If successful, this will herald the dawn of a new era in the medical world.
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Editor’s note: It is reported that Baijian and its partner Eisai announced that it plans to submit Aducanumab’s “Biologics License Application” to FDA in early 2020. If approved, Aducanumab will be the first to improve Alzhai. A drug with clinical symptoms of silent disease. This seems to be an exciting news, but the relevant professionals still remind: according to the current research situation, it is far from the time to celebrate. So what did the research discover? Why is the FDA hesitant? Let’s take a look. This article is translated from the article in the New York Times titled “That New Alzheimer’s Drug? Don’t Get Your Hopes Up Yet.”
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October 22, Biogen and its partner Eisai announced that they plan to submit Aducanumab to FDA in early 2020 after consultation with the US Food and Drug Administration (FDA). The Biopharmaceutical License Application (BLA) (early Alzheimer’s treatment drug) and will continue to engage in dialogue with regulators in international markets, including Europe and Japan. If approved, Aducanumab will be the first drug to improve the clinical symptoms of Alzheimer’s disease, and it will also prove that reducing the accumulation of beta amyloid in the brain will help improve the symptoms of Alzheimer’s disease.
Alzheimer’s disease is a neurodegenerative disease. At present, there are 50 million Alzheimer’s patients worldwide, and there is an explosive growth, which is expected to reach 150 million by 2050. The company’s CEO, Michel Vounatsos, said that if the drug was approved, about 10 million Americans would be eligible for treatment.
However, even so, depending on the current research situation, there is still no time to celebrate.
Because the company has not released the latest analysis results, most of the experts are currently ignorant of the efficacy of this drug. Some media speculate that it may neither prevent nor treat Alzheimer’s disease: because the company only claims that Aducanumab can slow down the cognitive decline of some patients.
In fact, In March of this year, Baijian and Eisai announced the termination of Aducanumab’s two global studies of ENGAGE and EMERGE, which are based on predictions. The study is unlikely to reach its primary endpoint upon completion.
The decision to go public is based on a new analysis with the FDA. In the new analysis, the EMERGE group reached its pre-set primary endpoint, cognitive decline in patients treated with high-dose Aducanumab. Significantly reduced, significant gains in cognitive and functional indicators such as memory, orientation and language. Patients’ daily activities also benefit, including personal finance, housework, and independent travel (“just in time for Halloween, Aducanumab is also back to life”, a pharmaceutical industry analyst in the email Say).
Let’s take a look at some of the points that the company announced on Tuesday.
What is the new drug?
Information shows, Between 1998 and 2017, 146 Alzheimer’s drugs worldwide failed in clinical R&D centers, 40% died in early clinical stages, 39% in The mid-term clinical announcement failed and 18% failed in the late clinical stage.
Because this is a “blockbuster” that can swept the market as long as it succeeds, annual sales will start at least $1 billion. Alzheimer’s disease has long been considered the largest cake in biopharmaceutical research and development, with billions of dollars of potential.
Because of the huge gaps in the market and the pressure of social aging, even if the research and development difficulties, the pharmaceutical companies are still “going forward.” According to the registration data of clinicaltrials.gov, giants such as Roche, Baijian, Yangsen, Lilly and Takeda are still there.
A report published by the American Association of Pharmaceutical Research and Manufacturers (PhRMA) in 2018 states that despite the tremendous efforts, only six Alzheimer’s disease drugs have been approved for FDA approval. In order to treat this disease, pharmaceutical companies have now spent billions of dollars on theMost of the bed tests ended in failure, so that some companies have completely abandoned this aspect of exploration.
The drug to be marketed by Baijian is Aducanumab (BIIB037), a research monoclonal antibody used to study early treatment of Alzheimer’s disease. It attaches to a specific protein to make it ineffective. This drug removes a key protein in Alzheimer’s disease: beta-amyloid.
Aducanumab requires an intravenous infusion once a month. I hope that if the patient is treated early in the disease, the brain is likely to return to normal, and if not, the progress of the disease will slow down.
According to the cooperative development and licensing agreement, Baijian introduced Aducanumab from the license of Neuimmune. Since October 2017, Baijian and Eisai have cooperated in the development and commercialization of this monoclonal antibody.
What did the study discover?
The study involved approximately 1,600 patients, and brain scans showed that the drug reduced beta-amyloid levels in the brain. In one study, the risk of cognitive decline was also slowed; however, in another study, there was no substantial improvement in the patient’s clinical performance.
But the company changed the design of the second study midway, giving patients in this group a higher dose. But as of March this year, this change has not brought any clinical benefits. But the researchers said the situation changed as more data was entered.
Now, the data for Hundred Keys comes from more than twice as many patients in two clinical trials, and managers use these results to justify seeking approval. But the findings have not yet been published in peer-reviewed journals, and experts remain skeptical about whether the drug really works. If it does work, the effect remains to be seen.
It is reported that the company plans to submit an analysis report at a scientific meeting in December.
If approved, the drug will be the first treatment to reduce the clinical decline in Alzheimer’s disease and will be the first treatment to prove that amyloid beta protein can lead to better clinical outcomes.
In this regard, Michel Vounatsos, CEO of Baijian, said, “We hope to provide patients with the first treatment to reduce the clinical decline of Alzheimer’s disease, and These results have potential implications for similar approaches to amyloid beta.”
If the drug is considered to be a failure, why should we talk about it?
At present, there are 50 million Alzheimer’s patients worldwide. This data does not include those in the early stages. Due to the lack of effective treatments, the disease is almost a devastating blow to patients and the families that care for them.
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The staff of the 100-key group believes that additional research data show that the cognitive ability of the two groups taking the highest dose is slower. This finding, together with some positive results from another study, may be enough to convince the FDA that the drug is effective.
Warnasos said that the 100-key company met with officials of the FDA agency on Monday, and they told the company that the application for approval was “reasonable.”
However, an FDA spokesperson said the FDA declined to comment on research drug or drug approval applications.
Why does the FDA hesitate?
First, the Hundred Keys only provide a summary of their data and analysis. When Alzheimer’s experts see these details in December (preferably when a peer-reviewed paper is published as a supplement), they may be convinced that the drug will bring substantial benefits.
Second, new analysis of patients receiving high-dose treatment may not be reliable. In the so-called two-group analysis, it is easy to segment the data to prove the correctness of the conclusion—this is why large randomized trials with well-defined endpoints are so valuable. In general, subgroup analysis is considered a method of generating hypotheses, not a method of testing for correctness.
The Dr. Ronald Petersen, a researcher at Alcoa’s Alzheimer’s Clinic, advised Baijian, and he pointed out that the company has not yet convinced the FDA to believe the efficacy of the drug.
He said: “They are only allowed to apply and there is no guarantee of approval. But this also givesThe drug has a chance.
What if Aducanumab is really useful?
Dr. Michael Weiner, Alzheimer’s disease researcher at the University of California, San Francisco, said that if successful, this would herald the dawn of a new era in the medical world.
However, he added: “This is not a treatment and can only slow down the recession. The actual impact on patients is still to be seen.”
Of course, once approved, it will mean that the 100-key company will make a windfall. Guggenheim Securities said that if Aducanumab is approved, its annual sales could reach $10 billion or more.
But because the company published too little data, the researchers were cautious. Dr. Murali doreswami, an expert on Alzheimer’s disease at Duke University, said: “Although it seems very encouraging on the surface, I need to see more details.”