Othera drug candidate enters phase II study

Othera’s topical antioxidant eye drop, OT-551, will be investigated in patients with geographic atrophy (GA), a sign of dry or atrophic age-related macular degeneration (AMD). The study will assess OT-551’s potential to slow enlargement of the atrophic area in the macula and prevent progression to the vision-threatening advanced stages of AMD.

This phase II study will enroll ten patients with bilateral GA. Participants will receive three daily doses of OT-551 eye drop over two years. The primary efficacy measure will be percent change in area of GA in each eye, as measured by fundus photo grading. Secondary measures are to include: progression to advanced neovascular or wet AMD, changes in contrast sensitivity, and best-corrected visual acuity, and number of eyes developing advanced AMD.

OT-551 is a low molecular weight compound that has shown benefit in various animal models of macular degeneration, exhibiting both anti-inflammatory and anti-angiogenic effects. It has high bioavailability in the eye and distributes to the retina after topical dosing. OT-551 is also currently being tested for its ability to prevent or slow the progression of post-vitrectomy nuclear cataract. Further phase II studies are planned with OT-551 for AMD and other indications.

AMD is the leading cause of blindness in the US in persons over age 55. The only current therapy for dry AMD is high-dose oral vitamin and zinc supplements, which the NEI showed to be modestly effective in slowing progression of intermediate to advanced AMD.