英矽智能INS018_055完成中国IIa期临床试验全部患者入组

由生成式人工智能（AI）驱动的临床阶段生物科技公司英矽智能宣布，公司自主研发的小分子抑制剂INS018_055已经完成中国IIa期临床试验(NCT05938920)全部患者入组，该研究旨在评估INS018_055在患者群体中治疗特发性肺纤维化（IPF）的安全性、耐受性和初步疗效。

该项随机、双盲、安慰剂对照研究正在包括北京协和医院在内的中国29个研究中心进行，目前已经按计划完成了71名患者入组。该研究将分为3个实验组和1个安慰剂组，主要评估INS018_055与安慰剂相比，在成年IPF患者群体中口服给药12周的安全性和耐受性。此外，英矽智能正在筹备计划于2025年启动的IIb期概念验证研究，进一步探索INS018_055的疗效和安全性。

英矽智能首席医学官Sujata Rao表示，“目前，英矽智能已经完成该项临床试验的全部患者入组，期待在今年第四季度获得高质量的数据集。与此同时，我们已在美国开展了一项平行IIa期试验（NCT05975983），正在积极招募患者。我向临床合作伙伴、参与研究的患者和家属表示感谢。他们的关注和支持有助于我们了解人工智能驱动的创新疗法在治疗这一严重慢性疾病的研发进程。”

特发性肺纤维化（IPF）是一种慢性瘢痕性肺部疾病，其特点是肺功能进行性和不可逆的下降，影响全球约500万人。由于发病、进展较为隐秘，多数患者确诊时病情已发展到中晚期，确诊后中位生存期仅为3年。目前现有的抗纤维化药物，可能会减缓疾病的进展，但往往无法阻止或逆转疾病的发展。此外，药物产生的一系列副作用严重影响患者的生活质量。

英矽智能联合首席执行官兼首席科学官任峰博士表示，“英矽智能从未满足的临床需求出发，在自研人工智能平台支持下快速推进该项目早期药物发现工作，并于近期获得了激动人心的临床进展。这不仅是英矽智能自有管线的全新里程碑，也标志着人工智能驱动的药物研发验证又向前迈进了一步。我们期待分享更多临床数据，并尽快在概念验证研究中对INS018_055进行评估。”

2024年3月，英矽智能在Nature Biotechnology上发表了一项研究，详细介绍了INS018_055候选药物从人工智能算法开发到II期临床试验的研发流程，并首次披露了该款采用生成式人工智能发现和设计的潜在全球首创（first-in-class）TNIK抑制剂的原始实验数据，以及临床前和部分临床试验评估结果。

2016年，英矽智能全球首次在同行评审期刊上阐述了使用生成式人工智能设计新型分子的概念，为涵盖生成生物学、化学和医学等领域的商业化Pharma.AI平台奠定了基础。自2021年以来，英矽智能在自有人工智能平台Pharma.AI的支持下，建立了超过30条丰富的自研管线组合，并从中提名了18款临床前候选项目，其中7款化合物获得临床试验许可。

关于INS018_055

INS018_055是一款由英矽智能自主研发的潜在全球首创（first-in-class）小分子抑制剂。其新颖靶点由公司自有的人工智能驱动的靶点发现引擎PandaOmics识别，并且，英矽智能利用生成化学平台Chemistry42针对该新颖靶点生成和设计了全新的分子结构。2021年2月，英矽智能提名INS018_055为临床前候选化合物，用于特发性肺纤维化（IPF）治疗。同年11月，英矽智能启动该候选药物的首次微剂量组人体试验，首次将AI发现的全新候选药物推进到临床验证阶段。INS018_055于2023年2月获得美国FDA孤儿药认定，并先后于同年4月和6月在中美两地获得许可开展II期临床试验。

关于英矽智能

英矽智能是一家由生成式人工智能驱动的临床阶段生物医药科技公司，通过下一代人工智能系统连接生物学、化学和临床试验分析，利用深度生成模型、强化学习、转换模型等现代机器学习技术，构建强大且高效的人工智能药物研发平台，识别全新靶点并生成具有特定属性分子结构的候选药物。英矽智能聚焦癌症、纤维化、免疫、中枢神经系统疾病、衰老相关疾病等未被满足医疗需求领域，推进并加速创新药物研发。

更多信息，请访问网站 www.insilico.com

Insilico Medicine(“Insilico”), a clinical-stage generative artificial intelligence (AI)-driven drug discovery company, today announced the completion of patient enrollment in a Phase IIa study (NCT05938920) in China, which is investigating the safety, tolerability, and preliminary efficacy of INS018_055 for idiopathic Pulmonary Fibrosis (IPF).

