We are executing a multi-pronged approach to fully explore the potential of our owned assets as well as investigate new and interesting ophthalmic opportunities outside of our current domain.

Starting with Rhopressa™ (netarsudil ophthalmic solution) 0.02% and Roclatan™ (netarsudil/latanoprost ophthalmic solution) 0.02%/0.005%, we are exploring the potential longer-term impact of these treatments on the trabecular meshwork. By increasing trabecular outflow, we believe these products may have the potential to stop the degeneration of outflow tissues. This may be possible because as the trabecular meshwork becomes relaxed and opens as a result of the action of our products, the increased throughput of eye fluid, or aqueous humor, helps deliver nutrients and antioxidants to regions of the trabecular outflow pathway that were otherwise blocked from passage. The flow of fluid through the trabecular meshwork is the only known mechanism for delivering such nutrients to the diseased tissue, as there are no blood vessels present. Work is underway to explore whether our products may be able to prevent, or possibly even reverse, damage to the trabecular meshwork pathway through this effect. We recently completed preclinical studies demonstrating that Rhopressa™ ophthalmic solution may have the potential for disease modification, including stopping and reversing fibrosis and increasing perfusion in the trabecular meshwork, which would represent a breakthrough in glaucoma treatment. The illustrations below show the ability of netarsudil (AR-13324), the active ingredient in Rhopressa™ ophthalmic solution, to block TGF-beta-induced expression of fibrosis proteins in human trabecular meshwork cells and expand the trabecular meshwork tissue, opening spaces for increased outflow.

We also have a library of Rho kinase (ROCK) inhibitors of over 3,000 compounds. All of these compounds are wholly owned by Aerie. ROCK inhibitors may be neuroprotective, and we are currently exploring whether any of our compounds have the ability to directly protect the optic nerve from further damage in glaucoma patients. Our research, as illustrated below, has shown preclinically the potential of Rhopressa™ ophthalmic solution to promote retinal ganglion cell survival and axon regeneration following optic nerve injury, which may point to potential neuroprotective attributes.

Our expansive library of molecules is also being evaluated for potential benefit in the treatment of diseases beyond glaucoma, including AMD (age-related macular degeneration), due to the beneficial effect of ROCK inhibition on the underlying disease processes of inflammation, fibrosis and angiogenesis. If proven out, we may have the potential to provide new mechanisms and pathways to treat this disease and other sight-threatening diseases.

As we look at diseases at the back of the eye, we will also explore drug delivery alternatives for our small molecules, including devices, to optimize the duration of effect in the back of the eye. In addition, we are evaluating opportunities for the use of sustained delivery technologies for front of the eye delivery of our products for the treatment of glaucoma or ocular hypertension.


RhopressaTM Disease Modification Potential


Netarsudil* May Have Anti-Fibrotic Activity in Human Trabecular Meshwork Cells


Netarsudil* Causes Expansion of Trabecular Meshwork Tissue, Opening Spaces for Increased Outflow


RhopressaTM has shown preclinically the promotion of retinal ganglion cell survival and axon regeneration following optic nerve injury


ROCK Inhibitors for AMD


Early results of the research performed thus far on our preclinical molecule AR-13154 has shown lesion size decreases in a model of wet AMD at levels that are numerically higher than a current market-leading product. AR-13154 inhibits Rho kinase, PKC, JAK2 and PDGFR-β. If proven out, we may have the potential to provide new mechanisms and pathways to treat this disease. The graph below depicts the results of a recent preclinical study designed to show reduced neovascularization in an animal model. AR-13154 demonstrated greater lesion size reduction than Eylea® in this in vivo preclinical study involving laser-induced choroidal neovascularization. Additionally, in our preclinical studies and as depicted in the graph below, we have seen a promising effect of this molecule on reducing neovascularization in a model of proliferative diabetic retinopathy. We are also exploring if there is any benefit of our other compounds in DME (diabetic macular edema), as it is known that ROCK inhibitors reduce the weakening of vessel walls and microvascular damage in animal models of diabetes.


AR-13154 vs. Eylea in Preclinical AMD Model

Posters and Research Information
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March 2, 2017 149 KB
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August 8, 2016 859 KB
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February 24, 2016 593 KB
May 7, 2015 300.6 KB
Mar 2, 2015 679.9 KB
Mar 2, 2015 1.5 MB
May 30, 2014 748.4 KB
Feb 14, 2014 547.5 KB
May 15, 2013 469.5 KB
May 21, 2012 530.8 KB
May 21, 2012 1.3 MB