Drugs

Biogen, Denali Halt Development of Parkinson’s Drug in Idiopathic Disease

Biogen and Denali Therapeutics say they will limit the development of their experimental Parkinson’s disease drug BIIB122 after the therapy failed to meet primary and secondary endpoints in a Phase 2b clinical trial. The companies reported that the LUMA study showed BIIB122, also known as DNL151, did not slow progression of early-stage Parkinson’s disease compared with placebo.

“Based on these results, Biogen and Denali will discontinue further development of BIIB122 in idiopathic Parkinson’s disease,” the companies say. “Denali will continue to independently conduct the Phase 2a BEACON study evaluating the small molecule inhibitor in carriers of a pathogenic LRRK2 variant.

The investigational oral therapy targets LRRK2, or leucine-rich repeat kinase 2, an enzyme linked to genetic forms of Parkinson’s disease and viewed as a potential disease-modifying target, according to the companies. The failure represents a setback for efforts to develop therapies aimed at slowing the underlying neurodegenerative process of Parkinson’s disease rather than simply treating symptoms.

The randomized, placebo-controlled Phase 2b LUMA study enrolled 648 patients between ages 30 and 80 with early-stage Parkinson’s disease, including both patients with and without pathogenic LRRK2 gene variants. Participants received BIIB122 or placebo for between 48 and 144 weeks.

The trial’s primary endpoint measured time to confirmed worsening on a combined score from parts II and III of the modified Movement Disorder Society Unified Parkinson’s Disease Rating Scale, a standard measure of Parkinson’s progression. Although the study failed clinically, the companies said exploratory biomarker analyses showed evidence of target engagement. BIIB122 achieved more than 90% inhibition of peripheral LRRK2 kinase activity and reduced a cerebrospinal fluid biomarker associated with LRRK2 activity by approximately 30%.

The drug was generally well tolerated and demonstrated what the companies described as an acceptable safety profile.

While the companies will end development of BIIB122 in idiopathic Parkinson’s disease, Denali said it will continue independently running the Phase 2a BEACON study in Parkinson’s patients who carry pathogenic LRRK2 variants confirmed through genetic testing. That study is evaluating safety, pharmacokinetics and biomarker effects of the therapy in genetically defined patients whose disease is associated with elevated LRRK2 kinase activity. Data from BEACON are expected in the first half of 2027.

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