The EU decision to invest 3.36 MEUR in CombiGene’s development of gene therapy epilepsy treatment CG01 recently bore fruit as the company entered into an agreement with Spark Therapeutics for further development of the candidate. BioStock reached out to CEO Jan Nilsson for a comment as the company just received their last payment from the EU programme Horizon 2020. Strengthened by the large grant and triumphant deal, the company is looking to broaden its focus going forward.
CombiGene develops the gene therapy candidate CG01 for the treatment of drug-resistant epilepsy, a disease that affects a large population of approximately 47,000 patients annually in the US, EU4, UK, Japan and China. In May 2018, the major EU programme for research and innovation, Horizon 2020, announced that it had decided to grant CombiGene 3.36 MEUR to fund its continued development of CG01.
The EU grant
The first payment of approximately 1.5 MEUR was made in September 2018. After the EU project was successfully completed and evaluated in July of this year, the last payment of approximately EUR 500 000 has now been paid. This sealed CombiGene’s Horizon 2020 project and summed the accumulated total grant to 3.36 MEUR.
The agreement with Spark Therapeutics
Receiving non-dilutive research funding from Horizon 2020 is a quality stamp for the company as well as the project. Securing an agreement with a large global pharmaceutical company is something most smaller players strive to accomplish, but that, statistically speaking, rarely comes true. CombiGene has delivered on both counts.
»Thanks to the grant from Horizon 2020, we have been able to develop CombiGene as a company over the past three years. This, in turn, has enabled us to achieve the value-creating milestones that have made the CG01 epilepsy project really attractive to the international pharmaceutical industry and that led to our agreement with Spark. Without the funding from Horizon 2020, this journey would have taken considerably longer«—Karin Agerman, Chief Research and Development Officer at CombiGene
In October 2021, CombiGene announced that it had entered into an exclusive collaboration and licensing agreement with Spark Therapeutics regarding CG01. Under the terms of the agreement, CombiGene is eligible to receive up to 328.5 MUSD excluding royalties, including an upfront payment of 8.5 MUSD, as well as up to 50 MUSD for several preclinical and clinical milestones.
The CEO comments
BioStock talked to CombiGene’s CEO Jan Nilsson about the Horizon 2020 grant and the subsequent deal with Spark. From the interview, we also learn that the company’s two projects, CG01 targeting epilepsy and CGT2 – a gene therapy developed to treat partial lipodystrophy – are likely just the beginning of the company’s development journey towards a broader portfolio of gene therapies, based on the company’s development platform.
Did the research grants from Horizon 2020 contributed to you eventually reaching an agreement with Spark?
– It absolutely did. On a general level, research grants mean a lot to small companies in that they add both capital and credibility. For CombiGene, the contribution from Horizon 2020 has been very significant. Firstly, the company was provided with funds that allowed us to continue to CG01’s preclinical program according to our original plan. Secondly, the grant from Horizon 2020 has been a quality stamp that has been valuable both when we have raised additional capital, and when we have sought leading partners in the CRO and CDMO areas. In short, the grant from Horizon 2020 has made CombiGene more attractive both to our investors and to our suppliers.
– All in all, this has helped us to establish CG01 as a highly interesting project in the international pharmaceutical market in a relatively short time, a transition that culminated when we signed the agreement with Spark Therapeutics.
»CombiGene is now entering an intensive business development period with the goal of identifying and inlicensing new interesting projects with high commercial potential«—Jan Nilsson, CEO at CombiGene
Based on your platform and the knowledge that you continuously accumulate, have you identified any possibilities to expand your pipeline in the future?
– I see great opportunities for us to bring new projects into the company. Today, CombiGene is a stronger company than ever before. Through the agreement with Spark, we will strengthen our cash position by 8.5 MUSD. Meanwhile, Spark will cover the costs for finalizing the preclinical program as well as all costs of clinical development and future commercialisation. Our strengthened financial position, together with the great knowledge we have accumulated in recent years, means that we are now in a very favourable position to broaden our development portfolio.
– CombiGene is now entering an intensive business development period with the goal of identifying and inlicensing new, interesting projects with high commercial potential. We will particularly look for AAV-based projects as it is within this technology platform that the company has acquired knowledge in key areas, such as vector design (design of drug candidate), safety aspects and production. Similarly, the disease areas in focus are those where CombiGene has built up a solid knowledge base, i.e. diseases in the central nervous system and metabolic diseases. Having said that, we will keep an open attitude towards possible projects and evaluate each opportunity on its own merits.
What news flow can we expect over the next six months?
– My many years in the pharmaceutical industry, both in Swedish and internationally, have taught me that it is wise to wait to present news until they have occurred. However, we will of course report on the development of the CG01 and CGT2 projects and account for our efforts in CombiGene’s business development as far as possible, with obvious limitations in terms of commercial confidentiality.
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.