Acucela, a clinical-stage biotechnology company focused on developing new treatments for blinding eye diseases, announced today that a scientific paper co-authored by Ryo Kubota, M.D., Ph.D., chairman, president and chief executive officer of Acucela, has been published in Experimental Eye Research (Exp. Eye. Res. 2010, 91;153-61). The paper, "Visual Cycle Modulation in Neurovascular Retinopathy" was also co-authored by scientists from Children's Hospital and Harvard Medical School in Boston and demonstrates for the first time the impact of treatment with a visual cycle modulator (VCM) on retinopathy in an immature eye.
“These findings are key for illustrating the effect visual cycle modulation can have on the eye and blinding eye diseases”
"These findings are key for illustrating the effect visual cycle modulation can have on the eye and blinding eye diseases," stated Dr. Kubota. "Visual cycle modulators - like Acucela's own ACU-4429 - are designed to prevent or inhibit the generation of toxic by-products of the eye's visual cycle that can lead to degenerative eye conditions like dry age related macular degeneration, or dry AMD. In this paper we were able to demonstrate the possibility of reducing the oxygen demand in the retina by modulating the visual cycle to treat neurovascular retinopathy, like retinopathy of prematurity or the even more common condition like diabetic retinopathy. By showing that VCM treatments can regulate rod function in a preclinical model, my co-authors and I have demonstrated the early promise and potential of this treatment approach for patients suffering from more blinding eye diseases beyond AMD."
The Experimental Eye Research paper describes a preclinical study in in vivo models of oxygen-induced retinopathy (OIR), which is a preclinical model of the pediatric disease retinopathy of prematurity (ROP). The study results provide evidence that the eye's rod cells may be a possible therapeutic target in neurovascular diseases such as retinopathy of prematurity. Recent findings in OIR preclinical models imply a causal role for the rods in the ROP disease process. The study outlined in the publication was conducted to experimentally manipulate rod function and to establish this causal role conclusively. The authors concluded that visual cycle modulation resulted in altered rod function and in enhanced recovery of retinal vessels in OIR. In summary, these findings are the first demonstration of a treatment effect with a VCM on retinopathy in an immature eye and demonstrate a causal role for the rod photoreceptors in the development of retinal vasculopathy.
Acucela's proprietary visual cycle modulator, ACU-4429, is an investigational oral treatment for dry age-related macular degeneration (dry AMD) that is currently in Phase 2 clinical development as part of the ENVISION Clarity Trial. ACU-4429 is one of the only treatments in development that works to regulate the eye's visual cycle for processing light. By regulating this cycle, ACU-4429 has demonstrated the ability to decrease the levels of toxic by-products in the eye and thereby potentially slow the advancement of dry AMD. AMD is a leading cause of vision loss in people over the age of 50. Dry AMD accounts for 90% of the AMD patients, and there are no therapies currently approved to treat this condition.
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