A Hong Kong-based biotech startup called Insilico Medicine has developed the first-ever drug completely generated by artificial intelligence (AI), which has now entered clinical trials involving human patients. The drug, named INS018_055, aims to treat idiopathic pulmonary fibrosis (IPF), a chronic lung disease characterized by lung scarring. This condition, which has become more prevalent in recent years, currently affects approximately 100,000 individuals in the United States. If left untreated, IPF can lead to death within a span of two to five years, as stated by the National Institutes of Health.
Insilico Medicine, backed by over $400 million in funding, accomplished the remarkable feat of creating this AI-generated drug. The CEO and founder of the company, Alex Zhavoronkov, expressed that this drug is the first of its kind to reach human clinical trials, specifically Phase II trials involving patients. While other AI-designed drugs are also being tested, INS018_055 stands out as it not only features a novel AI-discovered target but also possesses a unique AI-generated design.
The development process for this groundbreaking drug commenced in 2020, driven by the desire to create a revolutionary treatment that surpasses the limitations and side effects associated with current therapies for IPF, which primarily focus
on slowing down disease progression. Zhavoronkov mentioned that Insilico decided to concentrate on IPF partly due to its implications in aging. However, the company has two other drugs in the clinical stage, both partially generated by AI. One is a COVID-19 drug currently undergoing phase one clinical trials, and the other is a cancer drug known as a “USP1 inhibitor,” which recently obtained FDA approval to initiate clinical trials for the treatment of solid tumors.Zhavoronkov admitted that when the company was first established, their primary focus was on algorithms and developing the technology necessary for discovering and designing new molecules. He never imagined in those early days that he would be involved in conducting clinical trials with patients using AI-generated drugs. However, the realization that validating their AI platform required not only designing a new drug for a new target but also bringing it to clinical trials to demonstrate the efficacy of their technology prompted this significant milestone.
The current study of the IPF drug is a randomized, double-blind, placebo-controlled trial taking place over a period of 12 weeks in China. Insilico intends to expand the testing to include 60 subjects at 40 sites in both the United States and China. If the ongoing phase two study proves successful, it will progress to another study involving a larger group of participants, potentially leading to phase three studies with hundreds of individuals.
Zhavoronkov shared his expectation of obtaining results from the current phase II trial next year, although predicting the exact timing of future phases is challenging, especially given the rarity of the disease and the specific criteria that patients must meet. However, he expressed optimism that this drug will be ready for market and made available to patients who could benefit from it within the next few years.
