The Edinburgh-based biotech firm demonstrates complete tumour eradication and durable protection in an aggressive brain cancer model.

Edinburgh-based cancer biotech company Trogenix has demonstrated complete tumour eradication and durable protection in an aggressive brain cancer model that closely mimics human glioblastoma (GBM).

The research supports an innovative approach to potentially achieving curative responses with long-term immunological protection against GBM, the most lethal form of brain cancer. It underpins, the company says, its transition to a clinical-stage oncology company, with patient dosing in its first clinical trial for glioblastoma expected by the end of the second quarter.

In October last year, the firm raised £70 million in Series A funding to help develop its pipeline of potentially curative cancer therapies across multiple aggressive solid tumours into the clinic. To date, the company has raised £85 million in funding, according to Crunchbase. It last raised £15 million in seed funding in January 2024.

“This pre-clinical work in an aggressive brain cancer model that closely mimics human glioblastoma has achieved what we thought impossible – complete tumour elimination and long-lasting protection against cancer recurrence without off-target toxicity using a single dose of a single agent,” said Steve Pollard, study lead at the University of Edinburgh and chief scientific officer of Trogenix.

Striking tumour regression

The findings focus on Trogenix’s proprietary Synthetic Super-Enhancers (SSEs), which are engineered genetic constructs that act as selective transcriptional switches, designed to harness glioblastoma stem cells’ own unique transcriptional machinery. The SSEs, delivered using adeno-associated virus vectors (AAVs), specifically target SOX2 and SOX9-driven gene networks that are activated in patient glioblastoma stem cells.

They showed that, when delivered as a single treatment dose, SSEs led to striking tumour regression within a couple of weeks, and complete tumour clearance in 83% of treated cases over the subsequent two to three weeks. More to the point, there was no further tumour regrowth after the initial treatment, with no toxicity over the next 11 months, and no detectable tumour formation even after re-challenge.

Critically, SSE activity was validated using fresh patient GBM tissue samples, demonstrating selective expression in tumour cells but not in the healthy normal brain cells. This precision targeting of glioblastoma cells while sparing normal tissue minimises off-target toxicities and represents a key validation step before advancing to patient trials.

Iain Foulkes, chief executive of Cancer Research Horizons, pointed out that around 3,200 people are diagnosed with glioblastoma every year in the UK, of which just 160 will survive for five years or more.

“That number is unacceptable and we urgently need better treatment options. This work lays the foundation for Trogenix’s next steps into early-stage clinical trials, steps that will hopefully take us closer to a world where fewer people lose their lives to brain cancer,” he added.