Macular Degeneration

Age-related macular degeneration afflicts over 29 million people worldwide and is segmented into "dry" and "wet" forms of the disease. The dry form is much more common than the wet: about 90% of AMD patients have the dry form. Unfortunately, there is no drug treatment available for dry form macular degeneration. One main reason for the lack of drug therapies is that there are few relevant disease models, which has proven to be a major obstacle for pharmaceutical development. Acucela has developed relevant disease models to discover drugs that combat dry form macular degeneration.

Phase I clinical trial of a non-retinoid visual cycle modulator for dry AMD is underway.

Stargardt Disease

Stargardt disease is a recessive, hereditary retinal disease, which consists of progressive loss of cone photoreceptors, leading to loss of central vision. It most commonly affects people under the age of twenty, starting with symptoms of difficulty in reading and seeing in dim lighting. Eventually, all patients with Stargardt disease are expected to have visual acuity between 20/200 and 20/400, not correctable by corrective lenses.

Recent pharmacology studies demonstrate that many of Acucela's compounds are efficacious in animal models, supporting its use in Stargardt disease.

Retinopathy of Prematurity (ROP)

ROP is a potentially blinding eye disease that can occur in premature infants and involves abnormal blood vessels growing and spreading throughout the retina, the tissue that lines the back of the eye. These abnormal blood vessels may leak, scarring the retina and pulling it out of position, which may cause detachment of the retina. Retinal detachment is the primary cause of vision impairment and blindness in ROP. There are approximately 15,000 annual cases of ROP in the U.S. of which approximately 10 percent are considered severe. There is currently no approved treatment for ROP.

Recent preclinical data demonstrate an improvement in retinal abnormalities following treatment with Acucela's VCM compound.

Diabetic Retinopathy

Diabetic retinopathy is the most common diabetic eye disease and a leading cause of blindness in American adults. It is caused by changes in the blood vessels of the retina.

In some people with diabetic retinopathy, blood vessels may swell and leak fluid. In other people, abnormal new blood vessels grow on the surface of the retina. The retina is the light-sensitive tissue at the back of the eye. A healthy retina is necessary for good vision.

If you have diabetic retinopathy, at first you may not notice changes to your vision. But over time, diabetic retinopathy can get worse and cause vision loss. Diabetic retinopathy usually affects both eyes.

Retinitis Pigmentosa

Retinitis pigmentosa is an inherited degenerative eye disease, usually diagnosed in young adults who experience a loss of their night and peripheral vision. Over time, people with retinitis pigmentosa steadily lose their vision as their photoreceptors atrophy and die. There are no current treatment options for patients with retinitis pigmentosa.

Animal models for retinitis pigmentosa do exist; however, these are low-throughput systems due to the cost and time involved. Acucela's high-throughput screening technologies could lead to the discovery of drug treatments for retinitis pigmentosa.