# Development of Filociclovir for the Treatment of Ocular Adenoviral Infections and Keratoconjunctivitis

> **NIH NIH R44** · MICROBIOTIX, INC · 2022 · $712,798

## Abstract

Project Summary/Abstract
 The long-term goal of this project, to develop filociclovir (FCV) for the safe and effective treatment of human
ocular adenovirus (AdV) infections, especially AdV-related epidemic keratoconjunctivitis (EKC), a stated National
Eye Institute (NEI) research priority. There are currently no treatments for patients suffering from adenoviral
conjunctivitis, which is responsible for up to 3.5 million lost school days and 8.5 million lost work days every year
in the U.S., or EKC, the most severe form of ocular adenoviral infection, which often causes long-term visual
sequelae, such as chronic keratitis and vision loss. As FCV has previously completed Phase 1 clinical studies
for a systemic indication of cytomegalovirus (CMV) infection in solid organ transplant recipients and is a potent
inhibitor of human adenoviruses, the objective of this SBIR proposal is to generate the remaining ocular-specific
preclinical (PC) data needed to support an investigational new drug (IND) submission to treat human ocular AdV
infections. In this Phase II SBIR, IND-enabling PC ocular pharmacokinetics (PK), biodistribution and GLP-
toxicology studies will be completed, along with confirmatory efficacy parameters, suitable for IND submission.
Additionally, we will confirm the mechanism of action of FCV against AdV and compare it with what is known for
the viral targets in CMV. The specific aims of this proposal, along with the research design and methods, are as
follows:
AIM 1. Optimize eye drop formulation with suitable excipients and confirm efficacy in the rabbit AdV
infection model (years 1-2). Milestones: Identify final formulation suitable for PC and human clinical ocular
safety and efficacy studies. Select most favorable dose level, frequency and duration. Experimental design:
formulation development, repeat Ad5/NZW rabbit eye infection model, dosage optimization in the same model.
AIM 2. Perform IND-enabling ocular biodistribution, systemic toxicokinetics, local dose range finding
ocular tolerance and repeat-dose ocular toxicology and supporting studies in albino rabbits and dogs
(years 1-2). Milestones: Determine ocular and systemic exposure and distribution to predict areas of potential
toxicity. Establish NOAEL to determine human clinical starting dose. Completion of acute local tolerance studies,
GLP 28-day subacute ocular toxicology and in vitro phototoxicity studies to support an IND filing. Experimental
design: complete remaining preclinical studies for the ocular route to support IND for ophthalmic product.
AIM 3. Conduct mechanism of action studies (years 1-2). Milestones: Confirm viral and host polymerase and
kinase targets. Experimental design: determine whether the mechanism of action identified for CMV is the same
for AdV by performing mutation experiments.

## Key facts

- **NIH application ID:** 10410533
- **Project number:** 5R44EY032837-02
- **Recipient organization:** MICROBIOTIX, INC
- **Principal Investigator:** Terry L. Bowlin
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $712,798
- **Award type:** 5
- **Project period:** 2021-06-01 → 2024-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10410533

## Citation

> US National Institutes of Health, RePORTER application 10410533, Development of Filociclovir for the Treatment of Ocular Adenoviral Infections and Keratoconjunctivitis (5R44EY032837-02). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10410533. Licensed CC0.

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