WALTHAM, Mass. & MONTREAL--(BUSINESS WIRE)--Ventus Therapeutics, a clinical-stage biopharmaceutical company utilizing its proprietary ReSOLVE™ platform to discover and develop differentiated small-molecule therapeutics, today announced results from its Phase 1 clinical trial of VENT-03, the first cyclic GMP-AMP synthase (cGAS) inhibitor to successfully complete a first-in-human study.

马萨诸塞州沃尔瑟姆和蒙特利尔——（商业新闻短讯）——Ventus Therapeutics是一家临床阶段的生物制药公司，利用其专有的ReSOLVE™平台发现和开发分化的小分子疗法，今天宣布了其VENT-03第一期临床试验的结果，VENT-03是第一个成功完成人体首次研究的环状GMP-AMP合酶（cGAS）抑制剂。

The Phase 1 trial evaluated the pharmacokinetics (PK), target engagement, safety, and tolerability of VENT-03 across a broad range of single and multiple ascending doses in 72 healthy adult volunteers..

第一阶段试验评估了72名健康成年志愿者在广泛的单次和多次递增剂量范围内VENT-03的药代动力学（PK），目标参与，安全性和耐受性。。

“The extraordinary profile demonstrated by this trial qualifies VENT-03 as a first- and best-in-class anti-cGAS medication and underscores the power of our ReSOLVE™ platform to develop high-quality small molecules for targets that have historically been undruggable,” said Marcelo Bigal, M.D., Ph.D., President and CEO of Ventus.

Ventus总裁兼首席执行官马塞洛·比加尔（Marcelo Bigal）医学博士说：“这项试验所证明的非凡特性使VENT-03成为一流和一流的抗cGAS药物，并强调了我们的ReSOLVE™平台为历史上无法治疗的目标开发高质量小分子的能力。”。

“These results unlock the path to exploring the broad potential of cGAS inhibition across a wide range of autoimmune disorders and cardiometabolic diseases, beginning with lupus. We’re excited to continue leading the way in cGAS development and look forward to initiating a Phase 2 trial with VENT-03 in SLE in 2025.”.

“这些结果为探索cGAS抑制在广泛的自身免疫性疾病和心脏代谢疾病中的广泛潜力开辟了道路，从狼疮开始。我们很高兴继续领导cGAS的发展，并期待在2025年在SLE中启动VENT-03的2期试验。”。

In the Phase 1 trial, VENT-03 was safe and well-tolerated at doses far exceeding those planned for use in Phase 2 trials. There were no dose-limiting or dose-related toxicities, serious adverse events, or changes in clinical laboratory parameters, electrocardiogram (ECG), or vital signs. All treatment-related adverse events were mild, transient, and easily managed..

在第一阶段试验中，VENT-03安全且耐受性良好，剂量远远超过计划在第二阶段试验中使用的剂量。没有剂量限制或剂量相关毒性，严重不良事件或临床实验室参数，心电图（ECG）或生命体征的变化。所有与治疗相关的不良事件均为轻度，短暂且易于控制。。

VENT-03 demonstrated a favorable PK profile that supports once-daily dosing. In addition, VENT-03 reached plasma concentrations required for full target inhibition and demonstrated robust pharmacodynamics. Ventus plans to present full data from the Phase 1 trial at a future medical conference.

VENT-03表现出良好的PK曲线，支持每日一次给药。此外，VENT-03达到了完全靶向抑制所需的血浆浓度，并表现出强大的药效学。Ventus计划在未来的医学会议上提供第一阶段试验的完整数据。

“Available treatments for lupus today include injectable medicines for the type I interferon or BAFF pathways that address only limited aspects of patients’ symptoms and disease course,” said Xavier Valencia, M.D., Head of Clinical Development at Ventus. “A once-daily oral cGAS inhibitor has the potential to modulate both pathways validated by biologics, impact multiple facets of SLE, and provide superior efficacy compared to existing treatments and therapies in development.”.

Ventus临床开发负责人Xavier Valencia医学博士说：“目前可用的狼疮治疗方法包括I型干扰素或BAFF途径的注射药物，这些药物只能解决患者症状和病程的有限方面。”。“每日一次的口服cGAS抑制剂有可能调节生物制剂验证的两种途径，影响SLE的多个方面，并且与开发中的现有治疗和疗法相比提供优异的疗效。”。

About cGAS

关于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, pronounced inflammation, and tissue damage.

cGAS是一种细胞内模式识别受体，在与细胞质中的双链DNA（dsDNA）结合后被激活。细胞质中dsDNA的存在通常是细胞功能障碍的结果，这是许多自身免疫性和炎性疾病的标志。cGAS的激活导致cGAMP形成，STING的激活，明显的炎症和组织损伤。

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, dermatomyositis, and Sjögren's disease..

在患者和临床前疾病模型中，cGAS途径已被证明是狼疮和其他炎症性疾病（如系统性硬化症，皮肌炎和干燥综合征）的关键驱动因素。。

About Ventus Therapeutics

关于Ventus Therapeutics

Ventus Therapeutics is a clinical-stage biopharmaceutical company deploying deep protein science expertise and a proprietary computational chemistry platform to develop novel small molecule therapeutics for immunology, inflammation, and neurology disorders. Ventus’ ReSOLVE™ platform combines the latest advances in artificial intelligence/machine learning (AI/ML), structural biology, and biophysics to model the full spectrum of protein dynamics at an unparalleled resolution.

Ventus Therapeutics是一家临床阶段的生物制药公司，部署了深度蛋白质科学专业知识和专有的计算化学平台，以开发针对免疫学，炎症和神经病学疾病的新型小分子疗法。Ventus的ReSOLVE™平台结合了人工智能/机器学习（AI/ML），结构生物学和生物物理学的最新进展，以无与伦比的分辨率对全谱蛋白质动力学进行建模。

Using ReSOLVE™, the company screened its first target in 2020, selected three development candidates in 2022, advanced its two wholly-owned product candidates into the clinic in 2023, and completed Phase 1 trials for both programs in 2024: VENT-03, a potent, selective, oral cGAS inhibitor, and VENT-02, a potent, brain-penetrant, oral NLRP3 inhibitor.

利用 ReSOLVE™，公司在 2020 年筛选出第一个目标，在 2022 年选出三个候选开发项目，在 2023 年将两个全资拥有的候选产品推向临床，并在 2024 年完成两个项目的一期试验： VENT-03 是一种强效、选择性口服 cGAS 抑制剂，VENT-02 是一种强效、脑穿透性口服 NLRP3 抑制剂。

In addition, Ventus has out-licensed VENT-01, a peripherally-restricted NLRP3 inhibitor in Phase 1, to Novo Nordisk A/S. For more information, please visit www.ventustx.com and engage with Ventus on LinkedIn..

此外，Ventus已向Novo Nordisk a/S颁发了VENT-01许可证，VENT-01是一种外围限制的NLRP3抑制剂。有关更多信息，请访问www.ventustx.com并在LinkedIn上与Ventus联系。。