Insilico’s AI drug for IPF gets official name, hits trial milestone

USA – Insilico Medicine, a clinical-stage biotech company, announced that its lead drug candidate for idiopathic pulmonary fibrosis (IPF) now has an official generic name: Rentosertib.

The name was granted by the United States Adopted Names (USAN) Council, marking a symbolic step forward for a drug discovered entirely through artificial intelligence.

Rentosertib is the first experimental drug where both the disease target and the compound itself were discovered using Insilico’s generative AI platforms.

“This is a breakthrough moment for the pharma and AI industries,” said Dr. Alex Zhavoronkov, CEO of Insilico. “Rentosertib shows how human intelligence and AI can work together to find solutions to tough diseases like IPF.”

IPF is a chronic, progressive lung disease that affects around 5 million people globally. It causes scarring of lung tissue, making it difficult to breathe.

Current treatments only slow the disease down; they don’t stop or reverse it. Most patients are given a life expectancy of just 3 to 4 years after diagnosis.

Insilico tackled this challenge using its Pharma.AI platform. The company’s biological analysis tool, PandaOmics, identified a novel protein target for IPF called TNIK.

Then, its chemistry engine, Chemistry42, designed molecules to inhibit TNIK. This resulted in the creation of Rentosertib—all in just 18 months from target discovery to a preclinical drug candidate.

The name Rentosertib also has personal meaning. It partly honors Dr. Feng Ren, Insilico’s Co-CEO and Chief Scientific Officer, who led the program and authored the company’s 2024 Nature Biotechnology paper on this discovery.

“Having Rentosertib officially named reflects its maturity as a drug candidate,” said Dr. Ren. “It also signals recognition of AI’s growing role in how we develop new treatments.”

The drug is showing promise in clinical trials. In Phase I trials in New Zealand and China, Rentosertib was tested on healthy volunteers and found to be safe and well-tolerated, with good pharmacokinetic results. That success paved the way for a Phase IIa trial in IPF patients.

Here’s what the Phase IIa study found:

• Improved lung function: Patients taking Rentosertib showed a dose-dependent improvement in lung capacity. At the highest dose, they gained nearly 100 mL in forced vital capacity (FVC), while those on placebo lost lung volume.
• Better quality of life: Patients on Rentosertib also reported relief in respiratory symptoms like coughing.
• Strong safety: Side effects were mostly mild or moderate, and no serious drug-related issues occurred.

With these results, Insilico now plans to begin larger trials and engage regulators globally, aiming to make Rentosertib the first AI-discovered drug approved for human use.