MacuSight's clinical development update on its proprietary ocular sirolimus product

Sep 30 2009

MacuSight, a developer of innovative therapeutics for the treatment of severe ocular diseases and conditions, today provided a clinical development update for Perceiva, the company's proprietary ocular sirolimus product. The company has completed patient enrollment in four ongoing Phase 2 clinical trials of Perceiva and plans to announce data from these studies in the first half of 2010. All four studies are being conducted at investigational sites in the United States. These studies, which are evaluating Perceiva as a treatment for diabetic macular edema (DME), neovascular (wet) age-related macular degeneration (wet AMD) and dry eye syndrome, are part of MacuSight's broad Phase 2 Perceiva clinical program which spans five major ocular disease indications.

"The completion of enrollment in these important clinical trials, which was achieved rapidly and, in some cases, ahead of our study timelines, represents a key milestone in our Phase 2 clinical program. Based on promising data from our two Phase 1 studies, as well as Perceiva's broad biological activity relevant to multiple ocular diseases, we elected to undertake this large Phase 2 program targeting five major ophthalmic disease markets," stated David A. Weber, Ph.D., president and chief executive officer of MacuSight. "We are excited by the progress that is being made with our ongoing clinical work and look forward to presenting the results early next year."

Perceiva Phase 2 Clinical Program Overview

MacuSight's DME study, known as the DIAMOND trial, has completed enrollment of 131 patients at 39 investigational sites. 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.

Additionally, the company is conducting two studies in patients with wet AMD. The first trial, which is a monotherapy study in newly diagnosed patients not previously treated for the disease, is intended to demonstrate Perceiva's biological activity in wet AMD. MacuSight has completed enrollment of 20 patients in this single-site, open-label, parallel study. Patients enrolled in this re-dose trial are administered Perceiva by either subconjunctival injection or the traditional, more invasive intravitreal injection at baseline, again at two months and a final time at four months. The primary endpoints for the trial are changes in visual acuity and retinal thickness measured at six months.

MacuSight's second wet AMD study, known as the EMERALD trial, is designed to evaluate the therapeutic potential of Perceiva in combination with Lucentis® in patients with wet AMD. The company has completed enrollment of 62 previously treated wet AMD patients at 28 investigational sites. Patients in this randomized, dose-ranging, placebo-controlled study are being administered Perceiva via subconjunctival injection, along with Lucentis, at baseline and again at two months. Roche's Lucentis is currently approved by the United States Food and Drug Administration for the treatment of wet AMD. The primary endpoints of this combination trial are changes in visual acuity and retinal thickness measured at four months.

"In conducting these two complementary studies in wet AMD, MacuSight is taking a comprehensive approach to assessing the therapeutic potential of Perceiva in this disease," stated Pravin U. Dugel, M.D., managing partner, Retinal Consultants of Arizona, Phoenix, AZ, clinical instructor, vitreoretinal diseases and surgery, department of ophthalmology, University of Arizona, and a lead investigator of both wet AMD studies. "While evaluating the biological activity of the product on its own is important, there is a rapidly advancing belief in the ophthalmology community that the future of wet AMD treatment lies in combination therapy. Furthermore, there is a critical need for safe and effective wet AMD treatment alternatives that have less frequent administration requirements. This product may possess the potential to address these needs."

MacuSight's fourth Phase 2 trial, in which the company is using Perceiva to evaluate the potential role for sirolimus in the treatment of dry eye syndrome, has successfully enrolled 143 patients at four investigational sites. For this trial, MacuSight worked with Ora, Inc., a leading global clinical research organization located in Andover, MA. As part of this randomized, dose-ranging, placebo-controlled study, patients receive a single subconjunctival injection of Perceiva at baseline. Investigators will evaluate signs and symptoms of dry eye over a period of 28 days following a single injection.

"In addition to these four separate studies that we are overseeing at MacuSight, our Perceiva Phase 2 program includes three trials sponsored by the National Eye Institute of the National Institutes of Health, as well as individual investigators," added Joel Naor, M.D., chief medical officer of MacuSight. "The NEI is conducting studies of Perceiva in DME, dry AMD and anterior uveitis. A separate investigator-sponsored trial in posterior uveitis is also currently enrolling patients. As a result of this extensive clinical development program, more than 500 patients across five major posterior and anterior segment ocular indications will have been dosed with Perceiva by the time the Phase 2 program is complete. This scope is virtually unprecedented for a Phase 2 program in the area of ocular drug development."

Perceiva Phase 1 Clinical Results

MacuSight has completed two Phase 1 clinical trials of Perceiva in patients with DME and wet AMD. 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.