CSL112 Misses Key Goal in Late-Stage Heart Attack Trial

Australian pharmaceutical company CSL reported that its cholesterol management drug, CSL112, did not meet its primary endpoint in a Phase III trial. The drug, designed to enhance cholesterol removal from the body, was evaluated in the AEGIS-II study, which involved a large-scale, double-blind, placebo-controlled trial with 18,200 participants from nearly 50 countries. The study aimed to assess the drug's impact on patients who had experienced a heart attack, but the results showed no significant reduction in major adverse cardiovascular events when compared to a placebo group within 90 days post-heart attack.

Despite the setback, the trial did not uncover any significant safety or tolerability concerns associated with CSL112. Following the announcement of the trial's results, CSL's share price experienced a decline of approximately 5%. The company's executive vice president and head of research and development, Bill Mezzanotte, described the AEGIS-II trial as the most extensive in CSL's history. He expressed pride in the quality of the study conducted and the capabilities developed during the process. Mezzanotte also indicated that CSL will further analyze the trial's findings to gain a deeper understanding and determine future steps for the drug.

CSL plans to present the AEGIS-II study outcomes at the American College of Cardiology Scientific Sessions in April 2024. The drug in question, CSL112, is a proprietary formulation of apolipoprotein A-I, a key component of high-density lipoprotein, which plays a crucial role in cholesterol regulation. The drug operates by enhancing the body's natural ability to clear cholesterol from arterial plaques and transport it to the liver for processing.

Previously, in 2016, CSL had reported positive findings from the Phase IIb AEGIS-I study, indicating that CSL112 was well-tolerated and safe, without causing significant changes in liver or kidney function. The earlier trial also confirmed the drug's mechanism of action and its potential to significantly increase cholesterol removal capacity.

CSL112's development and testing represent a significant effort by the company to address cardiovascular and metabolic conditions, a strategic therapeutic area for CSL. The company's commitment to applying the knowledge gained from the AEGIS-II trial to future medical needs in these areas underscores its ongoing dedication to advancing treatments for patients.