CombiGene has concluded the planned pharmacokinetic study in the CG01 epilepsy project with promising results.

Mar 20, 2020

CombiGene recently concluded the preclinical pharmacokinetic study in the CG01 project. The outcome of the study is very promising and means that CombiGene now has the answers to key questions with respect to the long-term expression of NPY and Y2.

Successful pharmacokinetic study confirms that CG01 creates long-term expression of NPY and Y2
CombiGene’s candidate drug, CG01, is being developed for treatment of patients with drug-resistant focal epilepsy. The aim is to enable a very long-term therapeutic effect through a one-time administration. To determine how the body takes up a drug and how lasting the effect is, so-called pharmacokinetic studies are conducted. CombiGene recently concluded a study of this kind, the results of which are extremely encouraging.

The concluded study clearly shows that the expression (occurrence) of neuropeptide Y (NPY) and its receptor, Y2, increases markedly after only one week following administration of CG01, and then continues to increase during the following two weeks, reaching a plateau after three weeks. It is particularly pleasing to note that the level achieved after three weeks remained stable throughout the duration of the study, i.e., six months.

“One question that often arises in our discussions with physicians is just how long we can expect to see an effect in humans after an injection and whether several injections will be necessary to maintain the effect. This study shows that the expression or occurrence of NPY/Y2 persists over a long period. This provides further evidence that a single injection should have an effect that lasts for many years. A rule of thumb is that six months in the experimental model we have used for our pharmacokinetic study corresponds to 15 years in humans. The results from the pharmacokinetic study are extremely encouraging,” says a very satisfied Annika Ericsson, Senior Project Manager, CombiGene.

Findings from the pharmacokinetic study are gratifying in other respects as well. Understanding how CG01 acts in the brain is also an important piece of knowledge that will aid planning of the coming safety studies (toxicology and biodistribution) in an optimal way. Knowledge gained from the pharmacokinetic study will also constitute a central component in the design of the first study in humans.

CombiGene’s lead project CG01 has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 823282

About CombiGene

CombiGene’s vision is to provide patients affected by severe life-altering diseases with the prospect of a better life through novel gene therapies. CombiGene’s business concept is to develop effective gene therapies for severe life-altering diseases where adequate treatment is currently lacking. Development assets are sourced from an external research network and developed to achieve clinical proof of concept. Drug candidates for common diseases will be co-developed and commercialized through strategic partnerships, while the company may manage this process on its own for drugs targeting niched patient populations. The company is public and listed on the Swedish marketplace Nasdaq First North Growth Market and the company’s Certified Advisor is FNCA Sweden AB, +46 (0)852 80 03 99


Combigene AB, Medicon Village, SE-223-81 Lund, Visiting adress: Scheelevägen 2, Lund, Sweden

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