First Alzheimer’s disease treatment
For the past 16 years, the searches for an Alzheimer’s disease treatment has been unsuccessful.
This scenario could start to change by next year with the resurrection of a drug once deemed failure.
But on Tuesday, Biogen said that their new analysis of larger datasets actually showed that the aducanumab was “pharmacologically and clinically active” in trial patients who have Alzheimer’s. The companies worked with the FDA to take a look at the larger datasets after the earlier failures.
The news sent Biogen’s stock skyrocketing. The company shares gained 37 percent to $305.37 in trading on Tuesday morning.
The new analysis found that the aducanumab was able to reduce the clinical decline in patients with early Alzheimer’s disease. That is, the drug kept patients from experiencing cognitive decline as fast as they might had they not been on the drug.
After calling the trials off, the scientists went back to the dataset and found the aducanumab appears to work, at least in one of the 2 studies that came with a higher dose of the medication.
Biogen said in a release Tuesday, Biogen strongly believes that the difference between the results of the larger dataset analysis and the outcome predicted by the futility analysis was largely due to patients’ greater exposure to the high dose aducanumab drug.
Biogen & Eisai are planning to file the aducanumab drug with the FDA in early 2020. If FDA approved, it would be the first new Alzheimer’s treatment in nearly two decades.
Alzheimer’s affects more than five million Americans, a number that is expected to balloon to 14 million by the year 2060. There are only 4 drugs that have been approved to treat the symptoms of Alzheimer’s disease, and the most recent drug approval happened in the year 2003. There are no drugs currently available that could reverse Alzheimer’s disease.
In recent years a number of Alzheimer’s disease trials have failed, including the one Biogen reported in March. The setbacks in the field have seen in recent years had people coming back to theory.
Research studies have determined that years – even decades – before a person might start showing symptoms, amyloid-beta deposits in the brain that are characteristic of Alzheimer’s disease can actually start to accumulate.
Starting earlier could benefit treatments that are targeting beta-amyloid deposits in the brain, with the hope that clearing them out could actually help to slow down the rate of cognitive decline. This idea of targeting beta-amyloid deposits in the brain to clear them out is known as the “amyloid hypothesis.”
But there is one major drawback to the amyloid-beta approach: In people who have Alzheimer’s, these amyloid deposits build up in certain parts of the brain, but it is still not known that whether the plaques cause Alzheimer’s, or if they are just byproducts. What does seem to be very well established is that in individuals with a genetic version of the disease, there is a very strong relationship between those DNA mutations and the amyloid plaques.
The amyloid hypothesis has been put to the test many times and has seen a few failures and this is why the reversal from Biogen now comes as a surprise. Merck’s failed BACE inhibitor was also acting on the amyloid hypothesis in order to prevent the protein from forming & keep the disease from progressing. Eli Lilly’s Solanezumab drug also acts on the amyloid hypothesis, failed some key clinical trials, though the company is still testing the drug in the preclinical stages of the disease.
Analysts, for their part, have their own doubts about the surprise reversal by Biogen. Brian Skorney, an analyst at Baird, said that he was skeptical of the new analytical results for Biogen’s Alzheimer’s treatment drug.
Skorney said in a research note that we expect Biogen will have an uphill climb as they look to convince regulators results from the EMERGE trial were nothing more than a random chance.
Brian Abrahams, RBC Capital Markets analyst, noted that it is hard to forget the earlier failure of ENGAGE, one of the 2 late-stage trials Biogen and Eisai were running alongside the EMERGE trial.
Abrahams wrote that even though FDA apparently provided some endorsement for the idea of filing, which could maintain some probability of provability, we believe the new details suggest the data may be more mixed than initially perceived and believes that the ENGAGE failure will be a point of controversy as investors debate aducanumab drug’s prospects.
Elsewhere, groups like the Alzheimer’s Association said on Tuesday that they were encouraged by the reversal filing.
Alzheimer’s Association said in a release that they have never been as optimistic as they are today.
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