Advanced Vision Therapies, Inc. (AVT) is focused on the treatment of sight-threatening eye diseases, such as age-related macular degeneration and finding a cure that works. The market, which is of a multi-billion dollar magnitude, and represents significant unmet medical needs, has been underserved by large pharma. Recent clinical trial data suggest that these diseases will be treatable, but with therapeutics that must be repeatedly injected directly into the eyes. AVT recognized that an improved delivery system is required to enable the broad application of ocular therapeutics. The company has identified two novel therapeutics and developed a proprietary delivery system, which, with a single administration, will provide sustained, possibly life-long therapy. The AVT business model is to leverage its expertise and that of its collaborators to rapidly advance its first product candidate into the clinic and then form a partnership with large pharma for late stage clinical trials, product launch, and marketing. AVT was founded by three scientific directors, formerly with Genetic Therapy, Inc., a Novartis Company, to commercialize their pivotal research in localized, ocular-based gene therapy. AVT is the only gene-based medicine company that is devoted to eye applications. The founders bring with them the bulk of the technology that they developed at Novartis in a comprehensive and favorable licensing package. Importantly, AVT believes it has overcome the barriers that have encumbered previous gene therapy trials. A critical success factor is in targeting diseases of the eye where there are clear clinical pathways, predictable timelines, excellent pre-clinical models, and a high likelihood of therapeutic success. AVT is targeting large, underserved markets where its products are anticipated to have major competitive advantages. AVT's initial focus is on the development of a gene-based medicine product for wet age-related macular degeneration. The company's first product, AVT-1, will consist of an AVT vector engineered to deliver Kininostatin, a potent and broadly efficacious anti-angiogenic factor. Following a single intra-ocular administration, the vector will genetically modify a small number of cells in the back of the eye. The modified cells will then secrete Kininostatin, which will diffuse throughout the eye and block the growth of the new blood vessels that cause the disease. Importantly, the delivery of Kininostatin will be continuous and sustained for years. Moreover, Kininostatin blocks defective new blood vessel growth irrespective of the underlying angiogenic stimuli and has a significant advantage over competing products, which target only a single angiogenic stimulus, VEGF. Once having demonstrated efficacy in wet age-related macular degeneration, AVT will move into diabetic proliferative retinopathy with this same product. The company's second drug candidate, AVT-2, is designed to treat retinitis pigmentosa (RP) and dry age-related macular degeneration The product will consist of the AVT vector engineered to deliver RdCVF, a newly discovered, first-in-class, retinal survival factor that functions to protect and prolong the life of photoreceptors. This product has the potential to open the door to treatments for diseases that have previously been unapproachable. Finally, AVT is seeking to moderate investor risk by investigating an additional therapeutic gene, T2-TrpRS, for use in wet age-related macular degeneration. T2-TrpRS has potent anti-angiogenic activity in ocular animal models. Gene Transfer Technologies: AVT's leadership in ocular gene transfer technologies is evidenced in key areas. First, AVT has developed a highly advanced lentiviral gene transfer system with outstanding efficacy and safety profiles. The vector is uniquely suited for use in the eye and has yielded impressive data in animal modeling studies. Second, AVT has developed technology for scale-up and manufacturing for lentiviral vectors and believes it will be first to generate stable producer cell lines for commercial production. Third, AVT has an exceptionally broad intellectual property portfolio that provides a strong proprietary foundation for the business. The IP portfolio was obtained from Novartis and Temple University and consists of 25 patent families that cover the vector platform, therapeutic genes, and scale-up and manufacturing. Fourth, and most critical, AVT has the necessary expertise to fulfill the promise of gene therapy for the eyes and bring new, important drugs to market. These technologies also enable a broad pipeline, including therapies for dry age-related macular degeneration, glaucoma, and retinopathies. Markets for Macular Degeneration Therapies AVT's first product is focused on the treatment of wet age-related macular degeneration. Wet age-related macular degeneration is the leading cause of blindness in the US, Europe, and Japan. This progressive eye disease is caused by the growth of defective new blood vessels behind the retina that leak and bleed leading to a rapid loss of vision. Wet age-related macular degeneration accounts for approximately 200,000 new cases annually with a prevalence of 1.2 million cases in the United States alone. These numbers are expected to triple over the next 20 years due to the aging of baby boomers. An immediate spin-off application for the first product is the treatment of diabetic proliferative retinopathy, a second large market eye disease caused by aberrant blood vessel growth in the eye. AVT's second product is focused on the treatment of retinitis pigmentosa and dry age-related macular degeneration. Dry age-related macular degeneration affects 25% of people over 65, while RP affects 200,000 people in the US. The initial clinical application will likely be RP since this disease can inexorably lead to blindness and there is a strong compassionate need for treatment.
