Benzinga | Aug 2, 2021 12:38PM EDT

Avidity Biosciences Shares Gain As Its Oligonucleotide Candidate Enters Early-Stage Trial In Muscle Wasting Disorder

* The FDA has signed off Avidity Biosciences Inc's (NASDAQ:RNA) Phase 1/2 MARINA trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1).

* Myotonic dystrophy is characterized by progressive muscle wasting and weakness. AOC 1001, Avidity's lead program utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of DMPK, the disease-related mRNA.

* AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA) that targets DMPK mRNA.

* The 44-subject trial's primary objective is to evaluate the safety and tolerability of single and multiple ascending doses of AOC 1001 administered intravenously.

* The MARINA trial will assess the activity of AOC 1001 across key biomarkers, including spliceopathy, a key biomarker for DM1, and knockdown of DMPK mRNA, the disease-related mRNA responsible for DM1.

* Patients will have the option to enroll in an open-label extension study at the end of the post-treatment period.

* In 2H of 2022, Avidity plans to conduct a preliminary assessment in approximately half of the study participants.

* Recently, the FDA also granted Orphan Drug Designation to AOC 1001 for the treatment of DM1.

* Price Action: RNA shares are up 5.6% at $20.40 during the market session on the last check Monday.

* Related content: Benzinga's Full FDA Calendar.