OcuNexus Clinical Candidates and Indications
The active ingredient in OcuNexus lead drug product candidate Nexagon® is a natural, unmodified oligonucleotide (30-mer) that downregulates expression of the key gap junction protein Cx43. Cx43 is upregulated and overexpressed in pathological conditions such as chemical or thermal injuries to the eye, resulting in non-healing of the crucial epithelial layer of the cornea. Without an epithelium which is a major protective layer of the eye to external stimuli, as well as maintaining a moist and clear surface enabling good vision, the cornea would fibrose or perforate, resulting in loss of vision. Several patients experiencing the devastation of sight threatening chemical or thermal injury to the eye resulting in a persistent defect of the crucial first layer of the cornea, the epithelium, and that were non responsive to current standard of care (SOC) treatment, have been treated compassionately with Nexagon®. Nexagon® is formulated in a thermoreversible gel placed under a contact lens or amniotic membrane to ensure contact with the corneal/conjunctival surface for sufficient time to get the active drug into the cell.
The first patient treated with Nexagon® was injured when the hose he was using to spray concrete became blocked. In the process of unblocking the hose the patient received a blast of the alkali concrete substance in his eye. The treatment he received is outlined below along with his recovery following a single treatment with Nexagon® placed under the amniotic membrane that was in place as part of the standard of care treatment being provided. Besides the first patient described above, nine other patients have been treated compassionately following failure of SOC, five of whom are represented in the chart below. All experienced epithelial recovery and a majority restoration of vision following re-epithelialization.
Nexagon® has been discussed with the FDA and the clinical development plan is being finalized to take the product into a Pivotal Phase 3 Clinical Trial in Q2 2017. Nexagon® has orphan designation from the FDA.