ABIONYX Pharma Announces Positive Preclinical Results in a Model of Uveitis and Launches the Strategic Development of the First Class of Biomedicines in Ophthalmology Based on its bio-HDL
07 Octobre 2021 - 08:46AM
Business Wire
- Positive preclinical results in a model of uveitis
- Development of the first class of biomedicines in
ophthalmology with bio-HDL
Regulatory News:
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.
Positive preclinical results in a model of uveitis
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.”
Development of the first class of biomedicines in
ophthalmology with bio-HDL
Following the granting of 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 advanced biomedicines in France that 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 involvement.
As the orphan drug designation obtained covers both a renal and
an ophthalmological indication, the clinical development of bio-HDL
in ophthalmology may be very rapid.
Lipids are of major interest for 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 the University
Hospital of Dijon, University Professor, co-leader of the Eye,
Nutrition and Cellular Signaling team at the Taste and Food Science
Center of Dijon, and Dr. Niyazi Acar (PhD), leader of the
Eye, Nutrition and Cellular Signaling team at the Taste and Food
Science Center of Dijon state: "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,
particularly in the retina, and to provide our patients with 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 the new preclinical results
in uveitis, pave the way for clinical studies 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 biotech company that aims to
contribute to health through innovative therapies in indications
where there is no effective or existing treatment, even the rarest
ones. Thanks to its partners in research, medicine,
biopharmaceuticals and shareholding, the company innovates on a
daily basis to propose drugs for the treatment of renal and
ophthalmological diseases, or new HDL vectors used for targeted
drug delivery.
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NewCap Investor relations Louis-Victor Delouvrier
abionyx@newcap.eu +33 (0)1 44 71 98 53
NewCap Media relations Nicolas Merigeau abionyx@newcap.eu
+33 (0)1 44 71 94 98
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