Assessing the Efficacy, Safety Profile, and Tolerability of Anti-microRNA-328 Anti-sense (SHJ002) for Dry Eye Disease: A Randomized Phase 2 Trial.

PURPOSE: To evaluate the efficacy and safety profile of SHJ002 ophthalmic solution in patients with dry eye disease (DED).

METHODS

We conducted a multicenter, randomized, double-blind, vehicle-controlled phase 2 study. Adults with a history of DED ≥ 6 months, unanesthetized Schirmer test ≥ 5 mm, total corneal fluorescein staining (tCFS) score ≥ 5 (National Eye Institute scale), and total score ≥ 40 on the Symptom Assessment in Dry Eye (SANDE) questionnaire were included. Patients were randomly assigned 1:1 to receive SHJ002 or vehicle, administered twice daily for 12 weeks. The change from baseline to week 12 in tCFS was the primary end point, and the key secondary end points included the SANDE total score and 8 eye dryness-related symptoms on a visual analog scale (VAS).

FINDINGS: Among 85 randomly assigned patients, 75 without major protocol deviations were designated the per-protocol population. The improvement with SHJ002 versus vehicle was reported as the least squares mean difference in the per-protocol population: -1.66 (0.50) for tCFS (P = 0.001; 95% CI, -2.65 to -0.67), -8.97 (4.33) for the SANDE total score (P = 0.039; 95% CI, -17.5 to -0.45), -12.62 (4.63) for VAS-foreign body sensation (P = 0.007; 95% CI, -21.75 to -3.49), and -8.56 (4.49) for VAS-eye dryness (P = 0.058; 95% CI, -17.42 to 0.29). Analyzing the entire population of 85 patients yielded similar results. Most ocular adverse events (AEs) were mild in severity; no serious AEs occurred. No SHJ002-related AEs were reported.

IMPLICATIONS: SHJ002 demonstrated significant improvement in the signs and symptoms of DED patients and was well-tolerated.

GOV IDENTIFIER: NCT05486728.