Avidity Biosciences Announces 2021 Pipeline Updates and Research Collaboration with MyoKardia

Avidity Biosciences, Inc., a biopharmaceutical company pioneering a new class of oligonucleotide-based therapies called Antibody Oligonucleotide Conjugates (AOCs™), announced pipeline updates for 2021 and a research collaboration with MyoKardia, Inc., a wholly-owned subsidiary of Bristol Myers Squibb. 

"In 2021, we enter an important new phase for Avidity as AOC 1001 is expected to enter Phase 1/2 studies for the treatment of adults with myotonic dystrophy type 1, a rare, progressive muscle disease with no approved therapy," said Sarah Boyce, President and Chief Executive Officer. "This milestone is the culmination of years of engineering and research laying the groundwork for the progress of our muscle franchise and future expansion of our AOC platform."

2021 Pipeline Updates

• In 2H 2021, Avidity plans to initiate a Phase 1/2 clinical study of AOC 1001 in adults with myotonic dystrophy type 1 (DM1).

• AOC FSHD is a therapeutic program in development for the treatment of facioscapulohumeral muscular dystrophy (FSHD).  In 2021, Avidity plans to advance the program into IND-enabling studies.  In 2022, Avidity plans to submit a regulatory filing to support a clinical trial.

• AOC DMD is a therapeutic program in development for the treatment of Duchenne muscular dystrophy (DMD).  Avidity has expanded its efforts for this indication and is now advancing three programs for DMD which target different mutations that are amenable to skipping, including Exon 44, Exon 51 and Exon 45.  Avidity's lead AOC in development for DMD targets Exon 44. In 2022, Avidity plans to submit a regulatory filing to support a clinical trial.

• AOC Muscle Atrophy is a therapeutic program in development for the treatment of muscle atrophy (MA).  During 2021, Avidity researchers will evaluate AOCs in multiple disease models of MA to identify an optimal development path. 

Research Collaboration with MyoKardia, a Wholly-Owned Subsidiary of Bristol Myers Squibb

• Avidity has entered into a research collaboration with MyoKardia, a wholly-owned subsidiary of Bristol Myers Squibb, to demonstrate the potential utility of AOCs in cardiac tissue by leveraging MyoKardia's genetic cardiomyopathy platform including, among other aspects, its novel target discovery engine and proprietary cardiac disease models.   

"We are excited to partner with MyoKardia, the pioneers in precision medicine approaches to treating cardiomyopathies and other heart diseases," said Joseph Baroldi, Chief Operating Officer. "This research collaboration with MyoKardia furthers Avidity's strategic approach to broadening the use of our AOC platform beyond skeletal muscle. Looking ahead, our business development efforts will continue to focus on collaborating with high-quality partners to accelerate and expand the utility of our AOCs."

About Avidity Biosciences

Avidity Biosciences, Inc. is driven to change lives with a new class of therapies called Antibody Oligonucleotide Conjugates (AOCs) that are designed to overcome current limitations of oligonucleotide therapies in order to treat a wide range of serious diseases. Avidity's proprietary AOC platform combines the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide therapies to access previously undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, and its other muscle programs are focused on the treatment of Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, Pompe disease and muscle atrophy. In addition to its muscle franchise, Avidity has research efforts focused on immune, cardiac and other cell types.