LCAT

TOULOUSE, France & LAKELAND, Mich.--(BUSINESS WIRE)-- Access here the full press release ABIONYX Pharma, (FR0012616852 – ABNX – PEA PME eligible), a new generation biotech company dedicated to the discovery and development of innovative therapies based on the world’s only natural recombinant apoA-I, today acknowledges that the Phase 3 AEGIS-II study evaluating the efficacy and safety of CSL Behring’s human-plasma-derived apoA-I, CSL112, compared to placebo in reducing the risk of major adverse cardiovascular events (MACE) in patients following an acute myocardial infarction (AMI), did not meet its primary efficacy endpoint of MACE reduction at 90 days. About CER-001 CER-001 is a novel engineered recombinant human apoA-I that was designed to mimic the structural and functional biological properties of natural, nascent HDL, also known as pre-β HDL, and has been shown to perform all steps of the Reverse Lipid Transport pathway (RLT), the only natural pathway responsible for lipid elimination. Administered CER-001 particles increase transient apoA-I and the number of HDL particles and promote the elimination of trapped cholesterol and lipids in tissues in the absence of LCAT enzyme for example, but also the elimination of bacterial lipid endotoxin (LPS) in the case of sepsis. HDL particles are then recognized by the liver, leading to the elimination of these transported lipids via a process called Reverse Lipid Transport (RLT). About ABIONYX Pharma ABIONYX Pharma is a next-generation biotech company focused on developing innovative medicines in diseases where there is no effective or existing treatment, even the rarest ones. The company expedites the development of novel therapeutics through an extensive expertise in lipid science and a differentiated apoA-I -based technology platform. ABIONYX Pharma is committed to radically improving treatment outcomes in sepsis and critical care. View source version on businesswire.com: Contacts NewCap Investor relations Nicolas Fossiez Louis-Victor Delouvrier abionyx@newcap.eu +33 (0)1 44 71 98 53 NewCap Media relations Arthur Rouillé abionyx@newcap.eu +33 (0)1 44 71 00 15 Source: ABIONYX Pharma View this news release online at:

FRAMINGHAM, Mass. , Aug. 17, 2022 /PRNewswire/ -- Boston Heart Diagnostics, part of the Eurofins network of companies, announces the launch of LipidSeq™, a revolutionary genetic test for cardiovascular disease. This saliva-based NGS test identifies the most common genetic causes of lipid and lipoprotein disorders. These disorders are associated with premature cardiovascular, pancreatic, kidney, and neurological diseases. Continue Reading When patients have very high levels of low-density lipoprotein cholesterol, triglycerides, or plasma sterols and/or very low levels of high-density lipoprotein cholesterol, there is a likelihood of a genetic mutation. Conditions detected by this 23-gene test include familial forms of hypercholesterolemia, lysosomal acid lipase deficiency, sitosterolemia, cerebrotendinous xanthomatosis, dysbetalipoproteinemia, hypertriglyceridemia, and HDL deficiency states including ApoA-I deficiency, Tangier disease, and LCAT deficiency, as well as other disorders. If there is a genetic basis for the patient's condition, understanding the specific mutation and associated disease can help guide treatment considerations, educate the patient about health risks, and encourage relatives to seek testing to determine if they, too, may be at risk. Boston Heart is the only commercial laboratory able to test biochemical and genetic contributors when diagnosing inherited disorders of lipid metabolism (Reference: Schaefer EJ, Geller AS, Endress G. The biochemical and genetic diagnosis of lipid disorders. Current Opinion in Lipidology 2019; 30:56-62.) Boston Heart Diagnostics announces the launch of LipidSeq™, a revolutionary genetic test for cardiovascular disease. Tweet this Testing of appropriate patients can be done at any age, which favors early identification and treatment of genetically influenced lipid and lipoprotein disorders. Early diagnosis and precision treatment can save lives, and have a significant clinical impact for practitioners, patients, and their families. LipidSeq was developed in collaboration with Eurofins laboratory partner Clinical Enterprise, which performs the genetic sequencing, and in consultation with Dr. Robert A. Hegele of Robarts Research Institute in London, Ontario. Healthcare providers may access this testing for their patients by signing up for an account with Boston Heart. The Boston Heart Customer Care department is available at 877.425.1252 or [email protected]. About Boston Heart Diagnostics Corporation Boston Heart Diagnostics, a subsidiary of Eurofins Scientific (EUFI.PA), is transforming the treatment of cardiovascular disease and related conditions by providing healthcare providers and their patients with novel, personalized diagnostics and integrated customized lifestyle programs that have the power to change the way clinicians and patients communicate about disease and improve health. For more information on Boston Heart Diagnostics, please visit SOURCE Boston Heart Diagnostics

ABIONYX Pharma (FR0012616852 – ABNX – PEA PME eligible) (Paris:ABNX), a new generation biotech company dedicated to the discovery and development of innovative therapies for patients, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation (ODD) to the Bio-HDL CER-001 for the treatment of lecithin-cholesterol acyltransferase (LCAT) deficiency. The designation covers both partial LCAT deficiency, presenting as Fisheye Disease, and complete LCAT deficiency presenting with renal symptoms and corneal opacities. Progression of LCAT deficiency, for which there is no approved treatment, can ultimately lead to renal failure requiring dialysis or kidney transplant, and/or to complete corneal opacification requiring transplant. The European Medicines Administration (EMA) granted ODD status to CER-001 for the treatment of LCATdeficiency in July 2021. Positive clinical results from CER-001 in LCAT disease have previously been published. In the Annals of Internal Medicine in March 2021, a case study of a patient who was about to undergo dialysis due to the rapid decline in renal function was described. The patient was able to avoid the need for dialysis during her treatment with CER-001 and in addition, lipid deposits in her corneas which had caused significant visual blurring, improved with treatment. The improvement in visual function was still observed after 1 year of follow-up. A second case was described in the Journal of Internal Medicine in November 2021 and showed that CER-001 reduced glomerular lipid deposits and slowed the patient’s decline in renal function. Furthermore, CER-001 remodeled his plasma lipoproteins by reducing the level of LpX, large abnormal lipid complexes known to be renally toxic. “We are pleased to have received ODD for CER-001 just eight months after the ODD in Europe and one week after the first positive clinical results in COVID-19. These OD designations from the FDA underscore the importance of bringing this important therapeutic option to patients with LCAT Deficiency both as a kidney disease and as an ophthalmic disease,” commented Cyrille TUPIN, CEO of ABIONYX Pharma. “We look forward to presenting new clinical results in the coming months and providing additional insight into the potential of our Bio-HDL therapy platform in both kidney diseases and ophthalmologic diseases. The ODD paves the way for ABIONYX Pharma to launch a new strategic clinical development of the bio-HDL in kidney diseases and ophthalmologic diseases in the US.” Orphan-drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes a disease or condition that affects fewer than 200,000 individuals in the U.S. Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The designation is granted based on the mechanism of action of the drug or biologic taken into consideration with the pathogenesis of the disease or condition, its course and prognosis as well as the availability of treatments and/or resistance to available treatments. Orphan drug designation qualifies sponsors for incentives including: tax credits for qualified clinical trials, exemption from user fees, and a potential for seven years of market exclusivity after approval.