FDA Eases Partial Hold On Avidity Biosciences' Investigational Drug Study In Type Of Dystrophy

The FDA has eased the partial clinical hold on Avidity Biosciences Inc's RNA AOC 1001, allowing Avidity to double the number of participants in the MARINA Open-Label Extension (MARINA-OLE) study receiving 4 mg/kg of AOC 1001. 

The FDA is also allowing new participant enrollment for AOC 1001 at 2 mg/kg. 

The FDA placed a partial clinical hold on new participant enrollment in the trial in September last year.

Data from the MARINA-OLE study will be used to finalize the AOC 1001 pivotal dose and Phase 3 study design for adults with myotonic dystrophy type 1 (DM1), a fatal neuromuscular disease.

Avidity announced topline AOC 1001 data from the Phase 1/2 MARINA trial demonstrating directional improvement in multiple functional assessments, including myotonia, strength, and mobility measures.

In March, Avidity Biosciences decided to conclude the Phase 1/2 MARINA trial with the 38 participants enrolled at 1mg/kg, 2mg/kg, and 4mg/kg of AOC 1001 and not to move forward with the 8 mg/kg dose in myotonic dystrophy type 1 (DM1).

 Avidity remains on track to share a first look at the data from the MARINA-OLE study at the end of 2023.

Price Action: RNA shares are up 1.60% at $11.13 on the last check Wednesday.