AVI BioPharma Data Safety Monitoring Board Reports No Safety Concerns Identified in Phase IIb DMD Clinical Trial, Recommends Continuation of Trial
BOTHELL, WA -- (MARKET WIRE) -- 01/04/12 -- AVI BioPharma, Inc. (AVII), a developer of RNA-based therapeutics, announced today that the independent Data Safety Monitoring Board (DSMB) providing oversight for AVI's Phase IIb study of eteplirsen in Duchenne Muscular Dystrophy (DMD) patients has reviewed the 12-week biopsy data from the highest dose cohort (50 mg/kg). Following its assessment of this biopsy data, along with other laboratory results and safety outcomes, the DSMB did not identify any safety concerns and determined it was safe to proceed with the trial as planned.
"This DSMB review is an important milestone for our Phase IIb study of eteplirsen in DMD patients," said Chris Garabedian, President and CEO of AVI. "The decision to proceed with the highest dose of eteplirsen beyond 12 weeks continues to support our belief in eteplirsen's favorable safety profile."
Eteplirsen is AVI's lead drug candidate that is systemically delivered for the treatment of a substantial subgroup of patients with DMD. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.
Eteplirsen uses AVI's novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene. By skipping exon 51, eteplirsen may restore the gene's ability to make a shorter, but still functional, form of dystrophin from mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to improve, stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD.
AVI is also developing other PMO-based exon-skipping drug candidates intended to treat additional patients with DMD.
About AVI BioPharma
AVI BioPharma is focused on the discovery and development of novel RNA-based therapeutics for rare and infectious diseases, as well as other select disease targets. Applying pioneering technologies developed and optimized by AVI, the Company is able to target a broad range of diseases and disorders through distinct RNA-based mechanisms of action. Unlike other RNA-based approaches, AVI's technologies can be used to directly target both messenger RNA (mRNA) and precursor messenger RNA (pre-mRNA) to either down-regulate (inhibit) or up-regulate (promote) the expression of targeted genes or proteins. By leveraging its highly differentiated RNA-based technology platform, AVI has built a pipeline of potentially transformative therapeutic agents, including eteplirsen, which is in clinical development for the treatment of Duchenne muscular dystrophy, and multiple drug candidates that are in clinical development for the treatment of infectious disease. For more information, please visit www.avibio.com.
Forward-Looking Statements and Information
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These forward-looking statements involve risks and uncertainties, many of which are beyond AVI's control. Known risk factors include, among others: clinical trials may not demonstrate safety and efficacy of any of AVI's drug candidates and/or AVI's antisense-based technology platform; and any of AVI's drug candidates may fail in development, may not receive required regulatory approvals, or be delayed to a point where they do not become commercially viable.
Any of the foregoing risks could materially and adversely affect AVI's business, results of operations and the trading price of AVI's common stock. For a detailed description of risks and uncertainties AVI faces, you are encouraged to review the official corporate documents filed with the Securities and Exchange Commission. AVI does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.
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Source: AVI BioPharma, Inc.