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.
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.
macusight