About Acucela Inc.

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.

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. ACU-4429 for dry AMD is being co-developed by Acucela and Otsuka Pharmaceutical Co., Ltd. in North America, and the companies are also co-developing Rebamipide for dry eye and OPA-6566 for glaucoma in the United States.

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.

Acucela’s VCM technology may provide therapeutic benefit for additional retinal disorders, such as:

-          Diabetic Retinopathy, the most common diabetic eye disease and a leading cause of blindness in American adults.

-          Retinopathy of Prematurity (ROP), a potentially blinding eye disease that can occur in premature infants. In ROP, abnormal blood vessels grow and spread throughout the retina. There is no approved treatment for ROP.

-          Stargardt Disease, a recessive, hereditary retinal disease that results in progressive loss of rod and cone photoreceptors, leading to loss of central vision.

Acucela was founded in 2002 by eminent ophthalmologist Ryo Kubota, M.D., Ph.D., the discoverer of the gene that causes glaucoma.

ACU-4429 for dry AMD is being co-developed by Acucela and Otsuka Pharmaceutical Co., Ltd. in North America, and the companies are also co-developing Rebamipide for dry eye and OPA-6566 for glaucoma in the United States.

The Company is headquartered in Seattle, Washington. For more information, visit: www.acucela.com.