Arvinas (NASDAQ:ARVN) today presented positive data from its Phase 1 clinical trial of ARV-102, an investigational oral PROTAC (PROteolysis TArgeting Chimera) designed to treat neurodegenerative diseases.
Data showcased during the 2026 International Conference on Alzheimer’s and Parkinson’s Diseases (AD/PD) in Copenhagen confirmed that the drug successfully crossed the blood-brain barrier and achieved significant target engagement in patients.
In the multiple ascending dose (MAD) cohort, ARV-102 demonstrated approximately 50% or greater degradation of LRRK2 protein in the cerebrospinal fluid (CSF) across daily doses ranging from 20 mg to 80 mg.
This reduction was achieved by day 14 and was consistently maintained through day 28.
Notably, the treatment also led to a dose-dependent reduction in endolysosomal and neuroinflammatory biomarkers, such as CD68 and GPNMB, which are typically elevated in patients with Parkinson's and other tauopathies.
The safety profile of ARV-102 remains a key highlight for the program.
The drug was generally well tolerated with no serious adverse events (SAEs), discontinuations, or reported deaths.
Furthermore, researchers observed no significant changes in lung function or respiratory symptoms—a critical finding given that some traditional LRRK2 inhibitors have historically faced safety concerns regarding pulmonary toxicity.
Building on this momentum, Arvinas confirmed plans to broaden the clinical scope of ARV-102 beyond Parkinson’s disease.
The company intends to initiate a Phase 1b clinical trial in patients with progressive supranuclear palsy (PSP), a rapidly progressing rare brain disorder, in the second quarter of 2026.