Dr. Derek Lowe, from In the Pipeline, writes about another disappointing failure to treat Alzheimer's Disease:
Merck announced last night that the first Phase III trial of their beta-secretase (BACE) inhibitor verubecestat was stopped because of futility. The monitoring committee, after looking over the data so far (the trial's been running since 2012) concluded that there was no real chance of seeing efficacy.
[...] The list of Alzheimer's clinical failures is impressive, but the list of failures to clinically validate the amyloid hypothesis is even more so.
[...] Beta-secretase inhibitors have failed in the clinic. Gamma-secretase inhibitors have failed in the clinic. Anti-amyloid antibodies have failed in the clinic. Everything has failed in the clinic. You can make excuses and find reasons – wrong patients, wrong compound, wrong pharmacokinetics, wrong dose, but after a while, you wonder if perhaps there might not be something a bit off with our understanding of the disease. Remember, every time one of these therapies comes around, it builds on the failures before it. Better and better attempts are made – I mean, verubecestat seems to be a pretty good compound, from the preclinical drug discovery perspective. It's surely the best swing anyone's taken at beta-secretase (and there have been many). But it just flat out did not work.
The good news about this study is that it adds to the evidence that the amyloid hypothesis of Alzheimer's Disease is a blind alley and that the presence of amyloid plaques is simply correlative and not causative. As more data comes in from the study, I hope that the evidence will be conclusive enough that more effort will be spent on pursuing other therapeutic targets.
Pfizer has announced that it will halt efforts to find new treatments for Alzheimer's and Parkinson's diseases. Meanwhile, Axovant Sciences will halt its studies of intepirdine after it failed to show any improvement for dementia and Alzheimer's patients. The company's stock price has declined around 90% in 3 months:
Pfizer has announced plans to end its research efforts to discover new drugs for Alzheimer's and Parkinson's diseases. The pharmaceutical giant explained its decision, which will entail roughly 300 layoffs, as a move to better position itself "to bring new therapies to patients who need them."
"As a result of a recent comprehensive review, we have made the decision to end our neuroscience discovery and early development efforts and re-allocate [spending] to those areas where we have strong scientific leadership and that will allow us to provide the greatest impact for patients," Pfizer said in a statement emailed to NPR.
[...] Despite heavily funding research efforts into potential treatments in the past, Pfizer has faced high-profile disappointment in recent years, as Reuters notes: "In 2012, Pfizer and partner Johnson & Johnson (JNJ.N) called off additional work on the drug bapineuzumab after it failed to help patients with mild to moderate Alzheimer's in its second round of clinical trials."
Another potential treatment for neurodegenerative disorders — this one developed by Axovant, another pharmaceutical company — also found itself recently abandoned. The company dropped its experimental drug intepirdine after it failed to improve motor function in patients with a certain form of dementia — just three months after it also failed to show positive effects in Alzheimer's patients.
Looks like GlaxoSmithKline got a good deal when they sold the rights to intepirdine to Axovant Sciences in 2014.
Also at Bloomberg.
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Dr. Lowe, from In the Pipeline, writes of how the efficacy requirements of the FDA save US taxpayers money:
Remember solanezumab? That was the amyloid-targeting antibody that Eli Lilly kept on investigating in trial after trial, looking for some effect on Alzheimer’s. Last November, the final, final word finally came down that it really, truly, does not work. To recap, mouse model results with a similar antibody were published in 2001. Phase I results of solanezumab itself were published in 2010, and Phase II results were published in 2012.
The authors of the NEJM [New England Journal of Medicine] paper would like to point out that under the current system, the cost of investigating all this was largely borne by the drug’s developers, not the patients and not the taxpayers
[...] Under a system designed to speed up drug approvals, people might have started taking it back in 2010-2012, when the Phase I and II results showed no adverse effects.
[...] We have a very tightly regulated and opaque market indeed in this country for prescription drugs and every other form of health care, and it’s not a very good place to discover prices or utilities. You could imagine a system where these things could be done better than we’re doing them, but such a system would be pretty far from what we have going now.
[...] The NEJM paper estimates, pretty conservatively, that had solanezumab been given conditional approval back in 2012 or so, that we – meaning Medicare, for the most part, which is to say all taxpayers, but also insurance companies and patients – would have spent at least ten billion dollars injecting Alzheimer’s patients with an expensive placebo. No one would have gotten the tiniest bit better. False hope all around, with no benefit, and billions of dollars down the tubes.
Note: Bold added by submitter.