Shares of Ventyx Biosciences (NASDAQ:VTYX) traded flat on Tuesday, even after the company released promising top-line results from a Phase 2a trial evaluating its investigational drug VTX3232 in patients with early-stage Parkinson’s disease.
The biotech firm announced that the study successfully met its primary goal of establishing the drug’s safety and tolerability. Importantly, no treatment-emergent adverse events linked to the drug were reported during the dosing period. Additionally, the trial met key pharmacokinetic and pharmacodynamic objectives, with high concentrations of VTX3232 observed in both plasma and cerebrospinal fluid, along with suppression of biomarkers associated with NLRP3 inflammasome activity—indicating successful target engagement.
The study, conducted as a single-center, open-label trial, involved 10 patients who received a daily 40mg oral dose of VTX3232 over 28 days. Ventyx noted that the drug maintained plasma levels exceeding three times the IC90 for NLRP3 inhibition, sustained for a full 24 hours post-dose.
“We’re excited by these findings, which show that once-daily VTX3232 can safely achieve and maintain therapeutic levels in both the bloodstream and central nervous system,” said Dr. Mark Forman, Chief Medical Officer at Ventyx. “These results support our confidence in the drug’s potential as a treatment for neuroinflammatory conditions like Parkinson’s.”
While the company also observed improvements in patients’ motor and non-motor symptoms as measured by the MDS-UPDRS scale, the results are preliminary, given the small sample size and lack of a placebo control group.
Ventyx stated that it is currently planning a more rigorous follow-up study—a double-blind, placebo-controlled Phase 2 trial that will include dose-ranging components. The company also hinted at possible future applications of VTX3232 in other neurodegenerative conditions, including Alzheimer’s disease.