Antisense Therapeutics announces outcomes in DMD combination animal study

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Antisense Therapeutics (ASX:ANP) has reported what it describes as initial positive muscle functional data from a DMD mdx animal study assessing the use of the combination of antisense (ASO) to CD49d with a dystrophin exon skipping restoration drug.

The company said the use of the combination improved the specific maximum force of the extensor digitorum longus (EDL) muscle, a lower leg muscle, and the eccentric muscle force remaining following induced damage to the EDL.

"This functional data supports the potential use of ATL1102 in combination with dystrophin restoration drugs to improve therapeutic outcomes in patients with DMD," it said.

Under the collaborative research agreement with the Murdoch Children’s Research Institute’s (MCRI), six groups of DMD mdx mice (n=8 per group) were treated for six weeks with antisense oligonucleotide to CD49d (mouse equivalent of ATL1102), or control oligonucleotide mismatch or saline treatments, or the morpholino exon skipping dystrophin restoration drug alone and in combination.

The muscle physiology of the EDL was assessed for force parameters including specific maximum force and the force drop following 1 to 10 eccentric (lengthening) contraction each involving induced muscle damage by the stretching of the muscle by 10%.

Dr Peter Houweling is the team leader of the MCRI Muscle Research group.

“These early pre-clinical findings suggest that a combination therapy using an established dystrophin restorative ASO and CD49d result in improved functional outputs in the mdx mouse model of Duchenne muscular dystrophy," he said.

"These effects appear to be beneficial beyond the individual monotherapy and we look forward to continuing our investigations into the biological mechanisms that may be at play”.

According to Dr George Tachas, the director of drug discovery and patents at Antisense Therapeutics, “The encouraging effects observed in our study of the combination treatment on the EDL muscle function suggest the potential for the combination treatment to show benefit beyond monotherapy is on the right track. The study design and functional endpoints assessed have featured in many mdx mouse studies published in the scientific literature, and as such supports the validity of our study and its outcomes."