Insilico Medicine („Insilico“), an AI-based drug discovery company, announced today the nomination of ISM8001, a novel molecule targeting FGFR2/3, for the treatment of tissue-agnostic solid tumors. FGFR2/3 play a crucial role in various biological processes and have been linked to several types of solid tumors, making them potential targets for the treatment of tissue-agnostic solid tumors. However, existing pan-inhibitors face limitations due to adverse events, suboptimal responses, and acquired drug resistance.
Clinical cancer research reports FGFR aberrations in approximately 7.1% of cancer patients. Currently, pan-inhibitors are only approved for UC and ICC indications. By utilizing our revolutionary AI platform, Insilico has developed a unique molecular design strategy to address current challenges, maintaining desired profiles and overcoming the drawbacks of previous generations for a broader population with FGFR2/3 aberrations.”
Feng Ren, PhD, Co-CEO, and Chief Scientific Officer of Insilico Medicine
Insilico is generating a series of novel molecules with desired profiles and, with the support of Chemistry42, delivering the new generation of covalent FGFR inhibitors. ISM8001 is an orally available, highly selective dual inhibitor targeting FGFR2/3 while sparing FGFR1/4. The compound showed superior efficacy in several FGFR2/3-driven efficacy models, as well as gatekeeper and molecular brake mutant-resistant models. It also demonstrated favorable DMPK profiles, a low effective dose, and a desirable safety margin to support higher dosing in humans.
“We expect to collaborate with experienced partners to advance this program to the next milestone,” said Alex Zhavoronkov, PhD, Founder, and Co-CEO of Insilico Medicine. “The nomination of ISM8001 clearly validates the capabilities of Pharma.AI, Insilico’s integrated AI platform. We are committed to continuously improving and expanding cutting-edge technologies and AI algorithms, with the ultimate goal of advancing drug research and meeting unmet clinical needs.”
Driven by generative artificial intelligence, Insilico is at the forefront of artificial intelligence and cutting-edge technologies. In 2023, with the support of Pharma.AI, the company achieved six nominations for preclinical candidates (PCCs), all exclusively owned by the company, bringing the total number of PCCs nominated since 2021 to 17. The company has advanced one program to phase-2 trials, while the most advanced is an anti-fibrosis program currently in phase-1 studies.