NEW YORK – Denali Therapeutics on Thursday said it has treated the first patient within the global Phase IIa BEACON trial with BIIB122, a LRRK2 inhibitor it is developing as a treatment for Parkinson's disease associated with mutations in the LRRK2 gene.
LRRK2 mutations are the most common cause of inherited Parkinson's, accounting for up to 5 percent of familial cases and 2 percent of sporadic Parkinson's cases. BIIB122 is a small molecule inhibitor designed to slow progression of the chronic, progressive neurodegenerative disease by blocking LRRK2 and improving lysosomal dysfunction.
South San Francisco, California-based Denali is enrolling patients into the randomized-controlled BEACON trial to evaluate the safety, tolerability, and pharmacodynamics of BIIB122. About 50 participants will take the oral medication daily for 12 weeks in the clinical trial, followed by an open-label extension study.
The BEACON study is supported through a collaboration and development funding agreement between Denali and an unnamed third party. Denali filed the investigational new drug application seeking permission from the US Food and Drug Administration to begin the Phase IIa trial and is in charge of designing and executing the study.
"We look forward to continued collaboration with the Parkinson's community as we aim to generate biomarker and safety data to inform how LRRK2 inhibition may have an impact on the course of this disease," Denali Chief Medical Officer Carole Ho said in a statement.
Separately, through a collaboration with Biogen, BIIB122 is being investigated in patients with early-stage Parkinson's with or without an LRRK2 mutation in the global Phase IIb LUMA trial. Denali in 2020 announced it would codevelop BIIB122, previously known as DNL151, and advance it into late-stage clinical trials in a collaboration with Biogen.