muscular Dystrophy, Duchenne Muscular Dystrophy, Muscle Atrophy, and Pompe Disease, all of which are in preclinical testing.

Avidity plans to submit a regulatory filing to support a clinical trial for AOC FSHD, a therapeutic program in development for the treatment of facioscapulohumeral muscular dystrophy, in 2022.

The company has a research collaboration with MyoKardia, a wholly-owned subsidiary of Bristol Myers Squibb, to demonstrate the potential utility of Antibody Oligonucleotide Conjugates in cardiac tissue. Avidity's proprietary AOC platform is also being used by pharma giant Eli Lilly and Co. (LLY) to develop new treatments in immunology.

At year-end 2020, the company had cash, cash equivalents and marketable securities of $328.1 million.

Avidity Biosciences went public on the Nasdaq Global Market on June 12, 2020, offering its shares at a price of $18 each.

The stock has thus far hit a low of $19.82 and a high of $37.46. RNA closed Monday's trading at $20.54, down 7.06%.

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