Avidity Biosciences Touts Encouraging Data From Myotonic Dystrophy Type 1 Study

Avidity Biosciences Inc RNA announced topline data from the Phase 1/2 MARINA trial of AOC 1001 for myotonic dystrophy type 1 (DM1).

In March, the company concluded 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.

Directional improvement in multiple functional assessments was observed, including myotonia, strength, and mobility measures.

Meaningful DMPK reduction and splicing changes in participants treated with AOC 1001

Splicing changes followed by directional improvements in functional measures at 2 mg/kg and 4mg/kg doses of AOC 1001

AOC 1001 demonstrated broad splicing improvements in more than a thousand genes impacted by DM1, confirming activity in the nucleus

Favorable safety and tolerability profile of AOC 1001 with most adverse events mild or moderate

Avidity continues to work with the FDA regarding the partial clinical hold on new participant enrollment in the AOC 1001 program. 

The company continues to dose the participants at 2 mg/kg and 4 mg/kg of AOC 1001 in the MARINA open-label extension (MARINA-OLE). 

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 down 7.08% at $13.52 during the premarket session on the last check Friday.