# Mitochondrial-Targeted Therapy for Macular Degeneration

> **NIH NIH R44** · ECLIPSE LIFE SCIENCES, INC. · 2024 · $735,619

## Abstract

SUMMARY
Age-related macular degeneration (AMD) is the most common cause of blindness in the elderly. Most AMD
cases are the nonexudative (or “dry”) form, which affects up to 200 million patients globally and is comprised of
intermediate dry AMD, characterized by formation of sub-retinal pigment epithelium (RPE) deposits called
drusen, and geographic atrophy (GA), more advanced disease characterized by loss of RPE and
photoreceptors. Currently, there are no available treatments for any form of dry AMD. Thus, there is a
tremendous unmet need for any effective therapy. Mitochondrial dysfunction at the RPE has been established
as a major disease mechanism for dry AMD. While systemically administered mitochondria-targeted drugs
have shown promise in preclinical and early-phase clinical studies of dry AMD, they have limitations, including
insufficient bioavailability at the retina in some patients. The purpose of this Direct to Phase 2 SBIR grant
application is to develop a novel intravitreal extended release mitochondria targeted drug (IVT Mito XR) for the
treatment of dry AMD. Eclipse Life Sciences has designed novel mitochondria targeted prodrugs of EY005
(lead and backups). Preliminary studies demonstrate that the lead EY005 prodrug and a pilot formulation of the
prodrug in IVT Mito XR has excellent efficacy in both in vitro and in vivo models of mitochondrial dysfunction
that are relevant to dry AMD. The proposed project is focused on developing lead and backup formulations of
IVT Mito XR using Eclipse’s proprietary extended release drug delivery system (XRDDS), to achieve target
product specification of 3 months’ sustained release of EY005 following a single intravitreal injection. Aim 1 will
finalize IVT Mito XR formulations of lead and backup prodrugs. Aim 2 will be to perform nonGLP (good
laboratory practice) pharmacokinetics, toxicology, and proof of concept efficacy studies of IVT Mito XR in
rabbit models. Aim 3 will be to execute GMP (good manufacturing practice) production and preliminary
characterization of lead EY005 prodrug. The end deliverable will be submission of a pre-IND package in
preparation for scheduling a pre-IND meeting with FDA.

## Key facts

- **NIH application ID:** 10844533
- **Project number:** 5R44EY034732-02
- **Recipient organization:** ECLIPSE LIFE SCIENCES, INC.
- **Principal Investigator:** Scott William Cousins
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $735,619
- **Award type:** 5
- **Project period:** 2023-06-01 → 2026-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10844533

## Citation

> US National Institutes of Health, RePORTER application 10844533, Mitochondrial-Targeted Therapy for Macular Degeneration (5R44EY034732-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10844533. Licensed CC0.

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