Antisense Therapeutics have received approval from the Royal Children’s Hospital (RCH), Melbourne Human Research Ethics Committee, to undertake a Phase II clinical trial of its immunomodulatory therapy, ATL1102, in patients with Duchenne muscular dystrophy. The trial is on track for commencement in Q2’18 and will be conducted at the RCH neuromuscular centre, which operates the largest clinic in the southern hemisphere treating children with Duchenne.

The study is a single dose investigation of 25mg of ATL1102 administered weekly in wheel chair bound boys with Duchenne who are 10 to 18 years of age and weigh between 25 to 60kg. The primary goal of the study is to establish ATL1102’s safety and tolerability in this DMD patient population at the dose being investigated. The potential efficacy of ATL1102 will also be assessed via ATL1102’s effects on important blood and imaging (MRI) markers of inflammation and muscle damage associated with Duchenne. Notably, the extended (6 month) dosing period of this clinical trial may also allow for ATL1102 to show an improvement in key clinical endpoints that are relevant to Duchenne disease progression (e.g. the upper limb function of the boys) 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 Neurology Department at RCH. The clinical development of ATL1102 trial in boys with Duchenne is to be directed by an Advisory Board of international experts in the field including inventors of the FDA approved antisense drug, eteplirsen, used to increase muscle dystrophin in Duchenne patients and marketed by Sarepta Therapeutics (NASDAQ:SRPT, US$4bn). The Advisory Board is chaired by Mr William Goolsbee, a non-executive director of ANP and ex-Chairman of Sarepta Therapeutics.

About ATL1102

ATL1102 is an antisense inhibitor of CD49d, a subunit of VLA-4 (Very Late Antigen-4). Antisense inhibition of VLA-4 expression has demonstrated activity in a number of animal models of inflammatory disease including asthma and MS with the MS animal data having been published in a peer reviewed scientific journal. ATL1102 was shown to be highly effective in reducing MS lesions in a Phase IIa clinical trial in RR-MS patients. The ATL1102 Phase IIa clinical data has been published in the medical Journal Neurology (Limmroth, V. et al Neurology, 2014; 83(20): 1780-1788).

See page 3 for full protocol synopsis