FDA Lifts Partial Clinical Hold on Avidity Biosciences' Myotonic Dystrophy Drug, Delpacibart Etedesiran

Key Insights

• The FDA has lifted the partial clinical hold on Avidity Biosciences' delpacibart etedesiran (AOC 1001) for myotonic dystrophy type 1, allowing the company to resume patient recruitment.
• The hold was initiated in 2022 following a serious adverse event in a patient, the details of which were not disclosed by Avidity Biosciences.
• Goldman Sachs analyst Corinne Johnson reiterated a buy recommendation for Avidity's stock with a price target of $59, citing reassurance regarding the drug's safety profile.

Avidity Biosciences (NASDAQ: RNA) experienced a surge in its stock value this week after the U.S. Food and Drug Administration (FDA) released its partial clinical hold on delpacibart etedesiran (del-desiran), also known as AOC 1001, an investigational drug for myotonic dystrophy type 1. The stock rose almost 12% by the close of trading on Friday following the announcement.

The FDA had placed the partial clinical hold in 2022 after a patient experienced a serious adverse event during the phase 3 clinical trial. Avidity Biosciences did not disclose the specific nature of the adverse event, but the hold paused the recruitment of new patients. With the hold lifted, the company can now resume recruitment for the ongoing phase 3 trial of del-desiran, which began earlier this year.

Delpacibart etedesiran already holds breakthrough therapy, orphan drug, and fast track designations from the FDA, underscoring the unmet need in treating myotonic dystrophy type 1.

Analyst Confidence

The FDA's decision was met positively by investors and analysts. Corinne Johnson, an analyst at Goldman Sachs, reaffirmed her buy recommendation on Avidity Biosciences' stock, setting a price target of $59 per share. Johnson noted that the FDA's action "is incrementally reassuring of the agent's safety profile which has been the primary source of investor pushback."