Insilico Medicine Reports Positive Phase IIa Results for AI-Designed Drug Targeting Idiopathic Pulmonary Fibrosis

ISM001-055, developed using Insilico’s proprietary AI platform, targets TNIK (Traf2- and Nck-interacting kinase), a protein linked to fibrosis, the key pathological process behind IPF. The 12-week Phase IIa trial, which enrolled 71 patients across 21 sites in China, tested the drug’s safety and impact on lung function. The trial met its primary endpoint of safety and tolerability at all dose levels, while also showing a dose-dependent improvement in forced vital capacity (FVC), a critical measure of lung function. Patients receiving the highest dose (60mg once daily) demonstrated the greatest improvement in FVC.

Dr. Toby M. Maher, an investigator in the trial, noted that the dose-dependent response in FVC is particularly encouraging, suggesting that ISM001-055 may offer a new therapeutic option for IPF patients. He also mentioned that a Phase IIa trial in the U.S. is actively recruiting patients.

Reflecting on the growing impact of AI in drug discovery, Nobel laureate Dr. Michael Levitt stated:

"Last year, I presented a lecture on how generative AI can help with end-to-end drug discovery from disease modeling and target identification to generation of novel drugs with the desired properties and purposing it to a specific disease. I used Insilico's TRAF2 and NCK-interacting kinase (TNIK) inhibitor as a case study going from 0 to Phase I. The fact that this same drug demonstrated efficacy in addition to safety in a Phase IIa study is extraordinary and represents a true first in this new era of AI-powered drug discovery."

See also: It’s Been a Decade of AI in the Drug Discovery Race. What’s Next?

IPF, a rare and progressive lung disease affecting around 5 million people worldwide, has limited treatment options, with current therapies only slowing its progression. ISM001-055, which received FDA Orphan Drug Designation in early 2023, aims to halt or even reverse the fibrosis process in IPF, offering hope for more effective treatment.

Insilico Medicine’s Co-CEO, Dr. Alex Zhavoronkov, commented on the trial results:

"While we expected the drug to be safe, we did not expect to see such a clear dose-dependent efficacy signal after such a short dosing period. IPF is a very diverse disease, and it is very rare to see improvement in FVC. With our novel TNIK inhibitor, we attempted to go after what we think is a common mechanism in fibrotic diseases and in aging to maximize indication expansion potential."

Following the Phase IIa success, Insilico Medicine plans to engage with regulatory authorities to design a Phase IIb trial. The next phase will aim to explore extended treatment durations and involve larger patient cohorts, further evaluating ISM001-055’s potential as a disease-modifying therapy.