UNION CITY, Calif., Feb. 25 /PRNewswire/ -- MacuSight(TM), Inc., a developer of innovative therapeutics for the treatment of severe ocular diseases and conditions, today announced positive preliminary data from a Phase 1 study of its lead product candidate in patients with wet (exudative) age-related macular edema (wet AMD). Similar to its Phase 1 study in patients with diabetic macular edema (DME), preliminary results from this prospective study of 30 patients demonstrated that MacuSight's proprietary formulation of sirolimus (rapamycin) was safe and well-tolerated in all doses tested with two different routes of administration. In addition, improvements in visual acuity consistent with anatomical retinal changes were observed following a single administration of sirolimus. These findings were presented at Bascom Palmer Eye Institute's Angiogenesis, Exudation and Degeneration 2008 conference by Pravin U. Dugel, M.D., member of MacuSight's scientific advisory board, managing partner, Retinal Consultants of Arizona, Phoenix AZ, clinical instructor, vitreoretinal diseases and surgery, department of ophthalmology, University of Arizona, and the study's lead investigator.
As part of the design of this randomized, open-label study, investigators evaluated the safety, tolerability and biological activity of sirolimus when delivered by either a subconjunctival or intravitreal injection. The trial showed no evidence of increased intraocular pressure, inflammatory response to treatment, or indication of progression of cataracts. Furthermore, the study provided an initial assessment of sirolimus' biological activity in AMD with patients demonstrating improvements in visual acuity despite extended duration of disease prior to entering the study. Additionally, patients also experienced anatomical improvements as demonstrated by a reduction in retinal thickness. These preliminary findings demonstrated that sirolimus administered via subconjunctival injections was as effective, if not more so, than sirolimus administered via intravitreal injections.
Intravitreal injections (into the central cavity of the eye), while the standard route of administration for current therapies, are uncomfortable for many patients and are accompanied by the risk of serious infection in a small percentage of patients. In contrast, subconjunctival injections (just under the outer layer over the white of the eye) are designed to offer physicians and patients a less invasive and more convenient procedure.
"We are pleased to see the trial's positive safety and tolerability measures combined with promising indications of efficacy marked by consistent and rapid improvements in visual acuity and retinal thickness following a single injection of sirolimus," said Dr. Dugel. "Furthermore, the fact that patients in the subconjunctival injection arms exhibited efficacy measures equal to, and in some cases greater than, those in the intravitreal injection arms indicates that this product also has the potential to offer physicians and patients significant safety and ease of administration advantages."
Also at the Angiogenesis, Exudation and Degeneration 2008 conference, Mark Blumenkranz, M.D., chairman of MacuSight's scientific advisory board and professor and chairman of the department of ophthalmology, Stanford University School of Medicine, presented the final results of MacuSight's Phase 1 study of sirolimus in patients with DME. These findings corroborate the preliminary results which MacuSight announced in October 2007 at the 40th Annual Meeting of The Retina Society.
"We are very encouraged by this study's findings as they provide a compelling demonstration of sirolimus' potential therapeutic activity in patients with wet AMD. This is particularly exciting as these results are consistent with previously announced Phase 1 data for our sirolimus product in patients with diabetic macular edema," stated Joel Naor, M.D., chief medical officer of MacuSight. "Based on the positive findings from both our wet AMD and DME studies, we are preparing to initiate Phase 2 trials in both of these important indications, as well as additional ocular diseases and conditions."
MacuSight intends to complete its collection and analysis of all data from its wet AMD trial and present final findings at the Retinal Physician Symposium in Naples, Florida in May 2008.
Sirolimus, originally known as rapamycin, is a highly-potent, broad-acting compound that has demonstrated the ability to combat disease through multiple mechanisms of action including immunosuppressive, anti-angiogenic, anti- migratory, anti-proliferative, anti-fibrotic and anti-permeability activity. Based on the versatility associated with these multiple mechanisms of action, MacuSight believes that its sirolimus product may serve as a potentially highly-efficacious therapeutic for a wide range of ocular diseases and conditions, including the treatment and prevention of wet AMD.
As the active pharmaceutical ingredient in the FDA-approved products Rapamune(R) and CYPHER(R) Sirolimus-eluting Coronary Stent, sirolimus has been safely administered to humans for more than eight years. MacuSight has developed a proprietary minimally-invasive, sustained administration approach for its novel liquid sirolimus formulation which it believes will provide the product with significant competitive advantages related to convenience, ease- of-use, compliance and safety.
About Wet Age-Related Macular Degeneration (AMD)
Age-related macular degeneration (AMD) is the leading cause of visual loss in the western world. This disease of the aging eye results in loss of the sharp, central vision that is necessary for clearly seeing objects and undertaking routine tasks including reading and driving. AMD occurs in both wet and dry forms, with the wet form accounting for the vast majority of cases of AMD-related blindness and progressing much more rapidly than dry.
It is presently estimated that wet AMD affects approximately two million Americans. Growing at an annual rate of approximately 13%, it is expected that the total number of patients will exceed 3.5 million by 2010. Industry experts expect the total U.S. market opportunity for wet AMD therapeutics to reach close to $2 billion by that time.
MacuSight is a privately-held pharmaceutical company focused on developing innovative therapeutics for the treatment of severe ocular diseases and conditions. The company is dedicated to preserving patients' vision by identifying known, highly-potent and broad-acting small molecule drug compounds that may possess efficacy in treating and/or preventing diseases or conditions of the eye. As part of its unique product development philosophy, MacuSight also concentrates on the optimal delivery of these compounds into the eye. By combining its compounds with innovative delivery approaches, the company strives to optimize the efficacy, safety, convenience and cost- effectiveness of its product candidates.
The company's lead development program is centered on advancing sirolimus (rapamycin) as a potential next-generation therapeutic for the treatment and prevention of wet age-related macular degeneration (wet AMD) and the treatment of diabetic macular edema (DME), a manifestation of diabetic retinopathy.
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