CSL Behring, the blood product arm and major breadwinner of CSL Limited (ASX: CSL), this morning released an update on its 'CSL112' infusion drug to treat plaque build-up in the veins.
CSL reported that its Phase 2b trial into the treatment was a success, with the CSL112 being well tolerated when administered after a heart attack, and additionally causing no adverse effects to liver or kidney function. Additionally, the study confirmed the treatment's mechanism of action, with 'cholesterol efflux' (see below) rising by up to 4x above baseline.
Why should we care?
A special tip of the hat goes to CSL for translating today's update into English the layperson will understand. In simplest terms, cholesterol efflux is the body's natural process of removing cholesterol from the veins. Cholesterol is transported in the blood by two types of lipoprotein, HDL and LDL.
Cardiac events ('heart attacks' and similar) are caused by Low Density Lipoprotein (LDL), which deposits cholesterol inside the veins, forming plaque that makes veins narrower and thus increases blood pressure. Over many years of LDLs depositing cholesterol, the veins get narrower and blood pressure increases, until the veins either block completely (think 'heart attack') or rupture (think 'stroke').
The CSL112 therapy, which is itself actually a novel version of 'healthy' high-density lipoprotein (HDL), works by stimulating the body to increase its cholesterol efflux capacity, i.e., its ability to remove cholesterol from the veins. Today's results showed that CSL112 is successful at this.
In theory, accelerating the removal of cholesterol from the plaque veins should help stabilise the cardiovascular system in the event of a heart attack. CSL intends to use CSL112 as a therapy immediately following a cardiac event, in order to reduce cholesterol levels and hopefully reduce the likelihood of subsequent cardiac events. In theory, there are obvious pre-heart attack applications as well, for example in people with obesity and high blood pressure. That's getting ahead of ourselves however, because…
Phase III Trials
CSL first has to prove that CSL112 actually leads to improved cardiac outcomes. All we know so far is that CSL112 safely improves the speed of cholesterol efflux – now it has to prove that more rapid cholesterol efflux is measurably better for cardiovascular health.
That will take time and money. Fortunately a Phase III trial shouldn't take too long to conduct, with a likely endpoint for the next study being at 12 months follow up from the initial treatment with CSL112. So don't expect to hear about CSL112 in the next year at least.
Foolish takeaway
With companies like CSL that are always researching treatments, it can be tempting to gloss over the incomprehensible headlines about something called "AFSTYLA rFVIII-SC" (another recent announcement) which uses a "single polypeptide-chain to improve the stability of factor VIII" in patients. However, it's important to remind yourself that this is the next generation of treatments in development, which means that this is where tomorrow's profits and dividends are coming from.
CSL does a pretty good job of explaining in plain terms why its treatments are important, and investors should be sure to check in on its updates every now and then.
However, it's also important to remember that these treatments take a long time to come to fruition. So even though investment bank UBS upgraded CSL to buy today (on the strength of its treatment results) and shares are up 1.5%, you don't need to rush out and buy CSL right now.
