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Age-Related Macular Degeneration

The Disease: Age-Related Macular Degeneration (AMD) is a condition affecting the central area of vision and can lead to blurred vision, visual distortions, the appearance of dark spots in the central vision field and blindness.

There are two forms of AMD:

Atrophic (dry) AMD is characterized by the formation of fatty deposits, termed drusen, in the macula. Atrophic AMD leads to slow, but progressive central vision loss. This form of AMD only affects the central vision; peripheral or side vision is spared generally. Roughly 90% of AMD patients suffer from atrophic AMD.

Neovascular (wet) AMD occurs when abnormal blood vessels break through Bruch's membrane (a layer beneath the retina) of the eye. The blood vessels leak fluid and blood into the space beneath the retina in the macula. This can lead to sudden and severe visual impairment. This form of the disease affects roughly 10% of AMD patients, however, 90% of severe vision loss due to AMD is caused by neovascular AMD.

Risk factors for AMD include family history, age, obesity, and smoking. There is no cure for AMD. Novel therapies targeting the formation of blood vessels by way of blocking vascular endothelial growth factor (VEGF) have recently become available and represent a major advance in the treatment of the disease. However, only approximately a third of patients respond to VEGF blockers. Hence, there remains a high medical need for therapeutic approaches that could complement VEGF blockade, by targeting alternative biological aspects of this complex disease.

Although not yet supported by clinical proof of concept scientific evidence suggests that chronic inflammation may lead to the progression of the dry form of AMD to the neovascular state of the disease, and is also a factor in progression of the wet form of the disease. In fact, one of the hallmarks of the disease, the deposition of cell debris in the retina (drusen), may be mechanistically related to the fatty depositions in atherosclerotic plaque. Hence, a treatment targeting chronic inflammation in the retinal space could provide a new approach to the disease.

   Neovascular AMD (NEI, Ref#: EDA24)

The Market Opportunity: Of the approximately 25 million patients in North America and Europe affected by AMD, 90% are diagnosed with the dry form of AMD, and the remaining 10% with the wet form. Any medication that could complement, e.g., a VEGF blocker in the treatment of the wet form, or be effective in the pre-angiogenic form of the disease would represent a major commercial opportunity.