Antisense Therapeutics (ASX: ANP) has now completed enrolment for its phase two clinical trial using its immunomodulatory therapy ATL1102 drug on Duchenne Muscular Dystrophy (DMD), with results expected before the end of the year.
The trial is underway at a neuromuscular centre which is part of Melbourne’s Royal Children’s Hospital.
According to Antisense, the centre operates the largest clinic in the southern hemisphere treating children with DMD.
As part of the trial, nine non-ambulant patients aged between 10 and 18 with DMD are being treated with ATL1102 over six months.
Three patients have already completed 24 weeks of dosing, four are currently undergoing dosing, with the final two patients due to begin treatment before the end of the month.
“We are very pleased to have achieved the key clinical trial milestone of completion of patient enrolment into our phase two clinical trial of ATL1102 in DMD patients,” Antisense chief executive officer Mark Diamond said.
“The Data Safety Monitoring Board has been periodically evaluating the safety related trial data and has, 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 further quarter this year, which presents as a very exciting prospect for the company and potentially for future treatment of DMD patients,” Mr Diamond added.
About ATL1102 and Duchenne Muscular Dystrophy
Antisense has developed ATL1102 to treat inflammation that exacerbates muscle fibre damage in DMD sufferers.
Patients are currently given corticosteroids to reduce inflammation. However, these have a number of serious side effects when used for prolonged periods.
According to Antisense, there are a limited number of anti-inflammatory drugs in development for treating DMD.
Antisense anticipates the extended dosing period of the study may allow ATL1102 to show an improvement in disease progression.
“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 stated.
DMD is known as an X-linked diseased that affects one in 3,600-6,000 male babies who are born.
The disease is caused by mutations in the dystrophin gene, which results in the absence of dystrophin protein – or a severe reduction.
Males born with the disease suffer from muscle deterioration and are often consigned to a wheelchair during their early teenage years.
As the disease progresses, patients experience respiratory, cardiac and cognitive dysfunction.
Without intervention, the mean life span for a DMD sufferer is 19 years.
This morning’s news sent Antisense’s share price rocketing to $0.067 before settling at $0.06 before midday – up 20%.