In short
Delpacibart zotadirsen (AOC 1044) is an investigational protein-based medicine being studied for Duchenne muscular dystrophy (DMD) with gene mutations amenable to exon 44 skipping. It is given as an intravenous infusion and has no marketing authorisation. One authorised Phase 3 trial is evaluating its efficacy and safety in 70 participants.
Key points
- Delpacibart zotadirsen (AOC 1044) is an investigational medicine for Duchenne muscular dystrophy.
- It is being studied in people with DMD gene mutations amenable to exon 44 skipping.
- The trial is a Phase 3, randomised, double-blind, placebo-controlled global study.
- It plans to enrol 70 participants and includes an open-label extension.
- The main measure is change in time to rise from the floor (TTR velocity) from baseline to Week 54.
- Delpacibart zotadirsen is given by intravenous infusion and has orphan designation.
What is delpacibart zotadirsen?
Delpacibart zotadirsen, also known by its development code AOC 1044, is a protein-based substance classified under the therapeutic area of musculoskeletal diseases. It is an investigational medicine with no marketing authorisation in the product records, meaning it is not yet approved as a treatment. The product is supplied by Avidity Biosciences and has received orphan designation, which is granted for medicines intended to treat rare diseases.
How does delpacibart zotadirsen work?
Delpacibart zotadirsen is being studied for Duchenne muscular dystrophy (DMD) with gene mutations amenable to exon 44 skipping. DMD is a genetic condition caused by mutations in the dystrophin gene that lead to progressive muscle weakness. Exon skipping is an approach that aims to restore the reading frame of the dystrophin gene so that a shorter but functional dystrophin protein can be produced. The trial is designed to evaluate the efficacy of AOC 1044 on muscle function.
Which conditions is delpacibart zotadirsen studied for?
- Duchenne muscular dystrophy — a rare genetic disorder causing progressive muscle weakness, studied here specifically in people with mutations amenable to exon 44 skipping.
How is delpacibart zotadirsen given?
Delpacibart zotadirsen is given as a solution for infusion by the intravenous route. In the Phase 3 trial, the dose studied is 5 mg per kilogram of body weight.
What do the clinical trials show?
One authorised Phase 3 trial is investigating delpacibart zotadirsen in Duchenne muscular dystrophy. The study is randomised, double-blind, and placebo-controlled, with a global design and an open-label extension. It plans to enrol 70 participants. The primary outcome is the change from baseline to Week 54 in time to rise from the floor velocity (TTR velocity), a measure of muscle function. The comparison arm receives sodium chloride 0.9% solution as placebo.
What side effects are known?
No side effect data from supplementary sources were provided for delpacibart zotadirsen. The Phase 3 trial is designed to evaluate both efficacy and safety, and its results will provide information on side effects once available.