Jefferies analysts said on Tuesday that Ventyx could leverage its mid-stage data for VTX3232 to position the oral drug candidate as a treatment for other neurodegenerative diseases, including Alzheimer’s.
Ventyx Biosciences’ oral drug candidate VTX3232 can durably knock down its target biomarker in patients with Parkinson’s disease, an outcome that analysts at Jefferies said could support studies in other neurodegenerative diseases.
“Today’s data supports advancing to a larger Phase II study in Parkinson’s and perhaps Alzheimer’s, too,” the analysts wrote in a Tuesday note to investors, contending that Ventyx could also leverage these results in potential business development talks with pharma companies.
Tuesday’s data come from a small Phase IIa study which gave a daily 40-mg oral dose of VTX3232 to 10 patients with early-stage, idiopathic Parkinson’s disease. Treatment lasted for 28 days. Results showed that the drug was present at high levels in both the patients’ plasma and the cerebrospinal fluid (CSF). According to Jefferies, this supports its once-daily dosing profile.
VTX3232 treatment also reduced levels of its target biomarker, an immune protein called NLRP3, as well as downstream signaling molecules such as IL-6, hsCRP and SAA. Ventyx said patients showed notable improvements in motor and non-motor symptoms, with significant gains across the different domains of the MDS-Unified Parkinson’s Disease Rating Scale.
The biotech was quick to point out, however, that the study was open-label and had no placebo control. Patients were also on active symptomatic treatment.
Jefferies said that VTX3232 showed a “clean and safe profile.” Ventyx’s announcement noted that all side effects were mild or moderate in severity and were deemed unrelated to VTX3232. There were no serious adverse events documented.
Taken together, these findings support the continued clinical development of VTX3232 in Parkinson’s disease, for which the company is currently planning a double-blind and placebo-controlled Phase II study, Ventyx said on Tuesday. The biotech is also looking at the possibility of expanding beyond Parkinson’s and into other neurodegenerative diseases, including Alzheimer’s. The company is already testing VTX3232 in a Phase II trial for lowering inflammation in patients with cardiometabolic conditions and obesity, with data expected in the second half of 2025.
Ventyx CEO Raju Mohan on Tuesday said that the biotech has “initiated internal and external planning discussions” for the further development of VTX3232, though he did not specify whether these discussions were with potential industry partners or with regulators.
Jefferies believes Ventyx would be smart to look for a partner for VTX323. “Given the significant capital requirement, it makes sense to engage a Pharma partner to advance this program into larger [placebo]-controlled CNS studies,” its analysts wrote.
