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NYPharmer1

10/30/09 5:24 PM

#20624 RE: NYPharmer1 #20623

Part 3

Understanding ACTC's Position

Market Opportunity: Age related macular degeneration (AMD) represents a “tremendous” market opportunity given the size of the patient population and the lack of treatment alternatives. Diseases of the eye affect more than 30 M people worldwide and represent a $20+B market.
Existing therapies only treat the condition and are not curative but palliative and do not regenerate cells of the macula that have been damaged. Additionally, existing therapies only treat Wet AMD not Dry AMD hence leaving a significant unmet need for an effective treatment for the 90% of AMD patients who have the dry variety,
AMD is a common eye disease that leads to deterioration of the macula which is the central area of the retina in the back of the eye, the paper-thin tissue where light-sensitive cells send visual signals to the brain. In particular, sharp, clear, “straight ahead” vision is processed by the macula. Injury to the macula results in the formation of blind spots and blurred or distorted vision. When the macula becomes damaged, many daily activities such as driving and reading become increasingly difficulty,
AMD most often affects individuals over 50 years of age. There are two forms of macular degeneration: dry macular degeneration or wet macular degeneration. Dry AMD takes place when the light-sensitive cells in the macula slowly break down, gradually blurring central vision in the affected eye. As dry AMD gets worse, the patient may see a blurred spot in the center of their vision. As the disorder progresses, and less of the macula functions, central vision is gradually lost in the affected eye. The most common symptom of dry AMD is slightly blurred vision. The affected individual may need more light for reading and other tasks,
Wet AMD occurs when abnormal blood vessels behind the retina start to grow under the macula. As the dry form worsens, some people begin to have abnormal blood vessels growing behind the macula. These new blood vessels tend to be very fragile and often leak blood and fluid (hence ‘wet’ macular degeneration). The 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. Wet AMD is also known as advanced AMD. It does not have stages like dry AMD. The wet form is diagnosed in approximately 10 percent of cases; however, it results in 90% of the visual impairment and is considered advanced AMD (there is no early or intermediate stage of wet AMD). Wet AMD is always preceded by the dry form of the disease.