ABIONYX Pharma announced that the company has been granted a new Compassionate Access Authorization (CAA) in Europe for CER-001 in the rare, untreated kidney disease LCAT Deficiency. This is the fourth patient suffering from LCAT deficiency to be granted Compassionate Access to CER-001, in development by ABIONYX Pharma, in Europe. The four patients suffering from this rare disease, which has a major impact on quality of life and on lifespan, represent very different stages in the evolution of the disease, for which CER-001 may prove effective, whether or not the patients are transplanted, and whatever their age.

CER-001 would represent a unique therapeutic breakthrough for patients who need to be diagnosed as early as possible to avoid dialysis, followed by one or more transplants during their lifetime. The early administration of CER-001 in young patients with Norum disease could have a real beneficial effect on these patients, transforming their lives and, above all, delaying the end of their lives, while very significantly reducing the management of their disease, whether with dialysis or by one or more transplants. The safety and tolerability data for this treatment are as positive as ever, with no adverse events reported for any of the patients.

Constructive discussions have been initiated with the European regulatory authorities to define a pathway for the rapid advancement of CER-001's clinical development in the patient population affected by this rare disease. The treatment of LCAT deficiency or Norum disease is a major medical challenge, given the constant progression of the disease, leading to rapid renal decline and often premature death, and the total absence of approved treatments to slow or modify the progression of the disease. ABIONYX Pharma continues to receive and fulfill Compassionate Access requests from hospitals around the world.

The biomanufacturing of the latest and subsequent batches of CER-001 to the highest GMP quality standards will enable these requests to be met, and the clinical results of the first patients will be communicated exclusively through scientific publication.