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

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

Presentation at 2021 EMBO Inflammasome Workshop describes new drug discovery approach resulting in differentiated structure and pharmacologic properties for NLRP3 inhibitors

WALTHAM, Mass. & MONTREAL, September 24, 2021 – Ventus Therapeutics, Inc., a biotechnology company using structure-based drug design to advance a pipeline of small molecule medicines for high-value targets, presented data for its NLRP3 inhibitor program at today’s 2021 EMBO Inflammasome Workshop. The presentation highlighted results demonstrating how Ventus’ proprietary technology enabled the design of chemically distinct NLRP3-targeted small molecule therapeutics capable of addressing systemic and neurological disease applications.

Highlights of the data presented for the NLRP3 inhibitor program include:

  • Demonstrated the ability to experimentally interrogate NLRP3 protein directly in its monomeric form, followed by high throughput screening andsuccessful identification of small molecules that directly bind to the target and inhibit NLRP3 activation
  • Identified novel inhibitors of NLRP3 from multiple chemical series with structures and properties, including brain penetrance, differentiated from those of publicly known NLRP3 inhibitors
  • First company to utilize structure-based drug design for the target, achieving a high resolution 2.5 angstrom structure of monomeric NLRP3

“With these results that we share today with the scientific community, we believe that Ventus is breaking new ground in successfully performing structure-based drug design for NLRP3 for the first time ever,” said Michael Crackower, PhD, Chief Scientific Officer of Ventus. “Following on the seminal work of our co-founder Hao Wu of Harvard Medical School, we have established unmatched capabilities to stably express high concentrations of the monomeric form of NLRP3, enabling the first application of structure-based drug design to target NLRP3 and our rapid generation of differentiated inhibitors.”

“We are highly encouraged by our ability to overcome the historical challenges to identify structurally and pharmacologically diverse compounds directly targeting NLRP3, and we look forward to advancing our two NLRP3 inhibitor programs toward the clinic. Inhibition of NLRP3 has the potential to significantly impact the lives of patients suffering from severe neurological and immunological diseases,” said Anick Auger, lead biologist of the NLRP3 program and head of pharmacology at Ventus who presented the discovery of these novel inhibitors at the EMBO.

About NLRP3
NLRP3 (NLR pyrin domain-containing 3) is a member of a family of proteins known as inflammasome receptors and is integral in the formation of the NLRP3 inflammasome. 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, via generation of IL-1β and IL-18. 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. Aberrant activation of the NLRP3 inflammasome has been associated with systemic conditions including fibrotic, dermatological and rheumatological diseases and figures prominently in several neurological disorders including Alzheimer’s disease, Parkinson’s disease and multiple sclerosis.[1]

About Ventus Therapeutics
Ventus Therapeutics is a biopharmaceutical company discovering and developing novel small-molecule medicines by pioneering structure-based drug discovery for biologically validated, but challenging, drug targets.  Our ReSOLVE™ platform reveals novel druggable sites on traditionally undruggable targets, enables virtual screening, and accelerates medicinal chemistry and SAR.  Using ReSOLVE™, we are rapidly advancing a pipeline of new medicines to treat autoimmune diseases, inflammatory diseases and cancer. Our most advanced programs are directed to critical targets in innate immune pathways. For more information, please visit www.ventustx.com and engage with us on Twitter @Ventus_Tx or on LinkedIn. View perspectives from the Ventus team and luminary scientists in the fields of immunology, structural biology, and computational chemistry on new ways to design medicines to reach elusive drug targets.

Media Contact:
Kathryn Morris
The Yates Network

Investor Relations Contact:
Peter Vozzo

[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