Insilico Medicine announces phase 2 success for AI-designed drug

Insilico Medicine, a biotechnology company with operations in both China and the United States, recently announced that its leading drug candidate has successfully met its primary safety endpoint and secondary efficacy endpoints. The drug, INS018_055 (also known as ISM001-055), is a small molecule targeting TRAF2- and NCK-interacting kinase (TNIK). While TNIK inhibitors are typically studied for their potential anticancer properties, they are also being explored for their anti-fibrotic capabilities, which could be beneficial in treating lung diseases like idiopathic pulmonary fibrosis (IPF).

The phase 2a clinical trial of INS018_055/ISM001-055 involved 71 IPF patients in China. These participants were randomly assigned to receive either a placebo, 30mg once daily, 30mg twice daily, or 60mg once daily over a 12-week period. Concurrently, a similar phase 2a trial is in the recruitment phase in the United States.

In a recent statement, Insilico Medicine declared that its drug candidate had achieved its primary endpoint of safety and tolerability across all dosage levels. Additionally, the secondary efficacy endpoints were also met, with the highest dose of 60mg once daily showing the most significant improvement in forced vital capacity (FVC). Following these promising results, the company plans to design a phase 2b trial in consultation with regulatory authorities.

Despite these encouraging findings, Insilico has not yet released the complete data from the trial, which makes it difficult to fully assess the drug's efficacy. The company mentioned in a press statement that full topline data would be presented at an upcoming medical conference and that the clinical trial results would be submitted for publication in a peer-reviewed journal.

Toby Maher, an expert in interstitial lung disease who is actively involved in the Insilico clinical trials program, commented positively on the results. He emphasized that the dose-dependent response in FVC is particularly encouraging. "IPF is a devastating disease, and seeing improvements in lung function over just 12 weeks of treatment is a promising indication that ISM001-055 may provide a new therapeutic option for patients," Maher said.

Insilico is entering an increasingly competitive space targeting IPF. Earlier this week, Boehringer Ingelheim reported positive phase 3 results for its new IPF candidate. Other companies including Vicore Pharma, Bridge Therapeutics, and Pliant Therapeutics have also advanced their IPF candidates to phase 2 trials. Whether Insilico’s drug will be effective enough to stand out among these competitors remains to be seen.

Specializing in generative artificial intelligence (AI) for drug discovery, Insilico Medicine leverages AI to identify new drug targets and accelerate the early stages of drug development before clinical trials. The company was among the first to apply generative AI to drug development, having published a paper on the subject in 2016.

Since 2021, Insilico has announced 12 different preclinical therapeutic candidates aimed at treating conditions such as cancer, Covid-19, inflammatory bowel disease, and kidney fibrosis. Three of these candidates have advanced to clinical trials. INS018_055, which targets IPF, is the first drug designed using generative AI to enter phase 2 trials in 2023.

Alex Zhavoronkov, co-CEO of Insilico Medicine, remarked on the significant milestone these results represent in the realm of AI-driven drug discovery. "This study result represents a critical milestone in AI-powered drug discovery and in my life to date," Zhavoronkov stated. "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."