NLRP3 inhibitor for neuroinflammation enters trials

Neurons in a brain with Alzheimer's disease

The Phase I clinical trial of VENT-02, an oral, brain-penetrant NLRP3 inhibitor from Ventus Therapeutics, has launched with the dosing of the first patient.

Based on the safety margins demonstrated in IND-enabling studies, the Phase I 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.

Ventus President and CEO Marcelo Bigal, said: “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.”

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.

“NLRP3 is a key driver of many neuroinflammatory diseases,” said Ventus Chief Scientific Officer Mike Crackower. “Our extensive characterisation 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.”

Edited by Diana Spencer, Senior Digital Content Editor, Drug Discovery World

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