Following positive Phase IIa results for oral Rentosertib in GENESIS-IPF with good tolerability, a favorable PK profile, and dose-dependent efficacy improvements, the inhalation solution has received IND clearance from the CDE.

在GENESIS-IPF研究中，口服Rentosertib的IIa期结果积极，显示出良好的耐受性、理想的药代动力学特征以及剂量依赖性的疗效提升，其吸入溶液已获得CDE的IND批准。

The inhalation solution is designed for targeted lung delivery to achieve rapid onset and high local bioavailability at lower doses, while reducing systemic exposure and related side effects.

吸入溶液旨在实现靶向肺部递送，以较低剂量达到快速起效和高局部生物利用度，同时减少全身暴露及相关副作用。

Rentosertib inhalation solution is the 13th program from Insilico's AI-driven pipeline to receive IND clearance, further demonstrating how

雷托塞替吸入溶液是英矽智能第13个获得IND批准的AI驱动项目，进一步证明了如何

Pharma.AI

药企人工智能

could reproducibly drive the discovery and development of novel molecules from preclinical development to clinical validation.

可以重复地推动新分子从临床前开发到临床验证的发现与开发。

CAMBRIDGE, Mass.

马萨诸塞州剑桥市

,

，

April 28, 2026

2026年4月28日

/PRNewswire/ --

/PRNewswire/ --

Insilico Medicine (

英硅智能 (

'Insilico', HKEX:3696 ), a clinical-stage, generative AI–driven drug discovery company

‘英矽智能’（港交所代码：3696），一家临床阶段的生成式人工智能驱动的药物发现公司

, today announced that the inhalation solution of Rentosertib (ISM001-055), the company's first-in-class drug candidate with AI-identified novel target and AI-developed molecule structure, received IND clearance from CDE. Notably, Rentosertib inhalation solution is the 13th program from Insilico's AI-driven pipeline to receive IND clearance, further demonstrating how Pharma.AI could reproducibly drive the discovery and development of novel molecules from preclinical development to clinical validation..

今天宣布，其首创新药候选物Rentosertib（ISM001-055）吸入溶液获得了CDE的IND批准。值得一提的是，Rentosertib吸入溶液是Insilico公司AI驱动管线中第13个获得IND批准的项目，进一步证明了Pharma.AI能够持续推动新分子从临床前开发到临床验证的发现与开发过程。

The IND clearance supports a Phase I study to evaluate the safety, tolerability, and pharmacokinetic (PK) profiles of Rentosertib inhalation solution. The study will consist of two parts: (1) a randomized, double-blind, placebo-controlled Phase I trial in healthy participants involving single ascending dose (SAD) and multiple ascending dose (MAD) cohorts; and (2) a non-randomized, open-label evaluation in patients with Idiopathic Pulmonary Fibrosis (IPF) receiving multiple doses.

IND批准支持了一项I期研究，以评估Rentosertib吸入溶液的安全性、耐受性和药代动力学（PK）特征。该研究将分为两部分：（1）在健康参与者中进行的随机、双盲、安慰剂对照的I期试验，包括单次递增剂量（SAD）和多次递增剂量（MAD）队列；以及（2）在特发性肺纤维化（IPF）患者中进行的非随机、开放标签的多剂量评估。

Approximately 80 subjects are expected to be enrolled..

预计约有80名受试者将被招募。

'We are pleased to receive CDE IND approval for Rentosertib inhalation solution.'

“我们很高兴收到CDE对Rentosertib吸入溶液的IND批准。”

says Feng Ren, Ph.D., Co-CEO and CSO of Insilico Medicine.

英矽智能首席执行官兼首席科学官任峰博士表示。

'In prior clinical studies, oral Rentosertib showed good tolerability, a favorable PK profile, and dose-dependent efficacy trend, strengthening our confidence in its mechanism and clinical potential. We look forward to seeing the Rentosertib inhalation solution deliver positive clinical results again in both healthy volunteers and patient populations.

“在之前的临床研究中，口服Rentosertib显示出良好的耐受性、理想的药代动力学特征以及剂量依赖性的疗效趋势，这增强了我们对其作用机制和临床潜力的信心。我们期待看到Rentosertib吸入溶液在健康志愿者和患者群体中再次取得积极的临床结果。”

The inhalation solution is designed for targeted lung delivery, with the goal of achieving higher pulmonary exposure and faster onset at lower doses while reducing systemic exposure and optimizing the benefit–risk profile. In parallel, we continue to advance the oral Rentosertib program and remain on track to initiate Phase III trials in the second half of this year.'.

