AVI BioPharma, Inc. Presents Update on AVI-4658 at TREAT-NMD / NIH International Conference
- Category: DNA RNA and Cells
- Published on Thursday, 19 November 2009 02:00
- Hits: 1245
AVI BioPharma announced presentations on the company's exon skipping therapy for Duchenne muscular dystrophy at the TREAT-NMD / NIH International Conference taking place Nov. 17-19 in Brussels, Belgium
BOTHELL, WA, USA | November 19, 2009 | AVI BioPharma, Inc. (NASDAQ:AVII - News), a developer of RNA-based drugs, today announced presentations on the company's exon skipping therapy for Duchenne muscular dystrophy at the TREAT-NMD / NIH International Conference taking place Nov. 17-19 in Brussels, Belgium.
At the conference, data from the company's clinical and preclinical programs were presented. An update on the company's ongoing systemic Phase 1b/2 trial of AVI-4658 was provided by Professor Francesco Muntoni, of the MDEX consortium in the UK, during an oral presentation and also summarized in a poster presentation. The most recent data from the ongoing Phase 1b/2 trial at two MDEX sites in the UK demonstrate that AVI-4658 was well tolerated by DMD patients in a dose escalation study that is now up to the sixth and final cohort (20 mg/kg). The maximum cumulative dose administered to date is 3132 mg and the maximum single dose is 900 mg with no adverse safety signals, in either case.
A series of posters were also presented by Peter Sazani, Ph.D., Executive Director of Preclinical Development. Data presented included preclinical findings demonstrating that treatment with AVI-4658 was tolerated at dosages up to 960 mg/kg in mice and up to 320/mg/kg in primates -- both maximum feasible doses.
The aim of the TREAT-NMD Conference is to share progress in the area of translational medicine in inherited neuromuscular diseases and to set the future collaborative agenda.
The open label dose-finding clinical trial of AVI-4658 is evaluating the systemic delivery of AVI-4658 once per week for 12-weeks by intravenous infusion. It is being conducted in London, UK at the UCL Institute of Child Health / Great Ormond Street Hospital NHS Trust facilities by members of the MDEX Consortium led by Professor Muntoni and by Professor Kate Bushby at the Royal Victoria Infirmary, Newcastle-Upon-Tyne, UK, which is the coordinating center for the European Treat Neuromuscular Diseases (Treat-NMD) initiative. The clinical costs for the trial are provided, in part, by the UK Medical Research Council.
TREAT-NMD is a Network of Excellence facilitating collaborative research in neuromuscular disease that aims to create the infrastructure to ensure that the most promising new therapies reach patients as quickly as possible. Since the network was launched in January 2007 it has built up the tools that industry and researchers need to bring promising new therapies more quickly from the lab to the clinic. One of the key TREAT-NMD infrastructures built up in the last two years is a global patient registry for DMD and SMA comprising more than 30 national patient registries worldwide. The DMD registries now hold more than 10,000 individual patient entries with standardized items and patient consent, facilitating and accelerating clinical research and trials while giving patients improved access to relevant information on standards of diagnosis and care. These registries have been set up in collaboration with clinicians and patient organizations across the world and contain the key information needed to establish whether a particular patient might be eligible for a trial, together with the means of contacting them. Registries for other conditions are also in preparation. For more information, please visit www.treat-nmd.eu.
About the MDEX Consortium
The MDEX consortium led by Professor Muntoni, is a mutlidisciplinary enterprise to promote translational research into muscular dystrophies, and is formed by the clinical groups of Professor Francesco Muntoni (Imperial College London and UCL Institute of Child Health) and Professor Kate Bushby and Professor Volker Straub (Newcastle University), and scientists from Imperial College London (Professor Dominic Wells), UCL Institute of Child Health (Dr Jennifer Morgan), Royal Holloway University of London (Professor George Dickson and Dr Ian Graham), Oxford University (Dr Matthew Wood) and University of Western Australia (Prof Steve Wilton). In addition, the charities Muscular Dystrophy Campaign (MDC), Action Duchenne and Duchenne Family Support Group also participate in the Consortium. For more information visit www.mdex.org.uk.
About AVI BioPharma
AVI BioPharma is focused on the discovery and development of RNA-based drugs utilizing proprietary derivatives of its antisense chemistry (morpholino-modified phosphorodiamidate oligomers or PMOs) that can be applied to a wide range of diseases and genetic disorders through several distinct mechanisms of action. Unlike other RNA therapeutic approaches, AVI's antisense technology has been used to directly target both messenger RNA (mRNA) and its precursor (pre-mRNA), allowing for both up- and down-regulation of targeted genes and proteins. AVI's RNA-based drug programs are being evaluated for the treatment of Duchenne muscular dystrophy, including an ongoing systemic Phase 1b/2 clinical trial of exon skipping AVI-4658. AVI's antiviral programs have demonstrated promising outcomes in Ebola Zaire and Marburg Musoke virus infections and may prove applicable to other viral targets such as Junin, influenza, HCV or Dengue viruses. For more information, visit www.avibio.com.
SOURCE: AVI BioPharma, Inc.