Iolyx Therapeutics Announces Phase 2 Results for ILYX-002 in Autoimmune Dry Eye Disease

Results shared during a R&D Update featuring prominent experts in the field of ophthalmology

A favorable safety profile plus significant ocular surface healing support advancement to Phase 3

BURLINGAME, Calif., May 27, 2025 (GLOBE NEWSWIRE) — Iolyx Therapeutics, a clinical-stage biotechnology company focused on the development of therapeutics at the intersection of ocular inflammation and autoimmunity, today announced 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, during a virtual key-opinion-leader (KOL)-led event. A recording of the webcast has been made available on the Iolyx website.

With a clinically meaningful trend in the primary endpoint of total conjunctival staining and a statistically significant finding on the secondary endpoint of total corneal staining, the study showed a meaningful clinical improvement in ocular surface health of these hard-to-treat autoimmune patients. Notably, the study met significance for both staining endpoints at day 15, highlighting a rapid onset of action, and durable effects through day 57 when compared to a vehicle control.

During the R&D Event, Dr. Penny Asbell, M.D., FACS, MBA,FARO, Study Chair of the DREAM dry eye study and Investigator for the HEDS, and ZEDS trials, commented on the unmet need in DED patients: “Dry eye disease does not typically cause blindness, but is a chronic pain syndrome associated with variable vision and it profoundly impacts quality of life; dry eye disease is one of the most common reasons patients seek eye care. 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.”

On the importance of the data, Mark Hinds, BSc Optom, Founder of Ophthalmic Trials Australia, and Principal Investigator for the ILYX-002-201 Phase 2 trial added: “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 two weeks is something we normally see only with topical steroids, yet we recorded zero discontinuations for tolerability and no IOP concerns. Better still, the benefit was sustained through Week 8, confirming a durable response for patients who have had very few safe options.”

Key Highlights from the Phase 2 Trial

• Rapid and Durable Corneal Healing: ILYX-002 produced a -1.41-point LS-mean advantage versus the 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. In an indirect comparison with published trials, these results are roughly three-fold greater than effect sizes reported for currently marketed topical immunomodulators.
• Clinically Meaningful Conjunctival Response: On the conjunctival endpoint, measured by lissamine green conjunctival staining (tLGCS), ILYX-002 achieved statistical significance at day 15 with a -1.10-point LS-mean difference (p = 0.0425). At day 57, the drug maintained a -0.97-point separation (p = 0.0807) — In an indirect comparison with published trials, these results are nearly twice the level seen in previous studies with on market products.
• Consistent Symptom and Quality of Life Signals: Exploratory patient-reported outcomes showed meaningful 10- to 20-point improvements in multiple symptoms and quality of life sub-scores providing a clear roadmap for powering symptom success in Phase 3.
• Favorable Safety and Tolerability: Treatment-emergent adverse events were mostly mild to moderate, with no treatment-related SAE’s 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 an improvement in corneal staining, while intra-ocular pressure stayed within normal limits. No drug-related serious ocular events were observed, supporting chronic administration.
• Phase 3-Ready Design Insights: Planning is ongoing for a Phase 3 design similar to the design of ILYX-002-201 de-risking the planned registrational program. The study will continue to select for moderate to severe autoimmune DED and is slated to begin in late 2025.

On the significance of corneal health and the opportunity for ILYX-002, Dr. Houman Hemmati, M.D., Ph.D., Medical Director of Iolyx Therapeutics added, “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. We’re trying to create a product that is very differentiated from what’s on the market today and addresses unmet needs that current dry-eye drugs simply don’t meet.”

“ILYX-002 delivered the fastest and deepest corneal healing we have observed in immune-driven dry eye, while also providing additional clinically relevant benefits,” said Elizabeth Jeffords, Chief Executive Officer of Iolyx Therapeutics. “The totality of evidence—coupled with an excellent safety profile—supports our plan to move confidently into Phase 3 later this year.”

About ILYX-002-201

ILYX-002-201 is a first-in-human Phase 2 clinical trial designed to evaluate the safety, tolerability, and efficacy of ILYX-002 in patients with moderate-to-severe dry eye disease (DED) associated with systemic autoimmune or inflammatory disorders. Conducted across multiple sites in Australia, this randomized, double-masked, and vehicle-controlled study commenced with a closely monitored sentinel cohort (n=2) overseen by an independent Safety Review Committee. Following a 14-day vehicle run-in period, a total of 105 participants were randomized to receive either ILYX-002 or vehicle control, administered twice daily (BID) for eight weeks, followed by a two-week safety follow up. This trial represents an important milestone in establishing clinical proof of concept for ILYX-002 as a potential new standard of care for DED, providing key insights into its safety profile and immunomodulatory benefits for patients who have few effective treatment options, and unlocks development in other immuno-ophthalmology indications.

About Iolyx Therapeutics

Iolyx Therapeutics is an immuno-ophthalmology company dedicated to transforming the standard of care at the intersection of autoimmunity and inflammatory disease, from the ocular surface and anterior chamber to the retina. With optimized, locally administered formulations tailored to deliver potent therapeutics to relevant ocular tissues, Iolyx targets ocular inflammation at the source. Iolyx’s mission is to develop targeted therapeutics that maximize efficacy and convenience, with excellent tolerability, and the aim to displace steroids and older immunosuppressants for the benefit of a broad cross-section of patients.

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Heather Berger

Iolyx Therapeutics

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hberger@iolyx.com

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LifeSci Communications

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