Insilico Medicine has announced positive topline results from its Phase 2a trial of ISM001-055 for the treatment of idiopathic pulmonary fibrosis (IPF). The study (NCT05938920) demonstrated the tolerability, safety, and efficacy potential of the drug, with patients showing improvements in forced vital capacity (FVC) after 12 weeks.
ISM001-055: Targeting TNIK with AI
ISM001-055 was developed using Insilico Medicine’s generative artificial intelligence (AI) platform and is designed to target Traf2- and NCK-interacting kinase (TNIK). According to Zuojun Xu, MD, professor at Peking Union Medical College and principal investigator, AI is playing a crucial role in drug discovery and clinical research, offering real clinical benefits to patients.
Phase 2a Trial Details
The Phase 2a study enrolled 71 patients with IPF across 21 sites in China. The double-blind, placebo-controlled trial randomized participants to receive either 30 mg once daily (QD), 30 mg twice daily, or 60 mg QD for 12 weeks.
Key Findings
Notable findings from the trial indicated that ISM001-055 was well-tolerated across all dosage levels. The most common adverse events (AEs) were diarrhea (14.8%) and abnormal liver function (14.8%), with the majority of AEs classified as mild to moderate.
Researchers observed dose-dependent patient responses, with the 60 mg QD group showing an average FVC improvement of 98.4 mL at 12 weeks, compared to an average FVC decline of -62.3 mL in the placebo group. A 2% to 6% decline in FVC is considered clinically significant in IPF patients.
Quality of Life Improvements
The Leicester Cough Questionnaire was used to assess quality of life. Patients in the 60 mg QD group reported a meaningful improvement of 2 points from baseline to week 12, while other groups did not show significant improvements, further supporting the dose-dependent nature of ISM001-055.
Expert Commentary
Carol Satler, MD, PhD, vice president of clinical development at Insilico Medicine, highlighted the potential of ISM001-055 to modify the disease, suggesting it may play a role in preventing IPF progression and even promoting disease regression. She emphasized the value of future studies to further demonstrate ISM001-055's potential in transforming the IPF treatment landscape.
