Open-Label Uncontrolled Study of Lucentis Showed Improvement in Vision With Five to Six Doses at One Year.

MIAMI -- The University of Miami's Bascom Palmer Eye Institute announced today the one-year results of the PrONTO Study, a clinical study of 40 patients with neovascular age-related macular degeneration (AMD) who received ocular injections of ranibizumab (Lucentis(TM)). The PrONTO Study (Prospective Optical Coherence Tomography Imaging of Patients with Neovascular Age-Related Macular Degeneration Treated with Intra-Ocular Lucentis(TM)) is a prospective, open-label, uncontrolled study designed to evaluate the use of optical coherence tomography (OCT) as a way to determine when patients needed an injection of Lucentis. Lucentis, an investigational drug designed to bind and inhibit VEGF-A, a protein believed to play a critical role in angiogenesis, the body's process of making new blood vessels, is currently under review with the U.S. Food and Drug Administration for the treatment of neovascular AMD, the wet form of AMD.

The average patient in the PrONTO Study received five or six injections over a one-year period. OCT, a noninvasive, fast, quantitative imaging technique was used to follow the patients and determine when retreatment was necessary. Overall, average vision improved in the treated eye almost two lines after one year. Additionally, 82% of patients had the same or better vision after one year and 35% of patients experienced a two-fold improvement in vision as defined by gaining three lines of vision on a standardized visual acuity chart. The improvement in vision was associated with a decrease in the leakage of fluid from the abnormal blood vessels in these eyes. If left untreated, patients with wet AMD can often lose significant vision after one year.

"By using OCT in this uncontrolled study, we were able to give fewer injections into the eye and observed vision improvement for most of our 40 patients," said Philip J. Rosenfeld, M.D., Ph.D., associate professor of ophthalmology at the Bascom Palmer Eye Institute and the principal investigator of the PrONTO study. "In addition to the improved vision, Lucentis appears to have caused a reduction in leakage from the abnormal blood vessels, and we observed a restoration of normal macular anatomy in this study."

Another aspect of the PrONTO study was to determine how quickly Lucentis stopped the leakage of fluid and the growth of abnormal blood vessels in these wet AMD patients. Using OCT as a diagnostic tool, Rosenfeld found an improvement in the macular anatomy within one day following the injection resulting in vision improvement within two weeks and continued improvement in most patients over the first three months of the study.

Aside from the typical complaints of mild discomfort after an injection into the eye, there were no treatment-related or drug-related serious ocular or systemic adverse events during the first year of the study. Overall, the drug appeared to be safe and the injection process was very well tolerated by patients. To date, no one left the study or refused an injection, and the study is ongoing through two years.

After three months, the improvement appeared to be maintained in most patients with intermittent injections every 3 to 4 months as determined using OCT. However some patients may need more frequent injections and some patients may never need another injection. After 1 year, 17.5% of patients never needed another injection and 20% only needed 1 additional injection, so it's highly variable but easily monitored using OCT.

"It's not surprising that everyone is a little different and OCT allows us to tailor our therapy to the individual patient," said Carmen A. Puliafito, M.D., M.B.A., professor of ophthalmology and chairman of Bascom Palmer Eye Institute. OCT, now widely available throughout the world, was co-invented by Puliafito, who was also a co-investigator of the PrONTO study. OCT produces high speed, non-invasive, cross-sectional images of body tissue in high resolution, which is 10 times greater than magnetic resonance imaging (MRI) or ultrasound.

Rosenfeld presented the one-year PrONTO outcomes on May 2, 2006 at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) held in Fort Lauderdale.

Lucentis, also known as ranibizumab, is being developed by Genentech Inc., and the Novartis Ophthalmics Business Unit, and is in late phase III clinical trials, with a response from the FDA expected by the end of June 2006. The Phase III Lucentis studies are evaluating monthly injections of Lucentis for two years.

The neovascular or "wet" form of age-related macular degeneration, a degenerative condition, is the most common form of irreversible blindness and vision impairment among people 50 years of age and older in the U.S. An estimated 8 million Americans over the age of 50 suffer from AMD with 1.3 million at risk of developing wet AMD and severe vision loss. This number is expected to double by the year 2020 as baby boomers get older.

Patients with macular degeneration are thought to have elevated levels of vascular endothelial growth factor (VEGF) in their affected eyes. VEGF is a protein that causes abnormal blood vessels to grow, leak, bleed, and damage the macula resulting in vision loss. New anti-VEGF drugs work by blocking this protein and the formation of abnormal blood vessels that grow in the eye.

Treatment for AMD traditionally included thermal laser photocoagulation therapy and photodynamic therapy. Although neither treatment is a cure for wet AMD, each treatment may slow the progression of vision decline or stop future vision loss. "While these therapies are effective for certain types of wet AMD, pharmacotherapy (drug therapies) represents the new era in macular degeneration treatment," said Puliafito.

"Anti-VEGF drugs have the potential to revolutionize the care and treatment of this disease and there is now significant hope for patients with wet AMD," said Dr. Puliafito. "Bascom Palmer continues its leadership role as the world's best place for treatments for wet macular degeneration."

Rosenfeld emphasized that, "Lucentis is an investigational therapy that hasn't been approved for use by the FDA. It isn't a cure and it's not the right treatment for everyone with wet AMD. Individual results may vary."

Bascom Palmer Eye Institute

Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine is ranked the #1 eye hospital in the nation by U.S. News & World Report. Having earned an international reputation as one of the premier providers of eye care in the world, Bascom Palmer is also ranked #1 in patient care and residency training by Ophthalmology Times. As the largest ophthalmic care, research and educational center in the southeastern United States, more than 200,000 patients with nearly every ophthalmic condition are treated each year and more than 10,000 surgeries are performed annually. Founded in 1962, Bascom Palmer has patient care facilities in Miami, Palm Beach Gardens, Naples, and Plantation, Florida. For additional information about Bascom Palmer Eye Institute, please contact Cynthia Birch at [phone], [e-mail] or visit the website at http://www.bascompalmer.org

Risk Factors for Macular Degeneration

--Age - 60 years of age and older

--Race - Whites are much more likely to lose vision from AMD than Blacks

--Gender - Women tend to be at greater risk than men

--Family history of AMD

--Smoking may increase the risk

--Obesity

Symptoms of Macular Degeneration

--Words appear blurry while reading, requiring greater illumination to see details

--Inability to recognize faces at a distance

--Blurred or blind spot in the center of vision

--Straight lines appear wavy or crooked

--Rapid loss of central vision