In a significant breakthrough for artificial intelligence in healthcare, Insilico Medicine's AI-designed drug Rentosertib has demonstrated promising efficacy and safety in treating idiopathic pulmonary fibrosis (IPF), a chronic lung disease affecting approximately five million people worldwide.
Rentosertib, previously known as ISM001-055, is the first investigational drug where both the biological target and the therapeutic compound were discovered using generative AI technology. The drug works by inhibiting TNIK (TRAF2 and NCK-interacting kinase), a novel target identified by Insilico's AI platform PandaOmics as playing a crucial role in lung fibrosis development.
In the recently completed Phase IIa clinical trial, patients receiving the highest dose (60mg daily) of Rentosertib experienced a mean improvement of 98.4 mL in forced vital capacity (FVC), while the placebo group showed a mean decline of 20.3 mL. These results, published in Nature Medicine on June 3, 2025, and presented at the American Thoracic Society International Conference, mark the industry's first proof-of-concept clinical validation of an AI-driven drug discovery approach.
The development of Rentosertib showcases the remarkable efficiency of AI in pharmaceutical research. Using its Pharma.AI platform, Insilico compressed the traditional drug discovery timeline from 2.5-4 years to just 18 months, while testing only 60-200 molecules compared to the thousands typically required in conventional approaches.
With these encouraging results, Insilico Medicine has begun discussions with regulatory authorities to initiate larger pivotal trials. If successful, Rentosertib could become the first AI-discovered therapy to reach patients, potentially transforming treatment options for IPF, which currently has only two FDA-approved therapies (Pirfenidone and Nintedanib).
"These results demonstrate both safety and encouraging efficacy, warranting larger and longer studies," stated Alex Zhavoronkov, PhD, Founder and CEO of Insilico Medicine. "This represents a paradigm shift, underscoring AI's transformative potential to unlock therapies faster and at lower costs."