Ventyx Biosciences has published new Phase 2a data regarding its oral drug candidate VTX3232, which was tested in patients with early-stage, idiopathic Parkinson’s disease. The study, conducted over a 28-day period, involved administering a 40-mg daily dose of the compound to ten participants. According to the company, the data showed reductions in the immune protein NLRP3, as well as in other inflammatory biomarkers, and demonstrated improvements in both motor and non-motor symptoms.
The Phase 2a trial found that VTX3232 was detectable at high concentrations in both plasma and cerebrospinal fluid, a pharmacokinetic profile that supports once-daily oral dosing. Ventyx stated that patients in the study showed clinically meaningful progress across different areas of the MDS-Unified Parkinson’s Disease Rating Scale. However, the company also noted the study’s limitations, emphasizing that it was open-label with no placebo control and that participants were receiving concurrent symptomatic treatment.
VTX3232’s mechanism of action includes the inhibition of NLRP3 and its downstream signaling molecules such as IL-6, hsCRP, and SAA. The company said no serious adverse events were reported during the trial, and all side effects were mild to moderate and considered unrelated to the drug.
Jefferies analysts described the compound’s safety and biomarker response as a strong basis for further development. “Today’s data supports advancing to a larger Phase II study in Parkinson’s and perhaps Alzheimer’s, too,” the analysts wrote in a note released Tuesday. They also suggested that Ventyx could utilize the data for potential discussions with pharmaceutical companies.
The biotech has announced plans for a larger, double-blind, placebo-controlled Phase II study to further evaluate VTX3232 in Parkinson’s disease. In addition to pursuing further clinical testing for Parkinson’s, Ventyx is considering expanding the program to include other neurodegenerative diseases, including Alzheimer’s.
The company is concurrently running a separate Phase II trial of VTX3232 aimed at reducing inflammation in patients with obesity and cardiometabolic risk factors. Results from that study are anticipated in the second half of 2025. The outcome of this future readout may influence Ventyx’s collaboration opportunities, particularly with Sanofi, which holds a right of first negotiation on the program. T
his right was secured in conjunction with Sanofi’s $27 million investment in Ventyx and is scheduled to be activated following the second readout. However, according to Ventyx CEO Raju Mohan, the timeline for potential engagement is not limited. “Sanofi is not restricted to waiting until the second readout,” Mohan said.
Investor attention on Sanofi’s position has increased following its acquisition of Vigil Neuroscience, a company with which it held similar negotiation rights. Jefferies analysts noted that it could be prudent for Ventyx to consider a partnership to support further central nervous system studies, citing the high costs of conducting larger placebo-controlled trials.
Mohan added that the company has “initiated internal and external planning discussions” regarding the future of VTX3232, though he did not clarify whether these involve regulatory agencies or industry collaborators.
