FDA grants fast track designation to exon-skipping Duchenne muscular dystrophy therapy

Key takeaways:

• AOC 1044 is being evaluated in healthy volunteers and people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping.
• No approved therapies target exon 44.

The FDA has granted fast track designation to AOC 1044 for the treatment of Duchenne muscular dystrophy in people with mutations amenable to exon 44 skipping, Avidity Biosciences announced in a press release.

According to the release, AOC 1044 is an antibody oligonucleotide conjugate designed to deliver phosphorodiamidate morpholino oligomers to skeletal and heart tissue to skip exon 44 of the dystrophin gene, prompting dystrophin protein production in patients with Duchenne muscular dystrophy (DMD). Currently, there are no approved therapies that target exon 44, the company said.

AOC 1044 is being studied in the randomized, placebo-controlled, double-blind phase 1/2 EXPLORE44 trial, which is expected to enroll 24 individuals with DMD44 and 40 healthy volunteers, aged 7 to 27 years. The study will examine the safety, tolerability, pharmacokinetics and pharmacodynamic effects of single and multiple ascending IV doses of AOC 1044, as well as exon skipping and dystrophin protein levels in participants with DMD44, who will have the option to continue in an extension study.

“It is very encouraging to receive FDA fast track designation as it further validates the potential of AOC 1044 to target the underlying cause of DMD44 and the importance of bringing people living with this devastating disease an effective treatment option,” Steve Hughes, MD, Avidity chief medical officer, said in the release. “We will continue to work closely with the FDA as we advance AOC 1044.”

The company plans to release data from the healthy volunteer portion of the study in the second half of 2023.