Amarin Corporation plc (NASDAQ: AMRN) is hitting the spotlight in a big way as of late, but not necessarily in a positive way. After releasing data from a long term Phase 3 study of Vascepa, a debate has come in with some saying that the data should lead to an expanded label and others saying that the data was skewed.
Let’s Look At The Data First
The data we’re looking at comes from the Phase 3 clinical study of Vascepa that’s known as REDUCE-IT. In the study, hundreds of patients were given either Vascepa or a placebo. The results were overwhelmingly positive.
The primary endpoint was achieved in the study with a 25% relative risk reduction in first occurrence of major adverse cardiovascular events in the intent-to-treat population. These events consisted of cardiovascular death, nonfatal myocardial infraction, nonfatal stroke, coronary revascularization and unstable angina requiring hospitalization.
The company also achieved various secondary endpoints. The key secondary endpoint was achieved with a 26% relative risk reduction in 3-point major adverse cardiovascular events. These events included cardiovascular death, nonfatal heart attack and nonfatal stroke.
Finally, 7 additional secondary endpoints were met. These included:
- A 25% reduction in risk of cardiovacular death or nonfatal heart attack;
- a reduction of 31% in risk of fatal or nonfatal heart attack;
- a 35% reduction of risk in urgent or emergent revascularization;
- a 20% risk reduction in cardiovascular death;
- a 32% reduction in risk of hospitalization due to unstable angina;
- a 28% reduction in risk of fatal or nonfatal stroke;
- a 23% risk in total mortality as well as nonfatal heart attack and nonfatal stroke.
These results look amazing. In fact, they are the best results that we have ever seen in a large, cardiovascular risk trial of its like. However, this is where the debate comes in. There were concerns raised with regard to the placebo used by AMRN.
Digging Into The Placebo Drama
While the results of the REDUCE-IT trial were overwhelmingly positive, there has been quite a bit of drama unearthed as a result of it, and it all has to do with the placebo.
You see, during the clinical trial, AMRN used mineral oil pills as the placebo. Many argue that the use of this placebo skewed the data. The issue comes in when we see what happened with the placebo group over the time treated.
First and foremost, when it comes to triglyceride levels, those being treated with the placebo saw an increase of 2%. Going further, LDL cholesterol levels raised 10% in the placebo group. When it comes to other key markers of blood fat levels, more higher levels were seen in the placebo group.
The idea behind the debate is that the increase in blood fat levels in the placebo goup may have been caused, in part, by the placebo itself. If this is the case, then comparing the benfits of Vascepa against the decline in the placebo group would be meaningless. After all, higher levels of fat in blood will generally lead to higher cardiovascular risk.
Debunking The Placebo Concerns
While the placebo concerns have been somewhat damning when it comes to growth in the value of AMRN, the truth of the matter is that it’s not very hard to debunk these concerns. Recently, Dr. Weiss, cardiologist at the University of California, weighed in with regard to Amarin’s Vascepa results. First and foremost, Dr. Weiss pointed to the 10% increase in LDL-C in the placebo group and said that this may have led to more adverse cardiovascular events. He even explained that VASCEPA benefits may have been overstated. Nonetheless, Dr. Weiss is a believer in the benefit. That can be seen from this statement:
Perhaps the benefit of Vascepa is overestimated a little bit, but the higher LDL seen in the mineral oil group is still too small an effect to account for the large reduction in relative risk. This is a landmark trial and it confirms the omega-3 heart hypothesis.
While that is only one doctor’s opinion, the big dogs have weighed in with positive opinions as well. In fact, even the New England Journal of Medicine said that the mineral oil could not have caused such a significant improvement in risk of cardiovascular events. All in all, there are plenty of highly-respected professionals that are making it clear that they believe in the cardiovascular benefit of VASCEPA.
The takeaway here is a relatively simple one. While any medical data should be the subject of debate, this debate is a hands down win for AMRN. At the end of the day, even if the placebo concerns have some validity, there is no way that the mineral oil would have led to such a significant difference between VASCEPA and placebo.
With that said, AMRN is planning on submitting the new data to the FDA early next year. I believe that this submission will result in an expanded label, allowing for the treatment to be marketed as an option to reduce the risk of major cardiovascular events. When this happens, it will prove to be a game changer for AMRN and its investors!