CombiGene AB (publ) (“CombiGene”, the “Company”) today announces preliminary results from completed analyzes of the latest preclinical studies for the epilepsy project CG01. The results derive from two studies in the backup program initiated by the company's former partner and confirm a significant antiepileptic effect in a preclinical model compared to the control, as well as an administration method that looks promising for future human studies.

The backup program, another vector than AAV1 was tested in a preclinical genetic mouse model of temporal lobe epilepsy (TLE). The results show antiepileptic effects, although a significant increase in body weight was noted. Furthermore, the company has received results from a preclinical administration study performed with a commercially available catheter system that shows expression of the vector with adequate coverage of the hippocampus, the part of the brain intended to be treated. However, the current vector is not covered by CombiGene’s current patent families for CG01.

“We are pleased with the results showing a 50 percent seizure freedom after gene therapy with NPY and Y2 in yet another TLE model, as well as good coverage of the target area. The main findings from these two studies validate CombiGene's overall treatment concept. However, the side effect profile, including the noted weight gain, needs to be addressed. We now have the opportunity to present this new data to potential partners. Over the next few weeks, the management and board will evaluate the impact of the recent results on the company's overall strategy and outlook,” comments CombiGene's CEO Peter Ekolind.

In the project CG01, CombiGene has for a long time conducted research and development of a new gene therapy treatment for drug-resistant epilepsy. The drug candidate involves a construct of two genes, NPY and NPY receptor Y2, which are inserted into an AAV1 vector. The future drug is intended to be administered into the brain once, to offer a long-term or lifelong effect. After a period of collaboration with another company, CombiGene today owns all rights to the project. During the current year, CombiGene has been awaiting results from two animal experimental studies; an effect study and an administration study and has now obtained preliminary data.

About the epilepsy project CG01
CombiGene’s gene therapy candidate is aimed at a large patient population to solve an unmet need in epilepsy treatment. Epilepsy is a major global medical problem with approximately 47,000 drug-resistant patients with focal epilepsy estimated to be added each year in the US, EU4, UK, Japan, and China. CG01 is in preclinical stage.