Varenicline Treatment Associated with Dry Eye Sign, Symptom Reduction

The 4-week randomized trial saw an initial increase in eye dryness score, but positive results were observed at weeks 2 and 4.

This article is part of Ophthalmology Advisor’s Dry Eye Disease In-Depth coverage from the 2021 meeting of the American Academy of Ophthalmology, held in New Orleans from November 12 to 15, 2021. The team at Ophthalmology Advisor will be reporting on a variety of the research presented by the dry eye disease experts at the AAO. Check back for more from the AAO 2021 Meeting.


A twice-daily (b.i.d.) Varenicline (OC-1) treatment for dry eye disease (DED) showed improvement in signs and symptoms at weeks 2 (W2) and 4 (W4) of a randomized trial, despite baseline eye dryness score (EDS). This is according to findings presented at the American Academy of Ophthalmology (AAO) 2021 annual meeting, held in New Orleans from November 12-15.

Researchers conducted a study wherein 3 groups of participants (n=758) were chosen to receive 0.6 mg/mL OC-01, 1.2 mg/mL OC-01, or placebo b.i.d. for 4 weeks. Schirmer Test Score (STS) at week 4 and EDS at weeks 2 and 4 were outcome measures. The study evaluated treatment effects using Cochran Mantel-Haenszel and ANCOVA analyses.

The study found that both groups that received OC-01 b.i.d. saw statistically significant increases (P <.01) in proportion of participants achieving STS of at least 10 mm at Week 4: 0.6 mg/mL, 47.3%; 1.2 mg/mL, 49.2% versus placebo, 27.8%. Additionally, there was a significant reduction in EDS from baseline at W2 and W4 in the 0.6 mg/mL group (W2: -16.5; P <.05; W4: -19.8) and the 1.2 mg/mL group (W2: -17.9; P =.008; W4: -22.2) compared to placebo (W2: -12.7; W4: -15.4). In 84% of participants, sneezing was the most common adverse event. In fewer than 5% of participants, adverse events included cough, throat irritation, and instillation site irritation.

The researchers highlight the fact that new treatments are needed in order to improve signs and symptoms of DED, which is characterized by unstable and/or deficient tear film. 

Varenicline is a treatment traditionally used for cessation of smoking.

Visit Ophthalmology Advisor’s conference section for the complete Dry Eye Disease coverage from the AAO 2021.



Farid M, Gibson A, Hemphill M, Hendrix L. ONSET-2 phase 3 trial of OC-01 (varenicline) nasal spray for the treatment of the signs and symptoms of DED. Poster presented at: The American Academy of Ophthalmology 2021 annual meeting; November 12-15, 2021; New Orleans. Abstract PA029.