吸入溶液旨在实现靶向肺部递送，目标是在较低剂量下实现更高的肺部暴露和更快的起效，同时减少全身暴露并优化效益-风险特征。与此同时，我们继续推进口服Rentosertib项目，并有望在今年下半年启动第三阶段试验。

Administration by inhalation is a complex drug delivery technology that requires combining formulation and devices, presenting higher technical barriers. Inhalation solutions deliver treatment as vapor or aerosol through special devices into the respiratory tract (i.e. the target organ), which is considered one of the most efficient routes of drug delivery as it may provide advantages including rapid action onset, high bioavailability, lowered effective dose and reduced side effects with a noninvasive and targeted route of administration..

吸入给药是一种复杂的药物递送技术，需要结合配方和装置，存在较高的技术壁垒。吸入溶液通过特殊装置以蒸汽或气溶胶的形式将治疗传递到呼吸道（即目标器官），这被认为是最有效的药物递送途径之一，因为它可能提供快速起效、高生物利用度、有效剂量降低和副作用减少等优势，并且采用非侵入性和靶向的给药途径。

In preclinical studies, the inhalation solution of Rentosertib was able to achieve higher lung exposure with low systemic exposure and demonstrated antifibrotic and anti-inflammatory efficacy in animal models. Moreover, it showed good pharmacokinetic (PK) and safety profiles, as well as good stability and solubility..

在临床前研究中，Rentosertib 的吸入溶液能够实现较高的肺部暴露量，同时系统性暴露量较低，并在动物模型中展示了抗纤维化和抗炎效果。此外，它还表现出良好的药代动力学（PK）和安全性特征，以及良好的稳定性和溶解性。

'The CDE IND clearance for the Rentosertib inhalation solution further validates Insilico Medicine's sustainable, scalable, and reproducible AI-driven drug discovery and development workflow.'

“Rentosertib吸入溶液的CDE IND批准进一步验证了Insilico Medicine可持续、可扩展且可重复的AI驱动药物发现与开发工作流程。”

says Alex Zhavoronkov, Ph.D., Founder and CEO of Insilico Medicine

英硅智能创始人兼首席执行官亚历克斯·扎沃隆科夫博士说

. 'With our proprietary AI models, we empower the whole process from target discovery to clinical development, expanding into formulation types and geographic areas. To date, we have successfully nominated 30 preclinical candidate compounds (DC/PCC), with the latest emerging from the UAE, a country with no prior history of innovative drug development.

“通过我们专有的人工智能模型，我们赋能从靶点发现到临床开发的整个过程，并扩展到制剂类型和地理区域。迄今为止，我们已经成功提名了30个临床前候选化合物（DC/PCC），最新的一个来自阿联酋，这个国家之前没有创新药物开发的历史。”

At the same time, Insilico Medicine has initiated three Phase II clinical trials and achieved IND clearance for 13 programs across fibrosis, oncology, immunology, and central nervous system disorders.'.

同时，英矽智能已启动三项二期临床试验，并在纤维化、肿瘤学、免疫学和中枢神经系统疾病领域获得了13个项目的IND批准。

Insilico Medicine has established AI-driven drug discovery partnerships with global leading companies, including

英硅智能已与包括全球领先企业在内的公司建立了人工智能驱动的药物发现合作关系，包括

Eli Lilly

礼来公司

,

，

Hygtia Therapeutics

海吉提亚治疗学

,

，

Qilu Pharmaceutical

齐鲁制药

,

，

CMS

内容管理系统

,

，

TaiGen

泰根

,

，

ASKA Pharma

ASKA制药

,

，

Tenacia

坚韧

,

，

Fosun Pharma

复星医药

, and

，以及

Sanofi

赛诺菲

, and has achieved important R&D milestones across multiple collaborations. By integrating advanced AI and automation technologies, Insilico Medicine has demonstrated significant efficiency improvements in practical applications, setting a benchmark for AI-driven drug research and development. Compared to the typical 4.5 years required in traditional drug discovery, Insilico's 20+ nominated candidate drugs from 2021 to 2024 took only 12–18 months on average to progress from project initiation to nomination of preclinical candidates (PCCs), with each project requiring synthesis and testing of only about 60–200 molecules..

