UNION CITY, Calif., Oct. 31 /PRNewswire/ -- MacuSight(TM), Inc., a developer of innovative therapeutics for the treatment of severe ocular diseases and conditions, today announced the initiation of a Phase I study of its lead product candidate in patients with wet age-related macular degeneration (wet AMD). This trial, which will enroll a total of 30 patients, is designed to evaluate the safety and tolerability of MacuSight's proprietary formulation of sirolimus (rapamycin) when administered in various doses through two different types of ocular injections.
"Today's announcement is particularly exciting as it comes on the heels of our recently initiated sirolimus trial for the treatment of diabetic macular edema," said David A. Weber, Ph.D., MacuSight's president and chief executive officer. "We believe that conducting trials in two severe ocular indications concurrently serves as a true testament to the versatility and overall therapeutic potential of sirolimus in the eye. We look forward to successfully completing both of these trials and continuing to advance this compound through clinical development."
Investigators for this randomized, open-label, dose-escalation study will treat patients with a single subconjunctival (just under the lining layer over the white of the eye) or intravitreal (into the back of the eye) injection of MacuSight's sirolimus formulation. The trial will include six treatment arms with patients receiving one of three doses of sirolimus via subconjunctival injection or one of three doses of sirolimus via intravitreal injection. Each administration of sirolimus will provide the patient with exposure to the compound for up to approximately three months.
"Direct inhibitors of vascular endothelial growth factor or VEGF have rapidly become the standard of care for patients with exudative [or wet] AMD. It appears that the next opportunity to advance the treatment of this disease will likely involve VEGF inhibition in combination with the targeting of additional factors implicated in its development," said 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. "As such, we believe that sirolimus has the potential to play an important role in the evolution of therapeutics for exudative AMD. This is based on the compound's demonstrated ability to broadly inhibit VEGF and other key pro-angiogenic factors, while also impacting a number of important proliferative and inflammatory pathways."
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 well as the treatment of diabetic macular edema (DME). The company recently initiated a similar Phase I trial in patients with DME.
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 six 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.