Insilico Claims Phase 2a Success with Limited Efficacy Details in Latest AI Report

Insilico Medicine has announced the Phase 2a results of its lead program, highlighting that the study achieved its primary goal of ensuring safety and tolerability. However, the efficacy details provided were not as clear-cut. The study involved 71 patients with idiopathic pulmonary fibrosis (IPF) and examined the drug ISM001-055. According to Insilico, the drug demonstrated an improvement in forced vital capacity, a measure of breathing, compared to a placebo after 12 weeks. This improvement was dose-dependent, but the company did not disclose the absolute scores for each trial group or whether the differences were statistically significant.

In an interview, Insilico CEO Alex Zhavoronkov refrained from commenting on the statistical significance of the program, citing legal restrictions. Instead, he pointed to a trend observed by the company's researchers across various dosing groups as evidence of the drug's effectiveness. Zhavoronkov stated that the statistical significance could depend on the type of analysis used. He acknowledged that the current patient numbers were insufficient to claim statistical significance for approval. However, he noted that the data indicated a clear trend and response.

Zhavoronkov suggested that a longer trial focused on efficacy would likely show a continued dose response. While there is no set timeline for the next steps in the program, Zhavoronkov mentioned that the immediate priorities are to submit the results to a medical journal and engage with regulators.

ISM001-055 is a small molecule targeting TNIK, which stands for Traf2- and Nck-interacting kinase. Insilico claims that its AI software discovered TNIK and that inhibiting this target could stop or reverse fibrotic processes. The company is among several biotech firms using artificial intelligence to speed up drug development. The announcement comes shortly after another AI-focused company, Recursion, revealed its first Phase 2 data.

Recursion's data was also lacking in specifics, leading to a 16% drop in the company's stock price on the day of the announcement. In a trial for a rare neurological disease known as cerebral cavernous malformations (CCM), Recursion’s drug, REC-994, met safety and tolerability endpoints but had mixed efficacy results. Despite this, Recursion plans to advance the program, although it is unclear whether it will proceed with a Phase 3 study or another Phase 2 trial.

Zhavoronkov avoided making direct comparisons between Insilico’s and Recursion’s results, arguing that the program designs and diseases are different. He noted that IPF is a more heterogeneous disease compared to CCM. While expressing respect for Recursion and its CEO Chris Gibson, Zhavoronkov mentioned that some industry insiders were surprised that Recursion did not discontinue REC-994.

In summary, Insilico Medicine's Phase 2a results for ISM001-055 in IPF show promise in terms of safety and tolerability, with indications of efficacy that require further investigation. The company aims to continue its efforts by publishing the results and consulting with regulatory bodies. Meanwhile, the broader field of AI-driven drug development remains under scrutiny, as evidenced by the mixed responses to recent data from competitors like Recursion.