Inspire Pharmaceuticals, Inc. - diquafosol Clinical Trials

Phase 1 Trials
Diquafosol was shown to be well tolerated in Phase 1 clinical trials.

CORNEAL STAINING - An indication of the degree of damage on the corneal surface. A special dye is applied to the eye and the dye adheres to the cornea wherever there is damage (ulcerations or abrasions). This adherance of stain can be quantified by a standardized scoring procedure.

Phase 2 Trials
Trial 103
The Phase 2 trial, trial 103, was a 4-week environmental trial conducted in 158 patients. In this trial, diquafosol demonstrated statistically significant differences from placebo for mean corneal staining and mean conjunctival staining. The safety profile was comparable to placebo.

Phase 3 Studies
Trial 104
Trial 104, was a 3-month environmental trial with a 9-month safety extension, conducted in 558 patients. In this trial, diquafosol did not demonstrate statistically significant differences from placebo for mean corneal staining at the primary timepoint. However the trial did demonstrate statistically significant differences from placebo for mean corneal staining at 6 weeks using the secondary statistical analysis. The safety profile was comparable to placebo.

Trial 105
Trial 105, was a 6-month environmental trial conducted in 527 patients. In this trial, diquafosol demonstrated highly statistically significant differences from placebo for mean corneal staining and mean conjunctival staining at multiple timepoints throughout the trial, and also showed strong trends in dry eye symptoms. The safety profile was comparable to placebo.

Trial 108
Trial 108, was a 4-week trial conducted in 222 patients. The trial had an environmental component similar to the other phase 2 and 3 trials, and a "chamber" component during which patients are exposed to a warm, dry environment to exacerbate their dry eye condition. In the environmental analysis, diquafosol demonstrated statistically significant differences from placebo for ocular staining (the incidence of complete reduction of staining on the cornea and some of the conjunctiva) at multiple timepoints throughout the trial. In the "chamber" portion of the trial, diquafosol did not demonstrate statistically significant improvement in ocular staining or discomfort. The safety profile was comparable to placebo.

Trial 109
Trial 109 was a 6-week environmental trial conducted in 640 patients. In this trial (109), diquafosol failed to demonstrate statistically significant differences from placebo for the primary endpoint of the incidence of corneal clearing (complete reduction of corneal staining). Improvement compared to placebo was achieved for a number of secondary endpoints, including mean corneal staining, mean conjunctival staining and conjunctival clearing (complete reduction of conjunctival staining). The most common ocular adverse event, burning/stinging on instillation, occurred more frequently on diquafosol than on placebo, but the incidence was less than 5%.