Potentia Pharmaceuticals,
Inc. announced today that it is entering the clinical phase of development
for POT-4, its lead drug candidate for the treatment of age-related macular
degeneration (AMD). POT-4 is a complement inhibitor, which shuts down the
complement activation system that could lead to local inflammation, tissue
damage and upregulation of angiogenic factors such as vascular endothelial
growth factor (VEGF).
Four landmark studies published in April 2005 demonstrated a genetic
link between the complement system and AMD, providing evidence that
complement activation plays a significant role in the cause of the disease.
Less than two years after the publication of the studies, POT-4 will be the
first complement inhibitor tested in patients with AMD.
“These recent data have sparked hope that AMD can be treated with
complement inhibitors, which help treat the early stages of the disease. We
are hopeful that POT-4 may represent a new therapeutic option for patients
with dry and wet forms of the disease,” said Cedric Francois, M.D., Ph.D.,
Potentia’s President and CEO.
AMD is the leading cause of blindness in the elderly of the western
world and affects more than 10 million patients in the United States alone.
The current standard of care for AMD relies primarily on angiogenesis
inhibitors, an approach geared towards the approximately 10-15% of AMD
patients with complications resulting from ocular angiogenesis (growth of
new blood vessels and bleeding in the back of the eye). No drug currently
on the market has been approved for the treatment of the remaining
patients, who suffer from the so-called “dry” form of the disease.
About the Complement System and POT-4
Complement activation is an inflammatory process involving dozens of
plasma proteins, ultimately leading to cell membrane disruption through the
membrane attack complex. Activation of the complement system is an
important part of the body’s defensive immune response against pathogens
such as bacteria and viruses. In spite of its defensive function,
inappropriate or excessive complement activation can have pathological
consequences. Multiple studies published over the past 2 years have
strongly linked the complement system to the pathology of AMD.
POT-4 is a synthetic peptide discovered by Professor John Lambris at
the University of Pennsylvania. It binds tightly to complement component
C3, preventing its participation in the complement activation cascade. As
C3 is the central component of all major complement activation pathways,
its inhibition effectively shuts down all downstream complement activation
that could otherwise lead to local inflammation, tissue damage and
upregulation of angiogenic factors such as vascular endothelial growth
factor.
About Age-Related Macular Degeneration
AMD is the progressive deterioration of the critical central region of
the retina called the macula. This disorder leads to irreversible loss of
central vision. More than 25 million patients worldwide suffer from AMD,
including roughly one-quarter of those 70 years and older. Ten to fifteen
percent of patients with AMD develop a complication in which leaky blood
vessels grow into the retina. This form of the disease is referred to as
‘neovascular’ or ‘wet’ AMD, as opposed to ‘atrophic’ or ‘dry’ AMD when this
complication does not emerge. Pharmacological treatments of AMD are
primarily limited to drugs that inhibit blood vessel growth and leakage and
are therefore approved for use only in patients with wet AMD.
About Potentia’s AMD Program
With the ultimate goal of making AMD a preventable disease, Potentia’s
AMD program focuses on developing new therapies that target AMD early in
the disease process. Potentia’s AMD pipeline includes POT-4 formulated to
protect patients from the disease for prolonged periods of time after each
treatment.
About Potentia Pharmaceuticals, Inc.
Potentia Pharmaceuticals, Inc. (potentiapharma/) is a
privately held, biotechnology company based in Louisville, KY. Together
with its corporate and academic partners, Potentia is developing new
approaches to the treatment of complement-related inflammatory diseases
such as AMD.
This press release contains “forward-looking statements” regarding the
potential therapeutic benefits and progress of Potentia’s research and
development programs. These statements are hereby identified as
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