Initiation of the phase III LIGHTHOUSE study of BIIB 122 in Parkinson’s disease associated with LRRK2 pathogenic mutations
Biogen Inc. and Denali Therapeutics Inc. announced that dosing has commenced in the global Phase III LIGHTHOUSE study to evaluate the efficacy and safety profile of BIIB 122 (DNL 151), as compared to placebo in approximately 400 participants with Parkinson’s disease and a confirmed pathogenic mutation in the leucine-rich repeat kinase 2 (LRRK2) gene
The primary endpoint of the LIGHTHOUSE study is time to confirmed worsening, as assessed using the Movement Disorder Society-Sponsored Revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS) over the treatment period, up to 180 weeks. Participants will be randomized to receive oral BIIB122 or placebo once daily.
“Mutations in the LRRK2 gene comprise the most frequent mutations found in Parkinson’s disease, indicating that LRRK2 inhibition may be a promising therapeutic approach to the disease,” said Samantha Budd Haeberlein, Ph.D., Head of Neurodegeneration Development at Biogen. “The LIGHTHOUSE study will specifically recruit individuals with a pathogenic mutation in LRRK2, enabling us to test the genetic hypothesis and implicated lysosomal pathway. The LIGHTHOUSE study is the largest study ever undertaken in individuals with Parkinson’s disease caused by a LRKK2 mutation.”
BIIB 122 is an investigational small molecule inhibitor of LRRK2 that was discovered and initially developed by Denali. Denali and Biogen are co-developing and co-commercializing BIIB 122 for the potential treatment of Parkinson’s disease.
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