Insilico Medicine begins first human trial of its AI-designed drug for pulmonary fibrosis

Insilico Medicine launched its first trial in humans of its computer-designed drug candidate after demonstrating its artificial intelligence platforms could not only identify a potentially new cellular target for the treatment of idiopathic pulmonary fibrosis but also conjure up a novel drug in less than 18 months and for pennies-on-the-dollar compared to most Big Pharma R&D efforts.

The former Fierce 15 winner announced that the first healthy volunteer in an Australian study has received a limited, intravenous dose of the company’s ISM001-055, a small-molecule inhibitor aimed at the chronic lung disease.

“We believe this is a significant milestone in the history of AI-powered drug discovery because to our knowledge the drug candidate is the first ever AI-discovered novel molecule based on an AI-discovered novel target,” Insilico’s chief scientific officer, Feng Ren, Ph.D., said in a statement.

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Insilico previously outlined plans to ultimately deliver the compound as a once-daily oral medication with the goal of improving lung function and described its first clinical outing as a microdosing trial to study the drug's pharmacokinetic profile.

After less than a year and a half of AI-led development—and for a total cost of about $2.6 million—the company said it was able to discover and whittle down more than 20 disease targets and dozens of deep-learning-generated molecules to predict the most successful candidate through its Pharma.AI platform.

During that time, ISM001-055 went on to complete preclinical studies including in vitro and in vivo tests. Insilico then spent the following nine months focused on formulating its active pharmaceutical ingredients and establishing manufacturing processes ahead of clinical trials.

Insilico said the compound showed preliminary improvements in activating the myofibroblast cells that are essential for repairing damaged tissues. It also noted that the drug’s target could be potentially applied to other fibrotic diseases such as within the kidneys or liver.

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“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,” said Alex Zhavoronkov, Insilico’s founder and CEO. “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.”

“The failure rates in preclinical target discovery are very high and even after the targets are validated in animal models, over half of phase 2 clinical trials fail primarily due to the choice of target,” Zhavoronkov said. “Target discovery is the fundamental grand challenge of the pharmaceutical industry.”

This past summer, Insilico announced it had uncovered a separate preclinical drug candidate aimed at kidney fibrosis. The company’s AI suite includes PandaOmics for narrowing down disease targets, Chemistry42 for generating potentially druglike compounds and InClinico for aiding in the design of clinical trials and predicting their success through actuarial modeling.

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The company said it plans to complete preclinical testing of the kidney-focused molecule by the end of 2022. 

Both development programs got a large boost this summer from $255 million in venture capital money. The series C financing, led by Warburg Pincus and with about 20 backers in total, marked a serious leap above its previous fundraising round, which had topped just $37 million in 2019.