Insilico Medicine’s AI Drug Shows Promise for Idiopathic Pulmonary Fibrosis in New Trial
Insilico Medicine has announced positive topline results from its Phase IIa clinical trial of ISM001-055, a novel treatment for idiopathic pulmonary fibrosis (IPF) developed using generative AI. The drug, designed to target the TNIK protein, which plays a key role in cell signalling linked to the disease, proved to be safe, showed a good pharmacokinetics profile, and delivered clear dose-dependent improvements in lung function during the trial.
Dr. Zuojun Xu, principal investigator of the trial and a professor at Peking Union Medical College, described the findings as significant.
“I am very impressed by the positive results observed in IPF patients treated with ISM001-055, particularly the encouraging improvement in FVC. It not only reflects ISM001-055’s potential to slow disease progression but also suggests its capability to stop or even reverse it,” he said.
“AI, as an advanced technology, is already playing a crucial role in many aspects of medical practice, including drug discovery and clinical research, and we expect to see the real clinical benefits it brings to patients.”
A trial across 21 sites in China tested ISM001-055 in 71 patients with idiopathic pulmonary fibrosis, a serious lung disease. Patients received either a placebo or one of three doses of ISM001-055.
The results showed that the highest dose, 60 mg once daily, improved lung function by 98.4 mL in forced vital capacity, compared to a decline of 62.3 mL in the placebo group. This group also showed a 3.05% improvement in another measure of lung function, compared to a 1.84% decline in the placebo group.
Patients on the highest dose reported better quality of life, with a 2-point improvement in the Leicester Cough Questionnaire. However, the other two doses, 30 mg QD and 30 mg BID, showed no meaningful improvement.
Most side effects were mild or moderate, with diarrhoea and liver issues being the most common.
The drug’s behavior in the body also matched earlier studies, staying active for 7 to 12 hours, with stronger effects at the higher dose.
Following the promising results, Insilico Medicine has appointed Dr. Carol Satler as vice president of clinical development to lead the advancement of its non-oncology programs.
With over 20 years of experience in drug discovery, development, and strategic planning, Satler will play a “pivotal role” in further validating ISM001-055, the company’s leading program.
Describing the results as “highly promising,” Satler added: “The dose-dependent improvement in FVC in IPF patients treated with ISM001-055 compared to those treated with placebo suggests ISM001-055 may play a role in both the prevention of progression of IPF as well as disease regression, thus highlighting the disease-modifying potential.”
“Future studies will help delineate these benefits further with the hope that ISM001-055 may transform the current treatment paradigm and translate to better overall outcomes for patients with idiopathic pulmonary fibrosis, a progressive fatal debilitating disease.”
Moving forward, Insilico Medicine is actively enrolling patients for its Phase IIa clinical study in the U.S.
“These results are very encouraging, particularly the dose-dependent response in FVC,” said Toby Maher, principal investigator of the U.S. clinical trial and expert in interstitial lung disease.
“IPF is a devastating disease, and seeing improvements in lung function over just 12 weeks of treatment is a promising indication that ISM001-055 may provide a new therapeutic option for patients.”
The news comes as Insilico Medicine also reached a recent major milestone in its research collaboration with Sanofi, developing an AI-powered lead candidate with first-in-class potential to target an “undruggable” transcription factor involved in cancer.