Bothell, WA-based AVI Biopharma (NASDAQ: [[ticker:AVII]]) is trying to develop the first treatment to correct an underlying molecular abnormality in boys with Duchenne Muscular Dystrophy, and after taking an early peek at a small clinical trial, the drug appears to be doing what it’s supposed to do in at least one patient.
The preliminary results were from an initial phase trial from the first 9 out of 18 boys who completed dosing at a single site in the U.K., with once-weekly intravenous infusions of the treatment, AVI-4658, over a period of 12 weeks. The trial initially enrolled patients to receive extremely low doses of the drug, and when it appeared safe, enrolled more patients in higher dose groups. AVI Biopharma and its academic collaborators didn’t see evidence of an effect at the lowest doses, but they did among three boys who got mid-range doses of 2 and 4 milligrams per kilogram, which showed the drug corrected the molecular abnormality it is designed to fix. One of the boys was able to produce five-fold higher amounts of a protein called dystrophin, a key structural component of muscle that is lacking in patients with Duchenne Muscular Dystrophy, based on a biopsy of bicep muscle.
AVI doesn’t have data in hand that connects the dots to show that the molecular changes induced by the drug will actually reverse the disability associated with muscular dystrophy. Still, “these results suggest that we are on the right path towards developing a drug that could play a role in the treatment of Duchenne Muscular Dystrophy,” said Francesco Muntoni, Professor of Pediatric Neurology and Head of the Dubowitz Neuromuscular Centre at the University College London, and the trial’s lead investigator.
“We weren’t really expecting to see dystrophin expressed, based on the animal models, until we got to higher doses of 4 and 10 milligrams per kilogram,” says AVI chief medical officer Steve Shrewsbury. “It’s a nice way for us to round out the year and start the new year.”
Biotech companies haven’t traditionally shown much interest in muscular dystrophy, although that’s changing as researchers gather understanding of the genetic underpinnings of the disease. The AVI treatment is part of an emerging class of compounds known as antisense oligonucleotides.
What’s special about the AVI treatment is that it’s designed
