Antisense Therapeutics secures approval for clinical trial

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Antisense Therapeutics (ASX:ANP) has received approval from Melbourne's Royal Children’s Hospital to undertake a phase 2 clinical trial of its immunomodulatory therapy, ATL1102, in patients with Duchenne Muscular Dystrophy (DMD), an incurable muscle wasting disease of children.

The company said the trial is on track for commencement in the second quarter of calendar year 2018. It will be conducted at the Royal Children’s Hospital neuromuscular centre. It operates the largest clinic in the southern hemisphere treating children with DMD.

The study is a single dose investigation of 25mg of ATL1102 administered weekly in wheelchair bound boys with DMD who are 10 to 18 years of age and weigh between 25 and 60 kilograms.

According to the company, the primary goal of the study is to establish ATL1102’s safety and tolerability in this patient population at the dose being investigated. The potential efficacy of ATL1102 will also be assessed via its effect on important blood and imaging markers of inflammation and muscle damage associated with DMD.

It said the extended (six-month) dosing period of the trial may also allow for ATL1102 to show an improvement in key clinical endpoints that are relevant to DMD disease progression and that are of the type that would be required for future product registration.

The Principal Investigators for the trial are Dr Ian Woodcock, a neuromuscular fellow at the RCH and Professor Monique Ryan, director of the RCH neurology department.

“Duchenne Muscular Dystrophy is a common, debilitating and ultimately terminal degenerative condition causing muscle inflammation and wasting," said Dr Woodcock.

"There is a dire need for more effective therapies than those we have already. The approach of using ATL1102 to inhibit CD49d+ T cells to treat this inflammation is consistent with observations of international researchers and published studies.

"Every day I see the pain and suffering that patients with DMD and their families go through and I am highly motivated to help in the development of new drugs and therapies that will benefit these children”.

Mark Diamond, CEO of Antisense Therapeutics, said: “We are very pleased to have received approval for our Phase II study in DMD patients. DMD is a rare disease with high unmet medical need, and so we are keen to establish ATL1102’s effectiveness in treating children with this devastating condition."