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

More from Clinical Trials
RemeGen is planning to complete enrolment in the global Phase III RemeMG study with telitacicept in generalized myasthenia gravis by the end of 2025 or early 2026. The Chinese firm has already sidelined two other global Phase III trials with the molecule to prioritize the indication.
The biotech is banking on itolizumab’s longer-term efficacy as well as unmet need in frontline acute graft-versus-host disease as it prepares to meet with the FDA.
The head of the Spanish medical dermatology specialist told Scrip that maintaining the status quo will only result in the continent’s life sciences sector slipping further behind the US and China.
With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.
More from R&D
The Danish firm is spending nearly $580m to repurchase up to 2.2 million shares
The Texas-based firm is giving up on the investigational Alzheimer’s therapy after a second Phase III failure left it with nowhere to go in the disease.
A decision from the FDA is due by 28 September.