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Feb 22 2025
1Insilico Medicine, a leader in AI-powered drug discovery, has announced a groundbreaking development: its lead drug candidate, ISM001-055, has shown promising anti-aging properties. Initially designed to treat idiopathic pulmonary fibrosis (IPF), recent research suggests the drug could have broader applications for age-related diseases due to its ability to reduce cellular senescence.
The study, conducted at Insilico’s AI-driven robotics laboratory in Suzhou, China, showed that ISM001-055 works as a senomorphic agent. Unlike traditional senolytics that eliminate senescent cells, senomorphics like ISM001-055 modify the behavior of these cells. This approach allows beneficial senescent cells, which aid in tissue repair and tumor suppression, to continue functioning while mitigating their harmful effects.
Remarkably, ISM001-055 outperformed the FDA-approved anti-aging drug rapamycin in two key areas. It was more effective in reducing pro-inflammatory cytokines linked to tissue dysfunction and age-related pathologies. Additionally, cells treated with ISM001-055 showed a more pronounced return to youthful functionality compared to those treated with rapamycin, highlighting its superior rejuvenation potential.
The drug targets Traf2- and NCK-interacting kinase (TNIK), a key player in cellular pathways involved in fibrosis and aging. By inhibiting TNIK, ISM001-055 suppresses pro-inflammatory cytokines and controls pathways like TGF-β and Wnt/β-catenin, which are critical in the aging process.
“The strong evidence that TNIK plays a role in aging and senescence could influence us to expand the development of ISM001-055 beyond fibrosis into broader anti-aging applications,” said Alex Zhavoronkov, PhD, founder and CEO of Insilico Medicine.
While ISM001-055 has already shown positive results in early-phase clinical trials for IPF, including improved lung function and reduced fibrosis, the latest findings suggest a much wider potential impact. Future plans may involve expanding clinical trials to study its effectiveness in treating age-related diseases more broadly.
Insilico's use of generative AI platforms like PandaOmics and Chemistry42 in developing ISM001-055 marks a significant milestone. It represents the company’s first internally developed candidate where AI identified both the novel target and molecular structure. Notably, ISM001-055 also earned the FDA’s first Orphan Drug Designation for an AI-developed drug in 2023.
As Insilico continues to validate ISM001-055's anti-aging potential, additional studies, including animal model testing and expanded clinical trials, are anticipated. If successful, the drug could revolutionize treatments for aging-related conditions, offering hope for extending healthspan and improving quality of life.
