Acuity
Pharmaceuticals, an ophthalmic pharmaceutical company, today announced that
its lead clinical compound bevasiranib sodium, formerly known as Cand5,
appeared safe and showed clinical evidence of efficacy in the first results
from the Phase II C.A.R.E.(TM) trial for the treatment of wet AMD.
Bevasiranib is a first-in-class small interfering RNA (siRNA) therapeutic
designed to turn off or silence the gene that produces VEGF, the growth
factor believed largely responsible for wet age-related macular
degeneration (wet AMD), a leading cause of adult blindness. The findings
are being presented today at the annual meeting of the American Society of
Gene Therapy in Baltimore, Maryland.
“As a clinician I am encouraged by these initial results, which show a
trend toward dose dependent efficacy without discernable adverse effects in
these AMD patients with serious progressive disease,” said Lawrence
Singerman M.D., founder and executive secretary of the Macula Society,
clinical professor of ophthalmology at Case University and a principle
investigator for the study at its Cleveland site. “Bevasiranib and its
unique mechanism has the strong potential to be useful as a maintenance
therapy, first using a VEGF antagonist to ‘mop-up’ existing VEGF and then
using bevasiranib to stop further production in the eye, and it may also be
an effective new therapy for wet AMD on its own.”
Top-line Phase II results presented at the conference show bevasiranib
to be safe and well tolerated, with a dose related effect evident across
multiple endpoints including near vision, lesion size (CNV) and time to
rescue. Clinical data will be presented at the meeting of the American
Society of Retinal Specialists in September.
“These positive findings of safety and efficacy continue Acuity’s
leadership in developing gene silencing therapies for diseases of the eye,
and represent the first-ever clinical proof-of-concept for an siRNA-based
therapy,” said Dale Pfost, Ph.D., president and CEO of Acuity. “Overall
these positive initial results give us the foundation we need to take
bevasiranib into Phase III clinical trials, which we expect to begin next
year.”
The Acuity Cand5 Anti-VEGF RNAi Evaluation, or C.A.R.E. study, was a
randomized, double-masked trial that included three dose levels of
bevasiranib (Cand5) tested in 129 patients at 28 sites nationwide. The
study focused on patients with serious disease, including those who had
rapidly degenerating retinal lesions or had failed previous treatments and
it excluded patients with slow-growing occult lesions. Bevasiranib
demonstrated signs of efficacy at all dose levels.
“It is noteworthy that the groundbreaking C.A.R.E. trial was designed
and executed with the highest scientific standards and Acuity delivered
positive results from 28 clinical sites in just nine months from the
initiation of the trial,” said Jason Slakter, M.D., macular disease
specialist at the Vitreous Retina Macula Consultants of New York and
clinical professor of ophthalmology at N.Y.U. School of Medicine. “The
novel mechanism of bevasiranib has the potential to provide important
benefits in the treatment of wet AMD now reaching critical levels as our
population grows older, and I look forward to working with Acuity
researchers on the Phase III trials for bevasiranib that are now being
planned.”
Bevasiranib uses RNA interference (RNAi) to silence genes that promote
the overgrowth of blood vessels that lead to vision loss in wet AMD. This
shuts down the production of vascular endothelial growth factor (VEGF),
which has been shown to be the central stimulus in the development of wet
AMD. Bevasiranib is administered directly into the eye and does not affect
the patient systemically, an important safety consideration. The Phase III
clinical trial will further examine efficacy parameters, as well as dosing
and dose scheduling regimens.
About Wet AMD
Wet age-related macular degeneration (wet AMD) is the number one cause
of irreversible vision loss in the developed world, and its incidence is
growing rapidly. Advanced age is the main risk factor for wet AMD, and it
is expected to become an increasingly common condition as the population
grows older. An estimated 1.65 million Americans have wet AMD today and an
estimated 11 million people worldwide will have AMD by 2013. Existing
treatments for wet AMD are of limited efficacy and fail to halt disease
progression in many patients. In the search for more effective treatments,
researchers are targeting vascular endothelial growth factor (VEGF), which
has been shown to be a key cause of the excess growth of blood vessels that
results in loss of vision.
About Acuity Pharmaceuticals
Founded in 2002, Acuity Pharmaceuticals is an ophthalmic pharmaceutical
company applying proprietary technologies to the treatment and prevention
of ophthalmic diseases. Acuity’s lead clinical compound, bevasiranib, a
small- interfering RNA (siRNA) therapeutic targeting VEGF, is in clinical
trials for two of the leading causes of adult vision loss. Acuity recently
completed a Phase II trial of bevasiranib in age-related macular
degeneration and is currently conducting a Phase II trial for diabetic
macular edema. Acuity is applying its drug development expertise to a
growing pipeline of novel agents for ophthalmic conditions. In support of
these programs, Acuity is also developing proprietary technologies for
ocular drug delivery. For more information, see the company’s website at
.
Acuity Pharmaceuticals
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