Pharma Focus Europe

Initiation of Dosing Begins in Phase 1 Trial of VTX3232, an Innovative CNS-penetrant NLRP3 Inhibitor, by Ventyx Biosciences

Thursday, June 15, 2023

Ventyx Biosciences, Inc. (Nasdaq: VTYX) announced the initiation of a Phase 1 clinical trial for their new drug candidate, VTX3232. The company aims to develop novel oral therapies to address inflammatory diseases with significant unmet medical needs. VTX3232 is a CNS-penetrant NLRP3 inhibitor, designed to target neuroinflammatory conditions.

Raju Mohan, the CEO of Ventyx, expressed excitement about the progress, noting that VTX3232 is the fourth internally discovered compound to enter clinical trials. He emphasized the company's focus on developing a pipeline of orally-delivered small molecules that target large disease populations with high unmet needs. Ventyx believes that exploring NLRP3 inhibition and its impact on IL-1β biology through compounds like VTX3232 and VTX2735, a peripherally restricted NLRP3 inhibitor, will be beneficial in systemic and neuroinflammatory diseases.

The Phase 1 trial of VTX3232 will consist of two parts: a randomized, double-blind, placebo-controlled single ascending dose (SAD) and multiple ascending dose (MAD) trial. The primary objectives are to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VTX3232 in healthy adult volunteers. The trial will involve administering a range of doses and collecting cerebrospinal fluid (CSF) samples to assess brain exposure. The company expects to announce topline results from the trial in the first half of 2024. Ventyx believes that VTX3232, as an NLRP3 inhibitor, has the potential to address neuroinflammatory conditions such as Parkinson's disease, Alzheimer's disease, and amyotrophic lateral sclerosis (ALS), among others.

Alongside VTX3232, Ventyx is conducting a Phase 2 proof-of-mechanism trial for VTX2735, a peripherally restricted NLRP3 inhibitor. This trial focuses on patients with familial cold autoinflammatory syndrome (FCAS), which is a subset of cryopyrin-associated periodic syndrome (CAPS) caused by NLRP3 gene mutations. Ventyx believes that VTX2735 has the potential to treat various chronic inflammatory conditions characterized by NLRP3-induced excess IL-1β, including dermatologic, rheumatic, and cardiovascular diseases.

The NLRP3 inflammasome plays a crucial role in the body's inflammatory response. Upon activation, NLRP3 triggers the release of pro-inflammatory cytokines IL-1β and IL-18, leading to chronic inflammation. Ventyx aims to target this process to address systemic and neuroinflammatory diseases that have a significant impact on patients' lives.

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