Resolution Therapeutics has achieved a significant milestone in treating end-stage liver disease by successfully dosing the first patient with RTX001, a first-in-class engineered Regenerative Macrophage Therapy (RMT), in its Phase I/II EMERALD study. The patient, dosed at the Royal Infirmary of Edinburgh, has successfully cleared the safety period with no serious adverse events, marking the first-in-human administration of an engineered RMT for patients with advanced liver fibrosis.
Novel Therapeutic Approach for Critical Unmet Need
RTX001 represents a breakthrough in addressing end-stage liver disease, a condition characterized by high mortality and severely limited therapeutic options. As Dr. Amir Hefni, Chief Executive Officer of Resolution Therapeutics, explained: "The first patient dosed represents a significant milestone in advancing RTX001 as a potential first-in-class treatment option for patients with end-stage liver disease, a condition with high mortality and severely limited therapeutic options."
The therapy addresses a critical gap in liver disease treatment. Professor Jonathan Fallowfield, Chair of Translational Liver Research and Principal Investigator at the University of Edinburgh and Chief Investigator of the EMERALD study, noted: "Most liver-related hospitalisations and deaths result from decompensated cirrhosis, yet there are no licensed therapies that stabilise or reverse disease at this late stage. Liver transplantation is effective but limited by donor availability, cost, and suitability for only a small subset of patients."
Enhanced Engineering for Superior Efficacy
RTX001 has been specifically engineered with interleukin-10 (IL-10) and matrix metalloproteinase-9 (MMP-9) to enhance the anti-inflammatory and anti-fibrotic effects of RMT. This engineering aims to enable improved patient outcomes and longevity of effect in end-stage liver disease compared to non-engineered approaches.
The development builds on promising results from the academic MATCH Phase I and II studies, where non-engineered RMT demonstrated good tolerability with transformational efficacy in advanced liver cirrhosis. RTX001 has been developed as a commercial product with enhanced properties designed to deliver superior therapeutic benefits.
Study Design and Patient Impact
The Phase I/II EMERALD study is a multi-center, single-arm, first-in-human, open-label study designed to evaluate the safety and efficacy of RTX001 in patients with end-stage liver disease who have recovered from a recent hepatic decompensation. The study will enroll up to 25 patients across leading hepatology centers in the UK and Spain, with initial data readout expected in 2026.
Dr. Vijay Shah, a world-leading hepatologist and Resolution Therapeutics Advisory Board Member, emphasized the potential patient impact: "If this treatment can help stabilise liver disease for the long term and reduce the risk of clinical events, it could be an incredible benefit for so many patients. RTX001 may give patients suffering from decompensated cirrhosis the ability to live longer and potentially improve quality of life."
Addressing a Major Health Burden
The significance of this development is underscored by the substantial disease burden. Vanessa Hebditch, Director of Communications & Policy at the British Liver Trust, highlighted: "More than three-quarters of people are diagnosed with cirrhosis when it's too late for effective treatment, contributing to more than 11,000 deaths per year in the UK. For patients and their families who currently have very limited treatment options, we are really encouraged by the progress in advancing RTX001 as a novel treatment for liver disease."
Professor Fallowfield expressed optimism about the therapy's potential: "Our hope is that RTX001 can help prevent further clinical events in patients with decompensated cirrhosis, reducing the risk of further hospitalisations and delaying or avoiding the need for a transplant."
Broader Pipeline Development
Beyond liver disease, Resolution Therapeutics is developing RMT for additional fibrotic and inflammatory diseases with significant commercial opportunity. The company's initial focus includes Graft-versus-Host Disease (GvHD) and lung fibrosis, demonstrating the potential versatility of the regenerative macrophage therapy platform.
The EMERALD study represents a pivotal step in validating this novel therapeutic approach, with the potential to transform treatment options for patients facing one of the most challenging stages of liver disease.
