APOA1

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:

CSL Behring’s share price dropped around 5% when the Australian stock exchange opened Monday. CSL Behring’s largest-ever trial has ended in failure, as the Australian pharma revealed that its cholesterol efflux enhancer was unable to reduce the risk of heart attacks. The company had been testing CSL112, also known as apolipoprotein A-I, in a study of about 17,400 heart attack patients spread across 49 countries. The first participant was dosed in the AEGIS-II study in 2018. But the trial failed to hit its primary endpoint of showing a reduction in the risk of major adverse cardiovascular events after 90 days when compared to placebo, CSL disclosed in a Feb. 11 release. “As a result, there are no plans for a near-term regulatory filing,” the company concluded. CSL didn’t share many other details, although it did point out that there were no major safety or tolerability concerns with the drug. Instead, the company said it would share primary results at the American College of Cardiology Scientific Sessions in April. “Substantial work remains to fully analyze and understand the complete data and then to determine any development path ahead for this asset,” CSL’s head of R&D Bill Mezzanotte, M.D., said in the release. “AEGIS-II is the most ambitious study in our company's history and we are proud of the quality of the study we delivered and the enhanced capabilities we developed to do so,” Mezzanotte added. “We plan to apply these capabilities as well as our plasma protein platform to future unmet medical need in cardiovascular and metabolic conditions as well as those in our other strategic therapeutic areas.” CSL112 was developed using a new formulation of apoA-I, the main component of high-density lipoproteins, which was extracted from human plasma. By enhancing the body’s ability to remove cholesterol from plaque in the arteries, the hope was that CSL112 would reduce cardiovascular events among high-risk patients in what CSL had described as the “critical 90 days after a heart attack.” When the trial was designed back in 2017, CSL executives suggested that CSL112 had “the potential to change the current treatment paradigm for heart attack survivors and improve global health outcomes for the millions of people at risk for early recurrent cardiovascular events.” The company’s share price dropped around 5% when the Australian stock exchange opened Monday, with CSL’s shares ending the day’s trading at 290.24 Australian dollars compared to a Friday closing price of AU$305.

CSL’s AEGIS-II study for its MI drug CSL112 enrolled 18,200 participants across 850 sites worldwide. Image Credit: viewimage / Shutterstock. Australian CSL has released topline results from the Phase III AEGIS-II trial of CSL112 in patients following an acute myocardial infarction (MI). Results showed that the drug failed to meet its primary endpoint of a reduction in the risk of major adverse cardiovascular events (MACE) at 90 days. The company also stated that it had no near-term plans to file for regulatory approval for the drug. Following the news, CSL’s stock was down by more than 4.8% in trading today. The company’s market cap sits at A$143.36bn ($93.4bn). The placebo-controlled AEGIS-II study (NCT03473223) was one of the biggest undertaken by the company. It enrolled 18,200 patients with acute coronary syndrome – a precursor to MI, across 185 sites worldwide. The trial participants received four weekly doses of CSL112 or placebo, starting within five days of hospital admission. There were no major safety or tolerability concerns were observed in the treatment group, as per an 11 February press release. The company plans to publish the results and present data from the trial at the American College of Cardiology Scientific Sessions in Atlanta from 6 to 8 April. The risk of subsequent cardiac events, including a second MI, is high in patients who have suffered acute MI. A systematic review found that following an MI, these patients were at least 30% higher relative risk for all-cause death and cardiovascular outcomes such as recurrent MI and cardiovascular death, compared to the general population. See Also: CSL112 is a human apolipoprotein A-I (apoA-I) which was developed as an investigational cholesterol efflux enhancer. Cholesterol efflux is the ability of high-density lipoproteins (HDL) to carry cholesterol particles from plaques in the heart’s blood vessels and move those particles to the liver for disposal, with better cholesterol efflux proposed to result in lower likelihood of MACEs. CSL’s pipeline also consists of a haemophilia B gene therapy , Hemgenix (etranacogene dezaparvovec). In 2022, the US Food and Drug Administration (FDA) approved the adeno-associated virus vector-based gene therapy for haemophilia B treatment in adults. The company’s portfolio also consists of a self-amplifying messenger ribonucleic acid (sa-mRNA) vaccine for Covid-19. In November 2023, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved the ARCT-154 vaccine for preliminary vaccination and as a booster in adults. ARCT-154 vaccine was developed as part of a worldwide partnership and licensing deal with Arcturus Therapeutics.