In addition to assessing the safety and tolerability, pharmacokinetics, and pharmacodynamics of VENT-02, the trial will evaluate biomarkers to elucidate the role of inflammation in disease activity and explore the impact of VENT-02 on patient symptoms
Topline data expected in late 2025 or early 2026
WALTHAM, Mass. & MONTREAL--(BUSINESS WIRE)--Ventus Therapeutics, a clinical-stage biopharmaceutical company with two novel small-molecule programs entering Phase 2 development for immunological, inflammatory, and neurological disorders, today announced that the first patient has been dosed in a Phase 2a trial of VENT-02 in patients with mild to moderate Parkinson’s disease. VENT-02 is a novel, oral, brain-penetrant inhibitor of NLRP3, a key driver of inflammation and component of the innate immune system that plays a critical role in a wide range of inflammatory diseases.
“Ten million people globally are believed to have Parkinson’s disease, making it the second most common neurodegenerative disease worldwide. Treatment options are limited, and VENT-02’s unique mechanism of action has the potential to not only provide symptom relief to patients, but also slow disease progression,” said Xavier Valencia, M.D., Head of Clinical Development at Ventus. “Our Phase 2a trial incorporates digital health technologies to measure the effect of VENT-02 on symptoms and a comprehensive set of biomarkers that map inflammation to disease activity. We anticipate that these findings from this study will demonstrate VENT-02’s potential to be 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 evaluate the safety and tolerability, pharmacokinetics, and pharmacodynamics of VENT-02 over a 28-day treatment period in approximately 30 patients with mild to moderate Parkinson’s disease. The primary objective is safety and tolerability. Secondary and exploratory endpoints include changes in plasma and cerebrospinal fluid biomarkers of target engagement, inflammation, and disease activity, as well as improvements in motor function and other quality of life assessments as measured by digital health technologies customized for Parkinson’s disease. Topline data from the Phase 2a trial are expected in late 2025 or early 2026.
“NLRP3 is emerging as one of the most important targets in neurology, as it is a key component of the innate immune system and plays an instrumental role in neuroinflammation. Our study is designed to demonstrate VENT-02’s potential to change the treatment paradigm for Parkinson’s disease as a best-in-class brain-penetrant NLRP3 inhibitor,” said Marcelo Bigal, M.D., Ph.D., President and CEO of Ventus. “Beyond Parkinson’s disease, we have additional Phase 2 studies planned for VENT-02, including a Phase 2 trial for osteoarthritis in obese patients, underscoring the broad potential of NLRP3 as a key driver of inflammation across different diseases.”
About NLRP3
NLRP3 is a member of a family of proteins known as inflammasomes. Inflammasomes are multiprotein complexes that regulate the innate immune system and are involved in intracellular surveillance of danger and pathogen signals that trigger an intense inflammatory response, including the release of IL-1β and IL-18 and the induction of pyroptosis, an inflammatory form of cell death. Therapeutic inhibition of NLRP3 can prevent the formation of the NLRP3 inflammasome, which in turn inhibits the production of IL-1β and IL-18 as well as pyroptosis. Aberrant activation of the NLRP3 inflammasome has been associated with a broad range of systemic conditions, including rheumatological diseases, such as osteoarthritis, as well as fibrotic and dermatological diseases. In addition, NLRP3 is a key driver of disease pathology in several neurological disorders, including Parkinson’s disease, Alzheimer’s disease, obesity, and treatment-refractory epilepsy.
About Ventus Therapeutics
Ventus Therapeutics is a clinical-stage biopharmaceutical company with two novel small-molecule programs entering Phase 2 development for immunological, inflammatory, and neurological disorders. Using its proprietary drug discovery platform, ReSOLVE®, the company has established a robust pipeline, including two wholly-owned programs. VENT-03 is a first-in-class, oral cGAS inhibitor expected to enter Phase 2 development for lupus in 2025. VENT-02 is a best-in-class, brain-penetrant, oral NLRP3 inhibitor in Phase 2 development for Parkinson’s disease, and is expected to enter Phase 2 development in osteoarthritis in obese patients later in 2025. In addition, Ventus has out-licensed VENT-01, a peripherally-restricted, oral NLRP3 inhibitor in Phase 1, to Novo Nordisk A/S. For more information, please visit www.ventustx.com and engage with Ventus on LinkedIn.
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