Topline results from a phase 2 study investigating a novel topical cyclosporine product (CyclASol, Novaliq) are encouraging about its potential to provide effective, well-tolerated immunomodulatory treatment for dry eye disease (DED).
The investigational product is a preservative-free, water-free, multidose preparation that formulates the active ingredient in a proprietary semifluorinated alkane (SFA) vehicle (EyeSol Platform Technology). The SFA vehicle itself is marketed outside of the United States as an over-the-counter ocular lubricant for lipid deficiency DED (EvoTears, Ursapharm), and it is attractive as a carrier for cyclosporine because of its ability to dissolve the lipophilic molecule and increase its stability and bioavailability.
The phase 2 multicenter, double-masked study investigated two concentrations of the novel cyclosporine—0.05% and 0.1%—and compared it with the SFA vehicle itself and cyclosporine emulsion 0.05% (Restasis, Allergan). A total of 207 patients were randomly assigned equally into the four study groups and used their assigned treatment twice daily.
Both patient groups using the investigational cyclosporine product achieved significant improvement in corneal fluorescein staining (primary endpoint) compared with vehicle over the 4-month treatment period. The greatest benefit was seen in central corneal staining. Patients using the investigational cyclosporine product also achieved symptomatic improvement.
Most noteworthy, treatment with the investigational cyclosporine formulations was associated with significant reductions in corneal and conjunctival staining by day 14 and excellent tolerability. Ninety-eight percent of patients in both investigational cyclosporine groups completed the 4-month study.
“There is a huge unmet need for a better tolerated formulation of topical cyclosporine to treat inflammation in DED. The early data from this phase 2 trial indicate that cyclosporine in the SFA vehicle may address that need, and the results also show it has the potential for rapid onset of action,” said Claus Cursiefen, MD, PhD, Chairman and Professor, Department of Ophthalmology, University of Cologne. “We look forward to the release of more data from the phase 2 trial and to these very promising results being corroborated in larger, phase 3 studies.”