Ventus Therapeutics Begins Dosing in NLRP3 Inhibitor Study Licensed to Novo Nordisk

Ventus Therapeutics, Inc., a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, and Montreal, has announced a significant milestone in its partnership with Novo Nordisk. The first participant in a Phase 1 clinical study for NNC6022-0001 has been successfully dosed. This compound, an oral NLRP3 inhibitor, was initially known as VENT-01 and was licensed by Novo Nordisk in September 2022.

The initiation of this trial represents the first step in the clinical development program targeting the NLRP3 protein. Karin Conde-Knape, Ph.D., Senior Vice President of Global Drug Discovery at Novo Nordisk, emphasized the potential of NLRP3 inhibitors in treating a variety of cardiometabolic diseases, including obesity, metabolic dysfunction-associated steatohepatitis (MASH), and chronic kidney disease. She highlighted the additional evidence supporting the broad applications of targeting NLRP3, marking this trial as a significant development in their clinical endeavors.

The Phase 1 study aims to thoroughly investigate the pharmacokinetics, pharmacodynamics, and safety of NNC6022-0001 across a wide range of doses in healthy volunteers. Michael Crackower, Ph.D., Ventus' Chief Scientific Officer, expressed satisfaction with the progress made in collaboration with Novo Nordisk. He noted that this clinical milestone is the third Ventus-discovered compound to reach the clinical stage within the last nine months.

NLRP3 is a well-studied member of the inflammasome family, multiprotein complexes that regulate the innate immune system. These complexes play a crucial role in detecting danger and pathogen signals, inciting a strong inflammatory response. This response includes the release of cytokines IL-1β and IL-18 and the induction of pyroptosis, an inflammatory cell death mechanism. Inhibiting NLRP3 can prevent the formation of the inflammasome, thereby reducing the production of these cytokines and limiting pyroptosis. Abnormal activation of the NLRP3 inflammasome has been linked to various systemic conditions such as fibrotic, dermatological, and rheumatological diseases, and is a significant factor in several neurological disorders including Parkinson’s disease, Alzheimer’s disease, and treatment-resistant epilepsy.

In September 2022, Ventus and Novo Nordisk entered into an exclusive development and licensing agreement. This agreement granted Novo Nordisk worldwide rights to develop and commercialize Ventus’ lead NLRP3 inhibitor program across a spectrum of diseases, including MASH, chronic kidney disease, and other cardiometabolic conditions. Ventus received an upfront payment of $70 million and ongoing research and development funding. Additionally, the company is eligible to receive up to $633 million in potential clinical, regulatory, and commercial milestone payments, along with tiered royalties.

Ventus Therapeutics is focused on developing novel small molecule therapeutics for immunology, inflammation, and neurological disorders. Utilizing its proprietary ReSOLVE™ drug discovery platform, the company has made significant progress since its inception. In 2020, Ventus screened its first target and by 2022, had selected three development candidates. In 2023, Ventus advanced two wholly-owned product candidates into clinical trials: VENT-03, an oral cGAS inhibitor, and VENT-02, an oral NLRP3 inhibitor. The latter has already completed Phase 1. Moreover, Ventus has out-licensed VENT-01 to Novo Nordisk. These developments underscore Ventus' strong discovery capabilities and its commitment to advancing differentiated programs targeting multiple indications in immunology and neurology.