POT-4 well tolerated by all subjects and sustained levels of drug reported after one treatment
LOUISVILLE, Ky., May 4 /PRNewswire/ -- Potentia Pharmaceuticals, a privately held biotechnology company developing medicines for the treatment of age-related macular degeneration (AMD), announced today that it successfully completed its Phase I 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.
The ASaP trial was an open label, first-in-man, multi-center, single escalating dose study. The primary safety endpoint of this trial was achieved and the drug was well tolerated by all subjects at all doses tested, based on clinical signs, ophthalmic examinations, and laboratory results. No drug-related serious adverse events and no identifiable intraocular inflammation were reported. Additionally, significant sustained levels of POT-4 were consistently measured via serum analysis in subjects receiving the highest dose studied. The current POT-4 formulation is expected to provide sustained therapeutic ocular levels of the drug for several months following a single injection.
The data accumulated so far support the implementation of a comprehensive Phase II clinical program to further define the safety, efficacy and pharmacokinetic profile of POT-4 as a treatment for both dry and wet AMD.
"The safety and pharmacokinetic data strongly support the further development of POT-4 as a first-in-class treatment for patients with AMD," said Cedric Francois, President and CEO of Potentia Pharmaceuticals. "We believe that POT-4 has significant promise based on these early-stage findings and look forward to further testing of POT-4 in this disease, which is so prevalent and devastating to those affected by it."
POT-4 is a complement factor C3 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.
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. 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).
Potentia Pharmaceuticals, Inc. is a biotechnology company focused on developing novel therapeutics to address chronic inflammatory diseases, with an initial emphasis on diseases of the eye such as age-related macular degeneration.
|SOURCE Potentia Pharmaceuticals|
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