Insilico Medicine (“Insilico”), a clinical-stage generative artificial intelligence (AI)-driven drug discovery company, receives IND clearance from the FDA for ISM5939, a potential best-in-class oral small molecule inhibitor targeting ENPP1 announced that it had acquired the Treatment of solid tumors. This achievement marks Insilico’s 10th self-developed AI-driven molecule to be cleared to participate in clinical trials.
ENPP1 is an ectonucleotide pyrophosphatase that plays an important role in purinergic signaling that regulates the functions of the immune, cardiovascular, neurological, and blood systems. Elevated ENPP1 expression is associated with metastasis and poor prognosis in multiple tumor types. ENPP1 inhibition enhances the antitumor effects of the host immune system by modulating extracellular cGAMP levels and activating the cGAS-STING pathway.
In May 2023, Insilico Medicine announced that it had recommended ISM5939 as a preclinical candidate compound (PCC) targeting ENPP1 with potential for cancer immunotherapy. According to preclinical data, ISM5939 demonstrated strong antitumor effects in in vivo studies, while also exhibiting a favorable safety profile, in vitro ADMET, and in vivo pharmacokinetic profiles.
“We look forward to future clinical signals for ISM5939,” said Sujata Rao, MD, Chief Medical Officer, Insilico Medicine. “With favorable safety profiles and high potential for combination therapy, we are poised to significantly expand the range of treatment options for cancer patients.”
Utilizing Insilico’s proprietary generative chemistry engine Chemistry42, part of Pharma.AI, Insilico can generate clearly novel products within three months of project initiation, following dedicated optimization with structural modifications by Insilico’s medicinal chemists. We obtained a lead compound with a molecular structure.
The AI-driven drug discovery industry requires real proof-of-concept cases. Insilico has demonstrated the potential of its unique AI platform through consistent milestones in its ongoing pipeline, with 10 AI-enabled drug candidates successfully receiving IND approval to date . We look forward to further validating our internal R&D pipeline into the clinical stage and continuing to advance AI-powered drug discovery and development for the benefit of patients around the world. ”
Dr. Feng Ren, Co-CEO and Chief Scientific Officer, Insilico Medicine
In 2016, Insilico first described the concept of using generative AI to design novel molecules in a peer-reviewed journal, which laid the foundation for the commercially available Pharma.AI platform. Since then, Insilico continues to integrate technological breakthroughs into the Pharma.AI platform. The Pharma.AI platform is now a generative AI-powered solution spanning biology, chemistry, drug development, and scientific research. Powered by Pharma.AI, Insilico has named 20 preclinical candidates and received IND approval for 10 molecules since 2021 across a comprehensive portfolio of over 30 assets.
In early 2024, Insilico will complete the entire R&D process from AI algorithms to Phase II clinical trials for ISM001_055, the company’s flagship drug pipeline with AI-discovered targets and AI-designed structures. We published a paper in Nature Biotechnology showing the results. Following this, Insilico recently announced positive preliminary results from a Phase IIa study (NCT05938920). In this study, ISM001_055 demonstrated good safety and tolerability across all dose levels, as well as a dose-dependent response in forced vital capacity (FVC) after just 12 weeks. of dosage.
