"This study is a landmark in achieving the highest doses of any anti-sense drug, with no evidence of toxicity. The dystrophin expression is impressive, and given adequate time would likely lead to clinical benefit and the first molecular therapy for this most common form of muscular dystrophy".
"This study was very important for a number of reasons. It met the primary endpoint of dystrophin protein expression in patient muscle; the most to date by any method. It also achieved impressive doses of drug in patients, with repeated dosing nearly 10-times higher than most other studies to date.
Finally, the approach was impressively safe."
"The negative reaction of the stock market is the most surprising outcome of this phase II trial - Of course we all want all patients to jump out of wheelchairs and run a marathon the next day, but expectations must be balanced with realism, and this study achieved the primary outcome measures as expected.²
It is important to understand that in order to improve the lives of these boys, producing dystrophin in their muscle is an absolutely essential first step. AVI has achieved that. DMD is a disease in which the muscles waste away over a period of decades. To expect to see a functional benefit in 24 weeks is perhaps a bit extreme. This is cutting-edge research, which means we are in territory that has never been reached before. We really don¹t know how long it might take for a functional benefit to be achieved. I do think that the recent results of impressive dystrophin production with no toxicity are a reason to be extremely optimistic that AVI¹s exon-skipping approach may soon yield a functional benefit in these boys.
Eric P Hoffman, PhD
Chairman, Dept. Integrative Systems Biology George Washington University School of Medicine and Health Sciences Director, Research Center for Genetic Medicine Children's National Medical Center