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.
Avi IN FACT has received FDA A-P-P-R-O-V-A-L of its IND application for the US dmd human trial
AVI BioPharma, an Oregon-based drug discovery company, expects to partner its lead compound AVI-4658 and may license or sell early-stage drug candidates from its RNA-based platform, according to new CEO David Boyle
The FDA declared this past week the company’s investigational new drug (IND) application for AVI-4658 was open. AVI said it plans to initiate a phase Ib/II trial for AVI-4658, a treatment for the genetic muscle-wasting disease Duchenne muscular dystrophy (DMD), in the US before year’s end. The drug candidate already has completed a phase I trial in Europe.