AI Breakthrough: A Landmark Moment as an AI-Designed Drug for Inflammatory Bowel Disease Ventures into Human Clinical Trials - Addressing a Significant Medical Need

Inflammatory bowel disease (IBD) affects approximately 1.6 million people in the U.S., and a new artificial intelligence-generated drug could offer relief from its symptoms. Insilico Medicine, a biotech company driven by AI and based in Hong Kong and New York City, has announced that its AI-designed IBD drug, ISM5411, has entered Phase I clinical trials. This marks the fifth AI-designed drug from Insilico to enter the pipeline.

Alex Zhavoronkov, PhD, founder and CEO of Insilico Medicine, is pictured in the company's New York lab. (Insilico Medicine)

If approved, ISM5411 would be the first medication targeting IBD by inhibiting prolyl hydroxylase domain (PHD), a protein that regulates the body’s gut barrier protection genes. According to Alex Zhavoronkov, PhD, founder and CEO of Insilico Medicine, "ISM5411 is a potentially first-in-class candidate for the treatment of IBD – this means that it is designed to treat the disease in an entirely new way."

Why the need for a new drug?

Inflammatory bowel disease (IBD), encompassing conditions like Crohn’s disease and ulcerative colitis, is characterized by debilitating symptoms such as abdominal pain, diarrhea, fatigue, weight loss, and rectal bleeding, as per Mayo Clinic.

Insilico Medicine, an AI-driven biotech company based in Hong Kong and in New York City, recently announced that its new AI-designed IBD drug — ISM5411 — has entered Phase I clinical trials. Fox News Digital spoke with its founder and CEO. (Insilico Medicine)

Alex Zhavoronkov, CEO of Insilico Medicine, noted, "This condition is on the rise across the industrialized world, but currently there are no cures and few treatments."

Existing drugs for IBD typically have anti-inflammatory properties and rely on immunosuppression for their effectiveness. However, this approach comes with inherent risks, as immunosuppression can lead to chronic infections and an increased risk of tumors, Zhavoronkov added.

Many existing biologic drugs for IBD are administered through intravenous (IV) infusion or self-injection. Recognizing a substantial need in this field, Insilico Medicine aimed to develop a new approach to treating IBD. According to Alex Zhavoronkov, the goal was to focus on healing and rebuilding the intestinal lining, aiming for long-term improvement of the condition rather than merely addressing the symptoms.

Tapping into AI's power

Patients with inflammatory bowel disease (IBD) tend to produce significantly more of the PDH protein compared to those without the condition. Insilico Medicine's research and development team utilized Chemistry42, the company's generative AI chemistry engine, to design a molecule that blocks PDH and stimulates the production of genes protecting the gut barrier.

"We knew there was a significant need in this space, and we wanted to find a new way to treat IBD that focused on healing and rebuilding the intestinal lining to lead to long-term improvement of the condition rather than just treating the symptoms," Alex Zhavoronkov, PhD, founder and CEO of Insilico Medicine, told Fox News Digital. (Insilico Medicine)

Alex Zhavoronkov explained, "You can think of Chemistry42 as a ChatGPT for new molecules." He added, "But instead of providing text commands and having an AI model generate a new essay or poem based on existing data, our platform draws from biological and chemical data to design an entirely new molecule using our instructions."

Insilico's AI platform generated several possible molecules based on specific criteria, ranked them, and the R&D team synthesized and tested several before selecting ISM5411 as the most promising candidate. Described as "intestinal restrictive," ISM5411 is an oral medication that specifically targets the gut without impacting other parts of the body.

Alex Zhavoronkov explained, "You can think of Chemistry42 as a ChatGPT for new molecules." He emphasized the significance of the AI-designed drug, ISM5411, stating, "Very importantly, it promotes mucosal repair." The drug is specifically designed to contribute to the restoration of normal gut functioning.

Research has indicated that mucosal repair can be effective in reducing hospitalization and improving the long-term prognosis for individuals with inflammatory bowel disease (IBD). The focus on promoting mucosal repair distinguishes ISM5411 in its potential to address the underlying issues associated with IBD.

Progress toward approval

Insilico's IBD drug, ISM5411, has entered Phase 1a clinical trials involving 76 healthy volunteers in Australia to assess its safety and tolerability with gradually increasing doses. Following the completion of Phase 1a, the company plans to initiate an international multi-center clinical trial in the U.S., China, and other locations, incorporating three treatment groups and one placebo group.

Insilico’s IBD drug has officially begun trials in 76 healthy volunteers in Australia to evaluate its safety and tolerability in gradually increasing doses.  (Insilico Medicine )

Alex Zhavoronkov stated, "Our clinical team is working hard to design and execute the clinical trials in order to develop it further so we can begin helping patients in need." He also expressed openness to collaboration, saying, "As we continue to advance this drug, we also look forward to connecting with companies with relevant experience and resources who are interested in co-developing this asset with our team."

ISM5411 is one of over 30 drugs designed by Insilico's AI platform, covering areas such as cancer, fibrosis, central nervous system diseases, and COVID-19. The company's lead drug, the first AI-discovered and AI-generated drug to reach Phase II trials with patients, is intended for the treatment of idiopathic pulmonary fibrosis, a progressive lung condition with a grim prognosis.

Potential benefits and risks

Dr. Supriya Rao, a gastroenterologist based in Massachusetts, provided her perspective on ISM5411, emphasizing its promising nature but acknowledging the hurdles and the novelty of the data at this stage of development. She highlighted the importance of considering longer-term safety and efficacy in the evaluation of the drug's potential.

Inflammatory bowel disease (IBD) impacts 1.6 million people in the U.S., causing symptoms that include diarrhea, abdominal pain, fatigue, weight loss and rectal bleeding

Dr. Harvey Castro, an AI expert and emergency medicine physician from Dallas, Texas, expressed caution regarding AI-generated drugs, emphasizing the uncertainty surrounding their long-term effects. While acknowledging the promise, he highlighted the relatively new and evolving nature of AI-designed drugs in the medical field.

Dr. Harvey Castro highlighted challenges in ensuring that AI algorithms are trained on comprehensive and unbiased datasets to avoid misinterpretations or gaps that could lead to ineffective or harmful drugs. He pointed out that existing regulatory frameworks may not fully address the nuances of AI-driven drug development.

Castro emphasized the importance of public awareness, stating, "As these drugs move from research labs to clinical settings, the public needs to understand the potential and the limitations." He stressed the need for educating patients and healthcare providers about the development process and potential impact of AI-generated drugs on treatment paradigms. This awareness is crucial for fostering informed decision-making and understanding the implications of AI advancements in healthcare.