GSK and Alector Partner to Develop Immuno-Neurology Treatments


With $700 million in upfront cash, GlaxoSmithKline and Alector forged a strategic collaboration to co-develop monoclonal antibody candidates aimed at a range of neurodegenerative diseases, including frontotemporal dementia, amyotrophic lateral sclerosis, Parkinson’s disease and Alzheimer’s disease.

The two antibody candidates, AL001 and AL101, are seen as potential first-in-class medications aimed at these indications. The antibodies are designed to elevate levels of progranulin (PGRN), a key regulator of immune activity in the brain. 

There are genetic links between elevated levels of PGRN and multiple neurodegenerative disorders, which makes it “one of the most attractive genetically validated targets for the development of new immuno-neurology treatments.”

Enrollment is ongoing for a Phase III trial assessing AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). This is a severe form of dementia for which there are no approved treatments. 

FTD affects 50,000 to 60,000 people in the United States and approximately 110,000 in the European Union. FTD-GRN represents 5% to 10% of all people with FTD. 

Additionally, AL001 is currently being assessed in a Phase II study in symptomatic FTD patients with a mutation in the C9orf72 gene. There are plans to study the monoclonal antibody in a Phase II ALS trial beginning in the second half of the year. 

AL101 is in a Phase Ia study designed to treat patients suffering from more prevalent neurodegenerative diseases, including Parkinson’s disease and Alzheimer’s disease.

Hal Barron, chief scientific officer and head of GSK’s R&D, said the company’s focus on genetics and the immune system gives researchers unique insights into the potential of PGRN-aimed drugs in neurodegenerative diseases. The company noted that the collaboration with Alector provides it with promising antibodies in immuno-neurology, a core area of focus for the company. 

“Working with Alector’s world class scientists will allow us to investigate the potential of these immuno-neurology therapies to help patients with frontotemporal dementia, a devastating disease without any currently approved treatments, as well as explore the ability to help patients with other neurodegenerative diseases, such as ALS, Parkinson’s and Alzheimer’s,” Barron said in a statement. 

Alector Chief Executive Officer Arnon Rosenthal said the collaboration with GSK will expand and accelerate the development of the company’s progranulin franchise into large indications. He noted the partnership will also bolster Alector’s late-stage development and commercial capabilities.

“Importantly, this collaboration is designed to fully support AL001 and AL101’s development and to enable Alector to continue building a fully integrated company as we strive to address the high unmet medical need in patients suffering from neurodegenerative diseases,” Rosenthal said in a statement. 

"We are confident that GSK’s extensive experience launching ground-breaking medicines at the intersection of immunology and human genetics, will ensure that AL001 and AL101 are developed to their full potential.”

Under the terms of the agreement, GSK will pay Alector $700 million upfront, with potential milestone payments climbing to $1.5 billion. Alector will lead commercial efforts associated with AL001 in orphan indications and GSK will lead the commercialization of AL101 in Alzheimer’s and Parkinson’s disease. The companies will co-commercialize and share profits in the United States, while GSK will retain exclusive commercialization rights across the globe. 

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