Acucela

Disease Applications

Acucela’s primary focus is on safe and effective treatments for Age-related Macular Degeneration (AMD). Acucela also has programs underway to treat other potentially devastating ophthalmic diseases, including glaucoma, retinopathy of prematurity, Stargardt disease and diabetic retinopathy, as well as dry eye syndrome.

About macular degeneration

The macula is a small region in the center of the retina that enables us to see fine detail. Light-sensing cells in the macula, known as photoreceptors, convert light into electrical impulses and transfer the impulses to the brain via the optic nerve. Central vision loss from AMD occurs when photoreceptor cells in the macula degenerate.

Age-related macular degeneration (AMD) occurs in "dry" and "wet" forms. AMD is a leading cause of blindness in the United States, and is estimated to affect more than 10 million Americans and 120 million people worldwide. Dry AMD is the most common form, representing approximately 90% of all cases.
(Reference: The Retinal Pharmaceuticals Market Report, Market Scope, 2012)

Dry AMD occurs when light-sensitive cells in the macula slowly break down, gradually blurring central vision in the affected eye. As dry AMD worsens, a blurred spot may appear in the center of the patient’s vision. Over time, as less of the macula functions, central vision is gradually lost in the affected eye.
Wet AMD, also known as “advanced AMD,” occurs when abnormal blood vessels behind the retina start to grow under the macula. These new blood vessels are fragile and often leak blood and fluid. As blood and fluid raise the macula from its normal place at the back of the eye, damage to the macula occurs rapidly. With wet AMD, loss of central vision can occur quickly. An early symptom of wet AMD is that straight lines appear wavy.

For more information about AMD, visit the National Eye Institute.

ACU-4429

The lead compound in our VCM program, ACU-4429, is being evaluated in a Phase 2 clinical trial for the dry form of AMD, a degenerative disease of the eye that affects the part of the retina responsible for fine visual acuity and color vision. There is no approved treatment for dry AMD currently on the market.

Potential Non-AMD Retinal Indications

Acucela’s VCM compounds may provide therapeutic benefit for additional retinal disorders. Our VCM compounds will be investigated for potential use in these vision-impairing conditions. Potential therapeutic indications are described below:

Retinopathy of Prematurity

Retinopathy of prematurity (ROP) is a potentially blinding eye disease that can occur in premature infants. In ROP, abnormal blood vessels grow and spread throughout the retina. There are approximately 15,000 cases of ROP in the U.S. each year, of which about 10 percent are considered severe. These abnormal blood vessels may leak, scarring the retina and pulling it out of position, which can cause retinal detachment, the primary cause of vision impairment and blindness in ROP. There is no approved treatment for ROP.

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

Stargardt Disease

Stargardt disease is a recessive, hereditary retinal disease that results in progressive loss of rod and cone photoreceptors, leading to loss of central vision. It most commonly affects people under the age of 20, starting with symptoms of difficulty in reading and seeing in dim lighting. Eventually, all patients with Stargardt disease are expected to have visual acuity of between 20/200 and 20/400, which is not correctable with glasses. Recent studies demonstrate that several of Acucela's compounds are efficacious in animal models, supporting their use in Stargardt disease.

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 others, abnormal new blood vessels grow on the surface of the retina.

At first, patients with diabetic retinopathy, which usually affects both eyes, may not notice changes to their vision; however, over time, diabetic retinopathy can worsen and cause vision loss.

Non-Retinal Therapeutic Opportunities

Glaucoma

Glaucoma is a type of progressive or chronic optic neuropathy caused by intraocular pressure or by other factors of unknown origin. Persons affected with glaucoma experience gradual loss of vision over a long period of time. As the lost visual field cannot be recovered, continuous control of intraocular pressure is necessary for the rest of the patient’s life. For this reason, safe drugs that lower intraocular pressure are needed.

In the United States, over three million people suffer from glaucoma, and this number is expected to increase dramatically as the population ages. Common treatments for preventing progression of visual field loss in patients diagnosed with glaucoma include drug therapy (beta blockers, prostaglandins, etc.), laser therapy and surgery, depending on the necessity and urgency of treatment.

Dry Eye Syndrome

Dry eye syndrome affects more than 20 million Americans, making it one of the most common problems treated by eye physicians. It is usually caused by a problem with the quality of the tear film, the watery layer of the cornea that lubricates the eyes.

The normal aging process is one of the root causes of dry eye syndrome. As we age, our bodies produce less oil—60 percent less at age 65 than at age 18. Without as much oil to seal the watery layer, the tear film evaporates much faster that it should, leaving dry areas on the cornea. Although rare, blindness can result from serious cases of dry eye syndrome.