Antisense Therapeutics (ASX:ANP) has commenced dosing in a muscular dystrophy (mdx) mouse model of Duchenne muscular dystrophy (DMD) to assess the potential clinical utility of ATL1102 in combination with dystrophin restoration drugs.
Currently, dystrophin restoration drugs are used in combination with steroids and predominantly for the younger ambulant DMD population but are yet to demonstrate in controlled studies to be effective in further delaying loss of ambulation beyond the use of steroids alone.
The company that under its research agreement with the Murdoch Children’s Research Institute’s (MCRI), mice will be dosed with an antisense oligonucleotide designed to target CD49d (mouse equivalent of ATL1102) or control oligonucleotide or saline treatments in combination with a dystrophin restoration drug.
Antisense said inhibition of CD49d has previously demonstrated activity in an mdx mouse model as a monotherapy, reducing CD49d+ immune cells and both the CD49d target in the muscle and muscle damage.
It said the study is on track to be completed with results due in the fourth quarter of the calendar year 2022.