ABIONYX Pharma announced new positive results in ophthalmology for the first class of CER-001-based biomedicines for the treatment of ocular pathologies, and the appointment of Mr. Jérôme Martinez as ABIONYX Pharma's Senior Advisor in ophthalmology. New positive long-term preclinical results evaluating the efficacy of CER-001 after a single intraocular administration in a uveitis model with severe inflammation. Following the beneficial clinical findings related to the disappearance of visual blurring linked to corneal deposits in a patient suffering from LCAT deficiency treated under a Temporary Authorization for Use, and a marked improvement in the patient's visual function (results published exclusively in the scientific journal "Annals of Internal Medicine" in 2021), and still observed after more than a year of follow-up, ABIONYX Pharma conducted new preclinical studies in ophthalmology to qualify the efficacy spectrum of recombinant apoA-I alone and in combination with a corticosteroid, and to broaden its potential in new indications.

After demonstrating the safety of CER-001, the recombinant apoA-I was tested again to assess its action in reducing inflammatory reactions and its tolerability after a single intraocular administration (IVT) in a model of LPS-induced uveitis. 74 animals participated in this study, divided into 8 groups. Six hours after LPS injection, statistically significant reductions in inflammation, measured by slit-lamp examination, were observed for the groups treated with CER-001 alone or in combination with a corticosteroid compared with the vehicle-treated group.

CER-001 (cumulative score 3.1 ± 2.3, p= 0.0254) and best-in-class drug (cumulative score 3.1 ± 1.5, p= 0.0228) were superior to vehicle. No statistically significant differences were observed for the other groups treated with standard therapies. Twenty-four hours after induction, the significance observed at six hours for the CER-001 and best-in-class drug-treated groups versus the vehicle-treated group continued, demonstrating a reduction in inflammation (cumulative score 3.9 ± 1.7 and 4.9 ± 1.2, respectively and p < 0.0001 and = 0.0018, respectively).

The downward trend observed at six hours compared with the vehicle-treated group was confirmed by statistical significance for CER-001 alone and CER-001 in combination (cumulative score 5.3 ± 1.3 and 4.6 ± 2.1, respectively and p = 0.0081 and = 0.0018, respectively). No statistically significant differences were observed for all other groups. Results for the CER-001 and CER-001 combination groups were comparable or better than those for the best-in-class alone group.

Twenty-four hours after LPS injection, the high level of induced inflammation was reached in the vehicle-treated group, with median values of 5920 cells/µL. A statistically significant decrease in leukocyte infiltration was observed in the groups treated with CER-001 alone and CER-001 in combination with a corticosteroid, compared with the vehicle-treated group. For all other groups, no significance was observed. CER-001 alone or in combination with a corticosteroid, as tested in this preclinical study, proved safe and well tolerated on the ocular surface and inside the eye, when given by injection inside the eye.

These new preclinical results reconfirm the major therapeutic potential of the only recombinant apoA-I in ophthalmology. The anti-inflammatory and/or reverse lipid transport-enhancing properties of CER-001, and these new preclinical results in uveitis, pave the way for clinical trials testing apoA-I in patients with other severe inflammatory ocular diseases.