In a small Phase IIa trial, Insilico’s generative AI-designed idiopathic pulmonary fibrosis drug improved lung function and was well-tolerated across all dosing groups.
For the second time in two months, Insilico Medicine reported positive results from a mid-stage trial of its generative AI-developed idiopathic pulmonary fibrosis drug candidate.
Tuesday, Insilico announced positive topline results from a Phase IIa trial of ISM001-055, designed using the company’s generative AI engine to treat idiopathic pulmonary fibrosis (IPF), a progressive interstitial lung disease disproportionately affecting older adults.
The trial, which enrolled 71 people with IPF across 21 sites in China, showed a dose-dependent improvement in lung function across all doses (30 mg once daily, 30 mg twice daily and 60 mg once daily) for patients treated with ISM001-055 at 12 weeks, according to Insilico. Mean improvement in forced vital capacity (FVC) from baseline at the 60-mg dose was 98.4 mL compared to a mean decline in FVC change from baseline of -62.3 mL for placebo patients.
The improvement in FVC is notable, as no currently available treatments for IPF have achieved this objective. There is currently no cure for IPF and current treatment is designed to minimize symptoms, maintain quality-of-life and slow progression, Allison Reiss, an associate professor at NYU Grossman Long Island School of Medicine, previously told BioSpace.
Insilico’s candidate was well-tolerated across all dosing groups, according to the company, with the majority of drug-related adverse events being mild or moderate in severity. The most common adverse events were diarrhea and abnormal liver function.
“We are thrilled with the positive results from this Phase IIa clinical trial, which underscore the potential of generative AI and robotics to facilitate the discovery, design, and development of innovative therapies and improve patient outcomes,” Alex Zhavoronkov, founder and CEO of Insilico, said in a statement.
Insilico targets Traf2- and NCK- interacting kinase (TNIK). Zhavoronkov previously told BioSpace that due to the prevalence of IPF in people 65 years and older, Insilico was looking for targets that were also “very strongly implicated” in aging. TNIK scored high in six hallmarks of aging, and there was significant overlap in hallmarks of IPF, he said.
When ISM001-055 entered Phase II trials in August 2023, it became “the first drug discovered and developed using generative AI to have reached this clinical stage of development,” Sujata Rao, chief medical officer at Insilico, told BioSpace at the time.
Tuesday’s positive results follow those of a parallel Phase IIa trial. In September, Insilico reported that ISM001-055 met the primary endpoint of safety and secondary efficacy endpoints in that study, demonstrating a dose-dependent improvement in FVC over 12 weeks.
Insilico is far from alone in the IPF space. Also in September, Boehringer Ingelheim announced that its IPF candidate nerandomilast elicited a significant improvement in FVC at 52 weeks versus placebo in a much larger Phase III trial. Based on these results, Boehringer plans to file for regulatory approval of nerandomilast with the FDA and other health authorities, according to a press release issued at the time. Meanwhile, La Jolla, Calif.–based GRI Bio kicked off a Phase IIa biomarker study in December 2023 evaluating GRI-0621, a type 1 invariant NKT antagonist, for IPF.
