Insilico Medicine Reports Positive Topline Results for AI-Developed ISM001-055 in Treatment of Idiopathic Pulmonary Fibrosis (IPF)
Wednesday, November 13, 2024
Insilico Medicine has announced encouraging topline results from its Phase IIa trial of ISM001-055.
Idiopathic Pulmonary Fibrosis (IPF) is a chronic lung disease causing irreversible lung function decline, affecting around 5 million people globally. Current treatments slow progression but cannot stop or reverse the disease, underscoring the need for more effective therapies.
The drug candidate, developed in-house using AI, targets Traf2- and NCK-interacting kinase (TNIK) for the treatment of idiopathic pulmonary fibrosis (IPF). Findings from the 12-week trial indicate that ISM001-055 is safe, well-tolerated, and shows potential clinical efficacy, as evidenced by an improvement in forced vital capacity (FVC), a key measure of lung function.
The double-blind, placebo-controlled Phase IIa study (NCT05938920) enrolled 71 IPF patients across 21 sites in China.
Participants were randomly assigned to receive either a placebo or ISM001-055 at doses of 30 mg once daily (QD), 30 mg twice daily (BID), or 60 mg QD. Results showed that ISM001-055 was generally well-tolerated across all dosing groups, with most drug-related adverse events (AEs) being mild or moderate.
The most common AEs related to the drug were mild diarrhea and abnormal liver function, both occurring in 14.8% of patients.
Pharmacokinetic (PK) analysis of ISM001-055 indicated consistency with Phase I results, with a half-life of 7-12 hours and a proportional exposure increase at the highest dose.
The trial also revealed dose-dependent improvements in lung function, with the highest dose group (60 mg QD) showing a mean increase of 98.4 mL in FVC, compared to a mean decline of -62.3 mL in the placebo group.
Similarly, a mean improvement of 3.05% in percent predicted forced vital capacity (ppFVC) was observed at the highest dose, whereas the placebo group showed a decline of -1.84%.
Quality of life (QoL) and functional outcomes were also assessed via the Leicester Cough Questionnaire (LCQ) score.
Patients in the 60 mg QD group demonstrated a meaningful 2-point improvement in LCQ score by week 12, indicating a positive impact on QoL, while the other dose groups (30 mg QD and 30 mg BID) did not show significant changes.
Future trials will aim to confirm these initial findings and investigate the potential of ISM001-055 to modify disease progression in IPF, potentially offering a transformative treatment option for this progressive and often fatal disease.
Source: prnewswire.com
