Anti-amyloid antibody receives Breakthrough Therapy Designation in US

Roche’s gantenerumab, an anti-amyloid beta antibody developed for subcutaneous administration, has been granted Breakthrough Therapy Designation by the US Food and Drug Administration (FDA) for the treatment of people living with Alzheimer’s disease (AD).

This designation is based on data showing that gantenerumab significantly reduced brain amyloid plaque, a pathological hallmark of AD, in the ongoing SCarlet RoAD and Marguerite RoAD open-label extension trials, as well as other studies. Breakthrough Therapy Designation is designed to accelerate the development and review of medicines intended to treat serious or life-threatening conditions with preliminary evidence that indicates they may demonstrate a substantial improvement over available therapies that have received full FDA approval. This designation for gantenerumab marks the 39th Breakthrough Therapy Designation for Roche’s portfolio of medicines.

Gantenerumab is an investigational IgG1 antibody designed to bind to aggregated forms of beta-amyloid and remove brain amyloid plaques, a pathological hallmark of AD. Gantenerumab significantly lowered amyloid plaque in patients with sporadic AD in the SCarlet RoAD and Marguerite RoAD open-label extension studies, and with dominantly inherited AD in the DIAN-TU-001 study. Learnings from these studies have been incorporated into the optimised design of two ongoing parallel, global, placebo-controlled and randomised Phase III trials, GRADUATE 1 and 2.

The pivotal GRADUATE trials are investigating the effect of gantenerumab on amyloid load and downstream biomarkers of disease progression, as well as the safety and efficacy of gantenerumab in people with early (prodromal-to-mild) AD. The studies include more than 2,000 patients treated for more than two years in up to 350 study centres in more than 30 countries worldwide. It is evaluating a monthly target dose of 1,020mg with an optimised titration, aimed at maximising exposure and minimising dose interruption throughout the study period for better detection of a potential clinical benefit. Both trials are expected to be completed in the second half of 2022.

“For more than a decade, we have been committed to advancing the science of Alzheimer’s as well as our investigational medicine gantenerumab, and we look forward to delivering a comprehensive and robust data set that furthers our collective understanding of this devastating disease,” commented Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development. “This Breakthrough Therapy Designation reinforces our confidence in gantenerumab, which would be the first subcutaneous medicine for the treatment of Alzheimer’s disease with the potential for at-home administration.”