Neurodegenerative diseases such as Parkinson’s remain some of the most difficult conditions to treat, largely because they involve proteins that malfunction deep inside the brain. One such protein, LRRK2, plays a major role in certain forms of Parkinson’s disease, yet has historically been extremely difficult to target with traditional drugs. A new investigational therapy from Arvinas, known as ARV‑102, represents a major step forward in addressing this challenge. The company’s first‑in‑human clinical data, highlighted in their press release, demonstrate that this oral drug can reach the brain, degrade LRRK2, and do so safely in healthy volunteers.

 

ARV‑102 is part of a new class of medicines called PROTAC degraders. Unlike conventional drugs that merely block a protein’s activity, PROTACs work by eliminating the protein entirely. This is especially important for LRRK2, which becomes overactive or dysfunctional in certain genetic forms of Parkinson’s disease. According to the press release, ARV‑102 “achieved central and peripheral LRRK2 reduction indicating substantial LRRK2 protein degradation in healthy volunteers”. This means the drug not only reached the bloodstream but also crossed into the brain — a major hurdle for neurological therapies.

 

One of the most encouraging findings is that ARV‑102 was well tolerated. The company reports that the drug “was well tolerated, orally bioavailable, and brain‑penetrant”. This combination is rare: many neurological drugs fail because they cannot be taken orally or cannot cross the blood–brain barrier. ARV‑102’s ability to do both suggests it may be suitable for long‑term use, which is essential for chronic diseases like Parkinson’s.

 

The study also supports the broader scientific idea that targeted protein degradation may be a powerful strategy for treating neurodegenerative disorders. By removing LRRK2 rather than simply inhibiting it, ARV‑102 may avoid some of the limitations seen with earlier LRRK2 inhibitors, which sometimes caused side effects or incomplete suppression. The press release emphasizes that these findings “support continued evaluation of ARV‑102 in neurodegenerative diseases associated with LRRK2 dysfunction”. This includes not only Parkinson’s disease but potentially other conditions where LRRK2 plays a role.

 

Importantly, the company has already begun the next phase of research. A Phase 1 clinical trial in Parkinson’s patients “has been initiated and is currently enrolling”. This marks a significant milestone: moving from healthy volunteers to individuals who may directly benefit from the therapy. If future trials confirm safety and effectiveness, ARV‑102 could become one of the first brain‑penetrant PROTAC drugs to reach the clinic — a major achievement for both the field of protein degradation and the treatment of Parkinson’s disease.

 

Overall, the early data on ARV‑102 suggest that PROTAC technology may finally allow researchers to target proteins that were once considered “undruggable.” By demonstrating safety, oral dosing, brain penetration, and robust LRRK2 degradation, this investigational therapy opens the door to a new generation of treatments for neurodegenerative diseases.

 

Note: This page summarizes research findings published in Science Daily (2025). Readers interested in detailed methodologies and complete data should consult the original research article: Arvinas, Inc. “Arvinas Presents First‑in‑Human Data for Investigational Oral PROTAC ARV‑102 Demonstrating Blood‑Brain Barrier Penetration, and Central and Peripheral LRRK2 Degradation.” GlobeNewswire, 4 April 2025. Retrieved via search results.

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