Antisense Therapeutics announced that the US Food and Drug Administration (FDA) Office of Orphan Products Development has granted an Orphan Drug Designation (ODD) to ATL1102 for treatment of Duchenne muscular dystrophy (DMD). ATL1102 is an antisense drug designed to reduce inflammation in human disease.
It was recently assessed in a Phase II study in adolescent non-ambulant patients with DMD, meeting the primary endpoint of the study with confirmation of the drug’s safety and tolerability. ATL1102 also demonstrated strong effects on disease progression parameters with improvement or stabilization across multiple measures of muscle function and strength.
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 patients with RRMS. The ATL1102 Phase IIa clinical data has been published in the medical Journal Neurology (Limmroth, V. et al Neurology, 2014; 83(20): 1780-1788).
About ATL1102 DMD Trial
The Phase II clinical trial of ATL1102 in patients with Duchenne Muscular Dystrophy was an open label six-month dosing trial of ATL1102 administered SC at 25mg per week in nine non-ambulant patients with DMD aged between 10 and 18 years. The trial was conducted at the neuromuscular centre of the Royal Children’s Hospital (RCH) in Melbourne, Australia. The primary endpoints of the trial related to the safety and tolerability of ATL1102. The efficacy of ATL1102 was also assessed in terms of its effects on disease processes and progression (e.g. the upper limb strength and function of the boys).
Given the exploratory nature of this first trial in boys with DMD, it was not powered to see a statistical difference on these disease progression endpoints, which would be expected in future longer-term clinical studies in a larger number of patients. However, highly encouraging positive trends across multiple parameters have been reported in this Phase II clinical trial. Further details on the trial are available on the Australia and New Zealand Clinical Trials Registry.