Lux Biosciences announces results of its LX214 human safety study for dry eye syndrome

Sep 24 2009

Lux Biosciences, Inc. a privately held biotechnology company focused on the treatment of ophthalmic diseases, today announced results from a Phase 1 human safety and an open-label pilot efficacy study of the company’s potential best-in-class therapy for dry eye, LX214 (topical mixed nanomicellar formulation of voclosporin). Randomized, double-masked, placebo-controlled data from 30 healthy volunteers showed LX214 to be well tolerated at the two doses (0.02% and 0.2%) studied, with safety and tolerability measurements (pain, burning, reddening, photophobia, foreign body sensation and others) indistinguishable from placebo.

An additional cohort of five patients with severe dry eye syndrome was treated with LX214 in both eyes twice a day for 14 days at the target 0.2% concentration. Data from these patients confirmed that systemic exposure to voclosporin was very low and below the threshold level where measurement of voclosporin blood concentrations would be required in future studies. Signs and symptoms of disease were also assessed in this cohort of patients. Despite the small sample size and short duration of treatment, clinically meaningful improvements were noted in both signs (tear production) and symptoms (OSDI index) at both 7 and 14 days, a trend that bodes well for longer term, controlled studies to follow.

“The benign safety and tolerability results in humans demonstrated in both these trials confirm the positive profile exhibited by LX214 in preclinical studies, which showed this drug candidate to establish therapeutic levels in relevant ocular tissues and to be non-irritating when applied topically to the eye,” said Eddy Anglade, M.D., Lux Biosciences’ Chief Medical Officer. “Moreover, even though the 14 day results in severe dry eye patients were based on a small, uncontrolled sample, they presented quite encouraging indicators of efficacy.”

LX214, developed in cooperation with Ashim Mitra, Ph.D., University of Missouri Curator’s Professor of Pharmacy and Director of Translational Research at UMKC School of Medicine School of Pharmacy, University of Missouri-Kansas City, is a proprietary formulation of Lux Biosciences’ next generation calcineurin inhibitor, voclosporin. Utilizing voclosporin and Lux’s proprietary mixed nanomicellar formulation technology, LX214 establishes high levels of drug for 24 hours in relevant ocular tissues following a single administration, including the lacrimal gland and submandibular lymph nodes, suggesting that LX214 may be efficacious and suitable for once daily dosing.

“We are very pleased by the results of this initial human study, which supports the potential of LX214 to become a best-in-class therapy in dry eye, an indication that affects as many as 10 million patients in North America and Europe,” said Ulrich Grau, Ph.D., President and Chief Executive Officer of Lux Biosciences. “Lux has been able to transform voclosporin, a molecule with a mode of action that is applicable to a variety of ophthalmic diseases, into a pipeline of tailored, proprietary products that maximizes the value of this asset. LX214, the topical mixed nanomicellar formulation of voclosporin, was developed and advanced into clinical testing in less than two years, and we view it as the ‘crown jewel’ in the earlier stage Lux Biosciences pipeline. In addition, the mixed nanomicellar technology has now been validated in humans adding a highly valuable proprietary platform to the Lux portfolio.”