Resolution Therapeutics has announced the successful completion of a £63.5 million (US$83 million) Series B financing round, led by Syncona Ltd., to propel its lead candidate, RTX001, into clinical development. RTX001 is an engineered autologous macrophage cell therapy designed for the treatment of end-stage liver disease, a condition with limited effective therapeutic options beyond liver transplantation.
The funding will primarily support the advancement of RTX001 through the EMERALD Phase I/II clinical trial, scheduled to commence in the fourth quarter of 2024 in the UK and Spain. This trial represents a crucial step in evaluating the safety and efficacy of RTX001 as a potential first-in-class therapy for patients facing liver failure.
Beyond RTX001, Resolution Therapeutics plans to allocate resources to expand its macrophage therapy platform to address other inflammatory and fibrotic diseases, including graft-versus-host disease (GVHD) and lung fibrosis. This strategic move aims to leverage the regenerative potential of macrophages across a broader spectrum of conditions with unmet medical needs.
RTX001: A Novel Approach to Liver Regeneration
RTX001 is designed to enhance the inherent regenerative properties of macrophages, a type of white blood cell crucial in tissue repair. By engineering autologous macrophages with specific therapeutic genes, RTX001 aims to deliver increased anti-fibrotic and anti-inflammatory effects directly to the damaged liver tissue. Preclinical data suggest that RTX001 exhibits superior anti-fibrotic and anti-inflammatory advantages compared to non-engineered macrophages.
The therapy's development is rooted in the work of scientific co-founders Stuart Forbes from the University of Edinburgh and John Campbell from the Scottish National Blood Transfusion Service and the University of Glasgow and Edinburgh, who demonstrated the regenerative capabilities of macrophage cells in tissue repair following injury. Their early findings led to initial safety and Phase 2 trials in liver cirrhosis patients, paving the way for the formation of Resolution Therapeutics.
EMERALD Trial Design
The Phase I/II EMERALD trial is an open-label, first-in-human study designed to evaluate the safety and efficacy of RTX001 in patients with end-stage liver disease. The primary efficacy endpoint will measure clinical events, providing critical data on the therapy's impact on disease progression. The trial has received clinical trial authorization from the UK MHRA, signaling regulatory support for this innovative therapeutic approach.
To provide a robust control arm for the EMERALD study, Resolution Therapeutics is also conducting the OPAL study, a multi-center natural history study in patients with liver cirrhosis hospitalized for the first time with hepatic decompensation. The OPAL study aims to generate novel data on disease trajectory, enhancing the interpretability of the EMERALD trial results.
Leadership and Future Directions
Alongside the Series B financing, Resolution Therapeutics announced the appointment of Paul Sekhri as Chair of the Board of Directors. Sekhri brings over 35 years of experience in the life sciences industry, with a proven track record in drug development and business strategy. His expertise is expected to guide Resolution Therapeutics as it advances RTX001 through clinical development and expands its macrophage therapy platform.
"I am delighted to join Resolution at such an exciting time as it prepares to enter the clinic with RTX001 and expand pipeline efforts into new indications where significant unmet patient need exists," said Paul Sekhri. "I look forward to collaborating with Amir, the entire Resolution team and supporting the Company as it expands its leadership in macrophage therapy."
With the new funding and strategic leadership, Resolution Therapeutics is poised to make significant strides in unlocking the regenerative potential of macrophage therapy for end-stage liver disease and other inflammatory and fibrotic conditions.
