The Science

What is Age-Related Macular Degeneration (AMD)?

Age-related macular degeneration (AMD) is a debilitating eye disease and is the leading cause of legal blindness. Almost 1 in every 3 people over age 75 develop AMD. The disease is strongly age related.

There are two forms of AMD, dry and wet. It is the wet form of AMD that causes rapid vision loss and can lead to blindness. About 10 to 15 % of AMD patients have wet AMD.


The Retina and AMD


Dry AMD is caused by slow progressive, degenerative changes in the retinal epithelial cells and drusen deposits under the retina. Many people can go for a long time with dry AMD and not experience any vision problems.

Wet AMD is caused by the growth of abnormal, fragile blood vessels under the retina. This process is also known as choroidal neovascularization (CNV).

consequences of AMD

The macula is the region of the retina responsible for straight-ahead vision. The new abnormal blood vessels are fragile. As they grow, they leak fluid and blood (hence ‘wet’ macular degeneration) causing rapid damage to the macula and quickly leading to loss of central vision.

Treatment of wet AMD
Currently, options for the treatment of wet AMD are limited. The first generation therapy is based upon the destruction of existing abnormal vessels by photodynamic therapy (PDT), a combination of a photosensitive drug and laser therapy.


There are many factors that lead to AMD. However, effective second generation therapies are designed only to inhibit specific growth factors, such as Vascular Endothelial Growth Factor (VEGF). These angiogenesis inhibitors only slow the progression of wet AMD. This is not an optimal therapy.


OcuCure’s new generation of therapeutic compounds are vascular targeting agents. These selective tubulin inhibitors prevent new blood vessels from sprouting (antiangiogenic activity) as well as directly target the regression of newly formed blood vessels (angiolytic activity). This dual mechanism more completely addresses the uncontrolled blood vessel growth associated with AMD. Therefore, the OcuCure approach could lead to better long-term visual outcomes.

What is Diabetic Retinopathy (DR)?


As a complication of diabetes, people may face eye problems that result in severe vision loss or even blindness. Like AMD, diabetic retinopathy is another leading cause of blindness in American adults. It is caused by the growth of abnormal blood vessels on the surface the retina. These new blood vessels are fragile and may leak fluid and blood into the retina.



Retinopathy affects up to 80% of all diabetics who have had diabetes for 15 years or more. There are no drugs approved for the treatment of DR.

The OcuCure Lead Compound

OcuCure Therapeutics science is based on novel, proprietary antiangiogenic compounds. The OcuCure compounds are a new class of small molecules with a unique mechanism of action. They are selective inhibitors with a dual effect. These compounds have both antiangiogenic and angiolytic activity due to their specific binding activity. The OcuCure approach both inhibits new blood vessels from sprouting (antiangiogenic) and also causes regression of newly formed immature blood vessels (angiolytic).

As a chemically distinct class, OcuCure has a portfolio of over 100 novel analogs that were designed, synthesized and screened for antiangiogenic activity. The lead compound is a potent inhibitor of angiogenesis with a unique mechanism of action, as described above. It works further downstream, at the end of the common pathways that lead to AMD. This mechanism provides the dual therapeutic action independent of the multiple stimuli causing the formation of new blood vessels. Therefore, it may provide a more complete treatment for AMD and DR.


The OcuCure Lead Compound
OcuCure Therapeutics science is based on novel, proprietary antiangiogenic compounds. The OcuCure compounds are a new class of small molecules with a unique mechanism of action. They are selective inhibitors with a dual effect. These compounds have both antiangiogenic and angiolytic activity due to their specific binding activity. The OcuCure approach both inhibits new blood vessels from sprouting (antiangiogenic) and also causes regression of newly formed immature blood vessels (angiolytic).

As a chemically distinct class, OcuCure has a portfolio of over 100 novel analogs that were designed, synthesized and screened for antiangiogenic activity. The lead compound is a potent inhibitor of angiogenesis with a unique mechanism of action, as described above. It works further downstream, at the end of the common pathways that lead to AMD. This mechanism provides the dual therapeutic action independent of the multiple stimuli causing the formation of new blood vessels. Therefore, it can provide a more complete treatment for AMD.

Summary of Ocular Applications

Validation of Novel Therapeutic Target in Human Tissues:
AMD and Diabetic Retinopathy (DR)

Neovascular tissue from AMD and DR patients were tested for the presence of the b tubulin target.  The target b tubulin was present in significant amounts in both these samples.  Conversely, in normal retinal tissue, there were only trace amounts present.  This supports our basic scientific hypothesis that in AMD and DR diseased retina there is an overexpression of the target b tubulin, the site where our molecule binds.

OcuCure has developed a topical eye drop and achieved some significant milestones in animal studies including ocular safety and AMD efficacy.

Development of Topical Eye Drop
The lead compound has been formulated into a topical eye drop. Animal studies indicate the formulations to be nontoxic. The animal studies further show that the lead compound penetrates from the front (cornea) of the eye all the way back to the retina in clinically significant concentrations.

Animal Ocular Toxicity
Animals have been treated with lead compound delivered via intravitreal injections as well as topically via eye drops. Long-term (6 weeks) chronic administration studies have been conducted. Eyes were examined using a slit lamp, and confocal microscopy; and monitored systemically and for intraocular pressure. No toxic effects were observed.

Animal Ocular Efficacy: Wet AMD and Dry AMD Prophylaxis with Topical Eye Drops
When tested using the gold standard animal model for wet AMD (laser-induced CNV), the OcuCure compound, in a topical eye drop dosage form, inhibited new blood vessel growth as compared to the controls. Unlike the current drugs, like Macugen® and Lucentis™, the OcuCure compound has evidence of efficacy as a topical eye drop, and therefore could also be used as a prophylactic to manage the conversion from dry to wet AMD.


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