Investigational therapy ARV-102 now in Parkinson’s trial

Arvinas, a biopharmaceutical company, has started a Phase 1 clinical trial to test its investigational treatment, ARV-102, in people living with Parkinson’s disease (PD). This step follows promising early results from healthy volunteers, showing that ARV-102 was safe, well tolerated, and functioned as expected. What Is ARV-102? ARV-102 is being developed for neurodegenerative conditions like Parkinson’s and progressive supranuclear palsy, where a protein called LRRK2 plays a key role. In Parkinson’s, LRRK2 can become overactive—even in people without a genetic mutation—which may contribute to the loss of nerve cells. This oral medication uses Arvinas’ PROTAC protein degradation technology to tag LRRK2 for removal by the body’s natural disposal system, potentially slowing disease progression. Crossing a Major Barrier in Brain Treatment One of ARV-102’s most significant features is its ability to cross the blood-brain barrier (BBB)—a natural defense system that protects the brain but also blocks many medications. In studies with healthy volunteers, ARV-102 successfully reached the central nervous system (CNS), including the brain and spinal cord. Early Results in Healthy Volunteers In a study involving healthy adults aged 18–64, ARV-102 was tested in both single and multiple daily doses. So far: No serious side effects have been reported. Mild side effects included headaches and fatigue. ARV-102 showed dose-dependent increases in levels within the brain. At certain doses, the medication reduced LRRK2 protein levels by over 50% in the brain and over 90% in immune cells found in the blood. It also led to changes in biological markers related to Parkinson’s activity. These results are encouraging and support moving forward with testing in people who are directly affected by Parkinson’s. Current Trial in People with Parkinson’s The ongoing Phase 1 study (EUCT 2024-516888-84-00), which began in late 2024, is being conducted in the Netherlands and is still recruiting adult participants. It includes two parts: Single-ascending dose (SAD): Participants receive one dose to determine safety. Multiple-ascending dose (MAD): Participants receive daily doses to further assess safety and effects over time. Arvinas expects to complete the SAD phase and share early findings later in 2025, and will then move into the MAD phase. A Step Forward in Parkinson’s Research According to Dr. Noah Berkowitz, Chief Medical Officer at Arvinas, ARV-102’s ability to target LRRK2 and reach the brain could represent a transformative step in treating Parkinson’s and other neurodegenerative conditions. While this is still early in development, the research offers a sense of hope and progress for individuals and families affected by Parkinson’s. Further testing will be key to understanding how this treatment might improve lives.