ILYX-002 shows 3x greater corneal healing effect compared to current therapies on the market.
Iolyx Therapeutics (California, United States), a clinical-stage biotechnology company pioneering treatments at the intersection of ocular inflammation and autoimmunity, has announced positive top-line results from its Phase II trial of ILYX-002, a novel topical immunomodulator for moderate-to-severe dry eye disease (DED) associated with systemic autoimmune or inflammatory conditions.
“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 III later this year,” said CEO Elizabeth Jeffords.
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A closer look at the ILYX-002-201 phase II trial
The ILYX-002-201 Phase II trial was a randomized, double-masked, vehicle-controlled study conducted across multiple sites in Australia. It enrolled 105 patients with moderate-to-severe DED, following a closely monitored sentinel cohort and a 14-day vehicle run-in.
Participants received either ILYX-002 or vehicle control, administered twice daily (BID) for eight weeks, followed by a two-week safety follow-up. This approach has laid the groundwork for a Phase III program, which Iolyx plans to kick off in late 2025.
The clinical outcomes of the ILYX-002 Phase 2 trial were promising.
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- Rapid and durable corneal healing
ILYX-002 demonstrated a -1.41-point LS-mean advantage versus vehicle in total corneal fluorescein staining (tCFS) by day 15 (p = 0.0015), which increased to -1.78 points by day 57 (p=0.0021).
These results translate to a 36-44% improvement from baseline in the active arm, compared to 15-17% with the vehicle control. Notably, this effect is three times greater than what has been reported in published trials of existing topical immunomodulators.
- Clinically meaningful conjunctival response
On the conjunctival endpoint, measured by lissamine green conjunctival staining (tLGCS), ILYX-002 showed a -1.10-point LS-mean difference at day 15 (p=0.0425), with durable benefits through day 57 (-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 strong guidance for Phase III trial design.
- Favorable safety and tolerability
ILYX-002 was well tolerated, with no treatment-related serious adverse events, no discontinuations due to instillation discomfort, and no intraocular pressure (IOP) concerns. Mild, transient instillation-tolerability scores (<30/100) improved over time, aligning with the improvement in corneal staining.
READ MORE: A New Era of DED Relief
Expert voices on the results
Mark Hinds, BSc Optom, founder of Ophthalmic Trials Australia and principal investigator for the ILYX-002-201 trial, summed it up perfectly:
“In this Phase II study I observed clinically meaningful, statistically significant improvements on the ocular surface—and what’s remarkable is how fast the effect appeared. 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.”
Dr. Houman Hemmati, medical director of Iolyx Therapeutics, emphasized the clinical impact: “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.”
Moving forward
With these strong Phase II results, Iolyx Therapeutics is full steam ahead for Phase III. The upcoming trial will maintain a similar design, targeting patients with moderate-to-severe autoimmune DED—a population that has long been underserved by existing therapies.
Editor’s note: For more information, see the Iolyx Therapeutics press release here.