Je n’avais jamais entendu parler de Potentia Pharmaceuticals, mais leur approche thérapeutique de la DMLA pourrait être intéressante, basée sur l’inhibition du complément, dont on sait maintenant que certaines mutations dans le système d’activation sont un facteur de risque majeur de la maladie.
L’un des grands intérêts par rapport aux traitements actuels est qu’elle pourrait également être indiquée pour les formes atrophiques, pour lesquelles nous ne pouvons actuellement proposer aucun traitement (autre que préventif par les supplémentations vitaminiques, anti-oxydants et autres pigments maculaires) :
March 20, 2007—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.
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.
Compte tenu que la recherche croisée « Lambris JD » et « complement » dans PubMed retrouve 184 références (la première en 1980), nous avons manifestement à faire à un spécialiste du sujet, qui publiait dès 1996 un articles sur la « compstatin », inhibiteur de la fraction C3 du complément (32 références), dont dérive le POT-4 !
About Potentia Pharmaceuticals, Inc.
Potentia Pharmaceuticals, Inc. 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.
Autre intérêt : ils semblent également développer parallèlement un système pouvant délivrer le médicament pendant 1 an :
EOS Insertâ„¢ : Potentia’s first product containing POT-4 in a sustained delivery intravitreal implant formulation is called the EOS Insert. The insert is designed to be injected intravitreally through a 25-gauge syringe and to sustain a therapeutic concentration of POT-4 in the eye of the patient for a period of at least 12 months. The resulting drug product will be targeted at patients with either the dry or the wet forms of age-related macular degeneration.
A suivre…
Source : www.potentiapharma.com, via www.medicalnewstoday.com.