Insilico Medicine announced today that they have started the first human trial of ISM001-055 their AI-designed drug candidate for pulmonary fibrosis. This is a very significant milestone in the history of AI-powered drug discovery because this is the first time that a human being has been dosed with an AI-discovered novel molecule based on an AI-discovered novel target. A healthy volunteer in an Australian study has received a first-in-human intravenous dose of the small-molecule inhibitor aimed at the chronic lung disease. This is the first AI discovered compound to enter the clinic and more compounds are expected in the near future.
Insilico leveraged Pharma.AI, their AI-powered drug discovery platform, to analyze over 20 disease targets to predict the most successful candidate. The company completed the entire discovery process from target discovery to preclinical candidate nomination within 18 months on a budget of $2.6 million. Target discovery is the grand challenge in the pharmaceutical industry because the failure rate in preclinical target discovery is very high. Even after targets are validated in animal models, over half of Phase 2 clinical trials fail primarily due to the choice of target.
“Insilico Medicine has achieved this breakthrough milestone which is extremely impressive. Many drugs were discovered accidentally when scientists designed a drug for disease A and then they found out that it actually works for some different disease B. AI is a way to search for information and look for signals for drug discovery. This achievement didn’t happen by chance and is a reproducible method and procedure which is revolutionary.”
Michael Levitt, PhD, 2013 Nobel Laureate in Chemistry and member of Insilico Medicine’s Scientific Advisory Board
Since 2014, Insilico Medicine has demonstrated its ability to identify novel targets using deep learning techniques and the ability to generate drug-like hit molecules using generative AI for a well-known target in record time. The company has also demonstrated the target’s proof of concept by applying deep learning techniques for the identification of novel biological targets. This novel antifibrotic program combined both discovery and generative chemistry capabilities.
With ISM001-055 Insilico used end-to-end AI connecting biology, chemistry in order to assess activity and safety in multiple preclinical models. ISM001-055 demonstrated highly promising results in multiple preclinical studies including in vitro biological studies, pharmacokinetic, and safety studies. The compound significantly improved myofibroblast activation which contributes to the development of fibrosis. ISM001-055’s novel target is potentially relevant to a broad range of fibrotic indications.
“What impresses me the most about this achievement is not only the lower cost or faster speed of development but, also it demonstrated the Pharma AI platform can overcome the low probability of getting to this stage. There are very few examples of a pharmaceutical company discovering a new target for a broad range of diseases, designing a novel molecule, and initiating human clinical trials. To my knowledge, nobody has achieved this with AI to-date.”
Alex Zhavoronkov, PhD, Founder and CEO of Insilico Medicine
ISM001-055 is a potentially first-in-class small molecule inhibitor of a novel biological target discovered by Pharma.AI, Insilico Medicine’s end-to-end AI-powered drug discovery platform. It is being developed for the treatment of idiopathic pulmonary fibrosis a chronic lung disease which results in progressive and irreversible decline in lung function. After completing IND-enabling studies, Insilico Medicine initiated the microdose trial to begin characterizing the pharmacokinetic profile in humans.
In June, Insilico closed a $255 million oversubscribed Series C round led by Warburg Pincus. Since its inception in 2014 the company has raised over $310 million and received multiple industry awards. Insilico Medicine has also published over 130 peer-reviewed papers and has applied for over 30 patents. Proceeds from this round were used to advance therapeutic programs into human clinical trials, initiate multiple new programs for novel and difficult targets, and further develop its AI and drug discovery capabilities.
As a pioneer and leader in the industry, Insilico Medicine has built a strong drug discovery and drug development team, a broad distributed discovery partner network, and initiated multiple internal therapeutic programs. The company is making its technologies available for licensing for the leading pharmaceutical and biotechnology companies looking to gain a head start in AI and accelerate their drug discovery programs. It has collaborated with first-in-class biopharmaceutical companies, including Pfizer, Astellas, Johnson & Johnson, Taisho, and many others, with multiple partners reaching major milestones.
Insilico has developed AI platforms that utilize deep generative models, reinforcement learning, transformers, and other modern machine learning techniques to discover novel targets and to design novel molecular structures with desired properties. The company is delivering breakthrough solutions to discover and develop innovative drugs for cancer, fibrosis, immunity, central nervous system diseases and aging-related diseases. Insilco’s AI technologies are demonstrating impressive results and delivering valuable and absolutely novel therapeutic assets with high probability of clinical success.