Potentia Pharmaceuticals' Drug Candidate for Age-Related Macular Degeneration Shows Positive Safety Profile in Phase I Clinical Trial
The ASaP trial is a first-in-man, multi-center, single escalating dosestudy. The interim results of this trial revealed no drug-related toxicitybased on clinical signs, ophthalmic examinations, or laboratory results at anytime point monitored in patients treated with up to 150 microgram/dose of POT-4. Additionally, no serious adverse events and no identifiable intraocularinflammation were reported.
Preliminary results indicate that intravitreal POT-4 is safe, and the dataaccumulated so far support the continued investigation of POT-4 for thetreatment of both dry and wet AMD with larger randomized clinical trials tofurther define its efficacy profile.
"These safety data strongly support the further development of POT-4 as apotential treatment for patients with AMD," said Cedric Francois, Presidentand CEO of Potentia Pharmaceuticals. "We believe that the product hassignificant promise based on these early-stage findings and look forward tofurther testing of the compound in higher doses as we continue this trial."
POT-4 is a complement inhibitor, which shuts down the complementactivation cascade that could otherwise lead to local inflammation, tissuedamage and upregulation of angiogenic factors such as vascular endothelialgrowth factor (VEGF) in the eye. Based on this mechanism of action, POT-4holds the potential to be effective against both dry and wet AMD.
AMD is the leading cause of blindness in the elderly of the western worldand affects more than 10 million patients in the United States alone. Thecurrent standard of care for AMD relies primarily on angiogenesis inhibitors,an approach geared towards the approximately 10-15% of AMD patients withcomplications resulting from ocular angiogenesis (growth of new blood vesselsand bleeding in the back of the eye). No drug currently on the market has beenapproved 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 ofplasma proteins, ultimately leading to cell membrane disruption through themembrane attack complex (MAC). Activation of the complement system is animportant part of the body's defensive immune response against pathogens suchas bacteria and viruses. In spite of its defensive function, inappropriate orexcessive complement activation can have destructive consequences if leftunchecked. Over the past three years, multiple scientific publications havestrongly linked variants of genes encoding components of the complement systemwith a predisposition toward AMD.
POT-4 binds tightly to complement component C3, preventing itsparticipation in the complement activation cascade. As C3 is the centralcomponent of all major complement activation pathways, its inhibitioneffectively shuts down downstream complement activation that could otherwiselead to local inflammation, tissue damage and upregulation of angiogenicfactors such as vascular endothelial growth factors (VEGF).
Potentia Pharmaceuticals, Inc. is an early stage biotechnology companyfocused on developing novel therapeutics and drug delivery technologies toaddress chronic inflammatory diseases, with an initial emphasis on diseases ofthe eye such as age-related macular degeneration.Media Requests: Paul Kidwell (617) 296-3854 firstname.lastname@example.org Contact for Potentia Pharmaceuticals: Pascal Deschatelets (502) 569-1053
SOURCE Potentia Pharmaceuticals, Inc.
You May Also Like