Antisense Therapeutics Limited (ASX:ANP) has confirmed that dosing has commenced in a muscular dystrophy (mdx) mouse model of DMD to assess the potential clinical utility of ATL1102 in combination with dystrophin restoration drugs to improve on therapeutic outcomes for patients with DMD.
Under the collaborative 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 (morpholino oligonucleotide exon skipping drug of the same drug chemistry as the exon skipping treatments marketed in the US).
Antisense 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.
The combination study will assess the effects of antisense inhibition of CD49d in combination with a dystrophin restoration drug on markers of drug activity in the DMD mdx model including the potential of the combination to improve dystrophin expression levels beyond that achieved by the dystrophin restoration agent used alone, and thereby point to the potential utility of the combination treatment in the clinic.
This study is on track to be completed with results due Q4’CY22. Sales of the dystrophin restoration drugs in the US for the 2nd Quarter of 2022 alone were in excess US$233 million,
Currently these 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, which underlines the exciting opportunity for a new combination therapy that can deliver improved outcomes for DMD patients.