Ventyx Biosciences has shared promising top-line results from a phase 2a study of its investigational central nervous system (CNS) penetrant NLRP3 inhibitor in Parkinson’s disease (PD).
The company has been evaluating the once-daily oral drug, VTX3232, in ten patients with early stages of the neurodegenerative disorder over a 28-day treatment period.
The trial met its primary objective of showing that VTX3232 was safe and well tolerated, with no drug-related treatment-emergent adverse events seen throughout the dosing period.
Its pharmacokinetic and pharmacodynamic endpoints were also achieved, demonstrating high drug exposures in plasma and cerebrospinal fluid (CSF) as well as clear evidence of target engagement in plasma and CSF, with potent suppression of NLRP3-related biomarkers, Ventyx said.
More than ten million people worldwide are living with PD, which is characterised by symptoms such as tremor, muscle rigidity, slowness of movement and difficulty with balance.
NLRP3-mediated neuroinflammation is recognised as a key driver of neuronal degeneration in PD, and its inhibition in the CNS may offer a disease-modifying therapeutic approach.
Ventyx’s chief executive officer, Raju Mohan, said: “By inhibiting NLRP3-mediated cytokine production and inflammatory markers in the CNS, VTX3232 provides a unique opportunity for a disease-modifying therapy for PD.
“We are delighted that this trial met its goals of establishing that treatment with VTX3232 was safe and well tolerated, with high exposure levels in CSF and clear reductions in NLRP3-related biomarkers in a PD patient population.”
The company outlined that planning is underway for a placebo-controlled phase 2 trial of VTX3232 in PD, and potentially in additional neurodegenerative disorders such as Alzheimer’s disease.
The candidate is also currently being assessed in a 12-week phase 2 trial in patients with obesity and cardiometabolic risk factors, with top-line results from this expected in the second half of 2025.
Ventyx’s announcement came just one day after Roche said it would be advancing its own investigational PD drug, prasinezumab, into phase 3 clinical development. The trial will evaluate the Prothena-partnered monoclonal antibody in patients with early-stage PD, following results from two mid-stage trials and their ongoing open-label extensions.