First patient dosed in Parkinson’s clinical trial of Ventus’ VENT-02
Phase 2a study testing oral therapy in adults with mild to moderate disease
A Phase 2a clinical trial testing Ventus Therapeutics’ VENT-02 — a novel brain-penetrating oral medication — in adults with mild to moderate Parkinson’s disease has dosed its first patient.
The study (NCT06822517), expected to enroll about 30 people with Parkinson’s, is assessing the treatment’s safety, tolerability, effects on the body, and preliminary efficacy. Participants are still being recruited at a single site in Memphis, Tennessee.
Top-line data are expected by year’s end or at the beginning of 2026, according to the developer.
By targeting NLRP3, a protein involved in brain inflammation, “VENT-02’s unique mechanism of action has the potential to not only provide symptom relief to patients, but also slow disease progression,” Xavier Valencia, MD, head of clinical development at Ventus, said in a company press release.
“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,” Valencia said. “We anticipate that … findings from this study will demonstrate VENT-02’s potential to be the first symptomatic and disease-modifying drug for Parkinson’s.”
Novel brain-penetrating Parkinson’s therapy being evaluated in clinical trial
The NLRP3 protein is part of a molecular complex called the inflammasome, which regulates the immune system and works as a sensor for danger signals and disease-causing agents. It does so by triggering an intense inflammatory response.
However, in certain conditions, NLRP3 can become overly active and contribute to brain inflammation. This has been seen in neurological conditions like Parkinson’s, Alzheimer’s disease, and amyotrophic lateral sclerosis (ALS).
According to Marcelo Bigal, MD, PhD, Ventus’ president and CEO, “NLRP3 is emerging as one of the most important targets in neurology.” Bigal noted that “it is a key component of the innate immune system and plays an instrumental role in neuroinflammation.”
VENT-02 is an orally available molecule designed to enter the central nervous system, or CNS — which comprises the brain and spinal cord — and potently and selectively block NLRP3. This is expected to reduce brain inflammation and ease symptoms, as well as slow disease progression, per the company.
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 [protein suppressor].
In a previous Phase 1 trial that tested single and multiple ascending doses of VENT-02 in healthy adults, the treatment was found to be well tolerated, without reports of serious adverse events or dose-limiting toxicity.
Treatment-related adverse events were mild or moderate in severity, according to the company. Moderately severe side effects included headache and nausea, which were only reported at doses higher than the intended therapeutic dose.
Also, VENT-02 treatment was shown to reach the CNS, with significant levels of the medication being detected for 24 hours. Treatment also showed effective target engagement and reduced the levels of inflammatory molecules in the blood.
Data on VENT-02’s movement into, through, and out of the body supported a potential once-daily dosing, per Ventus.
In the ongoing Phase 2a clinical trial, adults with mild to moderate Parkinson’s are being randomly assigned to receive oral capsules of either VENT-02 or a placebo, twice daily for 28 days, followed by seven days of follow-up. The trial is enrolling patients ages 45 to 90.
The study’s main goal is to assess VENT-02’s safety and tolerability. Secondary goals include changes in biomarkers of inflammation, including NLRP3, and neurodegeneration in the blood and cerebrospinal fluid, which is the liquid that surrounds the brain and spinal cord.
As exploratory efficacy goals, researchers will look at changes in motor function and quality of life assessments via digital health technologies customized for Parkinson’s.
“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 [suppressor],” Bigal said.
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