Antisense Therapeutics confirms phase 2 DMD trial fully enrolled

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Antisense Therapeutics (ASX:ANP) has reported that the phase 2 clinical trial of its immunomodulatory therapy, ATL1102 for Duchenne Muscular Dystrophy (DMD), is now fully enrolled.

The open-label six-month dosing trial of ATL1102 in nine non-ambulant patients with DMD aged between 10 and 18 years is being conducted at the neuromuscular centre of the Royal Children’s Hospital in Melbourne. The hospital operates the largest clinic in the southern hemisphere treating children with DMD.

According to the company, three patients have completed their 24 weeks of dosing, four patients are in the treatment phase of the study and the final two patients have been screened and are scheduled to commence their dosing before the end of this month.

"No Serious Adverse Events have been reported to date," said the company, adding dosing of all patients in the trial will be completed in early November 2019.

ATL1102 is being developed as a novel treatment for the inflammation that exacerbates muscle fibre damage in DMD patients, currently treated with corticosteroids. Corticosteroids have a range of serious side effects when used for a prolonged period as required in DMD. Currently there are a limited number of anti-inflammatory drugs in clinical development for DMD.

The primary endpoints of the trial relate to the safety and tolerability of ATL1102. The efficacy of ATL1102 will also be assessed in terms of its effects on disease processes and progression.

"The extended dosing period (24 weeks) of this clinical trial may allow for ATL1102 to show an improvement in the clinical endpoints that are relevant to DMD 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," said the company.

Previous published studies in non-ambulant DMD boys have reported that after six months there is a significant decline in such functional endpoints even if the patients are on corticosteroids.

One of the objectives of the ATL1102 trial is to address the decline in upper limb strength and function of the non-ambulant DMD patients.

The company said it expects to report trial results shortly after the completion of dosing.

”We are very pleased to have achieved the key clinical trial milestone of completion of patient enrolment into our Phase II clinical trial of ATL1102 in DMD patients," said CEO Mark Diamond.

"The intensive safety monitoring required for this first study in boys with DMD requires frequent visitations to the hospital over the course of the trial period which is particularly challenging for wheelchair-bound boys and their families and so we greatly appreciate their commitment to participate in this trial.

"The Data Safety Monitoring Board have been periodically evaluating the safety related trial data and have on each occasion recommended continuation of the trial with no safety concerns. We are now set to have dosing completed and trial results reported in the fourth quarter of this year, which presents as a very exciting prospect for the Company and potentially for future treatment of DMD patients.”