Ventyx Biosciences initiates dosing in Phase 1 trial of VTX3232

Ventyx Biosciences announced that the first subject has been dosed in a Phase 1 trial of VTX3232, a novel central nervous system-penetrant NLRP3 inhibitor. The Phase 1 trial of VTX3232 is a two-part, randomized, double-blind, placebo-controlled single ascending dose and multiple ascending dose trial designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of VTX3232 in adult healthy volunteers. The trial will explore a broad range of doses and will include serial cerebrospinal fluid sampling to assess brain exposure. Topline results from the trial are expected in the first half of 2024. There is a growing body of preclinical data suggesting NLRP3’s role as a central driver of neuroinflammation and thus we believe VTX3232 may have therapeutic potential in a range of neuroinflammatory conditions with high unmet medical need, including Parkinson’s disease, Alzheimer’s disease and amyotrophic lateral sclerosis, among others. In addition to VTX3232, we are also evaluating VTX2735, a peripherally restricted NLRP3 inhibitor, in a Phase 2 proof-of-mechanism trial in patients with familial cold autoinflammatory syndrome. FCAS is the most common subset of cryopyrin-associated periodic syndrome, a group of rare autoinflammatory conditions caused by gain-of-function mutations in the NLRP3 gene. Beyond CAPS, we believe VTX2735 may have therapeutic potential across a broad range of chronic inflammatory conditions that are characterized by NLRP3-induced excess IL-1beta, including large dermatologic, rheumatic and cardiovascular disease.