Targeting Inflammation in Dry Eye with ILYX-002: Early Clinical Trial Results

Dry eye disease (DED) remains one of the most commonly reported ocular complaints, but there are limited treatment options that meaningfully address the inflammatory underpinnings of the disease, particularly in patients with systemic autoimmune or inflammatory conditions.

The Phase 2 trial examining ILYX-002, a topically administered immune modulator, introduces early but measurable evidence of efficacy in this difficult-to-treat population, according to data presented at the 2025 ARVO Annual Meeting.

Here’s a brief overview of what we know so far.

Study Design
In this first-in-human study, 105 patients with moderate-to-severe DED and coexisting autoimmune or inflammatory disease were enrolled following a 14-day vehicle run-in.

Participants were randomized to receive ILYX-002 0.3 percent or vehicle control twice daily for eight weeks, with safety and efficacy endpoints assessed throughout. The trial design included a closely monitored sentinel cohort to mitigate risk in the initial exposure phase.

Efficacy Findings
Clinical efficacy was measured using the total corneal fluorescein staining (tCFS) score based on a modified NEI grading scale.

Looking at the results, the active treatment arm demonstrated a statistically significant reduction in tCFS scores by Day 15, with a mean change of -2.41 compared to -1.00 in the vehicle group (P=0.0015). This early signal of effect was not only maintained but further improved by Week eight, where the mean difference from baseline reached -2.90 in the ILYX-002 group, translating to a significant treatment difference of -1.78 compared to vehicle (P=0.0021).

These results indicate a rapid onset of action, a feature often absent in current anti-inflammatory therapies for DED, where delays in effect can lead to poor adherence and treatment fatigue.

The sustained response through the trial’s endpoint suggests potential utility in both acute flares and chronic management, although durability beyond eight weeks remains to be determined in longer trials.

Safety Profile
When it comes to safety, signals remained favorable across the study period. Treatment-emergent adverse events were generally mild or moderate and occurred with similar frequency in both active and vehicle groups. Discontinuations due to adverse events were infrequent and did not reveal group-specific trends.

Intraocular pressure remained stable over the eight-week course, showing a mean change of -0.21 mmHg from baseline, with no concerning shifts observed. Tolerability, assessed using a visual analogue scale, showed no significant discomfort associated with ILYX-002 instillation.

These findings align with expectations for a topically delivered agent and may prove meaningful in clinical scenarios where systemic immunosuppression is not preferred or feasible.

The safety consistency across patients with varied autoimmune profiles further underscores the potential of localized treatment approaches for systemic inflammatory conditions manifesting in the eye.

Implications for Subpopulation-Specific Interventions
The trial’s focus on DED patients with underlying autoimmune or systemic inflammatory diseases represents a critical distinction from prior studies that often exclude these populations due to heterogeneity and perceived complexity.

The consistency of the response across this cohort, coupled with the favorable tolerability profile, suggests ILYX-002 may fill a therapeutic gap in an area where conventional lubricants and non-specific immunosuppressants offer limited benefit.

As therapeutic interest grows around biologically informed segmentation of DED, these data add to the argument for subtype-directed treatment selection. The agent’s potency as a selective immune modulator, as well as its topical administration route, position it for further development in inflammation-driven DED variants.

Next Steps in Clinical Development
The Phase 2 findings establish a foundation for larger, longer-duration studies to assess long-term efficacy, safety, and optimal duration of therapy.

Given the statistically significant improvements observed within two weeks, future protocols may also consider dose-response evaluations and comparison to existing standard-of-care agents. Importantly, the sustained efficacy through the eighth week without escalation or systemic exposure raises the possibility of integrating ILYX-002 into multimodal regimens for patients with systemic disease and ocular surface manifestations.

The investigational therapy’s performance in this early-phase trial affirms the feasibility of locally delivered immune modulation in targeted DED populations and invites broader exploration of non-systemic agents in autoimmune-associated ocular conditions.

Reference:
Hinds M,  Hemmati H,  Jeffords E,  Newman E. Phase 2 trial results of topically administered ILYX-002, a novel immune modulator for inflammatory dry eye disease. Invest Ophthalmol Vis Sci. 2025;66(8):5686.