Novel Therapy Gets Orphan Drug Status for Duchenne Muscular Dystrophy

The Food and Drug Administration (FDA) has granted Orphan Drug designation to AOC 1044 for the treatment of Duchenne muscular dystrophy in patients with mutations amenable to exon 44 skipping (DMD44).

Duchenne muscular dystrophy (DMD) is a rare genetic disorder caused by mutations in the DMD gene, which encodes the dystrophin protein. Lack of functional dystrophin results in the progressive loss of muscle function. In individuals with DMD44, AOC 1044 is designed to produce dystrophin protein by delivering phosphorodiamidate morpholino oligomers to skeletal muscle and heart tissue to specifically skip exon 44 of the DMD gene.

The safety, tolerability, pharmacokinetics, and pharmacodynamic effects of AOC 1044 is being evaluated in the phase 1/2 EXPLORE44 trial (ClinicalTrials.gov Identifier: NCT05670730). The trial will include both healthy volunteers and DMD patients; exon skipping and dystrophin protein levels will be assessed in DMD participants.

The Company expects to share trial data from the healthy volunteer portion in the fourth quarter of 2023.

“We are pleased that the FDA has granted both Orphan Drug and Fast Track designation to AOC 1044, highlighting the importance of advancing new treatments for people living with DMD,” said Steve Hughes, MD, chief medical officer at Avidity. “There are currently no treatment options that target the underlying cause of DMD44. AOC 1044 is designed to specifically skip exon 44 of the dystrophin gene to enable the production of functional dystrophin protein. We look forward to advancing AOC 1044 in clinical development and bringing this very important treatment to patients as quickly and safely as possible.”

This article originally appeared on MPR