CSL Behring has released disappointing top-line results from its phase 3 study assessing its investigational cholesterol efflux enhancer against placebo in reducing the risk of heart attacks.
The AEGIS-II trial did not meet its primary endpoint of major adverse cardiovascular events (MACE) reduction in patients post-acute myocardial infarction (AMI) at 90 days. As a result, CSL says it has no immediate plans for regulatory filing for the treatment, CSL112.
Further analysis of the trial is ongoing, with primary results scheduled for presentation at the American College of Cardiology Scientific Sessions in April 2024 and subsequent publication in a peer-reviewed journal.
The AEGIS-II trial, involving over 18,200 patients across 49 countries, was described by CSL's head of R&D as the company's "most ambitious study" to date. CSL112, Apolipoprotein A-I (Human), is a new type of drug designed to boost cholesterol removal from the body. It's made from a unique form of human plasma-derived apoA-I, which is a key part of HDL, often called ‘good cholesterol.’
CSL made headlines back in 2022, when the U.S. FDA approved Hemgenix as the first gene therapy for adult hemophilia B patients, and the $3.5 million therapy subsequently became the world's most expensive drug. Last year, the UK's pricing watchdog advised against England's NHS reimbursing Hemgenix.
