Insilico Medicine, a clinical-stage generative AI-driven drug discovery company, announced positive preliminary results from its Phase IIa clinical trial evaluating ISM001-055. ISM001-055 is a first-in-class small molecule targeting TNIK (Traf2- and Nck-interacting kinase) and was designed utilizing generative AI to treat idiopathic pulmonary fibrosis (IPF).

Insilico Medicine是一家临床阶段生成人工智能驱动的药物发现公司，宣布其评估ISM001-055的IIa期临床试验的初步结果为阳性。ISM001-055是靶向TNIK（Traf2和Nck相互作用激酶）的一流小分子，是利用生成性AI治疗特发性肺纤维化（IPF）的设计。

The study met both its primary endpoint of safety and its secondary efficacy endpoints, demonstrating dose-dependent response in forced vital capacity (FVC), a critical measure of lung function in IPF patients.Insilico's proprietary AI platform facilitated ISM001-055's target identification and molecular design.

该研究符合其主要安全性终点和次要疗效终点，证明了强制肺活量（FVC）的剂量依赖性反应，这是IPF患者肺功能的关键指标。Insilico专有的AI平台促进了ISM001-055的目标识别和分子设计。

Its development was recently described in a March 2024 Nature Biotechnology paper, which detailed TNIK's identification as a novel therapeutic target in IPF and ISM001-055 subsequent design. This comprehensive paper showcased ISM001-055's preclinical evaluation and positive Phase 0 & Phase I clinical studies justifying this intervention's potential as a disease-modifying agent for IPF.ISM001-055's Phase IIa study(NCT05938920)  was a randomized, double-blind, placebo-controlled trial that enrolled 71 patients with IPF across 21 sites in China.

最近在2024年3月的《自然生物技术》论文中描述了它的发展，该论文详细介绍了TNIK在IPF和ISM001-055后续设计中作为新型治疗靶标的鉴定。这篇综合性论文展示了ISM001-055的临床前评估和积极的0期和I期临床研究，证明了这种干预作为IPF疾病改良剂的潜力。ISM001-055的IIa期研究（NCT05938920）是一项随机，双盲，安慰剂对照试验，在中国21个地点招募了71名IPF患者。

Patients were randomized to receive either placebo, 30mg once daily (QD), 30mg twice daily (BID), or 60mg QD for 12 weeks. Patient enrollment was initiated in April 2023, and the last subject's follow-up visit was completed in August 2024. A parallel Phase IIa(NCT05975983) clinical trial in the U.S.

患者被随机分配接受安慰剂，每天一次30mg（QD），每天两次30mg（BID）或每天一次60mg，持续12周。患者登记于2023年4月开始，最后一名受试者的随访于2024年8月完成。美国的平行IIa期临床试验（NCT05975983）。

is ongoing and actively enrolling patients. In this 12-week Phase IIa study, ISM001-055 met its primary endpoint of safety and tolerability across all dose levels. Positive results were also reported for the secondary efficacy endpoint, wherein a dose-dependent FVC improveme.

正在进行并积极招募患者。在这项为期12周的IIa期研究中，ISM001-055在所有剂量水平上均达到了安全性和耐受性的主要终点。次要疗效终点也报告了阳性结果，其中剂量依赖性FVC改善。