US biotech major Biogen (Nasdaq: BIIB) and Canadian biopharma firm Alectos Therapeutics have entered into a license and collaboration agreement to develop and commercialize a novel pre-clinical selective GBA2 inhibitor, AL01811, which has first-in-class potential as an oral disease modifying treatment for patients with Parkinson’s disease (PD). This collaboration combines Alectos’ expertise in small-molecule therapeutics with Biogen’s development capabilities in movement disorders.
Increasing evidence suggests that lysosomal dysfunction plays a key role in the pathology of neurodegenerative disease, including PD. Small-molecule GBA2 inhibitors have been shown to reduce lysosomal pH and increase levels of the lysosomal proton pump vATPase, which is involved in maintaining the acidic pH required for lysosomal function. Currently, there are no approved disease modifying therapies for PD, creating significant unmet needs for patients seeking to slow disease progression. If approved, AL01811 has the potential to improve the standard of care for movement disorders such as PD, which affects approximately 10 million patients worldwide.
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