Overview
Acucela (pronounced ak-you-cell-la) is developing orally-delivered visual cycle modulation (VCM) therapies that selectively target cells within the retina to protect visual acuity. These compounds have the potential to treat blinding eye diseases, including the dry form of age-related macular degeneration (AMD), that affect tens of millions of people around the world.
Modulating the visual cycle has been shown to protect the retina from light damage, to improve retinal vasculature and to reduce the accumulation of retinal-related toxic by-products such as A2E, which is implicated in AMD.
In preclinical and clinical studies, Acucela’s novel proprietary VCM compounds demonstrated the ability to slow the visual cycle in multiple blinding eye diseases including dry AMD, retinopathy of prematurity, Stargardt disease and diabetic retinopathy.
AMD is a leading cause of blindness in the United States and is estimated to affect more than 29 million people worldwide – a number that is expected to double in the next 20 years due to the aging population of the world. About 90 percent of AMD patients suffer from the dry form of the disease.
Acucela launched the ENVISION (Evaluating a Novel VISION treatment for AMD) Clarity Trial, part of Acucela’s clinical program evaluating the investigational oral treatment ACU-4429, in patients with dry age-related macular degeneration (dry AMD) in January 2010. The Clarity Trial, a Phase 2 clinical trial, builds upon the promising preclinical findings and data from Acucela’s Phase 1 clinical studies. These Phase 1 data have been presented at key scientific meetings and provide support and clinical rationale for Acucela’s ACU-4429 clinical development program in patients with dry AMD. In March 2010, Acucela received Fast Track designation from the U.S. Food and Drug Administration (FDA) for ACU-4429 for the treatment of dry AMD.
Acucela's VCM compounds have a good safety profile: they are non-retinoid and can be administered orally, unlike current therapeutics for many eye diseases, which must be injected into the eye.
Although our primary focus is on developing safe and effective treatments for AMD, we also have programs underway to create next-generation therapeutics for other potentially devastating ophthalmic diseases, including dry eye syndrome, Stargardt disease, retinopathy of prematurity, and diabetic retinopathy. In addition, our OcuScreen™ technologies are very useful in helping ophthalmic researchers screen for drug leads using retinal neurons.
Acucela was founded in 2002 by eminent ophthalmologist Ryo Kubota, M.D., Ph.D., the discoverer of the gene that causes glaucoma. The company is based in Bothell, Washington.