Plans to request that OC-01 (varenicline), Oyster Point Pharma‘s lead therapy candidate for a series of eye disorders, be cleared for testing in a Phase 2 trial in people with neurotrophic keratitis (NK) remain on track, the company announced.

In a recent financial and business update, company officials noted an intent to submit the request, known as an investigational new drug (IND) application, to the U.S. Food and Drug Administration by year’s end.

If cleared, the trial, called OLYMPIA, could begin enrolling eligible patients in the first half of 2021. 

“I am excited for the Oyster Point team to initiate the clinical development of OC-01 to treat neurotrophic keratitis,” Jeffrey Nau, PhD, company president and CEO, said in a press release.

OC-01 is a preservative-free nasal spray initially developed to treat the signs and symptoms of dry eye disease, a common disorder in which inadequate tear production irritates the eyes. If untreated, the condition can cause inflammation and  damage the eyes’ surface tissues over time.

Administered as a nasal spray, OC-01 is a potent, selective, activator of the nicotinic acetylcholine receptor (nAChR). This protein receptor is found on the trigeminal nerve — a large and complex nerve that can be accessed through the nose and is known to control tear formation (via its parasympathetic function), in addition to facial movement and sensation. (Of note, the parasympathetic branch of the nervous system is  responsible for controlling automatic involuntary bodily functions while at rest.)

By stimulating the parasympathetic function of the trigeminal nerve, OC-01 is expected to restore tear production, alleviating the signs and symptoms of dry eye disease.

OC-01 is also being explored as a potential treatment of other eye disorders in which tear production is disrupted. These include NK, a rare degenerative eye disease in which the progressive destruction of the trigeminal nerve impairs tear production and compromises the cornea’s ability to self-repair, leading to tissue breakdown.

The cornea, the clear and outer protective layer of the eye, is responsible for controlling the amount of light that enters.

The Phase 2 OLYMPIA study will evaluate the efficacy and safety of OC-01 in patients with stage 1 and 2 NK (stage 1 refers to a mild disease of the epithelium, or the cornea’s outer layer; stage 2 refers to persistent damage to the epithelium).

It expects to enroll 200 patients, randomly assigned to either 1.2 mg/ml of OC-01 or a placebo nasal spray used twice a day for 56 days.

“Based on OC-01’s novel mechanism of action, we believe that the production of natural tear film may have the ability to provide a therapeutic benefit for a number of ocular surface diseases,” Nau said.