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AVI BioPharma Opens Investigational New Drug (IND) Application for AVI-4658 in Duchenne Muscular Dystrophy

AVI BioPharma, Inc. /quotes/comstock/15*!avii/quotes/nls/avii (AVII 1.45, -0.09, -5.84%) , a developer of RNA-based therapeutics, today announced that following review by the U.S. Food and Drug Administration the Company's Investigational New Drug (IND) application for AVI-4658 is open. AVI-4658 is AVI's lead drug candidate being developed as a systemically administered treatment for a substantial subgroup of patients with Duchenne muscular dystrophy (DMD), a genetic muscle wasting disease caused by failure to produce dystrophin. AVI plans to initiate a Phase 1b/2 clinical trial in DMD in the U.S. this year.

The intended site for the planned U.S. based study is Nationwide Children's Hospital in Columbus, Ohio, with Jerry R. Mendell, M.D. as the Principal Investigator. The clinical program design is being reviewed in consultation with Dr. Mendell, co-investigator Kevin Flanigan, M.D., and other DMD key opinion leaders. It is anticipated that future clinical evaluation will explore increasing doses of AVI-4658 considering the generally well tolerated nature of the drug candidate as exhibited in the clinical and preclinical studies to date, and the substantial, but variable, increases in dystrophin measurements demonstrated in patients with DMD in the U.K. based Phase 1b/2 clinical trial.

http://www.marketwatch.com/story/avi-biopharma-opens-investigationa...

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Permalink Reply by Conrad Klahn on July 12, 2010 at 8:19pm

I agree - good news. I bought another 250 shares of AVII today for my son's account. I don't expect any benefit from the stock, and may even lose money on it, but hopefully their success will continue.

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Permalink Reply by arnold on July 13, 2010 at 11:39pm

http://online.barrons.com/article/SB5000142405297020329600457529870... study results showed that the drug, when given intravenously, stimulated the production of dystrophin, a key muscle protein lacking in Duchenne sufferers, in many of the 19 boys in the trial—with one boy showing an increase in dystrophin levels to more than 50% of ornormal levels, the first time such high levels of dystrophin have been produced. The U.K. results should pave the way for a U.S. clinical-drug trial later this year for the drug, which is targeted at a subset of Duchenne sufferers with a particular genetic defect.

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Permalink Reply by arnold on July 14, 2010 at 10:10am

University of London Files Patent Application for Exon Skipping......
Royal Holloway, University of London has filed a US patent application for exon skipping to treat duchenne muscular dystrophy, specifically for treating DMD in boys with mutations at exons 44, 45, 46, and 53. Though its invention covers all antisense oligonucleotides, it mentions several times that it favors PMOs:

"]However, it should be noted that, relative to 2'OMePS AOs, PMOs have been shown to produce more consistent and sustained exon skipping in the mdx mouse model of DMD [12-14; A. Malerba et al, m@#$%cript submitted], in human muscle explants [15], and in dystrophic canine cells in vitro [16]. Most importantly, PMOs have excellent safety profiles from clinical and pre-clinical data [17]."

The University of London patent application was filed in September 2009, and published July 1, 2010. Kole's patent application for exon skipping for treatment of DMD was filed later, in October 2009, but published in May 2010.
http://appft.uspto.gov/netacgi/nph-Parser?Sect1=PTO2&Sect2=HITO...
http://appft.uspto.gov/netacgi/nph-Parser?Sect1=PTO2&Sect2=HITO...