Research & Development
Genentech's Anti-Amyloid Beta Antibody Gantenerumab Granted FDA Breakthrough Therapy Designation in Alzheimer's Disease
8 October 2021 - - US-based biotechnology company Genentech, a member of Switzerland's Roche Group (SIX: RO) (OTCQX: RHHBY), said that gantenerumab, an anti-amyloid beta antibody developed for subcutaneous administration, has been granted Breakthrough Therapy Designation by the US Food and Drug Administration for the treatment of people living with Alzheimer's disease.

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.

Learnings from these studies have been incorporated into the optimized design of two ongoing parallel, global, placebo-controlled and randomized Phase III trials, GRADUATE 1 and 2.

The pivotal trials are evaluating gantenerumab in more than 2,000 participants for more than two years and are expected to be completed in the second half of 2022.

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 Genentech's portfolio of medicines.

AD is a progressive, fatal disease of the brain characterized by a decline in memory, language, and other thinking skills as well as changes in mood and behavior.

Biological changes in the brain are believed to start decades before clinical symptoms of AD become evident. Alzheimer's is the most common form of dementia, which currently affects more than 55m people worldwide, and is projected to reach 78 m by 2030.

An enormous and growing public health challenge, it is predicted to cost the global economy a cumulative USD 20tn over the next decade, or the USD 2.8tn per year by 2030.

Approximately 10 m people are diagnosed with AD each year. Given the medical and societal complexities of AD, several tools and treatment options will likely be required to meet the multiple and diverse needs of people living with the disease.

Genentech is continuing to explore multiple approaches and molecules that may address key pathways of AD, including amyloid beta and tau, as well as innovative tools designed to more effectively diagnose AD and support clinicians in monitoring disease progression.

Gantenerumab is an investigational IgG1 antibody designed to bind to aggregated forms of amyloid beta and remove brain amyloid plaques, a pathological hallmark of Alzheimer's disease.

Gantenerumab significantly lowered brain amyloid plaques in patients with sporadic AD in the SCarlet RoAD and Marguerite RoAD open-label extension studies and in patients with dominantly inherited AD in the DIAN-TU-001 study.

Learnings from these studies have been incorporated into the optimized design of two ongoing parallel, global, placebo-controlled and randomized 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 participants treated for more than two years in up to 350 study centers in more than 30 countries worldwide.

It is evaluating a monthly target dose of 1,020 mg with an optimized titration, aimed at maximizing exposure and minimizing dose interruption throughout the study period for better detection of a potential clinical benefit.

Data from both trials are expected in the second half of 2022.

Genentech and Roche say they are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, stroke, Alzheimer's disease, Parkinson's disease, and autism spectrum disorder.


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