News & Events zu Denali Therapeutics Inc (DNLI)DNLIDenali Therapeutics Inc

Denali Therapeutics Sells Rare Pediatric Disease PRV for $195M

Denali Therapeutics announced it has entered into a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher, PRV, for gross proceeds of $195M. The U.S. Food and Drug Administration, FDA, awarded the PRV to Denali following accelerated approval of the enzyme replacement therapy Avlayah for the treatment of Hunter syndrome in March 2026. Avlayah is the first FDA-approved medicine in an emerging class of biotherapeutics designed to cross the blood-brain barrier via transferrin receptor-mediated transport.

Biogen and Denali Discontinue BIIB122 Development

Biogen (BIIB) and Denali Therapeutics (DNLI) announced topline results from the Phase 2b LUMA study evaluating BIIB122, an investigational small molecule inhibitor of LRRK2, in individuals with early-stage Parkinson's disease. Results from the study show that BIIB122 did not slow the progression of Parkinson's disease versus placebo, as measured by the primary endpoint of Time to Confirmed Worsening combined score. Secondary endpoints also did not show a benefit with BIIB122. Exploratory biomarker endpoints demonstrated greater than90% kinase inhibition of peripheral LRRK2 and, in a cerebrospinal fluid sub-study, up to approximately 30% reduction observed in a biomarker of LRRK2 activity. BIIB122 was generally well tolerated with an acceptable safety profile. Based on these results, Biogen and Denali will discontinue further development of BIIB122 in idiopathic Parkinson's disease. Denali will continue to independently conduct the Phase 2a BEACON study evaluating the small molecule inhibitor in carriers of a pathogenic LRRK2 variant. The global Phase 2a BEACON study is designed to assess safety, pharmacokinetics and biomarkers of lysosomal pathway engagement, which will inform the biomarker profile in LRRK2 pathogenic variant carriers and the impact of LRRK2 inhibition. Data from the BEACON study is anticipated in the first half of 2027.