Chilblains

May 25th, 2023 at 11:00 AM ET (Virtual Event) WALTHAM, Mass. & MONTREAL--(BUSINESS WIRE)-- Ventus Therapeutics, a biopharmaceutical company utilizing structural biology and a proprietary computational chemistry platform to identify and develop differentiated small molecule therapeutics across a broad range of diseases, announced today that it will host its inaugural Program Spotlight on VENT-03 on May 25th, 2023 at 11:00 AM ET. The virtual event will feature VENT-03, a first-in-class cGAS inhibitor in development for lupus and other inflammatory disorders. cGAS, a highly sought-after target, is an innate immune sensor of nucleic acids that, when aberrantly activated, leads to auto-inflammation and tissue damage. The pathway has been clinically validated in systemic lupus erythematosus (SLE) and is implicated in a wide range of immunological and neuroinflammatory conditions. Ventus has demonstrated proof-of-mechanism with its portfolio of cGAS inhibitors in multiple disease models. Despite significant efforts across the industry over the past decade, VENT-03 will be the first cGAS inhibitor to advance to the clinic, demonstrating Ventus’ ability to drug a target that has been undruggable to date and has the potential to significantly improve treatment options for patients. As part of the event, Ventus scientists, together with Dr. Victoria Werth, Professor of Dermatology and Medicine at Penn Medicine, will discuss the biology of the cGAS pathway and its importance in immunology. They will also review the current treatment landscape and the potential of VENT-03 as an effective treatment for SLE and present the clinical development plan for VENT-03, including an overview of Ventus’ differentiated translation to the clinic. A live question and answer session will follow. To register for the event, please click here. About cGAS cGAS is an intracellular pattern recognition receptor that is activated after binding to double-stranded DNA (dsDNA) in the cytoplasm. dsDNA in the cytoplasm is generally associated with cellular dysfunction, as seen in multiple diseases such as systemic lupus erythematosus (SLE), cutaneous lupus erythematosus (CLE), and dermatomyositis (DM). cGAS activation leads to cGAMP formation, activation of STING and downstream expression of type I interferons such as IFN-α, -ß and -κ, leading to pronounced inflammation and tissue damage. This pathway also leads to the expression of several other proinflammatory cytokines such as CXCL10. Genetic mutations that lead to the aberrant activation of cGAS/STING in humans are known to cause severe infant onset Aicardi-Goutières Syndrome (AGS) and genetic forms of chilblain lupus, demonstrating the relevance of the pathway in human diseases. Given that the pathophysiology and presentation of these genetic diseases have significant similarities with idiopathic complex diseases such as SLE, CLE, and DM, these diseases represent highly compelling opportunities for treatment with a cGAS inhibitor. About Ventus Therapeutics Ventus Therapeutics is a 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, ReSOLVE™, 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. Forward-Looking Statements This press release contains forward-looking statements about future expectations, plans, and prospects, including, but not limited to, statements related to the anticipated benefits of Ventus’ business strategy, pipeline programs, drug discovery platform, and financial position. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements due to the risks and uncertainties inherent in Ventus’ business, which include, without limitation, the timing, progress, and results of preclinical and clinical studies involving its development programs; the results of its earlier studies not being predictive of future results; its ability to enhance the ReSOLVE™ platform; its ability to continue to obtain funding for its operations and to implement its business strategy; the impact of the COVID-19 pandemic on its business; anticipated developments related to its competitors and its industry; the performance of third-party service providers, including suppliers, manufacturers, and CROs; and its ability to obtain, maintain, and protect its intellectual property. 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.

