Significant advances in the epilepsy project. Inlicensing of a new gene therapy project. Acquisition of Panion Animal Health.
This is an article from Ingenious number three.
Jan Nilsson, CombiGene’s CEO, is in a characteristically good mood when he sits down at the conference table for this interview with Ingeneious. It’s obvious that a lot is happening at CombiGene right now.
The most recent issue of Ingeneious was published in May. Can you tell us, briefly, what has happened at CombiGene since then?
”I’ll try,” says Jan with a laugh. ”In fact, a lot of exciting things are happening at CombiGene right now. You might say that the company is undergoing a metamorphosis: we’re moving from one development phase into the next. I’m not quite sure where to begin. The energy level in the company is higher than ever. We are really having fun and we’re working incredibly hard, as we have been for quite some time, and it’s starting to show results. That’s very satisfying. In the CG01 project, together with British Cell and Gene Therapy Catapult, we’re developing a manufacturing method for our candidate drug and we have recently selected both CDMO and CRO partners. This means that we have taken several very significant steps forward in the CG01 project. We have also in-licensed a new gene therapy project, of which the aim is to develop a treatment for the rare disorder: lipodystrophy. During the summer we also acquired the majority interest in the veterinary medicine company Panion, which uses the same technology platform as CombiGene.”
It sounds as though you have your hands full. Let’s start with the CG01 project and its progress. What does the choice of CDMO and CRO partners imply?
”Our CDMO partner is a Swedish-British company called Cobra Biologics and they will produce our candidate drug, CG01. Much remains to be done before we can produce the first batch of GMP-classed material, but we know who will do it. The relationship between CombiGene and Cobra has great potential to continue for many years to come.” Jan pauses briefly to catch his breath.
”The choice of a CRO partner is also very important. Northern Biomedical Research (NBR), our chosen CRO, will conduct the preclinical safety studies for CG01. We have been working for some time to find the best possible CDMO and CRO partners and I am very pleased with the choices we’ve made. I would also like to take this opportunity to thank my colleagues at CombiGene, Karin Agerman and Annika Ericsson, who have done a fantastic job and have put up with me without complaining during our innumerable trips in Europe and North America.”
Can you explain your new project, the lipodystrophy project?
”Absolutely. CombiGene’s strategy is not to conduct our own research, but to acquire or in-license interesting research assets, precisely as we did with the CG01 epilepsy project. We have in-licensed our new project from a company called Lipigon. The objective is to develop an effective gene therapy for the very rare disorder, lipodystrophy. Lipodystrophy is characterized by abnormal distribution of fat in the body, which means that body fat is lost. In the absence of normal body fat, different organs begin to accumulate fat, which subsequently leads to serious metabolic complications, among them, difficult-to-treat diabetes, acute inflammation of the pancreas and liver and elevated risk of cardiovascular disease. Since lipodystrophy is a very rare disorder for which there is currently no adequate treatment, there is the opportunity for a candidate drug to be designated as an orphan drug. Among other things, this entitles a sponsor to protocol assistance from the European Medicines Agency, reduced fees and commercial protection by data exclusivity for ten years after approval of the drug. The US Food and Drug Administration (FDA) applies a similar regulatory framework.”
So, orphan drug designation is a way for the authorities to stimulate development of medicines for small patient populations where the possibility of successful commercialization would otherwise be virtually non-existent?
”Yes, you could say that. Patients with rare disorders have the same right to care and treatment as sufferers of more common diseases. Both groups should be able to benefit similarly from advances in medical research. I should point out that our ambition is for the lipodystrophy project to receive orphan drug designation, but we don’t intend to submit an application until a much later stage
of the project.”
Let’s move on and discuss the acquisition of Panion. What does CombiGene achieve most through the acquisition?
”CombiGene now holds more than 90 percent of the shares in Panion. We have thereby achieved all of the original objectives for acquisition of Panion. Not least, we now have full control over our intangible assets, which is particularly important with respect to our relations with future strategic partners.”
It sounds as if CombiGene has taken major steps forward in just a few months.
”We think so. CombiGene now has a project (CG01) which, after the safety studies are completed, will be ready for the clinical phase and initial studies in humans, and an interesting project (the lipodystrophy project) in the preclinical phase. In addition, as mentioned, we’ve also acquired Panion. In-house, just for fun, we’re calling it CombiGene 2.0. We have two gene therapy projects, two new partners in the CG01 project, and we have two concluding preclinical studies (biodistribution and safety studies) that precede studies in humans. We have also acquired Panion. Overall, that makes CombiGene the Nordic region’s leading gene therapy company – a position which we intend to strengthen over the coming year.”
CombiGene’s lead project CG01 has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 823282
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 firstname.lastname@example.org.