MacuSight, a developer of innovative therapeutics for the treatment of severe ocular diseases and conditions, has received fast track designation from the United States Food and Drug Administration (FDA) for Perceiva, the company's proprietary ocular sirolimus product, for the treatment of diabetic macular edema (DME). MacuSight is currently advancing Perceiva in a broad phase-2 clinical programme across multiple large ocular indications. The company recently announced that it had completed enrolment in four separate phase-2 studies which are evaluating Perceiva as a treatment for DME, neovascular (wet) age-related macular degeneration (wet AMD) and dry eye syndrome. The company plans to announce data from these studies in the first half of 2010.

FDA's process for providing fast track designation is intended to facilitate the development and expedite the review of drugs that treat serious or life-threatening diseases and address an unmet medical need. With this designation, MacuSight will be provided the opportunity to submit portions of a New Drug Application (NDA) for Perceiva as they are completed instead of filing all completed portions of the NDA at one time. As a result, FDA may begin its review of the application as portions are received, potentially accelerating the review and approval process.

"By granting Fast Track status to Perceiva for the treatment of DME, the leading cause of vision loss among working-age adults, FDA is providing an accelerated pathway for delivering this potentially important therapeutic to patients in need of effective treatment options," stated Joel Naor, chief medical officer of MacuSight. "Based on our clinical research to date with Perceiva in DME patients, we believe that this drug candidate may offer those patients a safe and effective treatment. We remain focused on advancing the clinical development of Perceiva for DME and other ocular diseases in as expeditious a manner as possible and look forward to announcing data from our phase-2 clinical programme in the first half of 2010."

MacuSight recently announced that it had completed enrolment of 131 patients in its ongoing phase-2 DME study of Perceiva, known as the Diamond trial. This randomized, dose-ranging, placebo-controlled study consists of four treatment arms (three different doses and placebo) administered by a minimally invasive subconjunctival injection. Patients enrolled in this trial are administered Perceiva at baseline and again at three months. The primary endpoints for the trial are visual and anatomical improvements measured at six months, three months following the second administration of Perceiva. MacuSight plans to announce data from this study, along with the company's three additional ongoing phase-2 trials, in the first half of 2010.

Two phase-1 clinical trials of Perceiva in patients with DME and wet AMD have been completed. Findings from both studies, which included a total of 80 patients, demonstrated the product to be safe and well-tolerated in all doses tested with two different routes of administration (minimally invasive subconjunctival injection, as well as traditional intravitreal injection). In addition, investigators from both studies noted improvements in visual acuity consistent with observed beneficial anatomical changes following a single administration of Perceiva. Importantly, these findings were consistent across both routes of administration. These promising results for Perceiva delivered with subconjunctival injection provide the first evidence for the potential of treating retinal disease without direct injection into the back of the eye.

MacuSight is a privately held pharmaceutical company focused on developing proprietary ocular formulations of mTOR inhibitors, including sirolimus (rapamycin), for minimally invasive, sustained, local administration to treat a number of major sight-threatening diseases.