Insilico Medicine, a generative AI-driven, clinical-stage biotech made the announcement today, June 27.
The company says that INS018_055 is a potentially first-in-class small molecule inhibitor with a novel target discovered by its target identification engine, PandaOmics and a novel molecular structure designed by its generative chemistry engine, Chemistry42.
In February 2021, the company nominated INS018_055 as a preclinical candidate for the treatment of idiopathic pulmonary fibrosis (IPF), a chronic lung disease that results in progressive and irreversible decline in lung function, and advanced it to clinical validation in November 2021.
Positive topline data came after phase 1 trials and the US Food and Drug Administration (FDA)’s Orphan Drug Designation earlier this year (2023).
Phase 2 trials were carried out in New Zealand and China and the drug was tested in 78 and 48 healthy subjects, divided into cohorts focusing on a single ascending dose (SAD) study and multi-ascending dose (MAD) study.
The international multi-site phase 1 study yielded consistent results, demonstrating favorable safety, tolerability, and pharmacokinetics (PK) profiles of INS018_055, and supporting the initiation of the phase 2 study.
“Initiating phase 2 trials with this novel inhibitor for IPF represents a major milestone for AI drug discovery,” says Insilico Medicine founder and CEO Alex Zhavoronkov.
“We are eager to continue to advance this potentially best-in-class therapy forward to help patients in need.”
The company initiated the enrollment phase 2 clinical trial of INS018_055 in China in April 2023 and received FDA approval for a simultaneous phase 2 clinical trial in the US in June 2023. These two studies are randomized, double-blind, placebo-controlled trials to assess the safety, tolerability, pharmacokinetics, and preliminary efficacy of 12-week oral INS018_055 dosage in subjects with IPF divided into four parallel cohorts. To further evaluate the candidate in wider populations, the company plans to recruit 60 subjects with IPF at about 40 sites in both China and the US.
“With demonstrated potential against both fibrosis and inflammation, INS018_055 could offer another option for patients worldwide. The initiation of a phase 2 clinical trial for the AI-designed drug is a step forward for the AI drug discovery industry, and we are expecting more achievements powered by AI,” said Feng Ren, PhD, chief scientific officer, and co-CEO of Insilico Medicine.
Since its founding in 2014, Insilico has championed the use of generative AI in drug discovery and development. The company developed multiple approaches and new features for its generative adversarial network (GAN)-based AI platform and integrated them into the commercially available Pharma.AI platform, which includes generative biology, chemistry, and medicine. Powered by generative AI, the company is delivering breakthroughs for healthcare in multiple disease areas. Since 2021, it has nominated 12 preclinical candidates in nearly 30 internal research and development (R&D) programs and has advanced three of them to clinical validation.