CombiGene’s pilot study, which is aimed at optimising the administration of the drug candidate CG01, has given the company valuable insights for future development steps. The path towards clinical studies, which will take place with support from partner Spark Therapeutics, thus takes another step forward. BioStock contacted the company’s CEO Jan Nilsson who talks about the collaboration with Spark, and how the company views its intensified investment in business development.
Last summer, Swedish CombiGene, which develops the gene therapy CG01 for the treatment of epilepsy, initiated a collaboration with the British Neurochase.
Neurochase tailors therapeutic strategies for direct administration, Convection Enhanced Delivery, of drugs to different parts of the brain. CombiGene’s choice of partner was probably facilitated by Neurochase having extensive experience in administering the very type of gene therapy vectors used in CG01.
Before the first clinical studies with CG01 can be initiated, the preclinical programme must first be completed. An important part of this is to optimise how the drug itself should be administered in patients, which is what a study with Neurochase is meant to explore.
In this study, of which the practical part now has been completed, the administration has been monitored through real-time imaging, i.e., the scientists have tracked how the injected substance spreads in the brain, in order to ensure that the gene therapy vectors reach the intended part of the brain. Injection volume and injection rate have also been evaluated, which, according to CombiGene, has provided valuable information about Neurochase’s injection instruments for the upcoming long-term studies in toxicology and biodistribution in large animals.
Spark Therapeutics will lead the clinical studies
Since CombiGene and Spark Therapeutics entered into an exclusive global cooperation and licensing agreement for CG01 in October 2021, the companies have analysed which path forward will be the best. One such important step is to increase the number of clinics involved in the study to include the U.S., in addition to Europe.
During the remaining stages of the preclinical programme, all CG01-related R&D activities being run by CombiGene, internal as well as external, will be agreed upon and approved by Spark, which also assumes all agreed costs. As CG01 enters the clinical phase, Spark will take over the responsibility for the continuation of the project and thus also bear all costs during this development phase.
The CEO comments
BioStock spoke to CombiGene’s CEO Jan Nilsson, who shares his views on the completed sub-study with Neurochase, while also giving a brief insight into the collaboration so far with the pharmaceutical giant Spark.
Jan, you are now taking another step forward in the preclinical programme. How important is this particular part of the programme that has now been completed?
– First of all, I want to point out that all parts are important. But as we are the first in the world to hopefully one dayintroduce a potentially curative gene therapy treatment targeting a severely disabling condition such as drug-resistant focal epilepsy, every step forward is valuable. We now have more knowledge and data that will be importantfor the upcoming long-term studies in toxicology and biodistribution in large animals.
Just over four months have passed since the deal with Spark, unless you also include the long negotiation period that resulted in a huge deal for CombiGene. How has the cooperation developed since the agreement was signed?
– The collaboration with Spark has developed very favourably. It is clear that we have found a partner with exactly the qualities we want for the CG01 project, i.e., a partner with the know-how, capacity and willingness to run the CG01 project in an optimal way. What has so far pleased me the most is our joint decision to broaden the clinical development programme to include clinics in the U.S. In this way, CG01 gains a natural foothold in the U.S., by far the world’s largest pharmaceutical market.
In recent months, in addition to focusing on concluding the preclinical programme, you have intensified your focus on business development. Can you elaborate on this?
– Our focus in identifying new possible projects is based on two things. Technologically, we are most interested in AAV vectors because this is an area where we consider ourselves very knowledgeable. In terms of indications, we primarily look at diseases within the central nervous system and metabolic diseases where we also have good knowledge. At the same time, we will keep a very open attitude towards other technologies and indications and assess each potential project on its own merits. Identifying projects that could be of interest to CombiGene is a high priority within the company right now.The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.