TOULOUSE, France & LAKELAND, Michigan, October 07, 2021– (BUSINESS WIRE) – Regulatory news:
ABIONYX Pharma (Paris: ABNX) (FR0012616852 – ABNX – PEA PME eligible), a new generation biotechnology 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 of its bio-HDL for the treatment of ocular pathologies.
Positive preclinical results in a uveitis model
Following the positive clinical results which included the disappearance of visual blurring linked to corneal lipid deposits in a patient suffering from an LCAT deficiency, treated under a Temporary Authorization for Use, and the marked improvement in 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 carried out preclinical studies in ophthalmology to determine the ocular tolerance of its bio-HDL and the potential spectrum of efficacy in new indications.
Bio-HDL has been shown 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 on the surface of the eye or by injection inside the eye itself (into the vitreous).
Additionally, bio-HDL has been tested in a proof of concept efficacy study using a recognized and validated preclinical model of uveitis. In this model of endotoxin-induced uveitis (EIU), the uveitis was triggered by a dose of lipopolysaccharide (LPS) injected into the vitreous. Subsequent treatment with CER-001 injected into the vitreous body showed a statistical reduction in signs of uveitis, as measured by protein concentration and cellular infiltration into aqueous humor.
These first 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 National Hospital of Ophthalmology of the Quinze-Vingts Hospital (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: âThe latest scientific work in the field shows that lipids and their metabolism are involved in many eye pathologies, for example meibomian gland dysfunction and macular degeneration. By testing CER-001, a biomimetic HDL produced in France, in models of ocular pathology, we will be able to help choose the best ocular indication for this product, with the aim of offering patients a new effective treatment. “
Development of the first class of biomedicines in ophthalmology with bio-HDL
Following the obtaining of the orphan designation by the European Medicines Agency (EMA) for its drug candidate CER-001 as a potential treatment for LCAT deficiency, ABIONYX Pharma’s bio-HDL is one of the most popular biomedicines. more advanced in France which could soon be marketed in ophthalmology.
As a reminder, there are two forms of LCAT deficiency:
– familial LCAT deficiency (FLD), which results from a complete deficiency and is clinically characterized by hemolytic anemia, renal failure (frequently leading to renal transplantation) and corneal opacities; and
– “Fish Eye Disease” resulting from a partial deficiency and clinically characterized by more severe corneal opacities without renal damage.
As the orphan drug designation obtained covers both a renal indication and an ophthalmologic indication, the clinical development of bio-HDL in ophthalmology can be very rapid.
Lipids are of major interest to the eye in several respects: its structural role, its functional role, its pathophysiological links and its therapeutic potential.
Dr Catherine Creuzot-Garcher, Professor of Ophthalmology in Dijon, Head of the Ophthalmology Department at Dijon University Hospital, University Professor, co-head of the Eye, Nutrition and Cell Signaling team at the Center des Sciences du GoÃ»t et de l’Alimentation in Dijon, and Dr Niyazi Acar (PhD), head of the Eye, Nutrition and Cellular Signaling team at the Dijon Center for Taste and Food Sciences, declares: “The study and development of the therapeutic potential of CER-001, a biomimetic HDL, in the treatment of eye diseases will allow us to better understand the role of lipids in the physiology and dysfunctions of the eye, in particular of the retina,” and to offer our patients an innovative solution for the future. “
The anti-inflammatory and / or reverse lipid transport enhancing properties of CER-001, which may improve vision in patients with deficient LCAT activity, combined with new preclinical findings in uveitis, pave the way for studies clinical trials testing bio-HDL in patients developing corneal lipid deposits from other pathologies, and allow ABIONYX Pharma to launch a strategic development of the first class of biomedicines in ophthalmology based on its bio-HDL.
About ABIONYX Pharma
ABIONYX Pharma is a new generation biotechnology company that aims to contribute to health through innovative therapies in indications where there is no effective or existing treatment, even the rarest. Thanks to its partners in research, medicine, biopharmaceuticals and shareholders, the company innovates on a daily basis to offer drugs for the treatment of kidney and ophthalmological diseases, or new HDL vectors used for targeted drug delivery.
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