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Dr. Ryo Kubota, M.D., Ph.D., Acucela's founder, decided to leave his successful ophthalmology practice in favor of scientific research because he was frustrated with the lack of treatments available for his patients and came up with OcuScreenTM, Acucela's proprietary pharmaceutical compound screening systems using long-lived, fully-differentiated primary retinal neurons. Acucela later developed the visual cycle modulator (VCM) program. Drug Discovery & Development Division Acucela's VCM program is in clinical-stage development, targeting the dry form of age-related macular degeneration (AMD). A variety of in vivo models have established pre-clinical proof-of-concept for the VCM approach to AMD. Visual cycle modulators are proposed to work, at least in part, by reducing the buildup in the eye of a toxic by-product of the visual cycle, known as A2E. A2E has been shown to be toxic to retinal pigmented epithelium cells in vitro, and Acucela has shown that it is also toxic to photoreceptors. Furthermore, Acucela was able to demonstrate neuroprotective effects using mice models. Recently, VCM was shown to have potential therapeutic benefit in neovascular disease using a retinopathy of prematurity model. One of VCM compounds, which is orally available, is on a Phase I clinical trial for dry AMD. Using breakthrough technologies, Acucela's OcuScreenTM Division has two service contracts with customers and is in negotiations with several others. Three papers have been published on OcuScreenTM assays (see the "news" section for more details).
Other eye drug discovery technologies are very limited. The ability to screen for drug leads using primary retinal neurons has been severely limited since neurons usually die out very quickly after being cultured. This prevents adequate analysis in compound screening by the pharmaceutical industry, limiting their ability to commercialize new drugs for these eye diseases. Acucela's technologies address these problems, resulting in useful proprietary pharmaceutical screening systems. |
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