At the Annual General Meeting on May 25, 2023, Jonas Ekblom was elected new Chairman of the Board of CombiGene. At the Annual General Meeting, Board members Gunilla Lundmark, Peter Nilsson and Per Lundin were re-elected and Malin Almgren was elected as a new member of the Board. CombiGene’s Board of Directors thus has significant knowledge and experience from key areas for CombiGene’s continued development such as research, development, business development, intellectual property, marketing, and sales, as well as finance and accounting.
Ingeneious contacted Jonas Ekblom to see how he, as newly elected Chairman of the Board, views the continued development of CombiGene.
Jonas, can you briefly describe yourself and your background?
“I have a PhD in experimental neurology, and I am an associate professor in pharmacology at Uppsala University. I have worked in biotechnology and pharmaceuticals for almost thirty years. A significant part of that time I have been active in the US, where I still have a strong network. For the past fifteen years, I have mainly worked with smaller drug development companies. Over the years, I have had the privilege of being involved in many successful projects, but ultimately it is perhaps mistakes and setbacks that have offered the most instructive experiences. Over the past ten years, I have participated in the board work of more than a dozen biotech companies in Sweden, Switzerland, and the United States.”
In early 2023, CombiGene initiated a collaboration with the Danish company Zyneyro to jointly develop the pain program COZY. The program consists of a peptide treatment and a gene therapy. How would you like to comment on the science behind the pain program?
“The COZY program relates to a completely new pharmacological principle for pain relief. A major problem with several of today’s drugs for pain relief is tolerance development, i.e., that continuous treatment requires increasingly higher doses of the drug to achieve reasonable pain control. Data from experimental models with the COZY substances indicate that there does not seem to be any reduction in the pain-reducing effect over time. I also find it attractive to have two different types of pharmaceutical therapies, as this gives the company the opportunity to diversify risk. We get two bingo cards instead of one!”
Problems associated with pain are one of the biggest challenges in healthcare today. How do you see the commercial potential of the COZY program?
“It is well known that severe chronic pain is a very big problem. For the individual, chronic pain involves enormous physical suffering and can also lead to negative social consequences. An everpresent pain can make it difficult to work and participate in a normal social life. Today’s drugs are not developed to treat chronic pain and have a number of severe side effects, not least the addiction problems associated with the use of opioids. Chronic pain costs both healthcare and society huge amounts every year. All in all, this means that there is a very high demand for new therapies that do not have the side effects we see with today’s treatments. It is too early to quantify the commercial potential of CombiGene’s pain program, but if we are successful in our development and succeed in taking the pain program all the way to market, the economic upside will be very significant, to
put it mildly.”
The epilepsy project CG01 was outlicensed already in preclinical phase. Are CombiGene and Zyneyro planning a similar journey with the pain program?
“Absolutely! CombiGene’s strategy is to develop drug candidates that target large patient populations to late preclinical phase/early clinical phase and then enter into collaborations with larger pharmaceutical companies. However, finding the right partner and negotiating favorable agreements takes a lot of time and it is important that this crucial work starts at an early stage. This is what we did with the epilepsy project CG01, and we have now initiated corresponding work within the pain program through the meetings and discussions that CombiGene’s CEO Jan Nilsson and Zyneyro’s CEO Peter Horn Møller had at the BIO Convention 2023 in Boston in early June.”
In May 2018, CombiGene received a grant of EUR 3.36 million from the EU’s Horizon 2020. The company then had two employees and one project in the early preclinical phase. Today, CombiGene is a significantly larger company with three projects that the company itself is developing and the epilepsy project CG01 outlicensed to Spark Therapeutics –one of the world’s leading gene therapy companies. What milestones have been the most significant during this journey?
“We can start with one thing you mention in your question – the Horizon 2020 grant. This grant was an important part of the financing of the epilepsy project at the same time as it constituted a real quality stamp on CombiGene as a company. The choice of the most important service providers for the company’s manufacturing chain has also been crucial. By choosing good service providers, we have significantly increased the chances for being able to successfully run several gene therapy projects in the same development phase. The in-licensing of the pain program COZY has the potential to be very important for CombiGene given the major problems that exist with today’s pain treatment. The single most significant milestone for CombiGene so far is, of course, the licensing of the epilepsy project to Spark Therapeutics. This agreement has already provided CombiGene with USD 8.5 million and could potentially provide the company with an additional USD 320 million, excluding royalties. The agreement with Spark also meant a lot for CombiGene’s reputation and made us an even more attractive partner in a number of different contexts.”
CombiGene has the ambition to further broaden its project portfolio. Are there any particular disease areas that are particularly interesting – and if so, why?
“I would like to say that we have a broad outlook, but we have identified a number of projects that we are now evaluating to see if they could be of interest to us. These include diseases of the central nervous system, endocrine diseases, and genetic muscle diseases.”
Why is it important that CombiGene broadens the project portfolio?
“All drug development takes a long time and the costs of developing a new drug are significant. If you look at the pharmaceutical industry as a whole, only a small percentage of all drug candidates reach the market. The reasons for this vary from that the drug candidate does not have the intended effect to that there are safety problems associated with the treatment. All drug development includes a preclinical phase where the drug candidate is evaluated in several different ways to ensure as far as possible that the drug is both effective and safe before initiating clinical development, i.e. studies in humans. However, success in the preclinical phase does not guarantee success in the clinical phase, it is often not possible to easily translate the results of preclinical experiments into efficacy in humans. It is therefore not uncommon to discover that a drug candidate does not meet the requirements for efficacy and/or safety when initiating the clinical phase. Having a broad project portfolio is important because it significantly increases the chances of reaching all the way to market. In a way, it’s a bit like a football match. Not all chances result in goals, but the more chances you get, the more goals you will score.”
Finally, how do you view the future of CombiGene?
“I am very positive about the future of CombiGene. Through the epilepsy project, the company has shown that it can take a project to a late preclinical phase and out-license it to a world-leading player – Spark Therapeutics was one of the very first companies to get a gene therapy approved for the US market. The first generation of gene therapy companies did not have the luxury of being able to purchase qualified services for the production of investigational drugs from external service providers. The first companies in this sector were therefore forced to build up and validate internal production capacity, which is associated with large investments. CombiGene, on the other hand, has had the opportunity to outsource all the steps in the manufacturing process externally, which means that we have very small overheads compared to many other gene therapy companies. The fact that CombiGene has a good reputation among colleagues in the industry is demonstrated, among other things, by the fact that CEO Jan Nilsson was recently appointed Chairman of the Board of the newly formed CCRM Nordic AB – a national cluster for the commercialization of advanced therapies. As Chairman of the Board of CombiGene, I personally look forward to playing an active role in the continued development of CombiGene!”