Special report: A group of boys at the Royal Children’s Hospital in Melbourne will receive a unique opportunity this month.

Antisense Therapeutics (ASX:ANP) has launched its Phase 2 clinical trial for a treatment for Duchenne Muscular Dystrophy (DMD), a muscle-wasting disease that affects boys only, putting them in wheelchairs by their teens and cutting lives short in their 20s.

Recruitment is now underway and the trial will assess the safety and tolerability of Antisense’s immunomodulatory therapy ATL1102, as well as its efficacy on slowing disease progression.

ATL1102 is to be used as a treatment for a key aspect of the disease process, muscle inflammation.

The disease causes muscle wasting that is compounded by the body’s response which is expressed as inflammation — which further damages the muscles.

The inflammation is now treated by using corticosteroids, broad acting drugs with nasty side effects when used at high doses for long periods of time, including weight gain which is a major issue for a child or teen in a wheelchair.

Furthermore, corticosteroids do not appear to be as effective in patients with a greater number of white blood cells that express high levels of CD49d receptors — ATL1102’s biological target — on their surface.

The idea is that ATL1102 will be able to slow the disease progression to keep little boys out of wheelchairs or maintain their mobility for longer.

The trial, led by principal investigators Dr Ian Woodcock, a Neuromuscular Fellow at the Melbourne Royal Children’s Hospital and Professor Monique Ryan, director of the Neurology Department at the hospital.

The development of ATL1102 as a treatment for DMD is guided by a Scientific Advisory Board of experts including inventors of the FDA-approved drug eteplirsen, which is used to increase muscle dystrophin in DMD patients and marketed by US based and NASDAQ listed Sarepta Therapeutics.

Working together

Where Antisense’s drug tackles the inflammation, Sarepta’s drug looks to increase the levels of muscle dystrophin, the loss of which causes the disease.

Former Sarepta chairman and Antisense board member William Goolsbee believes the Australian company’s treatment may improve the outcomes for patients that are also on the Sarepta drug.

“What we want to do is to eliminate a substantial portion of continued muscle degeneration by ameliorating the damaging effects of the inflammation with the use of ATL1102 to help extend the period of mobility in the boys, and in particular the upper limb function of the wheel chair bound boys to be enrolled in our study,” he said.

“It is personally very gratifying to see our study successfully progressing in a disease area where I know there is a great need for better and safer treatments.”

The DMD treatment, ATL1102, is the same as that was previously used in a successful Phase II trial in Multiple Sclerosis, which showed significant activity in reducing the number of newly formed inflammatory brain lesions.


This special report is brought to you by Antisense Therapeutics.

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