« Back to news

Ventus Therapeutics to Present Data for Novel Brain-Penetrant NLRP3 Small Molecule Inhibitors for the Treatment of Inflammatory and Neurological Diseases

Presentation at 3rd Inflammasome Therapeutics Summit describes optimization of unique series of NLRP3 small molecule inhibitors

WALTHAM, MA & MONTREAL, QC, November 18, 2021 – Ventus Therapeutics U.S., Inc., a biopharmaceutical company utilizing structural biology and cutting-edge computational tools to identify and develop small molecule therapeutics across a broad range of disease indications, today announced that Michael Crackower, Ph.D., Chief Scientific Officer of Ventus, will be presenting at the 3rd Inflammasome Therapeutics Summit, being held virtually, November 16-18, 2021.

Ventus’ proprietary technology has enabled it to discover chemically distinct NLRP3 (NLR family pyrin domain-containing protein 3) inhibitors. The presentation includes data for Ventus’ NLRP3 targeted programs, which have demonstrated selectivity for NLRP3 and reduction of liver and kidney inflammation in a murine chronic repeat dose LPS (lipopolysaccharide) model, a systemic inflammation model used to determine the anti-inflammatory potential of test compounds.

Further data on Ventus’ brain-penetrant NLRP3 inhibitor program are also included, which describe high binding affinity in human whole blood and robust modulation of IL-1β activation, IL-6, and TNFα in murine brain. Compounds in this program showed pronounced exposure in the brain of mice, which may contribute to the optimization of a unique series of NLRP3 small molecule inhibitors for neurological indications associated with NLRP3 activation, such as Parkinson’s disease, traumatic brain injury and Alzheimer’s disease.

Dr. Crackower will also be discussing VENT-01, Ventus’ first lead program, a peripherally restricted small molecule inhibitor compound targeting NLRP3. The new data builds on previously presented results in September 2021 which demonstrated Ventus’ ability to experimentally interrogate NLRP3 protein directly in its monomeric form, followed by high throughput screening and successful identification of small molecules that directly bind to NLRP3 and inhibit its activation. Earlier results also showed Ventus’ novel NLRP3 inhibitors were differentiated from those of publicly known NLRP3 inhibitors and utilized structure-based drug design for the target, achieving a high resolution 2.6 angstrom structure of monomeric NLRP3.

Details of the presentation are as follows:

Oral Presentation:

Title: Identification of Structurally Distinct & Selective Brain Penetrant and Peripherally Restricted NLRP3 Inhibitors with High Binding Affinity

Presenter: Michael Crackower, PhD

Session: Thursday, November 18, 2:30 pm ET

 

About Ventus Therapeutics
Ventus Therapeutics is a biopharmaceutical company utilizing structural biology and cutting-edge computational tools to identify and develop small molecule therapeutics across a broad range of disease indications, with an initial focus on immunology, inflammation and neurology. We have developed a proprietary drug discovery platform, called ReSOLVE, which is built upon our structural biology and protein science expertise and our proprietary computational chemistry capabilities, to address the current limitations of small molecule drug discovery. We are leveraging our ReSOLVE platform to discover and characterize previously unknown or poorly understood pockets on the surface of proteins and identify small molecules that can bind to those pockets with optimal affinity. We are focused on high-value targets that have been extensively implicated in human diseases that were previously considered undruggable or where we believe there is a significant opportunity to improve upon existing therapies. Our lead programs target key innate immune modulators, including NLRP3 and cGAS. For more information, please visit www.ventustx.com and engage with us on Twitter @Ventus_Tx or on LinkedIn.

Forward-Looking Statements
This press release contains forward-looking statements about future expectations, plans and prospects, including, but not limited to, statements related to Ventus’ ability to develop small molecule inhibitors that target NLRP3 for neurological indications associated with NLRP3 activation, such as Parkinson’s disease, traumatic brain injury and Alzheimer’s disease and the objectives and results of Ventus’ preclinical studies and anticipated therapeutic benefits of its NLRP3 inhibitor program. These forward-looking statements are based on Ventus’ current expectations and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, the initiation, timing, progress and results of preclinical studies and clinical trials of its future product candidates; the results of Ventus’ earlier studies not being predictive of future results; ability to maintain and enhance the ReSOLVE platform and use it to effectively identify previously unknown pockets on the surface of proteins and small molecules that have optimal binding affinity for those pockets; ability to obtain funding for its operations and implement its business strategy; ability to meet regulatory standards, including any anticipated regulatory developments in the United States and foreign countries in which it may seek regulatory approval for its future product candidates; the impact of the COVID-19 pandemic on its business and efforts to address the impact on its business; anticipated developments related to its competitors and its industry; the performance of third-party service providers, including suppliers and manufacturers; and ability to obtain, maintain, protect and enforce intellectual property protection for its future product candidates and the ReSOLVE platform. Any forward-looking statements contained in this press release speak only as of the date hereof, and Ventus undertakes no duty or obligation to update any forward-looking statements as a result of new information, future events or otherwise.

[1] Ising, C., Heneka, M.T. Functional and structural damage of neurons by innate immune mechanisms during neurodegeneration. Cell Death Dis 9, 120 (2018). https://doi.org/10.1038/s41419-017-0153-x