WALTHAM, Mass. & MONTREAL--(BUSINESS WIRE)-- Ventus Therapeutics, Inc., a biopharmaceutical company utilizing structural biology and a proprietary computational chemistry platform to identify and develop differentiated small molecule therapeutics across a broad range of diseases, announced the details of two presentations to be made at the Keystone Symposium on Innate Immunity: From Innate Sensing to Adaptive Responses, being held April 16-19 in Snowbird, Utah. At the 8:00AM MDT Tuesday session, Ventus Chief Scientific Officer Michael Crackower, Ph.D., will present “Structure and Computation-Based Drug Discovery in Innate Immunity.” This presentation will mark the public debut of data showcasing the potency and pharmacokinetic pro the company’s potential first-in-class cGAS inhibitor, VENT-03. Ventus’ portfolio of cGAS inhibitors have demonstrated modulation of key endpoints in models of lupus, dermal inflammation, and chronic kidney disease. Additionally, Dr. Crackower will present new data on the company’s portfolio of brain-penetrant NLRP3 inhibitors in a seizure model. Additionally, Kelly Pike, Ph.D., Ventus head of translational sciences and scientific affairs, will present a poster during the 7:30PM MDT Monday poster session titled, “A Novel Selective Cyclic GMP-AMP Synthase (cGAS) Inhibitor Suppresses Systemic and Dermal Inflammation in a Model of Interferonopathy.” The poster outlines Ventus’ identification and optimization of a novel series of cGAS inhibitors, driven by unique structural insight. Dr. Pike will present evidence of robust suppression of systemic inflammation and protection against photosensitivity in a mouse model of auto-inflammation, supporting the therapeutic value of targeting cGAS for rheumatic diseases such as systemic lupus erythematosus (SLE). “We are eager to share the latest VENT-03 data with our peers at the Keystone Symposium, as this audience represents individuals and organizations that have been instrumental in elucidating the biology of cGAS for more than a decade,” said Dr. Crackower. “We expect VENT-03 to be the first cGAS inhibitor ever to enter clinical trials and believe that the scientific community shares our excitement for this candidate’s potential to treat a range of immunological diseases, including lupus, systemic sclerosis, and dermatomyositis.” About cGAS cGAS is an intracellular pattern recognition receptor that is activated after binding to double-stranded DNA (dsDNA) in the cytoplasm. dsDNA in the cytoplasm is generally associated with cellular dysfunction, as seen in multiple diseases such as systemic lupus erythematosus (SLE), cutaneous lupus erythematosus (CLE), and dermatomyositis (DM). cGAS activation leads to cGAMP formation, activation of STING and downstream expression of type I interferons such as IFN-α, -ß and -κ, leading to pronounced inflammation and tissue damage. This pathway also leads to the expression of several other proinflammatory cytokines such as CXCL10. Genetic mutations that lead to the aberrant activation of cGAS/STING in humans are known to cause severe infant onset Aicardi-Goutières Syndrome (AGS) and genetic forms of chilblain lupus, demonstrating the relevance of the pathway in human diseases. Given that the pathophysiology and presentation of these genetic diseases have significant similarities with idiopathic complex diseases such as SLE, CLE, and DM, these diseases represent highly compelling opportunities for treatment with a cGAS inhibitor. About NLRP3 NLRP3 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, including the release of IL-1β and IL-18 and the induction of pyroptosis, a lytic 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. 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 Alzheimer’s disease, Parkinson’s disease, and multiple sclerosis. About Ventus Therapeutics Ventus Therapeutics is a 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. Forward-Looking Statements This press release contains forward-looking statements about future expectations, plans, and prospects, including, but not limited to, statements related to the anticipated benefits of Ventus’ business strategy, pipeline programs, drug discovery platform, and financial position. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements due to the risks and uncertainties inherent in Ventus’ business, which include, without limitation, the timing, progress, and results of preclinical and clinical studies involving its development programs; the results of its earlier studies not being predictive of future results; its ability to enhance the ReSOLVETM platform; its ability to continue to obtain funding for its operations and to implement its business strategy; the impact of the COVID-19 pandemic on its business; anticipated developments related to its competitors and its industry; the performance of third-party service providers, including suppliers, manufacturers, and CROs; and its ability to obtain, maintain, and protect its intellectual property. 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.