The randomized, double-blind, placebo-controlled study is being conducted across 29 clinical centers in China and has completed enrollment of the planned target of 71 patients in four participant groups, including three experimental and one placebo. The primary objective is to evaluate the safety and tolerability of INS018_055 orally administered for up to 12 weeks in adult subjects with IPF compared to placebo. In addition, Insilico is preparing a Phase IIb proof-of-concept study to be initiated in 2025 to explore the efficacy and further safety of INS018_055.

“Having achieved full patient enrollment, we are looking forward to a robust dataset late in the fourth quarter of this year. Meanwhile, a parallel Phase IIa trial (NCT05975983) is ongoing and actively accruing patients in the U.S.”, said Sujata Rao, MD, Chief Medical Officer at Insilico Medicine. “We thank our clinical collaborators and participating patients and families for their continued interest and commitment. Their support contributes to our understanding of the discovery and development of innovative therapies driven by AI, in this serious chronic disease.”

Idiopathic pulmonary fibrosis (IPF), is a chronic scarring lung disease characterized by progressive and irreversible decline in lung function, affecting approximately five million people worldwide. Due to the lack of differential symptoms, patients diagnosed with IPF are often in advanced stages, with a median survival of three years. Current treatments, primarily antifibrotic agents, may slow disease progression but often fail to halt it, and they come with a range of side effects that can severely impact quality of life.

“Starting with an unmet clinical need, Insilico fast-tracked the program's early drug discovery with the support of our proprietary AI platform,” saidFeng Ren, Ph.D., Co-CEO and Chief scientific officer of Insilico Medicine. “I am excited about the clinical progress in this program, which is not only another milestone achieved by Insilico's wholly owned pipeline, but also marks a step forward in the validation of AI-driven drug discovery and development. We look forward to sharing more clinical data and evaluating INS018_055 in a proof-of-concept study soon.”

In March 2024, a study published in Nature Biotechnology explained the discovery and development of INS018_055 in detail from AI algorithms to Phase II clinical trials, revealing for the first time raw experimental data as well as preclinical and parts of clinical evaluations of the potentially first-in-class TNIK inhibitor discovered and designed through generative AI.

In 2016, Insilico first described the concept of using generative AI for the design of novel molecules in a peer-reviewed journal, which laid the foundation for the commercially available Pharma.AI platform spanning across biology, chemistry and clinical development. Powered by Pharma.AI, Insilico has nominated 18 preclinical candidates in its comprehensive portfolio of over 30 assets since 2021 and has received IND approval for 7 molecules.

About INS018_055

INS018_055 is a potentially first-in-class small molecule inhibitor with a novel target discovered by Insilico’s target identification engine, PandaOmics, and a novel molecular structure designed by its generative chemistry engine, Chemistry42. In February 2021, Insilico nominated INS018_055 as a preclinical candidate for the treatment of IPF, a chronic lung disease that results in progressive and irreversible decline in lung function, and started a first-in-human study in November 2021. The FDA granted Orphan Drug Designation to INS018_055 for the treatment of Idiopathic Pulmonary Fibrosis in February 2023. Insilico initiated the enrollment Phase II clinical trial of INS018_055 in China in April 2023 and received FDA approval for a simultaneous Phase II clinical trial in the US in June 2023.

About Insilico Medicine

Insilico Medicine, a global clinical-stage biotechnology company powered by generative AI, connects biology, chemistry, and clinical trial analysis using next-generation AI systems. The company has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques for novel target discovery and generating novel molecular structures with desired properties. Insilico Medicine is developing breakthrough solutions to discover and develop innovative drugs for cancer, fibrosis, immunity, central nervous system diseases, infectious diseases, autoimmune diseases, and aging-related diseases.

www.insilico.com

Reference

[1] Ren, F., et al. A small-molecule TNIK inhibitor targets fibrosis in preclinical and clinical models. Nat Biotechnol (2024). https://doi.org/10.1038/s41587-024-02143-0