Topline results: AI-established IPF drug well-tolerated, improves FVC

Key takeaways:

• Three differing doses of ISM001-055 were assessed against placebo.
• Each was “well-tolerated.”
• Each group also had improved FVC, but researchers reported that it was dose dependent.

Patients with idiopathic pulmonary fibrosis had improved FVC between baseline and 12 weeks with receipt of ISM001-055, a small molecule targeting Traf2- and Nck-interacting kinase, vs. placebo, according to topline results.

ISM001-055 (Insilico Medicine) was developed via generative AI, according to a press release from the manufacturer.

“I am very impressed by the positive results observed in IPF patients treated with ISM001-055, particularly the encouraging improvement in FVC,” Zuojun Xu, MD, professor of Peking Union Medical and principal investigator in the trial, said in the release. “It not only reflects ISM001-055’s potential to slow disease progression but also suggests its capability to stop or even reverse it.”

In this double-blind, placebo-controlled, randomized phase 2a study in China, Xu and colleagues assessed 71 patients with IPF to determine the impact of three differing doses of ISM001-055 — 30 mg once a day, 30 mg twice a day and 60 mg once a day — vs. placebo on lung function at 12 weeks.

Between baseline and 12 weeks, patients in the 60 mg ISM001-055 group vs. the placebo group had an improved rather than worse mean change in FVC (+98.4 mL vs. –62.3 mL), according to the release.

Each ISM001-055 group had improved FVC, but researchers reported that it was dose dependent.

In addition to FVC, the release said that the mean change in percent-predicted FVC between baseline and the 12-week mark was better among those receiving 60 mg ISM001-055 vs. placebo (+3.05% predicted vs. –1.84% predicted). This outcome was also dose dependent.

Using baseline and week 12 Leicester Cough Questionnaire total scores, researchers further observed that patients receiving 60 mg ISM001-055 vs. placebo had “a meaningful 2-point improvement” in this score, whereas this was not found in the 30 mg once daily and twice daily groups vs. placebo.

In terms of safety, the release reported that each dose of the drug was “well-tolerated,” with mostly mild or moderate drug-related adverse events. Among these patients, 14.8% experienced diarrhea, and 14.8% experienced abnormal liver function.

According to the release, Insilico stated it will do three things with this positive topline data: talk with regulatory bodies, pursue a pivotal trial and present and publish full study findings.