Pharma Focus Europe

Ventus Therapeutics Launches Phase 1 Clinical Trial for VENT-02: a New Orally Administered NLRP3 Inhibitor with Enhanced Brain Penetration

Wednesday, August 23, 2023

Ventus Therapeutics has commenced a Phase 1 clinical trial for VENT-02, a groundbreaking oral NLRP3 inhibitor engineered to address neuroinflammatory diseases. Marcelo Bigal, M.D., Ph.D., the CEO of the company, expressed enthusiasm about reaching this pivotal juncture, highlighting the distinctive characteristics of VENT-02, such as its exceptional structure, potent brain penetration, and efficacy. He underscored that VENT-02 has shown outstanding safety margins, enabling the formulation of an inventive clinical development strategy that encompasses diverse patient cohorts grappling with neuroinflammatory disorders.

The Phase 1 trial builds upon the promising safety margins established during the preclinical stages, with the objective of conducting a comprehensive evaluation of VENT-02's safety profile, pharmacodynamics, and tolerance across a spectrum of administered doses. Ventus is aiming to unveil preliminary outcomes by the first half of 2024.

Mike Crackower, Ph.D., Ventus's Chief Scientific Officer, shed light on the pivotal role played by NLRP3 in propelling neuroinflammatory diseases. Extensive scrutiny of VENT-02 in pertinent preclinical models has considerably mitigated the risks associated with disease treatment, indicating its potential to effectively manage a wide array of neuroinflammatory conditions, including Parkinson's, Alzheimer's, refractory epilepsy, and systemic disorders.

NLRP3 holds a significant place as a core member of the inflammasome receptor family, steering the regulation of the innate immune system. Inflammasomes, vital complexes responsible for surveilling internal danger signals that trigger robust inflammatory responses, including the release of IL-1β and IL-18, as well as inducing inflammatory cell death termed pyroptosis. Through the inhibition of NLRP3, it becomes feasible to impede the assembly of the NLRP3 inflammasome, ultimately leading to diminished production of IL-1β and IL-18, along with reduced pyroptosis. Anomalies in NLRP3 activation have been correlated with various systemic conditions, encompassing dermatological, fibrotic, and rheumatological ailments. Furthermore, it is instrumental in the progression of neurological disorders such as Parkinson's, Alzheimer's, and refractory epilepsy.

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