Spark to use CombiGene therapy to target focal epilepsy

Spark Therapeutics has secured the exclusive rights to CombiGene’s CG01 therapy, which aims to treat drug resistant focal epilepsy, in a deal worth $8.5m in advance with further payments due on reaching clinical milestones.

The news sent Swedish firm CombiGene’s shares soaring – almost doubling from SEK6.29 to SEK14.10 the following day, when the development was announced this week. CombiGene could receive up to $328.5 million excluding royalties and up to $50 million at preclinical and clinical milestones. When it is commercialised, the firm could reap royalties, depending on net sales.

CG01, which is at late pre-clinical stage, was jointly developed by CombiGene and its partners Cobra Biologics and Viralgen. CombiGene states that CG01 satisfies all quality requirements and “is scalable to meet all future needs, from limited volumes for preclinical and clinical trials to full commercial production.”

Focal epilepsy causes recurring seizures that affect one hemisphere of the brain. While the condition can be treated with medication, diet, nerve stimulation or surgery, these measures are not always successful and focal epilepsy remains a major global medical problem for drug-resistant patients.

There are “approximately 47,000 drug-resistant patients with focal epilepsy estimated to be added each year in the US, EU, UK, Japan, and China,” according to CombiGene.

Joseph La Barge, chief business officer, Spark Therapeutics, which is a member of the Roche Group, said: “We were impressed by CombiGene’s gene therapy platform and look forward to harnessing our collective expertise to tackle diseases that affect signalling in the central nervous system.”

Calling the deal momentous, Jan Nilsson, chief executive officer, CombiGene, praised the contribution of the EU Horizon 2020 programme, which aims to ensure that Europe produces world-class science, removes barriers to innovation and makes it easier for public and private sectors to work together.

Nilsson added: “Our epilepsy project CG01 has continued to show strength throughout the preclinical phase, and in Spark we have now found a perfect partner to take CG01 through the clinical phase to full commercialisation.

“CombiGene and Spark have had a productive ongoing dialogue during the latter parts of CG01’s preclinical development, and the entire CombiGene team have come to know Spark as a visionary and patient-focused organisation with the strength, know-how, and experience to exploit the full potential of CG01. We look forward to advancing this potentially transformative therapy together with Spark for the benefit of a patient group in need of better treatments.”