ILYX-002-201: Phase 2 Trial of ILYX-002 Shows Improved Conjunctival Staining in DED, with Penny Asbell, MD, MBA

Investigative topical immunomodulator ILYX-002 has proven its efficacy in treating moderate-to-severe dry eye disease (DED) by achieving its primary endpoint of total conjunctival staining in the recent ILYX-002-201 phase 2 trial.1

DED diagnosis is notoriously difficult, with signs and symptoms often failing to correlate with one another and many presenting similarly to other ocular conditions. Most clinicians diagnose based on a combination of relevant patient history and multiple diagnostics detecting tear film or ocular surface abnormalities.2

Management of DED is also challenging, as patients often require long-term care. Most treatments are chronic and multifaceted, aiming to mitigate all etiologic components and potentially involving both pharmacological and nonpharmacological interventions.2

HCPLive sat down with Penny Asbell, MD, MBA, clinical professor of ophthalmology at UTHSC and external professor of biomedical engineering at the University of Memphis, to discuss the implications of these new data.

“When you look at dry eye patients, they complain a lot about symptom-related issues: watching TV, driving, computer work, et cetera,” Asbell told HCPLive. “Those everyday activities are important to our dry eye patients, and that’s where they feel the impact of their dry eye disease. So whenever we do a dry eye trial, we measure symptoms, and the results did show improvement.”

ILYX-002-201 was a first-in-human randomized, double-masked, vehicle-controlled phase 2 clinical trial aiming to evaluate the safety, tolerability, and efficacy of ILYX-002 in patients with moderate-to-severe DED associated with systemic autoimmune or inflammatory disorders. The study was conducted in multiple sites across Australia and included a closely monitored sentinel cohort (n = 2), overseen by an independent Safety Review Committee.1

After a 14-day vehicle run-in period, 105 participants were randomized to receive either ILYX-002 or vehicle control, both of which were administered twice daily for 8 weeks. This was followed by a 2-week safety follow-up.1

Investigators found a -1.41-point least-squares-mean advantage versus vehicle control in total corneal fluorescein staining within 15 days (P = .0015), which rose to -1.78 points at day 57 (P = .0021). These data show a 36-44% improvement from baseline in the active arm versus 15-17% with vehicle control. Investigators noted that these results are approximately triple those of effect sizes for currently marketed immunomodulators.1

“One of the nice things that we saw in this trial is corneal staining, which had a significant improvement that seems clinically significant,” Asbell told HCPLive. “We want something that’s rapid, and we want something that really works, and that’s what we saw in this very first trial. So it’s not as big as some of the future trials we’ll do, but we already saw very significant results.”

Additionally, ILYX-002 reached statistical significance in its conjunctival endpoint with the achievement of a -1.10-point least-squares mean difference in lissamine green conjunctival staining at day 15 (P = .0425). A -0.97-point separation was maintained at day 57 (P = .0807). Investigators noted that these results were twice those seen in previous studies with other on-market products.1

Ultimately, ILYX-002 reached its endpoints with a favorable safety and tolerability profile. The only treatment-emergent adverse events during the study were mild to moderate, with no severe events associated with the medication and no discontinuations. Parent company Iolyx Therapeutics plans to move into phase 3 later in 2025.1

“I think this is sort of a new day for dry eye treatment, to hone in on what the patient has and what the best treatment is for that patient,” Asbell said.

Editors' Note: Asbell reports disclosures with NEI and the Office of Dietary Supplements.

References

1. Iolyx Therapeutics. Iolyx Therapeutics Announces Phase 2 Results for ILYX-002 in Autoimmune Dry Eye Disease. GlobeNewswire. May 27, 2025. Accessed August 27, 2025. https://www.globenewswire.com/news-release/2025/05/27/3088611/0/en/Iolyx-Therapeutics-Announces-Phase-2-Results-for-ILYX-002-in-Autoimmune-Dry-Eye-Disease.html

2. Sheppard J, Shen Lee B, Periman LM. Dry eye disease: identification and therapeutic strategies for primary care clinicians and clinical specialists. Ann Med. 2023;55(1):241-252. doi:10.1080/07853890.2022.2157477