The Medicines Company (NASDAQ:MDCO) just put out topline from its lead cholesterol trial, and the company has jumped on the back of the data. The release was a bit of a strange one, in all honesty, making numerous references to the preceding trial data, before settling on some closing remarks, but it seems positive nonetheless, and markets have obviously regarded it as so.
The data comes as a sort of preview to fuller data intended for presentation at the AHA Scientific Sessions (late breaking) come November 19. In anticipation of a little more clarity come AHA, and in advance of any further upside this clarity may initiate, let’s take a look at the drug in question and lay out what we are looking for when the detailed report hits.
The drug is called PCSK9si, and as we’ve mentioned, it’s targeting the lowering of cholesterol in adults. Some readers may already be familiar with the family of cholesterol drugs commonly referred to as PCSK9 inhibitors. Don’t get these confused with this one. They both act on the same enzyme (sort of) but they are very different as far as core MOA is concerned. PCSK9 is an enzyme that binds to the receptor on the liver that is normally responsible for destroying and clearing LDL-C (bad cholesterol). PCSK9 inhibitors bind to the PCSK9 enzyme, meaning the liver receptor remains intact, and can clear LDL-C for longer. In contrast, other SOCs like statins work to inhibit the production of LDL-C while increasing the production of the LDL-C clearing receptors.
PCSK9si is a synthesis inhibitor. We won’t go too much into the science behind it, as things could get complicated quickly when trying to differentiate between PCSK9si, PCSK9 inhibitors and statins, but essentially, it acts on the gene that codes for the PCSK9 enzyme at the RNA level, stopping the enzyme from being produced in the first place. A reduction in PCSK9 translates to more and longer lasting LDL-C clearing receptors, which – theoretically – should translate to a reduction in cholesterol on the aggregate.
With this MOA, the drug is first in class, and is targeting an approval in so far as it’s looking to provide an option for patients that have not responded to current SOC. If trial data demonstrates a boost in efficacy over the current treatments, there’s every chance this will extend to a first line a little further down the road.