ATL1102 phase II trial results published in Neurology


Antisense Therapeutics has reported the publication of previously generated phase IIa clinical trial data on ATL1102 in the medical journal Neurology.

The article titled “CD49d antisense drug ATL1102 reduces disease activity in patients with relapsing-remitting MS”, is currently available online and will be included in the print edition Volume 83, November 11, 2014.


The article highlights the successful outcomes of the Phase IIa clinical trial of ATL1102 in Multiple Sclerosis (MS) patients where in the randomised, double-blind, placebo-controlled study in 77 patients with relapsing-remitting multiple sclerosis (RRMS), ATL1102 met its primary end point after only two months of dosing, showing a significant reduction, by 54.4% (p=0.01) in the cumulative number of new active brain lesions in patients taking ATL1102 compared to placebo.


The efficacy outcomes from this study were viewed to be as good as, or superior to, those achieved with MS drug Tysabri at a similar stage in its clinical development. Tysabri (natalizumab) is a monoclonal antibody drug targeting the VLA-4 receptor (same target as ATL1102). In 2013, Tysabri generated sales in excess of US$1.6 billion. It is regarded as the current efficacy benchmark for the treatment of RRMS. ANP anticipates that ATL1102 could be as potent as Tysabri but potentially safer, cheaper to manufacture, and more conveniently dosed.


Principal investigator of the ATL1102 phase IIa study and lead author of the Neurology publication, Volker Limmroth (professor of Neurology, chairman, Department of Neurology and Palliative Care Medicine Cologne City Hospitals, University of Cologne) says:


“There are a number of unresolved issues with current MS drugs including the occurrence of neutralising antibodies to the antibody, protein and peptide MS drugs as well as long-term safety concerns with the more recently approved drugs. There is a clear need for more effective and safe drugs for the significant population of MS patients who have relapses and non-stable disease.


“The ATL1102 phase IIa trial provides evidence for the first time that antisense oligonucleotides may be used as a therapeutic approach in neuroimmunologic disorders such as MS. ATL1102 was shown to be highly effective in reducing brain lesions in RRMS patients with a quick onset of action and a clinical safety profile that strongly supports its ongoing development as a treatment for this disease.”