AVI Announces Top Line Results for Exon Skipping Trial

Another one of CureDuchenne's funded research projects achieves a significant milestone.

AVI Biopharma announced the top line results from their phase 2 trial for Eteplirsen, a drug which induces exon skipping in patients amenable to a skip of exon 51.

Chris Garabedian, CEO of AVI shared the top line results showing after 24 weeks, four patients on the 30 mg. dose had an average increase of dystrophin of 22.5 percent. The patients who received the 50 mg. dose for 12 weeks did not show an increase in dystrophin, suggesting duration of treatment is important.

"I always caution families, we still don't know if exon skipping will actually become a real treatment for our sons with Duchenne, or to what extent it will help them," said Debra Miller, president and founder of CureDuchenne. On a conference call on April 2 announcing these results, AVI's Chief Medical Officer Ed Kaye referred to "reasonable" dystrophin needed to function as 20 percent, so this study does indeed give us proof of concept and hope that we might have a treatment sometime in the future.

Dr. Eric Hoffman, at Children's National Medical Center in Washington D.C., spearheaded collaboration with CNMC along with the Foundation to Eradicate Duchenne and CureDuchenne several years ago. This collaboration gave critical funding to AVI at a very crucial time which allowed them to move forward with the preclinical work necessary to progress into this phase 2 human clinical trials. Dr. Jerry Mendell and Dr. Kevin Flanigan, at Nationwide Children's Hospital in Columbus, Ohio have played a pivotal role in conducting this trial over the last several months.

"We are very grateful to these investigators and organizations for their vision and dedication," said Miller.

What Comes Next?

There's a song named "Two Out Of Three Ain't Bad." We now have reasonable data that suggests the drug is safe, as there were no adverse event. And, we know the drug can produce dystrophin, and at a reasonable amount. Now, the big question is whether this quality/quantity of dystrophin will result in meaningful outcome measures and really help Duchenne patients live longer and healthier lives.

All patients, including the placebo groups that were on the phase 2, are now on the open label extension study at either the 30 mg. or 50 mg. dose. They will stay on this dose until they complete a total of 48 weeks dosing, at which time a biopsy will be taken and clinical outcome measures tested. In the meantime, AVI will be actively involved with the FDA, compiling a brief and planning the pivotal phase 3 study. Simultaneously, more animal studies will be conducted. Since the FDA most likely will not accept the presence of dystrophin alone as justification to move forward with Eteplirsen, this extension phase and the data they gather will be very important. The first patient to be dosed began in August of 2011, which would make this data available later this year.

As we await the clinical outcomes of the extension trial, AVI will be busy looking to the future, and CureDuchenne is equipped and ready to support their subsequent research on additional exons and next generation exon skipping compounds. CureDuchenne will always fund research projects that show more immediate potential to help our Duchenne sons, such as already approved FDA drugs to slow the progression. But CureDuchenne will never take its eye off the prize…to correct the actual defect that causes this terrible disease. CureDuchenne is committed to fund research, like exon skipping, that will replace the missing dystrophin protein, at the same time funding more immediate research that could get FDA approved or off-target drugs to keep our sons going in order to take advantage of these higher impact drugs.

CureDuchenne was also the first US organization to support Prosensa, another exon skipping biotech company. Prosensa was able to leverage our funds and received a commitment of up to $650 million to develop their drugs and is in the process of recruiting patients for its phase 3 pivotal trial.

Two out of three may not be bad, but it's not enough. So please rejoice in the possibility of an effective treatment, while at the same time show restraint with our expectations, since so many drugs do fail, even at late stage trials. One thing is for sure…we need all of you to support these research efforts.