Key points from article :
Insilico Medicine’s AI-designed drug, ISM001-055, shows promise for treating idiopathic pulmonary fibrosis (IPF), a progressive disease that scars lung tissue and impairs breathing. This phase 2a study involved 71 IPF patients across 21 sites in China, where participants were assigned to receive either a placebo, 30 mg of ISM001-055 once or twice daily, or 60 mg once daily over a three-month period. The study aimed to assess ISM001-055’s effectiveness in improving forced vital capacity (FVC), a key indicator of lung function that measures how much air a person can expel after a deep breath.
Top-line results indicated a dose-dependent improvement, with the 60 mg daily group showing a mean increase of 98.4 mL in FVC compared to a 62.3 mL decrease in the placebo group. This improvement suggests that ISM001-055 may help slow or even reverse the progression of lung scarring, as highlighted by Dr. Zuojun Xu, principal investigator from Peking Union Medical College. Importantly, ISM001-055 was reported as safe and well-tolerated, with most adverse events rated as mild to moderate. The most common side effects included mild diarrhea and abnormal liver function, each affecting around 14.8% of participants.
In addition to FVC improvements, the study used the Leicester Cough Questionnaire to assess quality of life, with patients in the 60 mg once-daily group reporting a notable two-point improvement in chronic cough symptoms by the end of Week 12. Although the other dose groups showed less meaningful changes, these findings reinforce ISM001-055’s potential in managing IPF symptoms. Insilico plans to present the complete findings at an upcoming medical conference and publish them in medical journals.