Topline data from the Phase 1 trial expected in the first half of 2024

WALTHAM, MA, USA & MONTREAL, Canada I August 22, 2023 IVentus Therapeutics, a clinical-stage biopharmaceutical company utilizing structural biology and a proprietary computational chemistry platform to develop differentiated small molecule therapeutics, announced today that the first participant has been dosed in a Phase 1 clinical trial of VENT-02, a novel, oral, brain-penetrant NLRP3 inhibitor.

“This trial marks a new chapter for Ventus as we become a clinical-stage organization,” said Ventus President and CEO Marcelo Bigal, M.D., Ph.D. “We are bringing a highly differentiated NLRP3 inhibitor into the clinic with VENT-02, which has best-in-class potential based on its unique structure, exceptional brain penetration, and exquisite potency. In addition, VENT-02 has demonstrated extraordinary safety margins – among the highest I have seen in 20-plus years of developing drugs. These benefits allow us to design an incredibly innovative clinical development plan for VENT-02 and to fully explore the potential impact of NLRP3 inhibition across multiple patient populations suffering from neuroinflammatory diseases.”

Based on the exceptional safety margins demonstrated in IND-enabling studies, the Phase 1 trial is designed to fully explore VENT-02’s pharmacodynamics, safety, and tolerability across a broad range of single and multiple ascending doses. Ventus expects preliminary results from the trial in the first half of 2024.

“NLRP3 is a key driver of many neuroinflammatory diseases,” said Ventus Chief Scientific Officer Mike Crackower, Ph.D. “Our extensive characterization of VENT-02 in multiple disease-relevant preclinical models has de-risked our disease selection process, and we believe VENT-02 has the potential to treat patients across a wide range of neuroinflammatory diseases, including Parkinson’s, Alzheimer’s, and refractory epilepsy, as well as systemic diseases.”

About NLRP3

NLRP3 is the best understood member of a family of proteins known as inflammasome receptors. Inflammasomes are multiprotein complexes that regulate the innate immune system and are involved in intracellular surveillance of danger signals that trigger an intense inflammatory response, including the release of IL-1β and IL-18 and the induction of pyroptosis, an inflammatory form of cell death. Therapeutic inhibition of NLRP3 can, therefore, prevent the formation of the NLRP3 inflammasome, which in turn inhibits the production of IL-1β and IL-18 as well as pyroptosis. Aberrant activation of the NLRP3 inflammasome has been associated with systemic conditions, including fibrotic, dermatological, and rheumatological diseases, and is a key driver of disease pathology in several neurological disorders, including Parkinson’s disease, Alzheimer’s disease, and refractory epilepsy.

About Ventus Therapeutics

Ventus Therapeutics is a clinical-stage biopharmaceutical company deploying leading-edge structural biology and computational chemistry tools to create novel small molecule medicines for challenging targets, with an initial focus on immunology, inflammation, and neurology. The company’s proprietary drug discovery platform, ReSOLVETM, gives it the ability to discover and develop medicines through unique insights into protein targets and to create dynamic blueprints that can guide the development of differentiated therapies at a fast pace. Ventus’ first target screen was conducted in 2020, which led to the nomination of three development candidates in 2022, including: VENT-03, a potential first-in-class cGAS inhibitor; VENT-02, a brain-penetrant NLRP3 inhibitor; and VENT-01, a peripherally restricted NLRP3 inhibitor. These programs exemplify Ventus’ unique discovery capabilities and focus on first- and best-in-class programs for multi-indication immunology targets with franchise potential. For more information, please visit and engage with Ventus on LinkedIn.

SOURCE: Ventus Therapeutics