Potentia Pharmaceuticals, a privately
held biotechnology company developing medicines for the treatment of
age-related macular degeneration (AMD), presented Phase I data last week
during the Retina Subspecialty Day at the American Academy of Ophthalmology
(AAO) Annual Meeting in Atlanta, GA. The data was from the ASaP (Assessment
of Safety of Intravitreal POT-4 Therapy for Patients with Neovascular
Age-Related Macular Degeneration) clinical trial for the company’s leading
drug candidate, POT-4, which is being developed for the treatment of AMD.
The ASaP trial is a first-in-man, multi-center, single escalating dose
study. The interim results of this trial revealed no drug-related toxicity
based on clinical signs, ophthalmic examinations, or laboratory results at
any time point monitored in patients treated with up to 150 microgram/dose
of POT- 4. Additionally, no serious adverse events and no identifiable
intraocular inflammation were reported.
Preliminary results indicate that intravitreal POT-4 is safe, and the
data accumulated so far support the continued investigation of POT-4 for
the treatment of both dry and wet AMD with larger randomized clinical
trials to further define its efficacy profile.
“These safety data strongly support the further development of POT-4 as
a potential treatment for patients with AMD,” said Cedric Francois,
President and CEO of Potentia Pharmaceuticals. “We believe that the product
has significant promise based on these early-stage findings and look
forward to further testing of the compound in higher doses as we continue
this trial.”
About POT-4
POT-4 is a complement inhibitor, which shuts down the complement
activation cascade that could otherwise lead to local inflammation, tissue
damage and upregulation of angiogenic factors such as vascular endothelial
growth factor (VEGF) in the eye. Based on this mechanism of action, POT-4
holds the potential to be effective against both dry and wet AMD.
About AMD
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 (MAC). 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 destructive
consequences if left unchecked. Over the past three years, multiple
scientific publications have strongly linked variants of genes encoding
components of the complement system with a predisposition toward AMD.
POT-4 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 downstream complement activation that could
otherwise lead to local inflammation, tissue damage and upregulation of
angiogenic factors such as vascular endothelial growth factors (VEGF).
About Potentia
Potentia Pharmaceuticals, Inc. is an early stage biotechnology company
focused on developing novel therapeutics and drug delivery technologies to
address chronic inflammatory diseases, with an initial emphasis on diseases
of the eye such as age-related macular degeneration.
Potentia Pharmaceuticals, Inc.
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