Verseon Reveals Preclinical Data on Novel Drug Candidates to Treat Diabetic Macular Edema

• Candidates show good pharmacokinetic properties for topical ophthalmic dosing.
• In preclinical testing, the candidates demonstrated reduced leakage into the retina and shorter mean circulation times – suggesting a slowing of the disease progression.
• Some of the candidates may be suitable for oral dosing.

Verseon presented preclinical data on multiple novel drug candidates for the treatment of diabetic macular edema (DME) at the 2017 BIO International Conference in San Diego, last week. Diabetic macular edema is the leading cause of blindness in the diabetic population according to the National Institutes of Health.1

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Left to right: normal vision; blurred vision due to DME; floating spots (hemorrhages) due to advanced diabetic retinopathy. Photos courtesy of the National Eye Institute, National Institutes of Health. (Photo: Business Wire)

Verseon is developing plasma kallikrein inhibitors that target a validated disease pathway2 and treat an underlying cause of DME. The current standard of care comprises monthly injections into the eye and laser treatments, which are associated with side effects such as inflammation, infections, and cataracts.3 To avoid these unwanted effects, Verseon is designing its inhibitors for eye-drop or oral delivery.

Studies estimate that one in three people living with diabetes for more than 20 years may develop DME.4 Chronically high blood sugar associated with diabetes can weaken the blood vessels in the eye, leading to fluid leaking into the retina (edema). Over time, fluid may accumulate in the macula, the central region of the retina (see illustration), resulting in swelling, blurred vision, and eventually central vision loss (see photos).i If current trends continue, researchers estimate that about one in three US adults could be suffering from diabetes by 2050,5 leading to a sharp increase in the number of people affected by DME.

Recent research suggests that the serine protease plasma kallikrein may be a promising new target for the treatment of DME.ii The level and activity of plasma kallikrein are both known to be upregulated in the eyes of DME patients. This results in the activation of inflammatory pathways and vasodilation in the retina, leading to edema. By inhibiting plasma kallikrein, Verseon’s drug candidates target the direct cause of downstream inflammation caused by this hyperactivity and can potentially lower leakage into the retina.

“We are designing several series of compounds in parallel, covering both topical and oral delivery,” said Dr. David Kita, Verseon’s Vice President of R&D, who presented preclinical data on the Company’s drug candidates at the recent BIO 2017 conference. “This includes preclinical experiments to test whether our drugs reach the back of the eye when applied as eye drops.”

“At BIO, we presented pharmacokinetic data for two series of compounds, which reach good exposure in the relevant tissues of the eye when administered topically,” he said. “In addition, some other drug candidates show high oral bioavailability, indicating that they may be suitable for oral dosing.”

Dr. Kita also presented an in-vivo efficacy study with systemic dosing. In this experiment, Verseon’s compounds demonstrated reduced leakage into the retina and shorter mean circulation times, both of which suggest a slowing of the disease progression.

The series of kallikrein inhibitors presented at the BIO conference are novel chemical entities discovered and developed by Verseon’s scientists using the Company’s platform. DME is Verseon’s second therapeutic area, following their anticoagulation program, which has produced multiple novel direct thrombin inhibitors with low bleeding risk.

About Verseon

Verseon (AIM: VSN) employs its proprietary, computational drug discovery platform to develop novel therapeutics that are unlikely to be found using conventional methods. The company has three drug programs in anticoagulation, diabetic macular edema, and oncology._______________________________1 “Managing diabetic macular edema” https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3158878/2 Bhat, M., Pouliot, M., Couture, R., and Vaucher, E. (2014) The Kallikrein-Kinin System in Diabetic Retinopathy. Prog. Drug Res. pp. 111–143.3 Facts about diabetic macular oedema – Moorfields Eye Hospital http://www.moorfields.nhs.uk/sites/default/files/diabetic-macular-oedema.pdf4 Klein, R., et al. The Wisconsin Epidemiologic Study of Diabetic Retinopathy. Ophthalmology, Volume 91, Issue 12, 1464 - 14745 Boyle, J. P., Thompson, T. J., Gregg, E. W., Barker, L. E., & Williamson, D. F. (2010). Projection of the year 2050 burden of diabetes in the US adult population: dynamic modeling of incidence, mortality, and prediabetes prevalence. Population Health Metrics, 8, 29. http://doi.org/10.1186/1478-7954-8-29

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