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Ventus Therapeutics Initiates Clinical Testing of VENT-03, a First-in-Class, Orally Administered cGAS Inhibitor

Activation of the cGAS pathway in response to cellular dysfunction is associated with severe inflammatory and cardiometabolic diseases

Data from the Phase 1 trial expected in the second half of 2024

WALTHAM, Mass. & MONTREAL — January 3, 2024 — (BUSINESS WIRE) — Ventus Therapeutics, a clinical-stage biopharmaceutical company utilizing its proprietary structural biology and computational chemistry platform, ReSOLVETM, to develop differentiated small molecule therapeutics, announced today that the first participant has been dosed in a Phase 1 clinical trial of VENT-03, the first cGAS inhibitor to advance into clinical development.

“cGAS has historically proven an elusive target despite significant industry efforts to develop a viable inhibitor. We have broken that cycle with the initiation of this Phase 1 clinical trial,” said Ventus President and CEO Marcelo Bigal, M.D., Ph.D. “We are eager to advance this therapeutic candidate and bring the potential of cGAS inhibition to patients suffering from inflammatory disorders with significant unmet needs. This trial continues our company’s momentum, being the second program that we have brought to the clinic in the last six months.”

This first-in-human Phase 1 trial is designed to fully explore the pharmacokinetics, pharmacodynamics, and safety of VENT-03 across a broad range of single and multiple ascending doses in healthy volunteers. Ventus expects results from the trial in the second half of 2024.

“The commencement of the Phase 1 VENT-03 trial demonstrates Ventus’ ability to drug a target that has been undruggable to date, made possible because of ReSOLVETM,” said Ventus Chief Scientific Officer Mike Crackower, Ph.D. “ReSOLVETM, which combines machine learning with physics and computational chemistry, provided us with unique insights about the binding pocket that allowed us to design VENT-03 as a first-in-class cGAS inhibitor with excellent potency.”

For more information about Ventus’ pipeline and ReSOLVETM platform, visit www.VentusTx.com.

About cGAS
cGAS is an intracellular pattern recognition receptor that is activated after binding to double-stranded DNA (dsDNA) in the cytoplasm. The presence of dsDNA in the cytoplasm is often the result of cellular dysfunction, which is a hallmark of many autoimmune and inflammatory diseases. Activation of cGAS leads to cGAMP formation, activation of STING, and downstream expression of type I interferons, leading to pronounced inflammation and tissue damage. In both patients and preclinical models of disease, the cGAS pathway has been shown to be a key driver of lupus and other inflammatory diseases, such as systemic sclerosis and dermatomyositis.

About Ventus Therapeutics
Ventus Therapeutics is a clinical-stage biopharmaceutical company deploying leading-edge structural biology and computational chemistry tools to create novel small molecule medicines for challenging targets in 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 hydrocophore™ 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 first-in-class cGAS inhibitor; VENT-02, a brain-penetrant NLRP3 inhibitor; and VENT-01, a peripherally restricted NLRP3 inhibitor. Ventus entered the clinic with its two most advanced programs, VENT-02 and VENT-03, in 2023, and the Phase 1 clinical trials are ongoing. These programs exemplify Ventus’ robust discovery capabilities and focus on differentiated programs for multi-indication immunology and neurology targets. For more information, please visit www.ventustx.com 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.

Media Contact
Alexis Feinberg
ICR Westwicke