Nicox’s Positive Post Hoc Results from NCX 4251 Phase 2b Mississippi Trial Suggest Path Forward in Dry Eye Disease

Nicox SA announced positive post hoc results from its Mississippi Phase 2b clinical trial suggesting that once-daily dosed NCX 4251, fluticasone propionate ophthalmic suspension 0.1%, is effective in reducing dry eye symptoms in a subgroup of patients. The new results show that, for a subgroup of patients scoring more highly for a key sign of dry eye, statistically significant improvements in several dry eye symptoms and improvements in one sign (p=0.0524) were seen. The top-line results from the trial, which did not meet the primary or secondary efficacy endpoints, were reported on September 23, 2021. 

The post hoc analyses identified a subgroup of patients (123 of 224 patients) with baseline scores ≥2.0 on a scale of 0 (none) to 4 (severe) for inferior cornea fluorescein staining, a key sign of dry eye disease. In this patient group, the analysis demonstrated a statistically significant difference against placebo for change from baseline in eye dryness scores as assessed on a Visual Analog Scale at Day 8 (p=0.0085), Day 11 (p=0.0020), and Day 15 (p<0.0016). 

Statistically significant differences against placebo were also observed in other symptoms of dry eye disease (photophobia, blurred vision, burning/stinging, foreign body sensation, ocular itching, pain) at all time points during treatment (Day 8, Day 11, and Day 15). In some symptoms, the effects persisted up to two weeks after the end of treatment. On Day 15, the difference in reduction from baseline in inferior cornea fluorescein staining reached a p-value of 0.0524, which we believe could reach statistical significance with a larger patient population. The next steps and timelines in the development of NCX 4251 will be announced following a meeting with the U.S. Food and Drug Administration, scheduled to take place at the beginning of 2022.

Michele Garufi, Chairman and Chief Executive Officer of Nicox, said “Dry eye disease is reported in some 70-80% of patients with blepharitis. The positive impact of NCX 4251 on the multitude of symptoms associated with dry eye disease in a substantial subgroup of blepharitis patients in the trial provides a compelling rationale to explore a targeted approach to the future development of the program with the FDA. We believe that there is still a significant unmet medical need in dry eye disease which could be filled with an efficacious short-term treatment applied only once-daily” 

NCX 4251 was found to be safe and well-tolerated over 14 days’ treatment, with no serious adverse events, and all of the adverse events for the NCX 4251 treatment arm were mild. There were no discontinuations in the study due to adverse events.

Mississippi was a Phase 2b clinical trial, which recruited 224 patients at multiple clinical centers in the U.S., the subpopulation described here comprises 123 patients (61 in placebo and 62 in NCX 4251), evaluating once-daily dosed NCX 4251, fluticasone propionate ophthalmic suspension 0.1%, versus placebo in patients with acute exacerbations of blepharitis. The primary outcome measure was the proportion of patients achieving complete cure in all three hallmark signs and symptoms of blepharitis, eyelid redness, eyelid debris, and eyelid discomfort, at Day 15. The study also explored two secondary outcome measures on signs and symptoms of dry eye.