AVI Biopharma
AVI BioPharma, Inc., a biopharmaceutical company, engages in the development and commercialization of therapeutic products based upon antisense and cancer immunotherapy technology. Development Programs The company’s RNA-based drug programs are being evaluated for genetic diseases (Duchenne muscular dystrophy), infectious diseases (Ebola and Marburg viruses), cardiovascular disease (restenosis) and other early discovery targets through its partner, Global Therapeutics, a Cook Medical Company. Duchenne Muscular Dystrophy (DMD) programs The company is developing a series of drugs for the potential treatment of DMD. It has two programs underway with drugs that skip exon 51 (AVI-4658) and exon 50 (AVI-5038). AVI-4658: The company’s splice switching oligomer (SSO) drug, AVI-4658, targets the frequent mutations resulting in DMD, forces the genetic machinery to skip over an adjacent piece (one or more codons) of RNA, thus restoring the ability of the cell to process genetic instructions, thereby, allowing for production of a new, albeit truncated, dystrophin protein. A phase 1 human clinical trial in boys with Duchenne Muscular Dystrophy (DMD) was completed. The company has opened a second, phase 1b systemic clinical trial with this product at the Institute of Child Health in London, the U.K. AVI-5038: The company is conducting preclinical evaluations with its SSO drug, AVI-5038, which is designed to skip exon 50 and potentially restore a range of associated mutations. This drug utilizes its phosphorodiamidate morpholino oligomer (PPMO) chemistry with the target to improve potency, tissue selectivity and bioavailability when compared to first generation phosphorodiamidate morpholino oligomers (PMO) drug candidates. AVI-5126 Prevention of Restenosis The company has partnered with Global Therapeutics, a Cook Medical Company, to evaluate its cardiovascular restenosis drug for use on a chromium cobalt drug-eluting stent (DES) for the treatment of cardiovascular restenosis. The application of AVI—5126 to the Global Therapeutics Silencer System is designed to offer drug release and reversion to a bare metal stent within hours of implantation. Infectious Disease Programs The company’s infectious disease program is focusing on single-stranded RNA viruses using its proprietary Translation Suppressing Oligomer (TSO) technology with its PMOplus chemistry backbone to target the often fatal diseases caused by Dengue, Ebola, Marburg and JunÃn viruses, as well as other items included on the Department of Homeland Security’s list of bioterrorism agents, including anthrax and ricin. AVI-6002 Ebola virus program: The company has demonstrated significant survival in mice, guinea pigs and monkeys when they are treated with AVI-6002 post infection with Ebola virus. It has received approval to move into the initial clinical study. AVI-6003 Marburg virus program: The company has demonstrated significant survival in mice, guinea pigs and monkeys when they are treated with AVI-6003 post-infection with Marburg virus. It has received approval to move into the initial clinical study. Strategic Alliances The company has strategic alliances with the following companies and institutions: Cook Group for vascular diseases using PPMO-based RNA therapeutic agents; U.S. Department of Defense for Ebola, Marburg and Dengue viruses; Ricin and Anthrax toxins; USAMRIID for BSL—4 threats, including Marburg and Ebola viruses; Eleos, Inc. to treat cancer with p53-related drugs; and Institute of Child Health at the University College, London for clinical trials in muscular dystrophy. Significant Events The company and Action Duchenne announced a collaboration to support the acceleration of research and development for AVI’s exon skipping candidate drugs for the treatment of DMD. Competition The company’s competitors include Prosensa, Santaris, Merck, and ISIS Pharmaceuticals. History AVI BioPharma, Inc. was founded in 1980.
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