# Development of a biomimetic small molecule therapeutic for neovascular retinal diseases

> **NIH NIH R44** · EYEMEDIX, INC. · 2020 · $939,493

## Abstract

PROJECT SUMMARY / ABSTRACT
 Retinal diseases, including age-related macular degeneration (AMD) and diabetic retinopathy (DR) are the
leading causes of vision loss and blindness in people over 55 years old. According to the NEI, in 2010 nearly
10 million people in the U.S. suffered from some form of these diseases, whose prevalence is expected to
increase to 15 million over 20 years.
 Recently approved therapies have focused on the exudative (wet) form of AMD, which is characterized by
abnormal new vessel formation (neovascularization), and affects an estimated 1.6 million people. Typically,
these treatments focus on inhibiting vascular endothelial growth factor (VEGF), a protein that promotes the
extravasation from existing vessels as well as the formation of new blood vessels that lack a non-leaky blood-
retinal barrier. The most commonly used therapeutics include the biologic drugs LucentisTM, AvastinTM and
EyleaTM, which must be administered via injections directly into the vitreous cavity every one or two months.
Although these drugs are designed to bind to VEGF and limit its actions, they do not eliminate the source that
produces VEGF, and although rare can also be associated with serious side effects including stroke. Moreover,
the majority of patients do not have a marked improvement in their vision (e.g., about one-third of the AMD
patients get improvement of 3 lines or more on the ETDRS vision chart). Therefore, despite these advances,
there is still a major unmet need to develop new treatments that can better address the pathogenesis of these
diseases.
 This project is based on a new therapeutic concept resulting from unique insights on the critical impact of
persistent local inflammation that is responsible for the production of VEGF, leading to the initiation and
progression of AMD. These efforts led to the identification of a family of locally formed small molecules that
reduce local inflammation in AMD and also suppress the production of VEGF. As a result, it became possible
to use these findings to design and validate new biomimetic small molecules as potential therapeutics for
neovascular retinal diseases. This proposal is for the development of a new small molecule drug candidate that
dramatically reduces abnormal VEGF-mediated effects without directly binding to VEGF. The efficacy of this
promising molecule has been validated in animal models, which showed that it is able to reduce the underlying
persistent inflammatory response, suppress the VEGF pathway, block neovascularization, and promote
regression of the disease. The proposed work will further advance this lead molecule towards the next steps of
pre-clinical development, setting the stage for translation into human clinical trials.

## Key facts

- **NIH application ID:** 10018003
- **Project number:** 5R44EY025490-03
- **Recipient organization:** EYEMEDIX, INC.
- **Principal Investigator:** Amy Wang
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $939,493
- **Award type:** 5
- **Project period:** 2015-05-01 → 2022-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10018003

## Citation

> US National Institutes of Health, RePORTER application 10018003, Development of a biomimetic small molecule therapeutic for neovascular retinal diseases (5R44EY025490-03). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10018003. Licensed CC0.

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