The world's first AI-developed drug to treat lung disease is undergoing clinical trials in China and the USA

An experimental drug developed using artificial intelligence (AI) to combat an aggressive and often fatal lung disease has entered phase 2 clinical trials in China and the United States. According to AI drug development company Insilico Medicine, this is the world's first drug created using AI.

The company said its AI-based methodology has enabled faster and more efficient drug discovery and is proof of the “promising potential of generative AI technologies to transform the industry.”

Insilico is a global biotechnology company with offices and researchers in Hong Kong and mainland China, Europe, the Middle East and North America.
The company's founder and CEO, Alex Zhavoronkov, told the Post that while generative AI has only become widely known in recent years, he has been exploring its applications in biomedical research for a decade.

“The integration of AI, robotics and research in the field of aging will allow us to find a complete cure for such complex diseases as Alzheimer’s and Parkinson’s, as well as many others,” Zhavoronkov said, adding that AI can provide humans with the tools to completely avoid these diseases .

In 2014, the company began training deep neural networks to understand the human aging process, using the power of AI to record, track and analyze people's health throughout their lives.

“AI can understand billions of people simply by understanding what aging is. It can then begin to understand the underlying biology of diseases, and not just to slow down their progression,” Zhavoronkov says.

“In an ideal scenario, we want to see the disease disappear completely or not exist at all.”

Zhavoronkov, who calls aging “biology in time,” is an expert in generative biology and chemistry, as well as in aging and longevity research.

“Human biology and the homeostasis (state of balance between body systems) of your body breaks down over time. This is what happens with disease. Diseases accelerate or are caused by this process. Therefore, without understanding the process of basic human aging, you cannot understand most diseases.” – he said.

Idiopathic pulmonary fibrosis (IPF) causes chronic scarring of the lung tissue, making it difficult to breathe. The disease affects 5 million people worldwide, mostly over 60 years of age, and has a high mortality rate. The median survival rate for patients who do not receive treatment is two to three years.

The cause of the disease is unknown and there is no cure, but some treatments can help relieve symptoms and slow the progression of the disease. Many patients receiving steroids suffer progressive decline in lung function and die from respiratory failure.

In the new study, researchers used generative artificial intelligence to find an antifibrotic target and its inhibitor, significantly reducing traditional drug development timelines, which can often take more than a decade.

“This work was completed in approximately 18 months from target discovery to preclinical candidate advancement and demonstrates the capabilities of our artificial intelligence-driven generative drug discovery system,” said the paper, published Friday in the peer-reviewed journal Nature Biotechnology.

The researchers first trained a target identification engine on the artificial intelligence platform Insilico using data and publications on fibrosis. This disease causes tissue to thicken or scar, which can reduce the elasticity of organs.

Fibrosis is closely related to the aging process, which results in chronic inflammation leading to fibrosis.

With the help of predictive artificial intelligence, a protein called TNIK has emerged as a leading antifibrotic target. The team then used generative chemistry to generate about 80 small molecule candidates to find the optimal inhibitor, known as INS018_055.

“[Ингибитор] exhibits desirable medicinal properties and antifibrotic activity in various organs… when administered orally, inhaled or topically,” the scientists write.

They said the study “proves that generative AI platforms offer time-efficient solutions for creating targeted drugs with potent antifibrotic activity.”

“We believe this study highlights the power of artificial intelligence approaches to drug discovery, which are likely to revolutionize drug discovery.”

According to the company, concurrent Phase 2a clinical trials of INS018_055, taking place in China and the United States, involving 60 patients each, will evaluate its safety, tolerability and pharmacokinetics – the body's interaction with a substance over time – as well as its preliminary effectiveness on function. lungs in patients with IPF.

When asked about the importance of artificial intelligence in drug discovery research, Insilico, an AI chatbot built on ChatGPT, said: “By simplifying the early stages of drug discovery, AI allows us to reach the clinical trial stage faster, focusing resources and efforts on these critical stages of development “.

“While AI can speed up early-stage drug discovery challenges, such as target identification and lead optimization, it does not significantly reduce clinical trial times.

“Clinical trial phases still require significant time for ethical and regulatory approvals, patient recruitment, treatment duration, and data analysis.”