·ISM001-055 is an innovative drug driven by generative artificial intelligence and fully developed independently by Insilicon Intelligence. It targets TNIK (Traf2/NCK interacting kinase) and has completed phase IIa clinical trials for the treatment of patients with idiopathic pulmonary fibrosis (IPF);

·This study lasted for 12 weeks, and its preliminary results showed that ISM001-055 exhibited good safety in IPF patients and showed a dose-dependent pharmacological trend in improving lung function indicators in patients;
·The positive clinical trial results of ISM001-055 also provide the first conceptual validation for AI driven drug development.

Hong Kong, China, September 18, 2024- Yingsi Intelligent, a clinical stage biotechnology company driven by generative artificial intelligence (AI), announced that its pipeline ISM001-055 has achieved positive preliminary research results in a phase IIa clinical trial. ISM001-055 is a "first in class" small molecule inhibitor driven by generative AI for drug discovery and design, targeting TNIK (Traf2/NCK interacting kinase) for the treatment of idiopathic pulmonary fibrosis (IPF). This study not only achieved the primary endpoint of drug safety validation, but also the secondary endpoint of preliminary efficacy validation. It showed a dose-dependent trend in the efficacy of forced vital capacity (FVC), an important indicator for evaluating lung function improvement in IPF patients.
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Previously, in March 2024, Yingsi Intelligence published a research paper in the top global journal Nature Biotechnology, detailing the discovery of a novel target TNIK for treating IPF using an artificial intelligence platform, as well as the entire process of designing ISM001-055 molecules using a generative chemistry platform. The paper also disclosed for the first time preclinical data of small molecule drugs empowered by artificial intelligence, as well as positive results from phase 0 low-dose human trials and phase 1 clinical studies, demonstrating the potential of the molecule to improve IPF disease.
This phase IIa clinical study (NCT05938920) targeting ISM001-055 is a randomized, double-blind, placebo-controlled trial that recruited 71 IPF patients from 21 clinical research centers in China. The patients were randomly assigned to receive placebo, once daily 30mg, twice daily 30mg, or once daily 60mg for 12 weeks of medication observation. The clinical trial started patient recruitment in April 2023 and completed the follow-up of the last participant in August 2024. Meanwhile, a parallel phase IIa trial of ISM001-055 (NCT05975983) is underway in the United States and patient recruitment has been initiated.
The preliminary results of the 12 week study indicate that ISM001-055 demonstrates good safety at all dose levels. The secondary endpoint, which mainly evaluated the improvement of lung function in IPF patients through FVC, showed a dose-dependent pharmacological trend. IPF patients who received 60mg once daily medication showed the greatest improvement in FVC. Yingsi Intelligent plans to release the complete data of this research at subsequent medical conferences and submit it to the journal for peer review and publication.
Dr. Alex Zhaovoronkov, founder and CEO of Insilicon Intelligence, said, "This research result represents a key milestone in AI driven drug discovery and an important milestone in my life so far. Although we expected the drug to be safe, we did not expect to see significant dose-dependent efficacy signals after such a short dosing period. IPF is a very diverse disease, and it is difficult to see improvement in FVC in patients. We are trying to find common biological mechanisms in fibrosis and aging, and maximize the potential of innovative TNIK inhibitors in expanding indications
Dr. Feng Ren, Co CEO and Chief Scientific Officer of Insilicon Intelligence, said, "We are pleased to see significant clinical efficacy of ISM001-055 in treating idiopathic pulmonary fibrosis (IPF) during the three-month treatment cycle. ISM001-055 provides conceptual validation for AI driven drug discovery. Although these data are still preliminary, they are undoubtedly very encouraging. I believe that the achievement of this milestone is attributed not only to the capabilities of our proprietary generative artificial intelligence platform, but also to the efforts of our multidisciplinary research and development team. We will continue to strive for the benefit of patients worldwide. "
After obtaining positive results from this Phase IIa trial, the British Silicon Intelligence program has discussed with regulatory agencies the design of the ISM001-055 Phase IIb study, aiming to further investigate the therapeutic potential of ISM001-055 for IPF over a longer dosing period and a wider patient cohort.
Regarding ISM001-055 and TNIK
ISM001-055 is a potential global first small molecule inhibitor that targets the novel target TNIK discovered through artificial intelligence empowerment. In IPF, activation of TNIK targets can drive pathological fibrosis of the lungs, leading to progressive decline in lung function. By inhibiting TNIK signaling, ISM001-055 aims to prevent or reverse the fibrotic process, providing an improved treatment for IPF patients. In February 2023, ISM001-055 was granted orphan drug qualification by the FDA.
Regarding this Phase IIa study
This double-blind, placebo-controlled phase IIa clinical trial (NCT05938920) evaluated the safety, tolerability, pharmacokinetics, and preliminary efficacy of ISM001-055 orally administered for 12 weeks in IPF subjects. 71 patients from 21 research centers were randomly assigned to four parallel groups: once daily 30mg administration, twice daily 30mg administration, once daily 60mg administration, and placebo. The preliminary results of the study show that ISM001-055 exhibits good safety in IPF patients and shows a dose-dependent pharmacological trend in improving lung function indicators in patients.
Regarding idiopathic pulmonary fibrosis (IPF)
Idiopathic pulmonary fibrosis (IPF) is a chronic, scarred lung disease characterized by progressive and irreversible decline in lung function. IPF affects approximately 5 million people worldwide, with poor prognosis and a median survival of only 3 to 4 years. The current treatment methods include anti fibrotic drugs, which can slow down disease progression but cannot stop or reverse the disease process. Therefore, there is an urgent need for more effective treatment methods to improve the disease. IPF is an age-related disease, with an average onset age typically between 60 and 70 years old, most commonly in the elderly, and less common in individuals under 50 years old.
About Yingsi Intelligent
Yingsi Intelligent is a clinical stage biopharmaceutical technology company driven by generative artificial intelligence. It connects biology, chemistry, and clinical trial analysis through next-generation artificial intelligence systems, and uses modern machine learning technologies such as deep generative models, reinforcement learning, and transformation models to build a powerful and efficient artificial intelligence drug development platform. It identifies new targets and generates candidate drugs with specific molecular structures. Yingsi Intelligent focuses on unmet medical needs such as cancer, fibrosis, immunity, central nervous system diseases, and aging related diseases, and promotes and accelerates innovative drug research and development.