OPKO Health, Inc announced that, following the recommendation of the Independent Data Monitoring Committee (IDMC), it had decided to terminate its phase III clinical study of Bevasiranib, a first-in-class siRNA, for the treatment of wet age-related macular degeneration (wet-AMD). Although preliminary data, needing further analysis, show activity of Bevasiranib when used adjunctively with Genentech's Lucentis, review of the data by the IDMC indicated that the trial, as structured, was unlikely to meet its primary end point. There were no systemic safety issues identified and local ocular safety was generally unremarkable.

"While we are clearly disappointed with the preliminary results of this fully-enrolled study, the indications of activity are encouraging and we look forward to fully analyzing the data in the coming weeks," said Dr Phillip Frost, chairman and CEO of OPKO Health. "We remain committed to the continued development of our siRNA portfolio targeting Vascular Endothelial Growth Factor (VEGF), including our recently announced VEGFA165b sparing siRNA." These new proprietary siRNA's are designed to inhibit the angiogenic Vascular Endothelial Growth Factor A165 (VEGFA165 isoform) but spare the anti-angiogenic VEGFA165b isoform.

VEGFA165 is known to play a critical role in diseases of the eye where the underlying cause of the problem is abnormal growth of blood vessels, such as in wet age-related macular degeneration. To the contrary, data show that VEGFA165b is an inhibitor of abnormal vessel growth.

Miami-based OPKO is a specialty healthcare company engaged in the development, marketing, and sales of novel agents and both diagnostic and therapeutic devices for the management of ophthalmic diseases.