AI Drug Prevents Lung Aging: New Human Trial

A drug discovered and designed by artificial intelligence (AI) improves the breathing capacity of individuals with age-related lung disease.

Key Points:

Rentosertib is the first drug discovered and designed by AI.
Rentosertib was shown to improve lung capacity in individuals with age-related lung disease.
Overall, rentosertib was safe and tolerable.

In a significant stride for both AI and medical science, a new drug for idiopathic pulmonary fibrosis (IPF), a debilitating and progressive lung disease, has successfully completed its first Phase 2a clinical trial. What makes this achievement particularly noteworthy is that the drug, rentosertib, was discovered and designed using generative AI. This marks a pivotal moment, demonstrating AI’s growing capacity to revolutionize the traditionally slow and costly process of drug development.

The Challenge of Idiopathic Pulmonary Fibrosis

IPF is a chronic and often fatal lung condition characterized by the scarring of lung tissue. This scarring, or fibrosis, thickens and stiffens the lungs, making it increasingly difficult to breathe. The term “idiopathic” means the cause is unknown, adding to the complexity of treating this relentless disease. Still, the primary risk factor for IPF is older age, making it an age-related lung disease. Current therapies can slow the progression of IPF but do not reverse the damage, leaving a significant unmet medical need for patients worldwide.

AI Steps In: A New Approach to Drug Discovery

The journey of drug discovery is notoriously arduous, often taking over a decade and billions of dollars to bring a single new medicine to market. This is where AI offers a transformative advantage. Generative AI, a branch of AI that can create new content, was employed to identify a novel therapeutic target for IPF: an enzyme called TNIK (Traf2- and Nck-interacting kinase). Beyond identifying the target, the AI also designed a small molecule, rentosertib, specifically to inhibit TNIK.

(Wan et al., 2025) **TRAF2 Activation Leads to Lung Disease.** Since TRAF2 is thought to lead to IPF, inhibiting it with rontoserib may counteract IPF.

This AI-driven approach dramatically streamlined the early stages of drug development. From target identification to the completion of initial clinical testing, the entire process took less than 30 months—a fraction of the time typically required. This efficiency highlights the potential for AI to accelerate the delivery of much-needed treatments to patients.

Safe and Tolerable

The recent Phase 2a clinical trial of rentosertib included patients with IPF, some of whom were taking existing anti-IPF drugs. The study was designed to assess the drug’s safety and tolerability, as well as to gather preliminary data on its effectiveness. Patients were randomly assigned to receive different doses of rentosertib or a placebo over a 12-week period.

The results showed that rentosertib was generally safe and well-tolerated. Namely, the percentage of patients experiencing side effects was similar across all treatment groups, including the placebo group. While some patients experienced common side effects like diarrhea, nausea, and fatigue, serious adverse events were rare and comparable to those seen with the placebo. A small number of patients discontinued treatment due to liver toxicity or diarrhea, which are important considerations for future studies.

Rentosertib Prevents Further Lung Aging

Patients receiving 60 mg/day of rentosertib showed a clinically important improvement in forced vital capacity (FVC). FVC is a critical measure of lung function, indicating the maximum amount of air a person can exhale after a deep breath. An increase in FVC suggests that rentosertib may have a disease-modifying effect, potentially slowing or even reversing the progression of lung damage in IPF. Indeed, the placebo group and the group taking 30 mg/day of rentosertib showed a decline in FVC.

(Xu et al., 2025) **Rentosertib Prevents Lung Function Decline.** The placebo group showed a 20.3 mL decrease in air breathed (FVC change from baseline) while the group taking 60 mg once per day (QD) of rentosertib showed a whopping 98.4 mL increase. Notably, those taking 30 mg QD saw a 27 mL decrease, and those taking 30 mg twice per day (BID) saw a 19.7 mL increase.

It’s important to note that this was a relatively small and short-duration study, and the trial was not specifically designed to prove efficacy. Therefore, these promising results will need to be confirmed in larger, longer-term Phase 3 clinical trials. Nevertheless, this early signal of efficacy is a significant step forward for patients battling IPF.

The Future of AI and Robotics in Medicine

The successful completion of this Phase 2a trial represents a landmark achievement for AI in drug discovery. It provides compelling evidence that AI can not only accelerate the identification of new drug candidates but also contribute to the development of effective treatments for complex diseases. The next steps will involve more extensive clinical trials to further evaluate rentosertib’s long-term safety and efficacy in a broader patient population.

This breakthrough offers renewed hope for individuals living with IPF and underscores the transformative potential of AI in shaping the future of medicine. As AI continues to evolve, we can anticipate even more innovative solutions emerging from this exciting intersection of technology and healthcare. For instance, Insilco Medicine, the company behind rentosertib, which already utilizes an automated lab, will soon be employing humanoid robots to conduct its experiments.