Antisense Therapeutics (ASX:ANP) is planning to undertake a clinical trial of its immunomodulatory therapy ATL1102, in patients with Duchenne Muscular Dystrophy (DMD).
The trial is designed to assess the drug’s effects on the inflammation associated with this rare and incurable muscle wasting disease in children.
DMD is caused by a mutation in the muscle dystrophin gene leading to severe progressive muscle loss and premature death.
One of the most common fatal genetic disorders, DMD affects circa one in every 3,500 to 5,000 males worldwide.
A key challenge in the management of DMD patients is to reduce the inflammation that exacerbates the muscle fibre damage.
Corticosteroids are the only approved treatments for muscle inflammation, however they are not sufficiently effective and have serious side effects including adversely affecting growth rate.
As a consequence, there is an acknowledged high need for new therapeutic approaches for the treatment of inflammation associated with DMD.
Antisense’s plan to undertake a clinical trial of ATL1102 in DMD patients is facilitated by its pre-clinical and clinical experience via ATL1102’s development for the treatment of multiple sclerosis (MS).
The clinical trial of ATL1102 is planned to be undertaken at the Royal Children’s Hospital in Melbourne.
As DMD is a rare disease with a high unmet medical need, Antisense is expected to benefit materially from development incentives, including orphan drug designation that are provided to support rare disease drug development.
Antisense’s shares closed 6% higher on Monday, at $0.035.