Antisense Therapeutics (ASX:ANP) has released more positive news about its drug candidate ATL1102, saying it looks like the antisense inhibitor injections could have a “profound” impact in the lives of boys with Duchenne muscular dystrophy.

The Melbourne biotech on Friday released to the market data presented at the 25th International Annual Congress of the World Muscle Society, on the phase-two study of the drug candidate that was completed in May.

Antisense says nine wheelchair-bound boys aged 12 to 18 with Duchenne muscular dystrophy at Royal Children’s Hospital in Melbourne showed significant improvement when treated with ATL1102 injections over 24 weeks, compared to 20 who weren’t.

Those 20 were selected from a natural history database of muscular dystrophy patients in Rome.

Duchenne muscular dystrophy is a muscle-wasting disease for which there is no cure, but four of the nine boys showed improvement in their upper limb function after 24 weeks, compared to just 18 per cent in the control group.

Overall on a measure of upper limb function called the PUL2.0 Score, the boys treated with ATL1102 improved by an average of 0.89 points, while boys in the control group declined 2.00 points, Antisense says.

“Such a significant gain in Total PUL2.0 in six months is to our knowledge yet to be reported with other drugs in non ambulant DMD, a patient group who have very limited treatment options,” Antisense said.


DMD drug has ‘profound’ impact

Antisense managing director and chief executive Mark Diamond told analysts on a Zoom call that retaining some upper limb function might mean that a boy with DMD would still be able to brush their teeth, use an iPad and feed themselves.

“That’s why the data is being regarded as clinically significant,” he said.

Antisense consultant medical director Dr Gil Price said that the word patient groups used when it came to retaining limb function was “profound”.

“Things that we may look in our lives as being small, may be profound in the life of a boy with Duchenne,” he said.

Antisense wasn’t able to keep the boys on ATL1102 after the study ended  but hopes to re-enrol them into a phase 2b study next year.

Morgans healthcare analyst Scott Power called the announcement by Antisense “very positive – further validation that the drug appears to be working”.