We’re going to have PCSK9 debate all over again

Monthly or bi-monthly treatments that drastically lower cholesterol haven’t exactly made a mint for their makers. But what if you could do the same job with a twice-a-year treatment?

That’s the proposition behind the Medicines Company and its inclisiran, an injected treatment that uses RNA interference, or RNAi, to shut down the production of PCSK9 and reduce cholesterol. Some time this quarter, the company will disclose data from three late-stage trials that will determine whether long-term exposure to inclisiran is safe and effective.

PCSK9 is the target of two antibody therapies from Amgen and the partnership of Sanofi and Regeneron Pharmaceuticals. Those two therapies proved to lower cholesterol and improve long-term patient outcomes, but they spent years as poster children for commercial disappointment, gaining traction only after their makers more than halved their prices to appease payers used to cheap, generic statins.

And thus the question becomes: Does the world need the Medicines Company’s every-six-months treatment? Inclisiran’s late-stage data, whenever they're available, will rekindle an old biotech debate.