LX214

LX214 is a proprietary topical formulation of voclosporin that has been developed for ophthalmic use in collaboration with the University of Missouri at Kansas City. LX214 is a formulation that contains mixed nanomicelles of voclosporin. Voclosporin is normally not soluble in aqueous media; the mixed nanomicelles encapsulate voclosporin and render it soluble, homogeneous and crystal-clear on appearance. LX214, when instilled in the eye, produces high concentrations of voclosporin in the relevant target tissues of the eye. Of note, and unlike the reference product, LX214 establishes therapeutic drug levels in the lacrimal gland and in lymph nodes surrounding the eye. Furthermore, it has been demonstrated that the drug levels in ocular tissues are sustained for 24 hours, presumably allowing for once-daily dosing. Based on testing in animals, LX214 has been found to be non-irritating to rabbit eyes, unlike the reference product. These features render LX214 potentially a best-in-class product.

A Phase 1 clinical study with LX214 was completed in mid 2009. Similar to the results from animals, healthy human volunteers receiving solutions of 0.02% and 0.2% LX214 tolerated either concentration of the product well and their response could not be distinguished from volunteers receiving vehicle control in a double-masked fashion. Moreover, in a small cohort of 5 patients with moderate to severe dry eye, both symptoms, as measured by the ocular surface disease index (OSDI), and signs, as measured by Schirmer’s tear test, were markedly improved. Side effects associated with therapy were no different than symptoms present on initiation of the study, though less in frequency and severity. Although this study was not placebo-controlled, these findings were, nevertheless, encouraging. The product will initially be developed for Dry Eye Syndrome, but also has the potential to be used for other ocular inflammatory diseases.