Cambridge-based pharma company Insilico Medicine has announced promising results from a clinical trial of ISM001-055, a groundbreaking drug developed using generative AI to treat idiopathic pulmonary fibrosis (IPF).
IPF affects around five million people globally, causes irreversible scarring that makes breathing progressively difficult. Current treatments only slow progression, leaving many sufferers in desperate need of new options.
In a Phase IIa trial conducted in China with 71 patients, those receiving the highest daily dose of ISM001-055 (60mg) showed improved lung function and quality of life compared to the placebo group. The drug was well-tolerated, with most side effects deemed mild or moderate.
The drug targets TNIK, a molecule driving lung fibrosis in IPF, with the goal of halting or reversing disease progression. Designed using Insilico’s AI-powered Pharma.AI platform, ISM001-055 is the result of advanced modeling tools like PandaOmics and Chemistry42, which accelerate drug discovery and development.
Since its establishment in 2014, Insilico Medicine has advanced AI-driven drug discovery, with its methods featured in top journals like Nature Biotechnology. Its Pharma.AI platform uses generative AI to speed up drug development, fostering collaborations with major pharmaceutical companies, universities, and institutions worldwide.
With strong investor backing and a global presence in regions including the U.S., Greater China, Canada, and the Middle East, Insilico has cemented its reputation as a leader in AI-powered biotechnology.
Insilico CEO Alex Zhavoronkov highlighted the "the potential of generative AI and robotics to facilitate the discovery, design, and development of innovative therapies," in his announcement.
The company's next step is to enroll patients for US-based trials and engaging regulatory agencies to advance ISM001-055 toward pivotal trials. Full data from the study will be presented at an upcoming medical conference.
If approved, ISM001-055 could not only offer hope for IPF patients but also usher in a new era for the development of new drugs and treatments using AI.