Iolyx Therapeutics announced positive top-line data from its Phase 2 trial of ILYX-002, a topical immunomodulator for moderate-to-severe dry eye disease (DED) associated with systemic autoimmune or inflammatory conditions. The randomized, double-masked, vehicle-controlled study demonstrated statistically significant improvements in ocular surface health with rapid onset of action and durable effects through day 57.
Trial Design and Patient Population
The Phase 2 ILYX-002-201 trial was conducted at multiple sites across Australia, enrolling 105 participants with moderate-to-severe dry eye disease associated with systemic autoimmune or inflammatory disorders. Following a 14-day vehicle run-in period, participants were randomized to receive either ILYX-002 or vehicle control, administered twice daily for eight weeks, followed by a two-week safety follow-up.
The trial's primary endpoint was total conjunctival staining, while the secondary endpoint was total corneal staining. According to the company, results showed "a meaningful clinical improvement in ocular surface health" with significance for both staining endpoints at day 15.
Rapid and Sustained Corneal Healing
ILYX-002 demonstrated robust corneal healing effects, producing a -1.41-point LS-mean advantage versus vehicle control in total corneal fluorescein staining (tCFS) as early as day 15 (p = 0.0015), increasing to -1.78 points at day 57 (p = 0.0021). These results indicate a 36-44% improvement from baseline in the active arm versus 15-17% with vehicle control.
Mark Hinds, BSc Optom, founder of Ophthalmic Trials Australia and principal investigator for the trial, commented: "In this Phase 2 study I observed clinically meaningful, statistically significant improvements on the ocular surface—and what's remarkable is how fast the effect appeared. A treatment difference of this magnitude in just 2 weeks is something we normally see only with topical steroids, yet we recorded zero discontinuations for tolerability and no IOP concerns."
Conjunctival Improvements
The conjunctival endpoint, measured by lissamine green conjunctival staining (tLGCS), showed statistical significance at day 15 with a -1.10-point LS-mean difference (p = 0.0425). At day 57, ILYX-002 maintained a -0.97-point separation (p = 0.0807).
Dr. Penny Asbell, M.D., FACS, MBA, FARO, Study Chair of the DREAM dry eye study, emphasized the clinical significance: "The consistency of the ILYX-002 data—conjunctival improvement, corneal healing, and excellent tolerability—gives clinicians real confidence. We desperately need a more precise therapy for autoimmune dry eye, and this targeted approach is exactly the leap forward our patients have been waiting for."
Safety and Tolerability Profile
The treatment demonstrated a favorable safety profile with treatment-emergent adverse events described as "mostly mild to moderate." Importantly, there were no treatment-related serious adverse events and no discontinuations related to product instillation. Mean instillation-tolerability scores remained in the mild range (< 30 out of 100), were described as transient, and declined over time with improvement in corneal staining. Intra-ocular pressure stayed within normal limits throughout the study.
Clinical Context and Comparative Efficacy
Dr. Houman Hemmati, M.D., Ph.D., Medical Director of Iolyx Therapeutics, noted the clinical relevance: "Corneal health drives both vision and comfort, which is why total corneal staining is the indicator the FDA relies on most in registrational trials; the numerical benefit we saw with ILYX-002 is robust and clinically meaningful to both patients and ophthalmologists."
In indirect comparisons with published trials, the corneal healing results are roughly three-fold greater than effect sizes reported for currently marketed topical immunomodulators, while conjunctival improvements are nearly twice the level seen in previous studies with on-market products.
Path to Phase 3
Elizabeth Jeffords, Chief Executive Officer of Iolyx Therapeutics, stated: "ILYX-002 delivered the fastest and deepest corneal healing we have observed in immune-driven dry eye, while also providing additional clinically relevant benefits. The totality of evidence—coupled with an excellent safety profile—supports our plan to move confidently into Phase 3 later this year."
Planning for a Phase 3 clinical trial, similar to the Phase 2 ILYX-002-201 trial design, is ongoing and slated to begin in late 2025. The study will continue to select for moderate to severe autoimmune DED, de-risking the planned registrational program.
