Ocular Therapeutix, Inc. (Nasdaq:OCUL), a biopharmaceutical company focused on the formulation, development, and commercialization of innovative therapies for diseases and conditions of the eye, today announced topline results from its Phase 2 clinical trial of OTX-CSI (cyclosporine intracanalicular insert) for the treatment of dry eye disease (DED).
The Phase 2, U.S.-based, randomized, double-masked, multi-center, vehicle-controlled clinical trial of OTX-CSI was designed to evaluate safety, tolerability, durability, and efficacy of two different formulations of OTX-CSI by measuring signs and symptoms of DED in 140 subjects treated in both eyes over approximately 16 weeks (a 12-week study period, with an additional 4-week safety follow-up). In the Phase 2 clinical trial, OTX-CSI was administered to 147 subjects with DED at 15 sites in the U.S. The four groups evaluated in this study were: OTX-CSI for a shorter duration (2-3 months formulation-F1, n=42), OTX-CSI for a longer duration (3-4 months formulation-F2a, n=40), vehicle insert for a longer duration (3-4 months formulation-F2b, n=43) and vehicle insert for a very short duration (1 week formulation-F3, n=22).
The study did not show separation between the OTX-CSI treated subjects (both formulations) and the vehicle treated subjects (both formulations) for the primary endpoint of increased tear production at 12 weeks as measured by the Schirmer’s Test. Change from baseline (improvement) in mean Schirmer’s Test scores for the four groups were as follows: OTX-CSI F1: 1.98 mm, OTX-CSI F2a: 1.91 mm, Vehicle F2b: 2.24 mm and Vehicle F3: 3.08 mm.
The study did show an improvement compared with baseline in signs of dry eye disease as measured by total corneal fluorescein staining (CFS) and symptoms of dry eye disease as measured by the visual analogue scale (VAS) eye dryness in subjects treated with the OTX-CSI insert (both formulations) starting as early as two weeks after insertion and continuing over the 12 weeks study period. These improvements were not statistically significant compared with vehicle insert (both formulations) for either CFS or VAS eye dryness (severity and frequency) at 12 weeks.
Overall, the OTX-CSI insert (both formulations) was generally observed to have a favorable safety profile and was well tolerated. There were no ocular serious adverse events. No subjects dropped out of the trial due to an adverse event. The most common ocular adverse event was ocular pruritis which was seen in less than 16% of subjects. The adverse events of ocular discomfort or pain were seen in less than 3% of subjects. The most common non-ocular event was COVID-19 and was seen in 3% of subjects.
“We would like to thank the patients and investigators who participated in the OTX-CSI clinical trial,” said Antony Mattessich, President and Chief Executive Officer of Ocular Therapeutix. “While we are disappointed by these results, demonstrating clinical benefit in patients with dry eye disease remains a significant unmet need and we will continue to review the data for additional information that may inform future development of this program. We remain confident in the potential of our hydrogel-based formulation technology and its ability to deliver innovative ophthalmology therapies. We look forward to our anticipated Phase 2 top-line read out for OTX-DED for the short-term treatment of signs and symptoms of dry eye disease. In addition, we expect to provide updates on other pipeline programs being developed to treat glaucoma and wet-AMD as well as updates on our currently marketed product, DEXTENZA®, which recently received FDA approval to expand its label for the treatment of ocular itching associated with allergic conjunctivitis.”