Avidity Prepares to Transition into Clinical Company in 2021

Sarah Boyce_Avidity

Avidity CEO Sarah Boyce pictured above. Photo courtesy of Avidity. 

California-based Avidity Biosciences has a goal of disrupting the way RNA-based therapies are delivered to patients through its Antibody-Oligonucleotide Conjugates (AOCs) platform.

Antibody-oligonucleotide conjugates combine the tissue selectivity of monoclonal antibodies and the precision of oligonucleotide-based therapeutics to overcome barriers to the delivery of oligonucleotides and target genetic drivers of disease.

Chief Executive Officer Sarah Boyce called the AOCs platform a combination approach where the monoclonal antibody is the vehicle, and the oligonucleotide is the therapy that can reach what were previously considered undruggable tissue and cell types and more effectively target underlying genetic drivers of diseases. 

In an interview with BioSpace ahead of the virtual 2021 J.P. Morgan Healthcare Conference, Boyce provided an update on Avidity’s plans for 2021, which includes the company’s transition into a clinical-stage company.

The biggest milestone in the near future for Avidity will be initiating a Phase I/II study of its program for Myotonic Dystrophy. Avidity's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1 (DM1), a rare, genetic, life-threatening neuromuscular disorder that causes impairment in muscle function, cognition and quality of life.

Myotonic dystrophy is the most common form of muscular dystrophy, affecting approximately 1 in 8,000 people. AOC-1001 is designed to address the underlying cause of DM1 by reducing levels of mutant DMPK mRNA. 

Avidity’s lead asset consists of a proprietary monoclonal antibody that binds to transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA). Preclinical data showed AOC 1001 successfully delivered siRNAs to muscle cells, resulting in a durable, dose-dependent reduction of DMPK RNA across a broad range of muscles including skeletal, cardiac and smooth muscles, according to the company.

There are currently no approved therapies indicated for the treatment of myotonic dystrophy. 

“This is just a really exciting time for us to see,” Boyce told BioSpace. “It’s the culmination of years of engineering and research that will demonstrate the progress of our muscle franchise and AOC platform. This is exciting because we’re providing options for patients in disease areas where there are not many real options.”

Trial design will be announced later this year, with plans for the Phase I portion to begin in the second half of 2021.

In September, Avidity and the Myotonic Dystrophy Clinical Research Network entered into a research agreement to support END-DM1, a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1. The study is designed to evaluate the full spectrum of disease severity and serve as the foundation for therapeutic development in myotonic dystrophy type 1.

Following on the heel of Avidity’s Phase I/II Myotonic dystrophy study are planned studies in Duchenne muscular dystrophy and Facioscapulohumeral muscular dystrophy. Other muscle-based disease programs being assessed by the company include Pompe disease and muscle atrophy.

At the beginning of January, Avidity announced a research collaboration with Bristol Myers Squibb subsidiary, MyoKardia. The partnership will demonstrate the potential use of AOCs in tissue by leveraging MyoKardia's genetic cardiomyopathy platform including, among other aspects, its novel target discovery engine and proprietary cardiac disease models. Boyce said the partnership with MyoKardia is part of the company’s strategy of expanding their therapeutic options.

That partnership builds on several positive key moments Avidity ended the year with, including an IPO that netted the company $259.2 million. That helped bring the company about $340 million to support the development of its programs. Avidity also expanded over the year, despite the ongoing pandemic. In July, the company hired Jae Kim as its chief medical officer. An Alnylam Pharmaceuticals veteran, Kim has extensive experience developing monoclonal antibodies and oligonucleotides for a range of serious diseases, including orphan indications.

In addition to Kim, Boyce said the company was able to recruit a number of key employees who have late-stage experience. That kind of experience will benefit Avidity as it transitions into a clinical company, she said. In all, the company brought on about 30 people over the past six months.

“We’ve created a strong clinical team. It’s just really exciting to see what is happening,” she said.

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