Accure Therapeutics' drug candidate can slow epilepsy progression, study shows

Accure Therapeutics, an R&D pharmaceutical company in the central nervous system (CNS) field, has announced the publication of a pivotal preclinical efficacy study showing that its new drug candidate ACT-03 (formerly IPR-179) is the first candidate able to slow the progression of epilepsy.

The study results, published in the January issue of the peer-reviewed Journal of Clinical Investigation (JCI), show that the drug, when administered in animal models over one week, can modulate epileptogenesis by reducing the severity and number of spontaneous seizures, up to seven weeks after initial drug administration. It was also shown to prevent cognitive decline – one of the main comorbidities associated with epilepsy.

Approximately 65 million people worldwide are living with epilepsy. Commercially available anti-epileptic drugs focus on seizures, a key symptom of the disease, but are yet to show anti-epileptogenic or disease modifying activity. ACT-03 is the first drug candidate that has been shown to slow the progression of epilepsy, and prevent the cognitive decline associated with the condition.

Research into the treatment was carried out on an international scale in collaboration with Dr Erwin Van Vliet and Dr Eleonora Aronica of the Department of NeuroPathology at the University of Amsterdam, and Dr Alexander Dityatev of the molecular neuroplasticity group at the German Center for Neurodegenerative Diseases. The study received funding from the European Union’s Horizon 2020 Research and Innovation Programme, the European Union’s Seventh Framework Programme, and the Dutch Epilepsy Foundation.

After screening more than 100,000 compounds, Accure Therapeutics developed ACT-03, a selective and blood-brain barrier permeable matrix metalloproteinase inhibitor. ACT-03 tackles remodeling of the extracellular matrix that takes place after a brain injury.

Laurent Nguyen, CEO at Accure Therapeutics, said: “The anti-epileptogenic capacity of our drug candidate ACT-03 paves the way for a new pharmacological treatment for epilepsy. It represents a significant first-in-class innovation in the development of disease modifying treatments for epilepsy.”

Professor Lawrence J Hirsch, Director of the Epilepsy Center at Yale University, and member of the scientific advisory board of Accure Therapeutics, commented: “This preclinical study is very interesting in that it shows that ACT-03 is able to slow the progression of the disease.

“If this is confirmed in humans, it will be of major importance for people who live with refractory epilepsy or those who have rare epileptic syndromes. Patients with recent onset of the disease could also benefit, as well as those with a high risk of developing epilepsy in the near future – for example, patients who have suffered severe traumatic brain injury or intracerebral haemorrhage.”

Darcy Jimenez