Could Biogens astonishing victory over Alzheimers drugs be temporary?
Biogen (NASDAQ: BIIB) recently obtained US Food and Drug Administration (FDA) approval for the Alzheimers disease drug Aduhelm. There was, however, an important catch with this approval. Motley Fool Live video recorded on June 9Motley Fool contributors Keith Speights and Brian Orelli discuss whether Biogens victory could only be temporary. Keith Speights: Now there was an asterisk with the FDA approval. The FDA has granted whats called fast-track approval for Biogens Alzheimers disease drug. Brian Orelli: Expedited approvals are based on preliminary data. These are often surrogates or small trials with a limited number of patients. The surrogate endpoint means that it is something that predicts clinical benefit. In cancer, it would be like progression-free survival. How long does it take for the tumor to start growing, but what really interests us is how long does the patient live. The progression of survival would be the surrogate endpoint for overall survival. Lowering blood pressure would be another example that reduces the likelihood of having a stroke, so you can get approval based solely on lowering blood pressure. In this case, the drug from Biogen was successful in reducing the amyloid plaques, and what were really interested in is the improved cognition. Expedited approval requires a company to conduct a confirmatory study. Biogen is due to conduct another study and if it fails, the FDA may take it off the market. In theory, this is a temporary victory. The drug was actually going to be on the market for years because it might take up to a year to get the study going. Im sure the FDA wants them to go faster, but it takes a while to design a study. Im not 100% sure how theyre actually going to conduct this study, because ideally youd like to compare it to a placebo, but who would participate in the study if, say, they do 2 to  1 drug to the placebo. , you still have a third chance to get a placebo. If you were serious about taking this medication, you would probably go and ask your doctor to prescribe it. I think it will be difficult for them to set up the study. Then they have to do the study, and it will take a few years to sign up and get everyone through. Lets say we look at cognition after a year, so the last person is going to sign up a few years later, then well have to follow the last person for a year, and then theyll have to analyze the data. I think the FDA is giving them nine years or something, I think I read yesterday   Speights: Yes. Orelli:  to actually perform the clinical trial. I think its going to be on the market for quite a while. Even then, I think we have seen that some cancer drugs have failed their confirmatory trials and are still approved for the indication. Even if this clinical trial fails, they will still be on the market for a bit longer, as it will take some time for the FDA to complete its market withdrawal process. Of course, as soon as clinical trial data is released, sales will likely decline because doctors likely wouldnt prescribe a drug that failed the most recent study. Then [laughs] maybe I can jump to my podium here for a minute and we can talk about the surrogates because thats really what got me upset because the amyloid hypothesis, the idea that amyloid, this are the misfolded protein plaques in the brain causing Alzheimers disease, is really an unproven and it has been for years. Its just if amyloid causes Alzheimers disease. What we really dont know is whether removing these plaques helps patients, and in fact, I think weve had studies where drugs lowered plaque levels and didnt improve disease. cognition. I am just stunned by this decision. It just seems like the FDA really wanted to improve the drug, but they felt like they couldnt give full approval so they approved it based on surrogate criteria. But I dont think these alternate endpoints are valid at this point. I dont think we have enough data to say that in the same way that progression-free survival leads to overall survival most of the time, or to a drop in blood pressure most of the time, or almost all of the time. , leads to a lower probability of stroke. I think that just opened up a whole new box of worms. Speights: Yes. Let me say that you are not the only person upset, as you said, by this decision. It is very controversial. I read that one of the FDA advisory committee members who had been on the panel for quite some time resigned in protest against this decision. To make matters worse, Brian, Biogen came out and announced a surprisingly high price for this Alzheimers drug. I think most analysts expected between $ 10,000 and $ 25,000 for the drug. They came out with a prize of $ 56,000. Orelli: I think they did it because you dont expect them to have more sales because their label is horrible and that doesnt show that it actually helps patients. I dont think they will have as many patients on treatment. This would be the same reason why the indications often have really expensive drugs because there are not many patients and therefore, you have to pay dearly to justify the development of the drug. Speights: I think you are absolutely right. To me, its surprising that Biogen is offering such a high price. I wouldnt have been shocked at all if they had come up with a price that was around the high end of the range that exports look for, but to come up with a price that was more than double the high end. of the range which most experts projected, to me, was surprising. I think that just further fuels this controversy. One of the things was the price. My point was that the lower the price, the more bullish I will be because I think that means Biogen thinks its going to be used a lot. The higher the price, the less bullish I was going to be because to me that means Biogen doesnt think it will be used by very many patients.