ABIONYX Announces Positive Clinical Results From CER-001 in the LCAT Deficiency Disease Published in the Annals of Internal Medicine

• Demonstrated efficacy in renal and ophthalmic indication
• Confirmation of the breakthrough therapy status of CER-001 in kidney and ophthalmic diseases
• Systemic mechanism of action of CER-001

TOULOUSE, France--(BUSINESS WIRE)--ABIONYX Pharma (FR0012616852 - ABNX - PEA PME eligible), a new generation biotech company dedicated to innovative therapies for patients, today announces positive clinical results from CER-001 in the LCAT (lecithin-cholesterol acyltransferase) deficiency disease published in the Annals of Internal Medicine.

The patient with inherited mutations in the lecithin-cholesterol acyltransferase (LCAT) gene developed glomerulopathy and corneal lipid deposits and displayed very low circulating levels of high-density lipoprotein (HDL) and apoA-I. The patient was treated with CER-001, an apoA-I-containing HDL mimetic, to help in preventing rapidly progressive kidney failure.

The publication states that CER-001 prevented further kidney function decline. The patient who was about to be dialysed due to rapidly declining kidney function, was able to avoid the need for dialysis during their treatment with CER-001.

No other treatment was introduced strongly suggesting that stabilization of the renal function relied on the administration of CER-001.

The publication mentions positive extra-renal clinical results and the disappearance of visual blurring secondary to corneal deposits. This clear improvement in visual function is still observed after 1 year of follow-up.

Prof. Stanislas Faguer, nephrologist at Department of Nephrology and Organ Transplantation Reference Center for Rare Kidney Diseases, Hospital Rangueil, CHU Toulouse, states: “These positive clinical data demonstrate that CER-001 prevented significantly kidney function decline. ApoA-I containing HDL mimetic CER-001 is the first treatment that has proven its ability to slow the progression of renal failure and reduce visual discomfort secondary to corneal lipid deposits in a patient with FLD. We look forward to conducting new clinical studies of CER-001 to assess its ability to improve the prognosis of other orphan or common kidney diseases for which no effective treatment is currently available.”

Cyrille Tupin, Managing Director of ABIONYX Pharma concludes: “This represents an important step forward in the clinical development of CER-001, an HDL mimetic. We are committed to advancing the clinical development of the mimetic HDL as quickly and safely as possible while investigating new indications in ophtalmology so that we can help address new rare or orphan diseases with no existing treatment.”