- A drug in development bestows powerful anti-cholesterol effects that may last even longer than newly approved PSCK9 inhibitors from Amgen and Sanofi/Regeneron.
- The drug is being co-developed by Alnylam Pharmaceuticals and the Medicines Co.
- Currently, the PSCK9 market in the U.S. is comprised solely of Sanofi/Regeneron's Praluent and Amgen's Repatha. Pfizer also has a PCSK9 inhibitor in development. The Alnylan/Medicines Co. drug—ALN-PCSsc—has a different mode of action (MOA).
The goal of all companies involved in this therapeutic space, which now includes Alnylam, Medicines Co., and Pfizer in addition to Amgen, Sanofi, and Regeneron, is to participate in what is sure to be a multi-billion dollar market over the long term. In fact, Thomson Reuters estimates that by 2020, the market will be worth more than $2 billion.
In a phase I trial that included 69 patients, one 300-mg shot of ALN-PCSsc reduced LDL-C by more than 50% at the 12-week mark, and even after 140 days, the reduction was still 44% lower than baseline. Add to that the fact that the drug was well tolerated and there were no significant adverse events, and it seems that ALN-PCSsc could be a major winner.
It's still early for this drug, however. Even if it is approved, it probably won't be available until 2020—at which point the main issue surrounding these medications will probably be price. The current crop of PSCK9 inhibitors cost more than $14,000 per year before discounts.