Ventus Therapeutics Doses First Patient in Phase 2a Trial of VENT-02 for Parkinson’s Disease
Ventus Therapeutics, a clinical-stage biopharmaceutical company focused on developing novel small-molecule therapies for immunological, inflammatory, and neurological disorders, has announced the dosing of the first patient in a Phase 2a clinical trial evaluating VENT-02 in patients with mild to moderate Parkinson’s disease. VENT-02 is an oral, brain-penetrant inhibitor of NLRP3, a key driver of inflammation and a critical component of the innate immune system involved in a variety of inflammatory diseases.
Parkinson’s disease affects approximately 10 million people worldwide, making it the second most common neurodegenerative disease. However, treatment options remain limited, and current therapies primarily provide symptom relief without addressing the underlying disease progression. According to Dr. Xavier Valencia, Head of Clinical Development at Ventus Therapeutics, VENT-02’s unique mechanism of action offers the potential to provide both symptom relief and, more importantly, slow disease progression. Dr. Valencia also highlighted that the Phase 2a trial integrates digital health technologies, which will measure the effects of VENT-02 on symptoms and assess a broad range of biomarkers linking inflammation to disease activity. He emphasized that the findings from this study could pave the way for VENT-02 to become the first symptomatic and disease-modifying drug for Parkinson’s disease.
The Phase 2a study is a randomized, double-blind, placebo-controlled trial designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of VENT-02 over a 28-day treatment period. Approximately 30 patients with mild to moderate Parkinson’s disease will participate. The primary objective of the study is to evaluate the safety and tolerability of VENT-02, while secondary and exploratory endpoints will assess changes in plasma and cerebrospinal fluid biomarkers related to target engagement, inflammation, and disease activity. Additionally, improvements in motor function and quality of life will be measured using digital health technologies tailored to Parkinson’s disease. The company expects topline data from the Phase 2a trial in late 2025 or early 2026.
Dr. Marcelo Bigal, President and CEO of Ventus, noted the growing importance of NLRP3 as a target in neurology. NLRP3 plays a central role in neuroinflammation, which is a key contributor to the pathology of many neurological disorders, including Parkinson’s disease.
Dr. Bigal emphasized that VENT-02, as a brain-penetrant NLRP3 inhibitor, has the potential to revolutionize the treatment of Parkinson’s disease by targeting the underlying inflammatory processes driving the disease. In addition to Parkinson’s disease, Ventus has plans to conduct further Phase 2 studies for VENT-02, including trials for osteoarthritis in obese patients, highlighting the broad therapeutic potential of targeting NLRP3 in various inflammatory conditions.
Understanding NLRP3
NLRP3 is part of a family of proteins known as inflammasomes, which are responsible for regulating the innate immune system and detecting harmful signals that can trigger an inflammatory response. When activated, inflammasomes promote the release of pro-inflammatory cytokines, such as IL-1β and IL-18, and induce pyroptosis, a form of inflammatory cell death. In many diseases, including osteoarthritis, fibrotic conditions, and several neurological disorders, the aberrant activation of the NLRP3 inflammasome contributes to disease pathology.
Therapeutically inhibiting NLRP3 can prevent inflammasome formation, thereby reducing the release of inflammatory cytokines and preventing pyroptosis. This mechanism of action is particularly relevant for diseases like Parkinson’s, where neuroinflammation plays a crucial role in disease progression. By targeting NLRP3, VENT-02 aims to mitigate neuroinflammation and potentially slow the progression of Parkinson’s disease, offering a novel approach to treatment.
About Ventus Therapeutics
Ventus Therapeutics is at the forefront of developing innovative therapies for complex inflammatory, immunological, and neurological disorders. The company’s proprietary drug discovery platform, ReSOLVE®, enables it to identify and develop novel small-molecule inhibitors that target key drivers of disease. Ventus’s pipeline includes two wholly-owned programs currently in Phase 2 development. In addition to VENT-02, which is being tested in Parkinson’s disease and osteoarthritis in obese patients, the company is also advancing VENT-03, a first-in-class oral cGAS inhibitor expected to enter Phase 2 development for lupus in 2025.
Ventus has also out-licensed VENT-01, a peripherally-restricted, oral NLRP3 inhibitor, to Novo Nordisk A/S for further development. Through these programs, Ventus Therapeutics is focused on transforming the treatment landscape for patients with debilitating diseases, leveraging cutting-edge science to develop therapies that not only address symptoms but also target the root causes of disease.
As Ventus continues to advance its clinical programs, the company remains committed to addressing significant unmet medical needs in inflammatory and neurological diseases, with the goal of improving patients’ lives through precision medicine and novel therapies.
