Single-center, Single-arm, Investigator-Initiated Trial to Evaluate the Efficacy of Varenicline Solution Nasal Spray on Cytokine Levels in the Tears of Subjects with Dry Eye Disease Associated with Moderate-to-Severe Sjogren’s Disease

Molecule Varenicline solution nasal spray (formerly known as OC-01)
Therapeutic focus Dry eye disease
Phase Phase 4
Status Completed
Participants 39
Completion Date August 31, 2023

Note: This clinical trial was not included in the submission which resulted in Tyrvaya® approval.
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file.

Trial Design

*A validated multiplex Luminex® bead-based immunoassay was used to analyze the Schirmer’s test strips at a lab. Luminex is a registered trademark of Luminex Corporation.
BID, twice daily; IFN, interferon; IL, interleukin; TNF, tumor necrosis factor.
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file.

Inclusion Criteria

  • ≥22 years old at screening
  • Moderate-to-severe Sjogren’s disease
  • A diagnosis of dry eye disease
  • Category ≥2 staining score for conjunctiva based on the van Bijsterveld and Utrecht scale, and ≥3 staining score for cornea based on the National Eye Institute scale
  • Have been using topical cyclosporine, lifitegrast, or autologous serum tears for ≥3 months, if on these treatments
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file.

Exclusion Criteria

  • Previous ocular surgery (e.g., intraocular, oculoplastic, corneal, or refractive)
  • Thermal pulsation or intense pulsed light (IPL) in prior 3 months
  • Use of topical ophthalmic corticosteroid therapy in prior 4 weeks
  • Cataract surgery in prior 6 months
  • Active ocular herpes simplex or herpes zoster infection
  • Ocular inflammation (uveitis, iritis, scleritis, episcleritis, keratitis, conjunctivitis)*
  • Ocular infection (e.g., viral, bacterial, mycobacterial, protozoan, or fungal infection of the cornea, conjunctiva, lacrimal gland, lacrimal sac, or eyelids, including hordeolum/stye)
  • Severe (Grade 3 or 4) inflammation of the eyelid (e.g., blepharochalasis, staphylococcal blepharitis or seborrheic blepharitis)
  • Eyelid abnormalities that significantly affect their function (e.g., entropion, ectropion, tumor, edema, blepharospasm, lagophthalmos, severe trichiasis, severe ptosis)
  • Ocular surface abnormality that may compromise the corneal integrity (e.g., prior chemical burn, recurrent corneal erosion, corneal epithelial defect, Grade 3 corneal fluorescein staining (CFS), map dot fingerprint dystrophy, or the effect of any other ophthalmic medication that might compromise the ocular surface integrity*)
*In the opinion of the investigator.
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file.

Efficacy Endpoints

  • Mean Schirmer’s test score (STS) at baseline and day 28 for all subjects
  • Tear cytokine levels at baseline and day 28 for all subjects
  • Mean STS at baseline and day 28 for subjects with STS <5 mm at baseline
  • Tear cytokine levels at baseline and day 28 for subjects with STS <5 mm at baseline
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file

Baseline Characteristics

*Data presented as mean (SD), unless otherwise specified.
SD, standard deviation; STS, Schirmer’s test score.
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file.

Efficacy Outcome Measures – Primary Endpoint

*3 subjects had baseline STS of 35 mm.
CI, confidence interval; STS, Schirmer's test score​.
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file.

Efficacy Outcome Measures – Primary Endpoint

LLOQ values have been imputed by smallest value per parameter by visit (Baseline: IFNγ=0.000282243, IL-10=0.0004984466, IL-12p70=0.0002603448, IL-17a=0.0000257386, IL-1β=0.0002193732, IL-2=0.0002064163,
IL-4=0.0001430653, IL-6=0.0004244109, TNF-α=0.0001842957; Postbaseline: IFNγ=0.0002129771, IL-10=0.0004984466, IL-12p70=0.0000531067, IL-17a=0.0000649204, IL-1β=0.0000285413, IL-2=0.0014717635,
IL-4=0.0002345545, IL-6=0.0002425353, TNF-α=0.0001093103). One sample t-tests were used to generate p-values.
IFN, interferon; IL, interleukin; LLOQ, lower limit of quantitation; TNF, tumor necrosis factor.
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file.

Efficacy Outcome Measures

*Schirmer’s test was conducted within 4 hours of VNS use.
CI, confidence interval; STS, Schirmer's test score​; VNS, varenicline solution nasal spray​.
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file.

Efficacy Outcome Measures

LLOQ values have been imputed by smallest value per parameter by visit (Baseline: IFNγ=0.000282243, IL-10=0.0004984466, IL-12p70=0.0002603448, IL-17a=0.0000257386, IL-1β=0.0002193732, IL-2=0.0002064163, IL-4=0.0001430653, IL-6=0.0004244109, TNF-α=0.0001842957; Postbaseline: IFNγ=0.0002129771, IL-10=0.0004984466, IL-12p70=0.0000531067, IL-17a=0.0000649204, IL-1β=0.0000285413, IL-2=0.0014717635, IL-4=0.0002345545, IL-6=0.0002425353, TNF-α=0.0001093103). One sample t-tests were used to generate p-values.
IFN, interferon; IL, interleukin; LLOQ, lower limit of quantitation; STS, Schirmer's test score​; TNF, tumor necrosis factor.
Cytokine Sub-Study Oyster Point Pharma, Inc. Data on file.