FIBROLEAD · A Fibroblast-Targeting Advanced Lead Mitigating Inflammation and Fibrosis in IBD

Inflammatory bowel disease (IBD), which comprises Crohn's disease (CD) and ulcerative colitis (UC), is a growing global health challenge affecting over 6.8 million people. Characterized by chronic inflammation leading to intestinal fibrosis, IBD significantly reduces quality of life and increases the risk of severe complications, including colon cancer. Current treatments primarily target inflammation, and fail to address the persistent activation of inflammatory fibroblast phenotypes and the resulting fibrosis, contributing to frequent relapses and a heightened risk of disease progression.To address this gap, FIBROLEAD introduces a pioneering approach by targeting fibroblasts, key mediators of chronic inflammation and fibrosis in IBD. Our recent research identified VM-313 to effectively disrupt pathogenic fibroblast activation. We now have evidence that this compound has a strong potential in mitigating both inflammation and fibrosis in vitro, ex vivo and in a mouse model of lung fibrosis. Additionally, it exhibits a favourable safety/toxicity profile and drug-like properties that are equal or superior to currently available anti-fibrotic drugs. These findings position VM-313 as a promising candidate for expanding and improving therapeutic strategies for IBD and other fibrotic diseases.This PoC project aims to develop VM-313 as an advanced lead and validate its clinical and commercial potential. Our approach includes assessing its in vivo safety and efficacy in animal models of IBD and determining its mode of action via target identification and validation studies. In parallel, we will develop a comprehensive business case, engage with key stakeholders, and conduct a commercial assessment to evaluate market entry potential. By leveraging our unique targeting strategy and the advantages of a non-invasive oral therapy, VM-313 has the potential to transform IBD treatment, delivering significant benefits to patients.