USA – Insilico Medicine’s artificial intelligence-driven drug, ISM001-055, has shown encouraging early clinical results for idiopathic pulmonary fibrosis (IPF), a severe lung disease with limited treatment options.
In a recent clinical trial, the company announced improvements in patients’ breathing, sparking optimism for both Insilico and the broader AI-powered drug discovery field.
IPF, a chronic disease that causes lung scarring and irreversible decline in function, affects about 5 million people globally, predominantly older adults.
Once diagnosed, most IPF patients face a limited lifespan of three to four years. Current treatments only slow the disease’s progression, leaving an unmet need for therapies that can alter its course.
Insilico’s drug targets TNIK, an enzyme associated with the fibrotic pathways in IPF. While TNIK inhibition has previously been explored for cancer treatments, Insilico’s AI technology identified it as a viable target for fibrosis.
The small molecule ISM001-055, designed by AI, is specifically aimed at blocking TNIK to prevent lung scarring.
This breakthrough was first detailed in Nature Biotechnology earlier this year, showcasing preclinical data and findings from initial human trials.
The new data comes from a Phase 2a placebo-controlled trial in China involving 71 IPF patients, where ISM001-055’s safety and tolerability were tested alongside its effect on forced vital capacity (FVC), an indicator of lung function.
Although Insilico has not yet disclosed exact figures, the company confirmed ISM001-055 met its primary safety goal, with positive results on FVC as well.
“The drug showed a dose-dependent improvement in FVC, with the highest dose cohort experiencing the greatest improvement,” Insilico stated.
The company plans to share further data at an upcoming medical conference and intends to publish the full trial results in a peer-reviewed journal.
Following these results, Insilico is preparing to engage regulatory authorities in designing a Phase 2b trial to test extended treatment durations with larger patient groups.
The trial outcome will determine the drug’s competitive position among several advanced IPF treatments in development.
Rival treatments in the race for IPF therapies
Other companies, such as Boehringer Ingelheim and Pliant Therapeutics, have been progressing with their own potential IPF therapies.
In September, Boehringer Ingelheim’s drug, nerandomilast, hit the primary endpoint in Phase 3 trials, setting the stage for global regulatory submissions.
The drug, a PDE4B inhibitor designed to reduce inflammation, was tested in two studies—one for IPF and another for progressive pulmonary fibrosis (PPF), a related disease.
Boehringer reported that nerandomilast achieved the desired change in FVC after 52 weeks, with full safety and efficacy data expected next year.
Pliant Therapeutics’ IPF candidate, bexotegrast, takes a different approach by targeting TGF-beta, a signaling protein that increases collagen production, leading to lung stiffness in IPF patients.
According to recent Phase 2 results presented at the European Respiratory Society meeting, bexotegrast led to reduced collagen levels and improvements in both lung function and cough severity over 12 weeks.
Commenting on the findings, Leerink Partners analyst Faisal Khurshid said, “Bexotegrast’s collagen data provide compelling proof of disease modification,” noting the drug’s clean safety profile and potential as a best-in-class treatment for IPF.
Meanwhile, PureTech Health is testing a modified version of pirfenidone, an existing IPF drug, under the name LYT-100.
The modified drug aims to improve tolerability and minimize common side effects associated with pirfenidone, such as gastrointestinal issues and skin reactions.
According to Khurshid, “We have a favorable view on LYT-100 as it improves on a known anti-fibrotic drug in a manner that should diminish well-recognized safety liabilities.”
PureTech’s Phase 2b trial will compare two doses of LYT-100 to both pirfenidone and a placebo, with hopes that the improved formula can achieve better efficacy while maintaining a safe profile.
