Biogen Alzheimer’s Plans Met with Hope, Some Skepticism


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Biogen’s plan to seek regulatory approval for aducanumab as a potential new treatment for Alzheimer’s disease is being met with hope for a needed therapy for a dreaded disease, but also some skepticism regarding its long-term efficacy and, ultimately, potential.

On Thursday, Biogen presented full aducanumab data at the 12th Clinical Trials on Alzheimer’s Disease conference in San Diego that showed a re-examination of the full data from the global Phase III trials, ENGAGE and EMERGE, as well as the Phase II EVOLVE trial,  showed promising results. The company said a subset of patients who had been given a higher doses saw significant benefits on measures of cognition and function, including memory, orientation, and language. Those patients also saw benefits in daily living activities. With that data in hand, Biogen plans to take this to regulators in hopes of seeking approval – a move that has thrilled the Alzheimer’s community, which hasn’t seen a new medication in two decades.

Maria C. Carrillo, chief science officer of Alzheimer’s Association, was hopeful. She called Biogen’s presentation an “important moment” for the Alzheimer’s field. Carrillo said she, the association and the more than 5 million Americans with Alzheimer’s disease, are looking forward to the FDA’s reactions to the data.

If the FDA approves this drug for the treatment of Alzheimer’s, Carrillo said the association’s expectation is that everyone for whom the drug will be beneficial will have access to treatment as soon as possible.

“No stone can be left unturned. The Alzheimer’s Association believes we must advance all potential treatment avenues, and explore methods for combining approaches that are proven to be successful,” Carrrillo said in a statement.

Carol Routledge, director of research at Alzheimer’s Research in the United Kingdom, said aducanumab shows a clear effect on key hallmarks of the disease. The data provided this week by Biogen provides an explanation for why the two phase III trials may have shown such different top-line results, suggesting people benefitted more when on a higher dose for a longer period of time, she said.

“Regulators now have a critical decision to make -- are the benefits seen in these two 18-month-long trials large enough to make a meaningful difference to the lives of people with dementia? That decision will ultimately determine who may get access to aducanumab, and other drugs like it, in future and under what restrictions. We are now in unchartered territory for an Alzheimer’s drug, and there will be huge anticipation around how the FDA decides to proceed,” Routledge said in a statement.

Howard Fillit, founding executive director and chief science officer of the Alzheimer’s Drug Discovery Foundation, said Biogen’s announcement suggests that aducanumab worked at higher doses in a percentage of the study population. However, Fillit said in an emailed statement that more data will be needed to show the drug has a meaningful clinical effect for patients.

“I believe we are at a pivotal time in Alzheimer’s research. This is not a magic bullet, but an incremental step forward in advancing the science and potentially bringing a new, disease-modifying treatment to patients,” Fillit said in a statement.

Though there are still questions about the data, Fillit said that if aducanumab is approved, it may be effective in combination with other therapies in development. The treatment of Alzheimer’s will likely follow the path set by cancer where precision medicine via combination therapies will provide individualized and targeted therapies for patients, Fillit noted.

“As more therapies with novel drug targets other than amyloid are developed, including inflammation, we need more novel biomarkers that can measure effectiveness. Such biomarkers will further revolutionize how we approach Alzheimer’s disease by allowing us to improve diagnosis, accelerate clinical trials and make them more efficient, and more accurately monitor response to treatment and disease progression,” he said.

While company stock jumped following the announcement, analysts and some in the Alzheimer’s community showed a little more trepidation. Alessio Brunello, senior pharma analyst at GlobalData, said Biogen has “skepticism and criticism” for the way the data from the aducanumab trials were analyzed, as well as its decision to move forward with seeking regulatory approval, which had been announced in October. Data from the EMERGE study were positive with convincing clinical efficacy in the high-dose arm, Brunello said. However, he added that the ENGAGE study did not show the same pattern of target engagement or clinical efficacy.

Brunello pointed out that the data presented at the CTAD conference did not show any new results from the data released by Biogen in October. But, while there may be some skepticism over the data, Brunellow said it’s highly likely the FDA will approve the medication even with a modest benefit due to the lack of available treatments. If it is approved, GlobalData estimates sales of aducanumab to hit $4.6 billion by 2026.

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