OC-01 (varenicline) Nasal Spray for Treatment of the Signs and Symptoms of Dry Eye Disease

Our lead product candidate, OC-01, a highly selective nicotinic acetylcholine receptor (nAChR) agonist, is being developed as a preservative-free nasal spray to treat the signs and symptoms of dry eye disease. The parasympathetic nervous system, the “rest and digest” system of the body, controls tear film homeostasis partially via the trigeminal nerve which is accessible within the nose. Our novel approach leverages the parasympathetic nervous system to promote natural tear film production and re-establish tear film homeostasis.

Human tear film is a complex mixture of more than 1,500 different proteins, including growth factors, antibodies, and numerous classes of lipids and mucins.  This complex tear film coating is responsible for forming the primary refracting surface of the cornea, as well as protecting and moisturizing the cornea. The Lacrimal Functional Unit (LFU), which is controlled by the parasympathetic nervous system, is comprised of glands and cells responsible for producing the layers that comprise healthy tear film. To stimulate the LFU, we are targeting a class of receptors called nicotinic acetylcholine receptors (nAChR) that are located on the trigeminal nerve and readily accessible within the anterior nasal cavity. Administered as a preservative-free, aqueous nasal spray, OC-01 (varenicline) nasal spray’s novel mechanism of action has been shown in preclinical and clinical studies to activate the trigeminal parasympathetic pathway to stimulate natural tear film production.

We believe that increasing tear film volume and re-establishing tear film homeostasis will address the fundamental characteristic of dry eye disease, regardless of etiology, and has the potential to treat a broad population of patients throughout the dry eye continuum.

OC-01 (varenicline) Nasal Spray for Treatment of the Neurotrophic Keratopathy

Neurotrophic Keratopathy (NK) is a disease characterized by decreased corneal sensitivity and poor corneal healing. The most common causes of corneal sensation loss are viral infection (herpes simplex virus and herpes zoster keratoconjunctivitis), followed by chemical burns, physical injuries, and ocular surface surgery. In addition, systemic diseases such as diabetes and multiple sclerosis may decrease sensory nerve function or damage sensory fibers. NK can be classified broadly into 3 stages: Stage 1 (mild) consists of ocular surface irregularities and reduced vision; Stage 2 (moderate) exhibits a non-healing persistent defect of the corneal epithelium; and Stage 3 (severe) exhibits corneal ulceration, which may progress to corneal melting and perforation. If not adequately addressed, NK can lead to vision loss and a breakdown of corneal integrity, potentially leading to cornea transplantation.

We believe that OC-01 nasal spray may activate natural tear production via the trigeminal parasympathetic pathway, bypassing the impaired corneal nerves to stimulate the production of natural tear film to treat or prevent progression of NK.

OC-01 Nasal Spray in Action

OC-01 nasal spray is an investigational new drug and has not been approved for any use in any country. The safety and efficacy of OC-01 nasal spray have not been established.