Effect of OTX-101, a novel nanomicellar formulation of cyclosporine A, on conjunctival staining in patients with keratoconjunctivitis sicca: a pooled analysis of phase 2b/3 and 3 clinical trials
Abstract
Abstract Purpose: Keratoconjunctivitis sicca (KCS), a multifactorial disease, is the most common ocular condition for patients seeking medical treatment and is characterized by ocular burning, stinging, and dryness. This pooled analysis examined the effect of OTX-101 0.09% versus vehicle on the total and individual conjunctival staining in patients with KCS from phase 2b/3 and phase 3 studies. Methods: In these randomized, multicenter, double-masked, and vehicle-controlled studies, patients received 1 drop of OTX-101 0.09% or vehicle in both eyes twice daily. The time points for the pooled analysis were baseline (day 0) and study days 28, 56, and 84/early discontinuation. Conjunctival staining was graded on a 0- to 3-point scale per zone and averaged over both eyes at each assessment. Pooled safety assessments included adverse event (AE) reporting. Results: The total mean (standard deviation) conjunctival staining scores at baseline were 5.4 (1.7) for OTX-101 ( n = 523) and 5.5 (1.7) for vehicle ( n = 525). OTX-101 versus vehicle significantly reduced the total conjunctival staining scores ( P = 0.0316, <0.0001, and 0.0002) for days 28, 56, and 84, respectively. The most common treatment-related AE was instillation site pain (21.8% OTX-101 vs. 4.0% vehicle); most AEs were mild in nature. Conclusions: Treatment with OTX-101 versus vehicle significantly improved the conjunctival staining in KCS as early as 4 weeks, and the improvement was maintained through 12 weeks. OTX-101 was effective and well tolerated for use in KCS.
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OTX-101 nanomicellar solution resulted in dose-dependent accumulation of CsA in corneal tissue and superior bulbar conjunctiva.
“It has been found that concentrations of CsA in corneal tissue and superior bulbar conjunctiva accumulated in a dose-dependent manner [124,125].”
From From design to Deployment: The innovative role of nanomicelles in targeted ophthalmic therapeutics (2025) · cites this paper as [125]
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“It has been found that concentrations of CsA in corneal tissue and superior bulbar conjunctiva accumulated in a dose-dependent manner [124,125].”
- Authors
- Smyth-Medina, R., Johnston, J., Devries, D. K., Jasper, A., Kannarr, S. R., Schechter, B. A., Shen Lee, B., Varghese, G., Ogundele, A., Darby, C. H., Karpecki, P., & Luchs, J.
- Affiliations
- North Valley Eye Medical Group (US)
- Georgia Eye Partners, Atlanta, Georgia.
- Eye Care Associates of Nevada, Sparks, Nevada.
- Advanced Eyecare Specialists, West Palm Beach, Florida.
- Kannarr Eye Care, Pittsburg, Kansas.
- Florida Eye Microsurgical Institute (US)
- Vision Optique, Houston, Texas.
- Sun Pharmaceutical Industries, Inc., Princeton, New Jersey.
- Sun Pharma Advanced Research Company, Ltd., Princeton, New Jersey.
- University of Kentucky (US)
- Hofstra University (US)
- Corresponding author
- Smyth-Medina, Robert
- Venue
- Journal of Ocular Pharmacology and Therapeutics
- Publisher
- SAGE Publications
- Published
- 2019-09-01
- Volume
- 35
- Issue
- 7
- Pages
- 388-394
- ISSN
- 1080-7683 (ISSN-L)
- 1557-7732
- OpenAlex
- W2965629330
- Cited by
- 18
- References
- 17
- Citations by year
- 2026: 3 · 2025: 4 · 2022: 4 · 2021: 3 · 2020: 4
- FWCI
- 2.55
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Journal of Ocular Pharmacology and Therapeutics (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ocular Surface and Contact Lens
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advanced Drug Delivery Systems
- Health Sciences › Medicine › Ophthalmology › Drug-Induced Ocular Toxicity
- Keywords
- cyclosporine, dry eye, conjunctival staining, ocular drug delivery, treatment-related adverse events
- MeSH terms
- Humans, Immunosuppressive Agents, Keratoconjunctivitis Sicca, Micelles, Time Factors, Randomized Controlled Trials as Topic, Cyclosporine, Treatment Outcome, Clinical Trials, Phase II as Topic, Clinical Trials, Phase III as Topic, Nanoparticles
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