The nomination of VENT-03 validates Ventus’ proprietary ReSOLVE™ drug discovery platform Phase 1 clinical trials will include patients with multiple distinct interferonopathies Robust analyses based on internally developed and validated skin and blood biomarkers will measure inflammation and target engagement in distinct diseases, de-risking late-stage development WALTHAM, Mass. & MONTREAL--(BUSINESS WIRE)-- Ventus Therapeutics, Inc., a biopharmaceutical company utilizing structural biology and a proprietary computational chemistry platform to identify and develop differentiated small molecule therapeutics across a broad range of diseases, announced today the nomination of a potential first-in-class cGAS inhibitor, VENT-03, as the company’s first development candidate directed against cGAS. This represents the third development candidate nominated in the past 12 months to Ventus’ pipeline, following the nominations of VENT-02, a brain-penetrant NLRP3 inhibitor, and VENT-01, a peripherally restricted NLRP3 inhibitor that is partnered with Novo Nordisk. cGAS activation leads to a type I interferon response, which is implicated in a broad range of diseases, including cutaneous lupus erythematosus, systemic lupus erythematosus, systemic sclerosis, and dermatomyositis, as well as neuroinflammatory and neurodegenerative diseases. In addition, genetic diseases, such as severe infant onset Aicardi-Goutières Syndrome and genetic forms of chilblain lupus, are driven by genetic mutations that lead to the activation of the pathway in humans. In disease-relevant preclinical studies, VENT-03 demonstrated robust in vivo pharmacodynamic activity and efficacy. “cGAS is a highly coveted target for pro-inflammatory and neurodegenerative diseases for which no inhibitor has advanced into clinical development to date, despite significant efforts from the drug discovery industry over the past decade,” said Ventus’ President and CEO Marcelo Bigal, M.D., Ph.D. “We expect VENT-03 to be the first cGAS inhibitor to enter the clinic, with a clinical development plan tailored to provide multiple data readouts prior to Phase 2 trials.” In December 2022, Ventus scientists published a state-of-the-art review on the industry’s progress to develop inhibitors in the cGAS-STING pathway. In May 2022, Ventus announced that it had been awarded a Lupus Research Program Idea Award from the U.S. Department of Defense, and, along with it, a multi-year grant to study the role of cGAS in lupus and the potential for treatment of the disease using a cGAS inhibitor. “The nomination of VENT-03 as our first development candidate targeting cGAS was made possible by the unique insights provided by ReSOLVE, our proprietary drug discovery platform,” said Ventus’ Chief Scientific Officer Michael Crackower, Ph.D. “After identifying a high-resolution co-crystal structure of cGAS that was different from what had been previously published, we used ReSOLVE to determine characteristics of the binding pocket that are unique to only some of the target’s many conformations, a major breakthrough that transformed our ability to achieve high potency in our cGAS inhibitors. Given the highly validated role of the cGAS pathway as a driver of disease, we are now developing additional cGAS inhibitors to achieve the full potential of this attractive target in distinct therapeutic areas.” About cGAS cGAS is an intracellular pattern recognition receptor that is activated after binding to double-stranded DNA (dsDNA) in the cytoplasm. dsDNA in the cytoplasm is generally associated with cellular dysfunction, as seen in multiple diseases such as cutaneous lupus erythematosus (CLE), systemic lupus erythematosus (SLE) and dermatomyositis (DM). cGAS activation leads to cGAMP formation, activation of STING and downstream expression of type I interferons such as IFN-α, -ß and -κ, leading to pronounced inflammation and tissue damage. This pathway also leads to the expression of several other proinflammatory cytokines such as CXCL10. Genetic mutations that lead to the aberrant activation of cGAS/STING in humans are known to cause severe infant onset Aicardi-Goutières Syndrome (AGS) and genetic forms of chilblain lupus, demonstrating the relevance of the pathway in human diseases. Given that the pathophysiology and presentation of these genetic diseases have significant similarities with idiopathic complex diseases such as CLE, SLE and DM, these diseases represent highly compelling opportunities for treatment with a cGAS inhibitor. About Ventus Therapeutics Ventus Therapeutics is a 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 ReSOLVE 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 small molecule medicines. The Company’s lead programs target key innate immune modulators, including NLRP3 and cGAS. The Company is using its ReSOLVE platform to improve the way medicines are discovered and developed, with the ultimate goal of restoring the health of people facing serious illnesses. For more information, please visit and engage with Ventus 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 the anticipated benefits of the executive leadership team, Ventus’ business strategy, current programs, future results of operations, and financial position. These forward-looking statements are based on the Company’s 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 timing, progress, and results of preclinical and clinical studies involving its development programs; the results of its earlier studies not being predictive of future results; its ability to enhance the ReSOLVE platform; its ability to continue to obtain funding for its operations and to implement its business strategy; the impact of the COVID-19 pandemic on its business and its 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 its ability to obtain, maintain, and protect its intellectual property. 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.