AVI Biopharma doled out an interesting little morsel of news on its muscular dystrophy drug right before Christmas that showed encouraging results in three boys. This year, the Bothell, WA-based biotech company is eagerly awaiting more meaningful follow-up data that could show it is on track with what could be the first treatment to fix the underlying molecular abnormality in boys with Duchenne Muscular Dystrophy.
I got the update on AVI’s game plan for 2010 when I met with a few senior executives, including chief financial officer David Boyle and chief medical officer Steve Shrewsbury, a couple of weeks ago in San Francisco. We talked a little bit about the company’s RNA-based treatments for hemorrhagic viruses like Ebola, as well as a new government-funded flu program. But there’s no doubt the main event this year will be what happens with the company’s treatment for Duchenne Muscular Dystrophy.
AVI Biopharma (NASDAQ: [[ticker:AVII]]) wants to be the first company to make a drug that silences a specific strand of RNA, and enables the body to produce a protein called dystrophin. This is a protein that’s essential for enabling muscles to rebuild themselves, and is lacking in boys with a birth defect known as Duchenne Muscular Dystrophy. This is a crippling disorder that affects about one out of every 3,500 boys born worldwide.
“Duchenne Muscular Dystrophy is certainly our lead program, and we think it has significant value,” Boyle says.
The AVI approach made some medical news in The Lancet last year, when the company showed that its RNA-based treatment was able to restore production of dystrophin proteins when injected directly into a foot muscle. That prompted the next step, in which AVI developed a version of the drug that could be delivered intravenously, and circulate throughout the body, where it could presumably have a much broader impact on muscles.
The first peek at data from this trial of the intravenous version came out in December. This initial slice of data was from the first nine patients who were enrolled in the four lowest dose groups. Researchers found that in three patients who got doses on the high end of that range, the molecular abnormalities dissipated. One boy was able to produce five-fold higher amounts of dystrophin. “These results suggest that we are on the right path,” said Francesco Muntoni, the trial’s lead investigator at University College London, in a statement.
I wanted to know about the next steps to watch for in the clinical development of this drug, called AVI-4658. It turns out that AVI has two major data releases planned for 2010, and both will be closely watched by parents, researchers, and shareholders.
The first release will be before the end of June. The trial, for those unfamiliar
