Roche thins Alzheimer's program after phase 3 failure, may seek 'external partnerships'

Roche’s Genentech unit is walking away from a clutch of studies for its leading Alzheimer’s therapy gantenerumab weeks after the monoclonal antibody failed a pair of phase 3 studies.

Reuters reported the decision to cut the programs Wednesday evening, but the writing was on the wall after gantenerumab failed to improve cognitive and functional decline in the two much-anticipated trials from the Graduate program in November. The miss was called a “resounding failure for gantenerumab,” by analysts.

Genentech confirmed the decision in an emailed statement to Fierce Biotech Thursday afternoon.

“While gantenerumab has not shown sufficient clinical benefit for people with early Alzheimer’s disease, we are very grateful for the dedication of everyone involved in this program,” a company spokesman said. “We remain committed to Alzheimer’s disease and will direct our focus to new and potentially improved approaches for new treatments within our pipeline and potentially through external collaborations and partnerships.”

On the chopping block are studies in patients with early disease, including the extension trials in the Graduate program and the Skyline phase 3 trial. Genentech’s Greg Rippon, M.D., touted the Skyline program during an interview with Fierce Biotech in August as a way to make up for a previous failure to recruit a diverse group of patients. Rippon serves as vice president and chief medical partner of neurology, ophthalmology and internal medicine.

“In the Graduate studies, the level of amyloid removal was lower than expected. We expect the same, lower effect in the Skyline population, and consider this insufficient to continue,” the spokesman said in the company's statement.

A decision on two other studies featuring gantenerumab, called DIAN-001 OLE and DIAN-002, will be made in conjunction with Washington University, which sponsored the research.

Meanwhile, Roche is developing a version of gantenerumab that uses “brain shuttle technology” to move the drug across the blood-brain barrier and boost concentrations of the active ingredient within the brain. Development of this formulation in a phase 1b/2a trial will continue, with the brain shuttle tech now to be referred to as trontinemab. The spokesman said that the Graduate data will be fully examined to inform future development of trontinemab.

Roche also recorded a failure for an AC Immune-partnered asset called crenezumab in June. The company has been tightlipped about the future for the drug and the spokesman said that participants in a one-year extension study are conducting final visits now.

“Additional analyses of these data and discussions with our collaborators will inform the next trials with this remarkable ADAD kindred,” the spokesman said.

The news comes as Genentech joined its peers in presenting data on the leading Alzheimer’s disease candidates at the Clinical Trials on Alzheimer’s Disease (CTAD) conference. The company detailed the failed studies, blaming a lack of amyloid clearance for the miss.

While Genentech’s studies were unsuccessful, Eisai and Biogen scored a major win in September with their candidate lecanemab, which did demonstrate a slowing of cognitive decline in a late-stage trial.

The Alzheimer’s Drug Discovery Foundation said in a statement that future drugs for the condition will need to do more than just clear amyloid plaques.

“The mixed data shows that while anti-amyloids are a promising starting point, we will need a combination of drugs aimed at different targets informed by the biology of aging to effectively treat this disease,” said Howard Fillit, M.D., co-founder and chief science officer at ADDF.