Is PCSK9 the Next Wonder Drug?

Henry R. Black, MD; Howard S. Weintraub, MD


September 27, 2013

In This Article

PCSK9 -- The Next Great Drug?

Dr. Black: So, how long is it going to take before we have these drugs available, do you think? We are assuming, of course, that they perform well in clinical trials that are properly done and that we can interpret.

Dr. Weintraub: PCSK9! I would have thought that the phase 2 trials [on PCSK9] have been amazing, just amazing. And they are astonishing, and I am afraid I always want to bite my tongue before I say this, but their toxicities have been minor. I mean, the other 2 drugs I talked about, the Apo-B anti-sense and the ATP inhibitor, have some notable adverse events, some of which are a nuisance and some of which are dangerous. But these [in PCSK9] have been remarkably benign.

The companies who are making these, and there are 2 out in the front right now, have already been doing studies to determine what the best delivery system would be. And they are focusing on an insulin pen kind of a deal. They haven't filed on any new drug applications, but I wouldn't be surprised if in 2014 or 2015 we would see this. That would change the landscape of lipids.

Dr. Black: Getting away from injections and getting back to injections -- that seems somewhat bizarre to me. We don't know if the average reader is going to do that.

Dr. Weintraub: And here is the problem with randomizing trials. Imagine telling people that we are going to want to treat your lipids, but half of you are going to get the placebo, and we want you to take a placebo for 2 or 3 years.

Dr. Black: And an injected placebo.

Dr. Weintraub: An injected placebo. Granted, one drug [is given] every 2 weeks and the other once every month, but that becomes a challenge. I don't know how that is going to pan out in the ultimate conduction of trials. Think about the FIELD study where people were not on statins.[4] Diabetics were given fenofibrate or placebo, and the doctors who took care of the placebo patients were looking at them and saying, "You know, I don't like how your lipids look, so I am going to start a statin." So there was all this statin drop-in in the placebo group, which totally messed up the study.

Dr. Black: Are you supposed to take your iPens, or whatever you use, on vacation with you and put them in the refrigerator in the hotel, which no longer has one? How is that going to work?

Dr. Weintraub: I haven't gotten that memo yet, so I am not sure. I would imagine that given the companies that are involved in this -- these are not Moe, Larry, and Curly companies -- they will address this problem. Also, given that you can take it every 2-4 weeks, unless you are on vacation for 6 weeks, you can probably be all right. And if you miss a dose, the world would probably not come to a screeching halt.

Dr. Black: You're not going to get a big rebound in your LDL?

Dr. Weintraub: And if you do, if you're on it for a year, so what? It's not like insulin where, without it, you are going to die or have serious side effects. I would think that that is the way that would be. I don't know; I'm not aware of the refrigeration necessities of these drugs, but I think they are trying to make it as user-friendly as possible.

Dr. Black: Howard, thank you very much. It has been a pleasure talking to you, as always. We'll have more with Dr. Weintraub soon.


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