Biogen and Alectos Team Up to Advance AL01811 for Parkinson’s

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by Vanda Pinto, PhD |

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Biogen and Alectos Therapeutics have signed an exclusive license and collaboration agreement to further develop and commercialize AL01811, a small molecule therapy, as a potential treatment for Parkinson’s disease.

“Through this collaboration with Alectos, we hope to improve the lives of people living with Parkinson’s disease by advancing the research and development of a potential-first-in-class oral treatment that may slow disease progression,” Priya Singhal, MD, interim head of research and development at Biogen, said in a press release. “At Biogen, we aim to advance therapies that serve people with movement disorders, enabling them to have greater independence and quality of life.”

One of the main features of Parkinson’s disease is the toxic accumulation of alpha-synuclein protein clumps in nerve cells. Malfunctioning lysosomes — small recycling structures in cells — are thought to play an important role in this process. When lysosomes fail to work properly, harmful substances can accumulate within the cell.

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An acidic — or low pH — environment is necessary for lysosomes to function efficiently. In fact, preclinical studies using a mouse model of Parkinson’s have shown that re-acidifying lysosomes protects against nerve cell loss.

AL01811 is a small molecule oral therapy designed to inhibit an enzyme called GBA2 and slow the progression of Parkinson’s. The inhibition of GBA2 is expected to decrease lysosomal pH and raise levels of vATPase, a lysosomal proton pump involved in maintaining the acidic pH needed for adequate lysosomal function.

According to Alectos, blocking GBA2 has shown beneficial effects in several mouse models of lysosomal storage disorders, such as Batten disease, and it has also been validated as a safe, potential target in people.

Under the agreement terms, Biogen will make an upfront payment of $15 million to Alectos Therapeutics for an exclusive global license to AL01811 and other undisclosed backup molecules.

Should the collaboration reach specific milestones, Alectos will be eligible to receive up to $77.5 million in development payments and $630 million in commercial payments. Additionally, Alectos may also receive tiered royalties.

Both companies will be involved in the preclinical studies of AL01811, and once the investigational therapy is used in clinical settings, Biogen will take on the sole responsibility for all development, regulatory, manufacturing, commercial activities, and costs.

“By combining Alectos’ expertise in small-molecule therapeutics with Biogen’s global development expertise in Parkinson’s disease, we believe we will be well-positioned to help bring AL01811 to patients in need,” said Ernest McEachern, PhD, Alectos’ president and CEO.

“We’re also energized by the opportunity to tap into Biogen’s commercial acumen in order to advance the standard of care in Parkinson’s disease and movement disorders more broadly,” he added.