A UK biotech company based on UCL research has raised $33 million from investors to begin early-phase trials of a potential new treatment for people with severe epilepsy.
EpilepsyGTx, a UCL spin-out company, will use the funding to test a novel gene therapy for people with focal refractory epilepsy - a condition in which seizures arise from one specific part of the brain and do not respond to existing anti-seizure medications.
For patients with the condition, treatment options are extremely limited. Many continue to experience uncontrolled and potentially life-threatening seizures despite trying multiple drugs, and some are not suitable for current surgical approaches.
The experimental treatment, known as EPY201, is designed as a one-off therapy, rather than a drug taken daily for life. It will be delivered via surgical procedure directly to the small region of the brain where seizures begin, aiming to calm abnormal electrical activity without affecting the rest of the brain.
By targeting the seizure focus itself, the therapy seeks to stop seizures altogether without removing or damaging brain tissue. If successful, it is hoped a single procedure could leave patients seizure-free.
The investment will allow EpilepsyGTx to carry out Phase 1/2 clinical trials, marking the first time the therapy will be tested in people. These early trials will assess whether the treatment is safe and whether it can reduce or eliminate seizures.
Around 10 million people worldwide are affected by focal refractory epilepsy, making the need for new treatment approaches urgent.
The scientific research behind EPY201 was developed at UCL’s Queen Square Institute of Neurology, with support from the NIHR Biomedical Research Centre at UCLH.
UCL Business (UCLB) supported the translation of this research into a commercial spin-out company.
Co-lead of the BRC’s Translational Neuroscience theme, Prof Matthew Walker, said: “This is good news for patients. The development and use of gene therapies has been a core component of the BRC, including in our translational neuroscience theme, and the BRC has enabled the infrastructure necessary for gene therapy trials. The success of epilepsyGtx has been founded on UCL and UCLH’s joint commitment to advanced therapies.”
Prof Karl Peggs, UCLH Director of Research and Director of the NIHR UCLH BRC, said: “Congratulations to all those involved in securing this investment. Today’s announcement is validation of the importance we as a BRC have given to the development of these innovative therapies. As well as the first-in-human trials now in the pipeline - which will bring hope to patients - this is an example of how NIHR BRCs can support research which goes on to be commercialised - benefitting the wealth as well as the health of the nation.”
Image: Adobe Stock / bashkirovaphoto
