This is an article from Ingenious number 1, 2020.

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CG01 update

Apr 6, 2020

Further steps towards studies
in humans

CombiGene has recently concluded two planed preclinical studies in the CG01 project: a pharmacokinetic study and a memory study. The outcomes from both studies are positive and mean that CombiGene now has answers to several key questions from both physicians and regulatory authorities.

Successful pharmacokinetic study confirms that CG01 has a very
lasting effect
CombiGene’s candidate drug, CG01, is being developed for treatment of patients with 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 the planning of the coming safety studies (toxicology and biodistribution). Knowledge gained from the pharmacokinetic study will also constitute a central component in the design of the first study in humans.

No negative effect on cognitive function
NPY, the active substances in CG01, is associated with several physiological processes in the body, among others, memory and learning. CombiGene has therefore received an inquiry from the FDA and the Swedish Medical Products Agency as to whether CG01 has any impact on cognitive functions. The concluded memory study answers that important question: increased expression of NPY and Y2 has no negative effect on memory or learning functions in the experimental model used in the study.

David Woldbye, one of CombiGene’s scientific founders, commented on the results of the study. “The results of earlier academic studies on the effect of NPY on memory and learning have been contradictory. It is therefore especially pleasing to note that we can now demonstrate that we do not affect memory and learning when we inject CG01 into one of the brain’s two hippocampi, precisely as we intend to do in humans.”

Karin Agerman, CombiGene’s Chief Research and Development Officer, is also very satisfied with the outcome of the two studies.“With the completion of the pharmacokinetic study and the memory study it is very reassuring to have successfully answered several questions from the FDA and the Swedish Medical Products Agency. This brings us one step closer to starting the first study in humans. More and more pieces of the CG01 project are now falling into place.”

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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