UNION CITY, Calif., Sept. 26 /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 diabetic macular edema (DME). 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.
"We have been eagerly anticipating this day for quite some time and are very excited to have reached the milestone of advancing our lead product into clinical trials," said David A. Weber, Ph.D., MacuSight's president and chief executive officer. "This trial is a critical first step in our examination of sirolimus as a treatment for severe ocular diseases and conditions. It will provide important information as it relates to the compound's safety, tolerability, dosing and administration and, in turn, will guide the product's future 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.
"The potential of sirolimus to impact at multiple points in the DME pathway positions the compound as an ideal candidate agent for therapy of this condition," stated Julia Haller, M.D., Robert Bond Welch Professor of Ophthalmology, Wilmer Institute, Johns Hopkins University School of Medicine and member of MacuSight's scientific advisory board. "Not only does sirolimus inhibit the production, signaling and activity of many cellular factors reported to be relevant to the development of DME, it can also reduce permeability and down-regulate the genes involved in the inflammatory processes that are a key part of the pathophysiology of diabetic retinopathy. For these reasons, we are very excited to have the opportunity to study sirolimus in patients suffering from this condition."
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 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.
As a result of MacuSight's recent product development milestones, the company has secured an additional $8 million in Series A funding from its existing investors. This brings the total of the company's financing to date to $26 million. MacuSight intends to use the proceeds from this financing to fund the development of its lead program, including the ongoing Phase I clinical trials in DME and the initiation of a Phase I study in wet age- related macular degeneration (wet AMD). The company intends to initiate the Phase I wet AMD trial during the fourth quarter of 2006.
About Diabetic Macular Edema
Diabetic macular edema (DME) is a serious manifestation of diabetic retinopathy that involves retinal swelling brought on by the leaking of fluid from small blood vessels within the macula. As the condition develops, central vision becomes blurred. DME can progress fairly rapidly and over just a few years can lead to permanent visual loss. There is a significant unmet need to both reverse the visual loss associated with DME and to help delay the progression of this condition.
About MacuSight
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.
MacuSight, Inc.CONTACT: investors, Stephanie Diaz, +1-415-675-7400, orsdiaz@vidacommunication.com, or media, Tim Brons, +1-415-675-7402, ortbrons@vidacommunication.com, both of Vida Communication (On behalf ofMacuSight)
