Insilico Medicine, a company that leverages artificial intelligence (AI) technology for drug discovery, has developed a drug candidate named ISM001-055 aimed at treating idiopathic pulmonary fibrosis (IPF), a chronic lung disorder that causes irreversible scarring and declines in lung function. The disease, affecting around 5 million people worldwide, primarily older adults, sees a median survival of three to four years post-diagnosis. Current treatments only slow its progression without altering the disease course.
ISM001-055 targets Traf2- and Nck-interacting kinase (TNIK), an enzyme involved in signaling pathways linked to disease processes, including fibrosis. Though TNIK inhibition was initially explored in cancer therapy, Insilico’s AI identified it as a viable target for IPF. Utilizing generative AI technology, the company designed the small molecule now undergoing clinical trials.
Published details in Nature Biotechnology in March concerned the drug’s development process, highlighting preclinical data and outcomes from Phase 0 and Phase 1 studies. Recently, results from a Phase 2a study conducted in China involving 71 IPF patients were announced. Patients received various dosages or a placebo, with the study’s primary aim being to assess the drug’s safety and tolerability across all doses over 12 weeks. A secondary endpoint measured improvement in forced vital capacity (FVC), a metric indicating the volume of air a person can forcibly exhale.
Though specific data hasn’t been fully disclosed, Insilico reported that ISM001-055 met the primary safety endpoint and displayed a dose-dependent improvement in FVC, particularly prominent in those administered the highest dosage. Full details are expected to be presented at an upcoming medical conference and later published in a peer-reviewed journal. Additionally, a parallel Phase 2 study is currently recruiting in the U.S.
Encouraged by these outcomes, Insilico is planning further discussions with regulatory bodies to design a Phase 2b study that would explore longer treatment durations and involve a larger patient group. Despite these advances, Insilico remains behind some competitors in the IPF drug development landscape.
One notable competitor is Boehringer Ingelheim, whose IPF drug candidate, nerandomilast, aims to inhibit phosphodiesterase 4B (PDE4B). This drug recently achieved its primary goals in pivotal studies, setting the stage for regulatory submissions. Full efficacy and safety data are slated for release next year.
Another competitor, Pliant Therapeutics, has developed bexotegrast, targeting TGF-beta, a protein contributing to IPF by fostering high collagen levels which degrade lung elasticity and function. Recent Phase 2 results presented at the European Respiratory Society meeting showed that bexotegrast reduced collagen and improved lung function and cough severity over 12 weeks, compared to a placebo group.
PureTech Health’s approach adapts the old IPF drug pirfenidone, known commercially as Esbriet, which, despite its efficacy, often leads patients to discontinue use due to its side effects. PureTech’s version, LYT-100, incorporates chemical modifications aimed at improving tolerability. Current Phase 2b studies are comparing two doses of LYT-100 against pirfenidone and a placebo, hoping to reveal safety improvements and potentially greater efficacy at higher dosages.
Together, these developments represent significant progress in the treatment of IPF, an area of high unmet medical need. Each new investigational drug brings hope of better management options for patients suffering from this debilitating disease.
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