Abstract
Purpose :
Dry eye is a multifactorial disease characterized by a persistently unstable and/or deficient tear film causing discomfort and/or visual impairment, accompanied by variable degrees of ocular surface epitheliopathy, inflammation and neurosensory abnormalities. OC-02 (simpinicline), a novel compound delivered via nasal spray, is a nicotinic acetylcholine receptor agonist that pharmacologically activates the trigeminal parasympathetic pathway to stimulate the lacrimal functional unit to reestablish the natural tear film. This Phase 2 study (PEARL) evaluated the efficacy and safety of OC-02 in the treatment of signs and symptoms of dry eye disease.
Methods :
A total of 182 subjects were randomized 1:1:1:1 with OC-02 1.1 mg/mL (n=41), OC-02 5.5 mg/mL (n=41), OC-02 11.1 mg/mL (n=41), or vehicle control (VC, n=42) nasal spray over two visits. Outcome measures were pre- to post-treatment in anesthetized categorical Schirmer’s Test Score (STS) outcomes (percentage of ≥10mm) at Visit 1 and Eye Dryness Score (EDS, 0-100 scale) under controlled adverse environment (CAE) exposure at Visit 2.
Results :
Subjects treated with 5.5 mg/mL and 11.1 mg/mL OC-02 nasal spray showed statistically significant improvement compared with VC as indicated by a percentage gain in STS ≥10 mm from baseline at Visit 1. The 5.5 mg/mL, 11.1 mg/mL, and VC groups were: 73%, 76%, and 7% (p<0.0001), respectively (fig. 1). Correspondingly, improvements in EDS at Visit 2 from pre- to 5 minutes post-treatment in CAE were -16.5 (p = 0.0067), -19.0 (p= 0.0006), and -6.8, respectively. Of the treated groups, significant improvement in the percentage of eyes was seen with 11.1 mg/mL in baseline EDS <60mm and 5.5 mg/mL and 11.1 mg/mL in baseline EDS >60mm (Fig.2). Most common adverse events reported in >5% of treated subjects was coughing, throat irritation, sneezing, and instillation site irritation.
Conclusions :
Compared to VC, OC-02 nasal spray demonstrated improvement in both signs and symptoms of dry eye disease and was safe and well-tolerated under conditions of the study.
This abstract was presented at the 2022 ARVO Annual Meeting, held in Denver, CO, May 1-4, 2022, and virtually.