TOULOUSE, France & LAKELAND, Mich.– ABIONYX Pharma (Paris:ABNX) (FR0012616852 – ABNX – PEA PME eligible), a next-generation biotech company dedicated to the discovery and development of innovative therapies, announces today, after having already obtained an Orphan Drug designation for CER-001 in the treatment of LCAT deficiency, positive preclinical results in ophthalmology and the launch of a strategic development of the first class of biomedicines from its bio-HDL for the treatment of ocular pathologies.
Following the positive clinical results which included the disappearance of visual blur related to corneal lipid deposits in a patient suffering from LCAT deficiency, treated within the framework of a Temporary Use Authorization, and the clear improvement of the patient’s visual functions which was still observed after 1 year of follow-up (initial results published exclusively in the scientific journal “Annals of Internal Medicine; follow-up information on file), ABIONYX Pharma conducted preclinical studies in ophthalmology to determine the ocular tolerance of its bio-HDL and the potential spectrum of efficacy in new indications.
Bio-HDL was found to be completely safe and very well tolerated on the ocular surface and inside the eye, regardless of the route of administration in a preclinical study, either by intravenous injection, by application of drops to the surface of the eye or by injection inside the eye itself (into the vitreous).
In addition, bio-HDL was tested in a proof of concept efficacy study using a recognized and validated preclinical model of uveitis. In this Endotoxin-Induced Uveitis (EIU) model, uveitis was triggered by a dose of lipopolysaccharide (LPS) injected in the vitreous. Subsequent treatment with CER-001 injected into the vitreous showed a statistical reduction in the signs of uveitis, as measured by protein concentration and cellular infiltration in the aqueous humor.
These initial preclinical results indicate the major therapeutic potential of bio-HDL in ophthalmology, and more broadly the role of lipids in ocular pathologies.
Dr. Christophe Baudouin, Professor of Ophthalmology in Paris, Head of the Ophthalmology Department at the Centre Hospitalier National d’Ophtalmologie de l’Hôpital des Quinze-Vingts (Paris), Director of the “S12” research team at the Institut de la Vision, and member of the prestigious international societies, American Society of Ophthalmology and Academia Ophthalmologica Internationalis, states: “The latest scientific work in the field shows that lipids and their metabolism are involved in many ocular pathologies, for example dysfunction of the meibomian glands and macular degeneration. By testing CER-001, a biomimetic HDL produced in France, in ocular pathology models, we will be able to help choose the best ocular indication for this product, with the aim of providing patients with a new and effective treatment.”
