AVI BioPharma to Present Data for RNA-Based Duchenne Muscular Dystrophy Drug Candidate
Product News Apr 13, 2011
AVI BioPharma, Inc. has announced data presentations for eteplirsen, the Company's investigational exon skipping therapy for the treatment of Duchenne muscular dystrophy, at the 63rd Annual Meeting of the American Academy of Neurology taking place April 9-16, 2011 in Honolulu, Hawaii.
Dr. Francesco Muntoni, Professor of Pediatric Neurology and Head of the Dubowitz Neuromuscular Centre at the UCL Institute of Child Health, London, England, will present an oral abstract at 3:15 p.m. HDT on Wednesday, April 13.
The presentation is titled "Safety, Pharmacokinetic and Exploratory Efficacy of AVI-4658 (eteplirsen) a Phosphorodiamidate Morpholino Oligomer (PMO) To Skip Exon 51 in Duchenne Muscular Dystrophy Patients." The presentation will feature complete data from a Phase 1b/2 study of eteplirsen in DMD patients.
Peter Sazani, Ph.D., Executive Director, Preclinical Development at AVI, will present during poster presentation session P02, Muscle Disease: Dystrophinopathies, at 7:30 a.m. HDT today, Tuesday, April 12. The poster, P02.066, is titled "Clinical and Preclinical Comparison of the Pharmacokinetic Parameters of AVI-4658 (eteplirsen), a Phosphorodiamidate Morpholino Oligomer for the Treatment of Duchenne Muscular Dystrophy."
Eteplirsen is AVI's lead systemically administered drug candidate for the treatment of a substantial subgroup of patients with DMD. Data from clinical studies of eteplirsen in DMD patients has demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.
AVI has announced plans to initiate a Phase 2 study of eteplirsen in June, and is currently conducting NDA-enabling activities to support the initiation of a pivotal study in the second half of 2012.