University of Edinburgh spinout Trogenix has announced that the first patient has been dosed in a Phase I/II clinical trial to evaluate its novel gene therapy platform targeting brain cancer.
It will treat patients with newly diagnosed or recurrent glioblastoma (GBM), one of the most common and aggressive forms of brain cancer. The trial, named ADePT, is being conducted at two leading clinical sites: NHS Lothian, Edinburgh, and Ohio State University Hospital, USA.
The trial design will identify the optimal biological dose of TGX-007 - a dual-payload adeno-associated virus (AAV)-based gene therapy - and evaluate its safety and efficacy in newly diagnosed and recurrent GBM patients. It will generate clear and early insights into biological activity, tumour selectivity, safety, target engagement, immune activation and overall survival in GBM patients.
The initial clinical data will immediately drive the Company’s broader vision to enable testing of a potentially transformational treatment approach across many different solid tumours.
Ken Macnamara, Chief Executive Officer of Trogenix, commented:
We’re excited to advance the ADePT clinical trial and bring a potentially transformative “one time” treatment to patients with significant unmet need.
With precisely controlled therapeutic delivery, a strong safety profile, and support from US and UK regulators, we are positioned to treat newly diagnosed and recurrent glioblastoma patients ahead of standard-of-care.
The trial will provide meaningful clinical endpoints for high-grade gliomas and inform development across our broader solid tumour pipeline. We are grateful to the patients, investigators and clinical teams for making this possible and look forward to reporting data from the trial in due course. ”
TGX-007 harnesses Trogenix's proprietary Synthetic Super-Enhancer (SSE) technology to exploit the core cancer cell identity. In doing so, the programmable immunotherapy can switch on combination payloads to precisely kill tumour cells and activate the immune response.
The dual mechanism of action, delivered via a single vector, creates synergistic effects that act as an in-situ vaccination that provides long lasting protection from a “one time” treatment.
The first patient dosing in the ADePT trial follows the publication in Nature in April of a breakthrough pre-clinical study led by Professor Steve Pollard of the University of Edinburgh’s Institute for Regeneration and Repair, Co-founder and Chief Scientific Officer of Trogenix, demonstrating that a single dose of SSE-based therapy achieved complete tumour eradication in 83% of treated cases in an aggressive brain cancer model that closely mimics human GBM, with no toxicity over 11 months and no tumour recurrence.
Dr Andrea Taylor, CEO of Edinburgh Innovations, which supported the translation of research and company formation for Trogenix, which span out in late 2023 including with seed funding from Old College Capital, the University’s in-house venture investment fund, said:
It is truly fantastic to see Trogenix charging forward with so much success already, and we are all watching the trial with cautious hope and excitement. ”
Faye Robertson, Chief Investigator, Consultant Clinical Oncologist and Honorary Clinical Senior Lecturer University of Edinburgh, added:
Despite decades of research, outcomes for patients with glioblastoma remain extremely poor, underscoring the urgent need for innovative therapeutic approaches.
The start of the Phase I/II ADePT trial is a critical moment for patients facing this challenging and devastating disease. It is the culmination of more than a decade of research in pursuit of a therapy that delivers dual therapeutic payloads with exceptional precision directly to glioblastoma cells, while sparing surrounding healthy tissue.
We are delighted to have treated the first patient in Edinburgh and look forward to progressing ADePT to determine whether this approach translates into meaningful clinical benefit for patients. ”
The ADePT trial will establish the clinical proof-of-concept that will guide the Company’s broader pipeline development across colorectal, liver, and lung cancers.
Further details of the ADePT trial can be found here: NCT07346144
