So what did the latest update tell us?
It was a 90 day follow up, and it looked at 501 patients that were part of a study called ORION-1. The data showed that the drug was well tolerated, and importantly, there was no investigational drug-related elevation of liver enzymes, neuropathy or change in renal function. These latter three points are important, as they can all be associated with various degrees of SOC. The data demonstrated a so-called substantial knockdown of LDL-C, and did so by way of a regimen that could potentially validate administration just two or three times a year, again giving it a one up on current SOC, which is far more regular (if not daily, in many instances).
So what are we looking for from the detailed analysis?
Well, we want to know exactly what the phrase Substantial Knockdown means. If the numbers are in line with the numbers seen in the phase I, which were evaluated as a secondary, then the drug looks good to move in to a pivotal. Before it does that, however, there’s a day 180 follow up for 200 of the 501 patients set to present, and this also is set for presentation at AHA. The 180-day data is more about dosing schedule than anything else. If the drug can maintain its efficacy across the period, 0-180, then it suggests a bi-annual dosing. Tri-annual is pretty much already established, but the 180 data could build on this and make the drug that little bit more competitive when it comes to commercialization.
So, that’s about it for now – we’ve got some decent data but there’s not too much insight into how it stands up to current SOC from an efficacy perspective available right now. AHA s where the real catalysts lie, and we’ll be watching it closely to see what insight we can garner come presentation day.
Note: This article is written by Mark Collins and was originally published at Market Exclusive.