Study finds AI-discovered and designed drug delivered a dose-dependent trend in lung function improvement in IPF patients.
Longevity focused AI-powered drug developer Insilico Medicine has published the results of its Phase 2a idiopathic pulmonary fibrosis (IPF) clinical trial results in Nature Medicine. The trial studied the efficacy of Rentosertib, a novel TNIK inhibitor developed using the company’s generative AI platform, in the treatment of patients with IPF.
According to Insilico, Rentosertib is the first known investigational therapy created entirely through generative AI for both its target and molecular design. Designed to treat IPF, a chronic and progressive lung disease characterized by irreversible scarring and functional decline, the drug targets TNIK (TRAF2 and NCK-interacting kinase), a key regulator in fibrotic signaling pathways, with the goal of interrupting the disease process at its root.
Having demonstrated dose-dependent improvements in lung capacity and quality-of-life scores after three months, Insilico is now initiating discussions with regulatory bodies to plan larger-scale studies of Rentosertib in broader patient populations.
“These results not only suggest that Rentosertib has a manageable safety and tolerability profile, but also warrants further investigation in larger-scale clinical trials of longer duration, demonstrating the transformative potential of AI in drug discovery and development and paving the way for faster and more innovative therapeutic advancements,” said Insilico CEO Dr Alex Zhavoronkov.
Earlier this year, Insilico secured $110 million in Series E funding, boosting its valuation above $1 billion for the first time. The company claims its AI-driven model of drug discovery delivers significant reductions in development timelines, with drug candidates typically progressing from concept to preclinical nomination within 13 months. Between 2021 and 2024, Insilico says it advanced 22 preclinical candidates in just 12 to 18 months per project. The company boasts a pipeline of around 30 drug candidates, with 10 having received FDA approval to initiate human trials, including Rentoserib.
The clinical trial, called GENESIS-IPF, was a randomized, double-blind, placebo-controlled study conducted across 22 sites in China. A total of 71 patients with IPF were enrolled and administered either a placebo or one of three Rentosertib dosing regimens: 30 mg once daily, 30 mg twice daily, or 60 mg once daily, over a 12-week period. The primary endpoint focused on safety and tolerability, while secondary outcomes evaluated efficacy through forced vital capacity measurements, the standard metric of lung function in IPF.
The study demonstrated that Rentosertib was well-tolerated across all dosing groups, with treatment-emergent adverse events comparable between arms. Most side effects were mild to moderate, and serious adverse events were infrequent and resolved upon treatment cessation. The 60 mg once-daily dose group experienced the most notable clinical benefit, showing a mean FVC increase of 98.4 mL, in contrast to a mean decline of 20.3 mL in the placebo group, indicating a dose-dependent trend in lung function improvement.
In addition to clinical outcomes, exploratory biomarker analyses were conducted to validate the drug’s mechanism of action and its biological effects. Serum protein samples were analyzed throughout the trial, revealing that high-dose Rentosertib led to reductions in fibrotic markers alongside an increase in the anti-inflammatory cytokine IL-10.
“IPF remains a highly challenging disease with significant unmet clinical needs,” said the trial’s lead investigator Dr Zuojun Xu, Professor at the Peking Union Medical College. “This study demonstrates that Rentosertib has the potential to provide meaningful clinical benefits for IPF patients, which is truly exciting. However, the sample size in each patient group was relatively limited, and these findings will need to be validated in larger cohort studies.”
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