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With FDA Hold Lifted, Avidity Works To Bring First DM1 Therapy To Market

 
• By Joseph Haas

Avidity hopes to complete enrollment in mid-2025 for an ongoing Phase III trial of its antibody-oligonucleotide conjugate in myotonic dystrophy type 1, which has no approved drug therapy.

Dystrophin, skeletal muscle cell
Avidity can go ahead with its Phase III for a rare muscular disorder • Source: Shutterstock

With a US Food and Drug Administration partial clinical hold lifted, Avidity Biosciences, Inc. is moving forward with a Phase III trial of its antibody-oligonucleotide conjugate (AOC) candidate delpacibart etedesiran (del-desiran, AOC 1001) that ultimately could bring the first approved therapy for myotonic dystrophy type 1 (DM1), a rare type of muscular dystrophy, to market.

Key Takeaways

  • Avidity got an FDA partial clinical hold lifted for its lead antibody-oligonucleotide conjugate (AOC) candidate del-desiran, in Phase III for myotonic dystrophy type 1.

Del-desiran is one of three AOCs that Avidity has in clinical development; its second most-advanced pipeline asset is delpacibart braxlosiran...

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