The U.S. Food and Drug Administration (FDA) has approved a re-dosing study for patients formerly enrolled in Biogen’s phase three clinical trials of its Alzheimer’s drug, aducanumab. The company said it expects patients to start receiving the drug again as early as March.
The announcement marks the latest development in the twisted tale of aducanumab. Almost a year ago, Biogen and its partner Eisai shocked the Alzheimer’s community by abruptly killing phase three studies of aducanumab, saying that the drug had failed “futility tests” and was therefore unlikely to be successful in treating the disease.
But in October, the company reversed itself and said that continued data analysis had instead demonstrated that the drug would be successful in treating Alzheimer’s.
At the time, Biogen said it would seek approval to market the drug from the FDA and, while awaiting a decision, would seek to begin offering the drug to the 2,400 people who had been enrolled in trials around the world.
In a statement emailed to Being Patient today, Biogen said that it is working with U.S. trial sites to start a new open-label clinical trial for all patients who were enrolled in the aducanumab studies. The company said it was also seeking approval for the new trial from regional regulators and institutional review boards in Europe and Japan.
“We wish to thank all patients who have participated in the development of aducanumab,” David Caouette, a spokesperson for Biogen, said. “We remain dedicated to working with urgency with the purpose of meaningfully changing the course of Alzheimer’s disease.”
Jeffrey Borghoff, a Being Patient adviser who had participated in the aducanumab trial, said he was excited to restart treatment.
“My family and I really felt that aducanumab was making a difference and we were all devastated when the trial was cancelled,” Borghoff said. “I am eager to see how I feel once I begin to receive the drug again in March.”
Participants in the new trial will receive monthly intravenous infusions of 10 mg/kg of aducanumab for a total of 100 weeks. Approximately 2,400 people who were enrolled in the trials as of March 2019 are eligible to participate in the new study. All participants in the new study will receive aducanumab; no one will receive a placebo.
In October, the company said that its new announcement was based on further data analysis that showed that patients who received aducanumab “experienced significant benefits” in memory and general cognition.
“Patients also experienced benefits on activities of daily living including conducting personal finances, performing household chores such as cleaning, shopping and doing laundry and independently traveling out of the home,” the company said.
Aducanumab targets the build-up of amyloid in the brain, which has long been suspected as a cause of Alzheimer’s. If approved by the FDA, aducanumab would be the first treatment to demonstrate that removing amyloid from the brain impacts Alzheimer’s.
Last October, Biogen presented data at the annual Clinical Trials for Alzheimer’s Disease (CTAD) conference that it said demonstrated that people with mild cognitive impairment or early Alzheimer’s disease who received a larger dose of aducanumab for a longer amount of time showed a statistically significant slowing of the disease.
At the conference, Stephen Salloway, Director of Neurology and of the Memory and Aging Program at Butler Hospital in Providence, R.I., called the news positive and that “dose matters with this drug.”
“Continued exposure to high doses benefited patients with early Alzheimer’s disease,” Salloway said.
Also at the conference, Dr. James Kupiec, Chief Medical Officer of ProMIS Neurosciences, said the Biogen presentation was “clearly positive.”
“This is not the best drug for Alzheimer’s that will ever be approved,” he said. “It will be the first drug that is approved.”
In a video interview with Being Patient, Dr. Sharon Cohen, Medical Director and Principal Investigator at the Toronto Memory Program, defended aducanumab against skeptics who continue to believe that Biogen has not yet provided enough data to win regulatory approval for the drug.
As a clinician who works with patients, Cohen said, she found the Biogen presentation “exhilarating” because of what the study found in terms of people’s ability to continue the activities of daily living, and maintain independence for longer, with the disease.
“People still being able to work, shop, travel,” Cohen said. “This matters a lot more to our patients than a score they get on a memory test.”