Comparison of the baseline clinical and HRCT characteristics of Insilico’s ISM001_055 phase 2a trial cohort in China with real-world IPF datasets

ISM001_055 was a randomised, double-blind Phase 2a trial (<ext-link>NCT05938920</ext-link>) which recruited 71 IPF patients at 21 sites in China. The current study evaluated the similarity on baseline characteristics between the trial cohort and IPF patients from different world regions in the OSIC dataset and two UK datasets. The similarity was assessed using univariate χ2- and t-tests and a multivariate kernel test. Figure 1 shows that except for sex, there are no statistically significant differences between the distribution of the <ext-link>NCT05938920</ext-link> cohort and IPF patients from South America and North America. The respective kernel p-values are 0.848 and 0.075. Filtering on age (> 40 years), FVC % predicted (> 40%) and FEV/FVC (> 0.7) did not lead to a qualitative change in the comparison. Dimensionality reduction demonstrated that the <ext-link>NCT05938920</ext-link> cohort is within the boundaries of the distributions of the two UK and the Western Europe groups (bottom row in Figure 1). Monte Carlo sub-setting of these groups in 1:1 ratio yielded external arms with no statistically significant differences from the trial cohort. These results demonstrate that <ext-link>NCT05938920</ext-link> has recruited patients whose baseline characteristics are representative of the broader IPF population. They provide promise that the positive results of <ext-link>NCT05938920</ext-link> could be replicated in an ongoing ISM001_055 trial in the USA (<ext-link>NCT05975983</ext-link>). <fig><object-id>erj;66/suppl_69/PA5063/F1</object-id><object-id>F1</object-id><object-id>F1</object-id><graphic></graphic></fig>