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Biogen, Alectos Announce Licensing Agreement for Parkinson Disease GBA2 Inhibitor AL01811

Alectos to receive a $15 million upfront payment from Biogen and is eligible to receive upward of $630 million pending the success of the collaboration and achieving future milestones for the investigational PD treatment.

According to a recent announcement, Biogen and Alectos Therapeutics have entered a license and collaboration agreement to develop and commercialize AL01811, a novel GBA2 inhibitor designed to treat patients with Parkinson disease (PD). The two companies will collaborate on plans for preclinical testing of the drug; however, Biogen will be solely responsible for all development, manufacturing, and commercial activates and costs.1

Under the terms and conditions, Biogen is expected to make an upfront payment of $15 million to Alectos for the exclusive global license to the therapeutic agent and additional backup molecules. Additionally, Alectos is eligible to receive up to $77.5 million in potential development payments and $630 million in potential commercial payments if the AL01811 development program achieves certain milestones.

"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, interim head, Research and Development, Biogen, said in a statement.1 "At Biogen, we aim to advance therapies that serve people with movement disorders, enabling them to have greater independence and quality of life."

With no current FDA-approved disease-modifying therapies for patients with PD, the unmet need has forced researchers to try new ways of targeting the disease. Small-molecule GBA2 inhibitors have been shown to reduce lysosomal pH and increase levels of the lysosomal proton pump V-ATPase, which is involved in maintaining the acidic pH required for lysosomal function.

Exisiting literature has shown that loss of GBA2 function results in accumulation of glucosylceramide. Mutations in the human GBA2 gene have been associated with hereditary spastic paraplegia and autosomal-recessive cerebellar ataxia, among other debilitating neuromuscular disorders, and patients with these disorders exhibit impaired locomotion and neurological abnormalities.2

"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,” Ernest McEachern, PhD, president and chief executive officer, Alectos Therapeutics, said in a statement.1 "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."

Gaucher disease (GD), a rare, inherited lysosomal storage disorder, is caused by mutations in the GBA gene leading to low or deficient levels of GBA1. Mutations in GBA1 are also the single most common risk factor for developing PD. Although, the majority of those with GD do not develop PD, suggesting the possibility of other disease-modifying factors, including GBA2.3

REFERENCES
1. Biogen and Alectos Therapeutics announce license and collaboration agreement for AL01811, a novel GBA2 inhibitor for the potential treatment of Parkinson disease. News release. June 6, 2022. Accessed July 1, 2022. https://investors.biogen.com/news-releases/news-release-details/biogen-and-alectos-therapeutics-announce-license-and
2. Woeste MA, Wachten D. The enigmatic role of GBA2 in controlling locomotor function. Front Mol Neurosci. Published online November 28, 2017. doi:10.3389/fnmol.2017.00386
3. Burke G. Interplay between glucocerebrosidase 1 and glucocerebrosidase 2; potential implications for the pathogenesis of Gaucher and Parkinson disease. UCL Discovery. Published online August 28, 2017. Accessed July 1, 2022. https://discovery.ucl.ac.uk/id/eprint/1571208/1/A%20thesis%20formatted%20final%20final%20edit.pdf