Insilico Medicine, a biotech company with offices in both China and the US, announced this week that its lead candidate drug had met its primary endpoint of safety and its secondary efficacy endpoints.
INS018_055 (also known as ISM001-055) is a small molecule drug candidate targeting TRAF2- and NCK-interacting kinase (TNIK). TNIK inhibitors are being investigated for potential anticancer properties, but are also thought to have anti-fibrotic properties that could be effective for treating lung diseases such as idiopathic pulmonary fibrosis (IPF).
The phase 2a trial of INS018_055/ISM001-055 enrolled 71 patients in China with IPF who were randomly assigned to receive: placebo, 30mg once daily, 30mg twice daily, or 60mg once daily for 12 weeks. A similar phase 2a trial is currently recruiting in the US.
In a statement released this week, the company reported that its candidate therapy had met its primary endpoint of safety and tolerability at all doses. It also said secondary efficacy endpoints were also met, with the highest improvement in forced vital capacity (FVC) seen with the highest dose of 60mg once daily. The company will now design a phase 2b trial in consultation with regulators.
Notably, Insilico has not yet released full data from the trial, making it difficult to judge the degree of efficacy of the candidate therapy. In a press statement it said that complete topline data would be released at an upcoming medical conference and that the clinical trial results would be submitted for publication in a peer-reviewed journal.
“These results are very encouraging, particularly the dose-dependent response in FVC. 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,” said Toby Maher, an expert in interstitial lung disease who is involved with the Insilico clinical trials program for this drug candidate, in the company press statement.
Insilico is not the only company targeting IPF, Boehringer Ingelheim reported positive phase 3 results for its new IPF candidate earlier this week and Vicore Pharma, Bridge Therapeutics and Pliant Therapeutics all have candidates that have reached phase 2. Whether Insilico’s candidate is effective enough to compete with others in the space remains to be seen.
AI-based drug design
Insilico specializes in using generative artificial intelligence (AI) to look for new drug targets and speed up the early stages of drug-discovery before the potential therapies enter clinical trials. It was one of the first company’s in the space to propose and apply the use of generative AI to drug development, publishing a paper on the topic in 2016.
Since 2021, the company has announced 12 different preclinical therapeutic candidates in development for treatment of conditions such as cancer, Covid-19, inflammatory bowel disease, and kidney fibrosis and advanced three of them to clinical trials.
Its lead candidate and indication targets IPF and INS018_055 was the first drug designed with generative AI to enter phase 2 trials in 2023.
“This study result represents a critical milestone in AI-powered drug discovery and in my life to date,” said Alex Zhavoronkov, co-CEO of Insilico Medicine. “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.”
