Biogen and Denali dose first patients with LRRK2 inhibitor LRRK2 inhibitor in Parkinson’s disease study

2022-10-04

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Biogen and Denali Therapeutics have begun dosing the first participants in a phase 3 study to evaluate the efficacy and safety profile of BIIB122 (DNL151) in approximately 400 patients with Parkinson’s disease (PD) and a confirmed pathogenic mutation in the leucine-rich repeat kinase 2 (LRRK2) gene, the companies announced. LRRK2 is a complex, multi-domain protein found in neurones and many other tissues and cell types throughout the body. Following discovery of the LRRK2 mutation as a pathogenic genetic factor for PD, further research has uncovered that it has the potential to be a novel therapeutic target for PD. BIIB122 is a selective, central nervous system-penetrant small molecule inhibitor of LRRK2 that is hypothesised to improve lysosomal dysfunction. The global, randomised, placebo-controlled, phase 3 LIGHTHOUSE study’s primary endpoint 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 of up to 180 weeks. Samantha Budd Haeberlein, head of neurodegeneration development at Biogen, said: “Mutations in the LRRK2 gene comprise the most frequent mutations found in PD, indicating that LRRK2 inhibition may be a promising therapeutic approach to the disease. “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 PD caused by a LRRK2 mutation.” PD is the fastest growing neurological condition worldwide, with 145,000 people in the UK living with the disease. Mutations in the LRRK2 gene account for 4-5% of familial and 1-2% of sporadic PD. There is currently no cure for PD and, though current treatment options can help to manage symptoms, they often come with side effects and become less effective over time. BIIB122 is an investigational drug that is not approved by any regulatory authority, and its safety and efficacy have not been established. Carole Ho, chief medical officer of Denali, said the initiation of the phase 3 trial “marks an important milestone in the BIIB122 development programme”. "Together with the recent initiation of the phase 2b LUMA study in early-stage PD, we hope to have the opportunity to bring a novel therapeutic option to people living with PD,” she added.

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