Varenicline Nasal Spray Reduces Dry Eye Signs and Symptoms in Autoimmune Disease

Varenicline nasal spray can improve both tear production and patient-reported symptoms in individuals with dry eye disease and autoimmune conditions.

Varenicline nasal spray demonstrates the ability to improve both tear production and dry eye disease (DED) symptoms in patients with autoimmune diseases, according to a study published in Clinical Ophthalmology. 

“DED is common in patients with autoimmune disease, and pathophysiology is likely related to the underlying mechanisms of autoimmunity,” according to the study authors. “Autoimmunity can directly involve tissues of the lacrimal functional unit, leading to tear film deficiency and instability and clinical signs and symptoms of DED.”

Investigators included 891 patients from the phase 2b ONSET-1 ( Identifier: NCT03636061) and phase 3 ONSET-2 trials ( Identifier: NCT04036292 ) and assessed a subgroup of 31 participants who self-reported autoimmune disease in the post hoc subgroup analysis. Participants underwent Schirmer assessment and reported eye dryness score (EDS) before and after 28 days of treatment with varenicline nasal spray (mean age, 57.5 years; 90% women; 0.03 mg, n=9; 0.06 mg, n=11) or vehicle (mean age, 63.6 years; 90.9% women; n=11). Rheumatoid arthritis (45.2%) and Sjögren syndrome (12.9%) were the 2 most commonly reported autoimmune diseases. 

OC-01 (varenicline solution) nasal spray represents a novel first-in-class drug therapy for DED sufferers, including those with associated autoimmune disease.

Individuals with autoimmune disease who were treated with varenicline nasal spray experienced a 13.6 mm mean increase in Schirmer score compared with 1.8 mm among control group participants with autoimmune disease. A treatment difference of 11.8 mm (95% CI, 6.5-17.1) was noted among participants with autoimmune disease compared with 5.3 mm (95% CI, 4.1-6.5) in patients without autoimmune disease, the report shows. 

Participants’ EDS improved as well, with decreases of 19.6 and 20.0 noted among individuals undergoing varenicline treatment with and without autoimmune disease, respectively, and 10.3 and 14.0 point decreases among patients with and without autoimmune disease who underwent vehicle treatment, respectively.  

Sneeze was the most common adverse event reported (82–84%), which was graded as mild by 98% of participants.

“OC-01 (varenicline solution) nasal spray represents a novel first-in-class drug therapy for DED sufferers, including those with associated autoimmune disease,” according to the researchers. “The drug is delivered via nasal rather than via topical ophthalmic administration. The intranasal route has the benefit of preventing disruption of the tear film or ocular surface at the time of dosing, an important consideration given that tear film instability and deficiency is a fundamental component of the pathophysiology of DED.”

Study limitations included failure to confirm participants’ self-reported diagnoses with clinical testing.

Disclosure: This research was supported by Oyster Point Pharma, Inc. Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.


Schallhorn JM, McGee S, Nau J, et al. OC-01 (varenicline solution) nasal spray for the treatment of dry eye disease signs and symptoms in subjects with autoimmune disease: integrated Data from ONSET-1 and ONSET-2Clin Ophthalmol. Published online March 3, 2023. doi:10.2147/OPTH.S403953