Biogen announced this week that it was cancelling the dementia drug it was testing in a Phase 2 trial that aimed to fight tau buildup in the brain. The drug, gosuranemab, was being tested on patients with progressive supranuclear palsy, as well as several other tauopathies, including chronic traumatic encephalopathy (CTE).
The gosuranemab trial was being conducted at the University of California San Francisco (UCSF), as part of several basket trials that were aiming to target tau protein across various types of dementia.
In a company announcement, Biogen noted that the primary endpoint of the Phase 2 study, called PASSPORT, “was not met, and further development of gosuranemab in progressive supranuclear palsy (PSP) will not be pursued.”
Progressive supranuclear palsy is a brain disorder impacting movement and balance, and is associated with brain cell deterioration. The trial was also testing gosuranemab’s efficacy on several other dementias and brain conditions associated with tau protein buildup, including corticobasal syndrome, nonfluent variant primary progressive aphasia, frontotemporal dementia and chronic traumatic encephalopathy (CTE).
PASSPORT had enrolled 490 patients who were receiving either a placebo or an infusion every four weeks. But Biogen said that after 52 weeks of the trial, the results were “not statistically significant” and the drug didn’t show it worked on important clinical standards.
“We are disappointed with the efficacy results of the Phase 2 PASSPORT study,” Dr. Alfred Sandrock Jr., Executive Vice President, Research and Development and Chief Medical Officer at Biogen, said in the news release. “We remain unwavering in our commitment to advancing therapies that have the potential to address the significant unmet medical needs of people with neurodegenerative diseases who are faced with limited to no treatment options.”
Earlier this month, Biogen presented its topline data for another Alzheimer’s drug, aducanumab, at the Clinical Trials on Alzheimer’s (CTAD) conference in San Diego. The new data came after a major announcement in October that Biogen would be reviving aducanumab after halting it in March. At the conference, experts called aducanumab’s revival and latest data a “milestone achievement” in the field of Alzheimer’s drug development.
Just a few weeks later, Biogen’s cancellation of its other drug, gosuranemab, comes as an unexpected surprise to trial participants. When asked for remark, Dr. Adam Boxer, director of UCSF’s Neuroscience Clinical Research Unit who was heading the trial, said he was unable to comment at this time.
Shock For Trial Participant After Dementia Drug Cancellation
Steven Barbieri, a former vice president in banking and active martial arts practitioner who was diagnosed with chronic traumatic encephalopathy (CTE) at age 51, was one of the participants on the CTE trial. He said he was walking through Target with his wife when he got the phone call from UCSF that the trial had been cancelled.
“It was a complete shock,” Barbieri told Being Patient. “I felt like someone did a cheap shot and hit me in the stomach when I wasn’t ready.”
Though CTE is a form of dementia, it’s caused by traumatic brain injuries — or repeated blows to the head causing sub-concussive hits — and is more prevalent among athletes who play contact sports like football or soccer. For Barbieri, the CTE was likely caused by repeated hits to the head during his lifetime of practicing martial arts. He was one of eight people with CTE in the basket trial.
But he said that during the few months on the trial, he had believed he wasn’t on the placebo and that the drug had been working.
“I always thought that I bet I was getting the drug, because I felt good — I felt like my memory was being unlocked,” Barbieri said. “My long-term memory was opening. I’d have all these unlocking of long-term memories that would flood in, randomly.”
That’s why the call was such a shock, he said.
For Barbieri and many other dementia patients who are hoping for a new therapy, trial cancellations can be devastating. Many patients, who have driven hours to sit through numerous MRIs, infusions and reviews, find themselves feeling confused and hopeless after a cancellation.
Earlier this year, a string of trial cancellations for drugs targeting beta-amyloid left researchers and patients reeling, and wondering what the next steps for an Alzheimer’s treatment would entail.
When Biogen announced it would be reviving aducanumab, some of those hopes were restored. But the FDA will still need to approve aducanumab before it can be put into clinical practice.
Struggling to Find CTE Trials
Aducanumab’s revival brought a boost of motivation for researchers working on Alzheimer’s treatments. And even though the 50 to 60 clinical trials that exist for Alzheimer’s is a smaller number compared to the hundreds and even thousands of trials for cancer or heart disease, it’s better than nothing.
Still, for people with CTE in particular, Barbieri says it’s nearly impossible to find a trial to join.
“It’s been very hard for me with CTE because I’ve been looking for a CTE-specific trial for close to three years before I finally got into the UCSF one,” Barbieri said.
He added that he had been searching for dementia trials to join for months. “I’d get connected by an email, fill out the information online, I’d do phone interviews and then we’d drive all the way down,” Barbieri said. “But somebody would open up my file and say, ‘oh you have CTE, that’s not organic dementia. Yours was caused by head injuries.’ I’d be very devastated when I was turned down.”
For the small amount of CTE trials that do exist, “they’re looking for those obvious prominent athletes,” Barbieri said. “They’re not looking at the average guy who got hit in the head or who got concussions, and not even military people with head injuries. It’s so difficult for the average person like me to get into a trial.”
Despite the disappointment in the cancelled PASSPORT trial, Barbieri says he won’t stop looking for a new trial to join.
“Maybe they need to tweak the drug and I might have that opportunity to rejoin,” Barbieri said. “I did tell them, can you put my name at the top of the list the next time something comes around.”