，并在多个合作中取得了重要的研发里程碑。通过整合先进的AI和自动化技术，英硅智能在实际应用中展示了显著的效率提升，为AI驱动的药物研发树立了标杆。相比传统药物发现通常所需的4.5年时间，英硅智能在2021年至2024年间提名的20多个候选药物，从项目启动到临床前候选药物（PCC）提名平均仅需12至18个月，且每个项目只需合成和测试约60至200个分子。

About Idiopathic Pulmonary Fibrosis (IPF)

关于特发性肺纤维化（IPF）

Idiopathic Pulmonary Fibrosis (IPF) is a chronic, scarring lung disease characterized by a progressive and irreversible decline in lung function. Affecting approximately 5 million people worldwide, IPF carries a poor prognosis, with a median survival of 3 to 4 years. Current approved treatments, including antifibrotic drugs, can slow disease progression but do not stop or reverse it, leaving a significant unmet need for more effective, disease-modifying therapies..

特发性肺纤维化（IPF）是一种慢性、瘢痕性肺部疾病，其特征是肺功能进行性和不可逆的下降。全球约有500万人受其影响，IPF预后较差，中位生存期为3至4年。目前获批的治疗方法，包括抗纤维化药物，可以减缓疾病进展，但无法阻止或逆转病情，因此对更有效、能够改变疾病进程的治疗方案仍存在显著的未满足需求。

About Rentosertib (Known asISM001-055)

关于Rentosertib（又称ISM001-055）

Rentosertib is a potentially first-in-class small-molecule TNIK inhibitor, discovered and developed with the support of Insilico's proprietary generative AI platform,

Rentosertib是一种潜在的首创小分子TNIK抑制剂，是在Insilico专有的生成式人工智能平台的支持下发现和开发的，

Pharma.AI

药物人工智能

. The

。这个

FDA granted Orphan Drug Designation

FDA授予孤儿药资格认定

(ODD) to Rentosertib for the treatment of IPF in February 2023. The CDE granted Breakthrough Therapy Designation (BTD) to Rentosertib in May 2025.

2023年2月，Rentosertib获得用于治疗IPF的孤儿药资格（ODD）。2025年5月，CDE授予Rentosertib突破性疗法资格（BTD）。

Rentosertib's early drug discovery and development process was completed in 18 months with less than 80 small molecules synthesized and tested. This dramatically shortens traditional R&D timeline through AI-enabled target identification and molecule design. The AI-driven workflow was reported in

雷托塞替布的早期药物发现和开发过程在18个月内完成，仅合成了不到80种小分子并进行了测试。通过人工智能辅助的靶点识别和分子设计，这一过程极大地缩短了传统的研发时间。该人工智能驱动的工作流程被报道于

Nature Biotechnology

《自然-生物技术》

in March 2024. More importantly, Rentosertib achieved the world's first proof-of-concept for the efficacy of AI-driven drug discovery with the positive results demonstrated in the GENSIS-IPF Phase IIa trial, and the results were published in

2024年3月。更重要的是，Rentosertib在GENSIS-IPF二期a试验中展示了积极的结果，实现了全球首个AI驱动药物发现的有效性概念验证，结果发表在

Nature Medicine

自然医学

in June 2025.

2025年6月。

About Insilico Medicine

关于英硅智能

Insilico Medicine is a pioneering global biotechnology company dedicated to integrating artificial intelligence and automation technologies to accelerate drug discovery, drive innovation in the life sciences, and extend healthy longevity to people on the planet. The company was listed on the Main Board of the Hong Kong Stock Exchange on December 30, 2025, under the stock code 03696.HK..

英矽智能是一家领先的全球生物技术公司，致力于整合人工智能和自动化技术，加速药物发现，推动生命科学领域的创新，并延长地球上人类的健康寿命。该公司于2025年12月30日在香港证券交易所主板上市，股票代码为03696.HK。

By integrating AI and automation technologies and deep in-house drug discovery capabilities, Insilico is delivering innovative drug solutions for unmet needs including fibrosis, oncology, immunology, pain, and obesity and metabolic disorders. Additionally, Insilico extends the reach of Pharma.AI across diverse industries, such as advanced materials, agriculture, nutritional products and veterinary medicine.

通过整合人工智能和自动化技术以及深厚的内部药物发现能力，英矽智能正在为包括纤维化、肿瘤学、免疫学、疼痛、肥胖和代谢紊乱等未满足的需求提供创新的药物解决方案。此外，英矽智能还扩展了Pharma.AI在多个行业的应用范围，例如先进材料、农业、营养产品和兽医学。

.

。

For more information, please visit

更多信息，请访问

www.insilico.com

www.insilico.com

SOURCE Insilico Medicine

来源：Insilico Medicine

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