AVI BioPharma Demonstrates Effectiveness of Antisense Drug In Treating Symptoms of Polycystic Kidney Disease
NeuGene Drug Significantly Reduced Cystic Renal Enlargement and Inhibited the Decline in Renal Function
PORTLAND, Ore. - May 10, 2002 - AVI BioPharma, Inc. (Nasdaq: AVII, AVIIW, AVIIZ) today announced the results of a preclinical study that measured the effectiveness of AVI's antisense drug, NeuGene ®, in treating specific manifestations of polycystic kidney disease (PKD). The results of the study, published in Kidney International, Vol. 61, Symposium 1 (2002), showed that NeuGene compounds slowed kidney cyst enlargement and arrested the decline in kidney function in mice. The study marks another important step toward developing treatments for the disease, which affects more than 600,000 Americans and an estimated 12.5 million people worldwide.
"We are encouraged by the results of our study," said Denis R. Burger, Ph.D., AVI's chief executive officer. "Currently, dialysis and transplantation are the only known treatments for polycystic kidney disease, so the chance to arrest the development of this disease is a great leap forward."
About the Study
Previous studies have shown that the c-myc gene is overexpressed in
human PKD kidneys, suggesting that the gene plays an important role in
the progression of the disease. AVI's study was designed to test this
hypothesis using a mouse model that mimics the recessive form of PKD.
Study results indicate that the cystic mice treated with the antisense
oligomer that inhibits production of the c-myc protein showed
significantly reduced enlargement of cysts in the kidneys and an
inhibited decline in kidney function.
About Polycystic Kidney Disease
According to the Polycystic Kidney Disease Foundation, PKD is currently
the most common inherited disease, with more people suffering from PKD
than cystic fibrosis, muscular dystrophy, hemophilia, Down syndrome and
sickle cell anemia, combined. The disease is characterized by the
formation of multiple, fluid-filled cysts in the kidneys, which cause
the kidneys to grow abnormally large and to have progressively less
functioning tissue. In more than 60 percent of the cases, individuals
with PKD develop kidney failure or end stage renal disease (ESRD).
The inheritance of PKD can take either dominant or recessive forms. Roughly 90 percent of all patients with PKD suffer from the dominant form, which usually begins to manifest symptoms in adulthood. Individuals with the recessive form of PKD are affected as newborns or young children.
"These results provide substantial scientific support for our PKD clinical programs," said David H. Mason, Jr., M.D., AVI's senior vice president of clinical development and regulatory affairs. "Our ongoing Phase 1b trial of AVI-4126 in patients with PKD is progressing nicely and is two-thirds complete. We expect to have full results of this study later this year."
About AVI BioPharma
AVI BioPharma develops therapeutic products for the treatment of life-threatening diseases using two technology platforms: NeuGene antisense drugs and cancer immunotherapy. Its lead cancer agent, AVICINE®, a therapeutic cancer vaccine, has completed three Phase II trials in colorectal and pancreatic cancer and is initiating a Phase III pivotal trial in pancreatic cancer, with a supporting study in colorectal cancer. The first application of its NeuGene compounds, Resten-NG™, is designed to treat cancer, cardiovascular restenosis and other cell proliferation disorders by inhibiting the production of a cellular transcription factor, the oncogene c-myc. It is currently in Phase II trials for restenosis and in Phase I/II trials for cancer and polycystic kidney disease. AVI has recently completed a Phase I NeuGene antisense study that successfully down-regulated the liver enzyme Cytochrome P450 and modified drug metabolism. More information about AVI is available on the Company's Web site at http://www.avibio.com/.
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