Update to the Duchenne Community from Percheron Therapeutics on Progress with Avicursen

Dear Members of the Duchenne Community,

Today, Percheron Therapeutics, a public company registered in Australia and focused on developing treatmentsfor rare diseases with unmet medical need, announced topline data from an ongoing phase IIb clinical trial ofavicursen versus placebo in Duchenne Muscular Dystrophy (DMD), and we wanted to share some of the keypoints with you directly. Avicursen is also known as ATL1102, and you may see it referred to by that nameelsewhere.BackgroundWe have been developing avicursen for several years. 

Our current phase IIb study was designed to investigatethe drug in non-ambulant boys ages 10-18 with DMD and allows for co-administration with steroid therapies.The trial is a randomized, double-blind study testing two doses of avicursen, 25 mg and 50 mg, vs placebo forsix months, with a follow-on open label treatment period of six months so that some in the study had up totwelve months of treatment with avicursen.