Parkinson’s disease

Arvinas presented promising Phase 1 data for ARV-102, a brain-penetrant PROTAC LRRK2 degrader, at MDS 2025. ARV-102 was well-tolerated and demonstrated dose-dependent CSF exposure, indicating brain penetration. It achieved >90% PBMC LRRK2 reductions at ≥20mg. In healthy volunteers, 80mg for 14 days reduced CSF lysosomal and microglial markers linked to Parkinson’s disease. Multiple-dose patient data is expected in 2026, with a Phase 1b trial in progressive supranuclear palsy planned for H1 2026, pending IND clearance. The data supports further clinical development of ARV-102.

Biohaven launched a global Phase 2/3 trial for BHV-8000, a first-in-clinic, brain-penetrant TYK2/JAK1 inhibitor, targeting neuroinflammation in Parkinson’s disease. The drug aims to address the underlying cause of disease progression, with no existing disease-modifying therapies currently available. The trial will enroll 550 patients across 13 countries to assess efficacy and safety, utilizing innovative endpoints. BHV-8000 has shown safety and tolerability, with robust brain penetration, representing a potential breakthrough in treating neurodegenerative diseases.