Pharma, BioPharma

Biogen projects confirmatory study for Alzheimer’s drug will take four years

Six months after Biogen was awarded regulatory approval for its Alzheimer’s disease drug Aduhelm, the company is laying out a timeline for the post-marketing study to confirm whether the drug works. The projected four-year study will enroll more than 1,300 patients and will include a placebo control arm.

 

The clinical trial that could show whether Biogen Alzheimer’s disease drug Aduhelm works is slated to start in May and will take about four years—roughly half the time initially projected. That study will recruit patients globally and is designed to include a placebo control.

The Thursday announcement of Cambridge, Massachusetts-based Biogen’s clinical trial plans come about six months after the FDA granted accelerated approval to Aduhelm. For diseases that have few treatment options, such as Alzheimer’s, this pathway enables a drug to more quickly reach the market. The FDA bases these speedy decisions on a surrogate endpoint, a signal in clinical trials that a drug might be working.

Aduhelm is an antibody drug designed to break up amyloid plaques in the brain. The clinical trial results for Aduhelm were murky, but the accelerated approval was based on the surrogate of reducing the plaques of amyloid protein in the brain. Confirming that this reduction actually benefits Alzheimer’s patients will require a post-marketing study that is a condition of all accelerated approval decisions.

The Aduhelm confirmatory study will enroll more than 1,300 patients who are in the early stages of Alzheimer’s disease. Biogen and partner Eisai said they expect to submit the final clinical trial protocol to the FDA in March, then begin patient screening in May.

When Aduhelm initially received accelerated FDA approval, the agency laid out an eight-year timeline for the confirmatory study. Clinical trials for neurological disease drugs are large, and they can last years because of the time needed to enroll patients and assess how well a drug is working in those patients. The main goal of the Aduhelm post-marketing study will be to assess patients at 18 months after the start of treatment. Biogen said that based on the enrollment rates of previous Aduhelm studies, it expects the primary completion date will be about four years after the study begins. A long term extension study will collect additional treatment data for up to 48 months.

The clinical trial plans come as scrutiny mounts on both Biogen and Aduhelm. The company took heat for placing a $56,000 a year price tag on the drug, a sum that many insurers have balked at covering. In October, the company said in its report of third quarter financial results that Aduhelm sales totaled just $300,000—well below expectations. Last month, a European Medicines Agency committee gave a negative trend vote on Aduhelm, a vote that bodes poorly for that body’s formal opinion on the drug’s application for marketing authorization.

On Friday, the EMA said that Aduhelm’s benefits do not outweigh its risks and the agency recommended refusal of Biogen’s application for marketing authorization. While the EMA acknowledged that the drug reduces amyloid in the brain, it said that a link between this effect and an improvement in patients has not been established. The EMA also said that clinical research did not show that the Biogen drug was sufficiently safe, as images from brain scans showed swelling and bleeding. Biogen has option to ask the EMA to re-examine the refusal opinion, and the company said that it plans to do so. [Story updated with EMA opinion.]

Photo: Adam Glanzman/Bloomberg, via Getty Images

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