• After completing the Phase 0 and Phase I human safety studies, INS018_055, Insilico's AI-discovered and AI-generated drug, has entered multi-regional Phase II clinical trials in the U.S and China.
• The first human patients were dosed with the drug.
• This is a potentially first-in-class anti-fibrotic drug candidate that has reached clinical Phase II, and it is fully delivered by generative AI, with a novel AI-discovered target and a novel AI-generated molecular structure.
• Powered by its integrated commercially available AI platform, Pharma.AI, Insilico has nominated and announced 12 preclinical candidates for internal drug discovery programs since 2021 and 3 of them have advanced into human clinical trials

The study is a randomized, double-blind, placebo-controlled trial to assess the safety, tolerability, pharmacokinetics and preliminary efficacy of 12-week oral INS018_055 dosage in subjects with IPF divided into 4 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 the U.S and China.

Previously, INS018_055 received positive topline data in Phase I in early 2023. In Phase I trials carried out in New Zealand and China, INS018_055 was tested in 78 and 48 healthy subjects, divided into cohorts focusing on a single ascending dose (SAD) study and multiple ascending dose (MAD) study. The international multi-sites Phase I studies yielded consistent results, demonstrating favorable safety, tolerability, and pharmacokinetics (PK) profiles of INS018_055, supporting the initiation of the Phase II study.

"With demonstrated potential against both fibrosis and inflammation, INS018_055 could offer another option for patients worldwide," said Feng Ren, PhD, co-CEO and Chief Scientific Officer of Insilico Medicine. "The achievement of the first dose for INS018_055 in the Phase II clinical trial is not only an important step for Insilico, but also a milestone for AI-driven drug discovery and development. Together, we are expecting more achievements powered by AI for global unmet medical needs."

"When we started developing generative AI for drug discovery, I never expected to see the clinical and preclinical results we have today," said Alex Zhavoronkov, PhD, Founder and co-CEO of Insilico Medicine. "Initiating Phase II trials with this novel inhibitor for IPF represents a major milestone for deep generative reinforcement learning in drug discovery. We will explore the efficacy for patients of AI-discovered and designed treatments in clinical trials, which is a true validation of our generative AI platform. We are eager to continue to advance this potentially first-in-class therapy forward to help patients in need and show the value of generative AI in drug discovery and development."

Insilico is one of the pioneers in utilizing generative AI for drug discovery and development. The company first described the concept of using generative AI for the design of novel molecules in a peer-reviewed journal in 2016. Then, Insilico developed and validated multiple approaches and features for its generative adversarial network (GAN)-based AI platform and integrated those algorithms into the commercially available Pharma.AI platform, which includes generative biology, chemistry, and medicine.

Moreover, Insilico Medicine launched its target discovery AI platform PandaOmics, generative chemistry AI platform Chemistry42, and clinical trial analysis platform inClinico commercially to enable the biotechnology and the pharmaceutical industry to utilize the advanced generative AI tools to power their internal research and development. Powered by generative AI, Insilico is delivering breakthroughs for healthcare in multiple disease areas. Since 2021, Insilico has nominated and announced 12 preclinical candidates in its comprehensive portfolio of over 30 assets and has advanced 3 of them to the clinical stage.