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#3 • 2017

CombiGene – World-leading Swedish gene-therapy research IN THIS ISSUE: Editorial............................................................. page 2 Historic day for gene therapy..................... page 2 CombiGene awarded Seal of Excellence... page 2 Report from BIO 2017 San Diego................. page 3 The epilepsy project – long-term study.... page 4

This newsletter has been produced by CombiGene AB, September 2017


A breakthrough for gene therapy We remember some days more than other days. Sometimes, these memories are personal in nature; sometimes, they have to do with major world events. In my career, I have experienced few days that can compare with August 30th 2017. That was the day that the FDA approved the first gene therapy for the US market; namely, Novartis’s treatment for acute lymphoblastic leukaemia.


FDA approves Novartis treatment for acute lymphoblastic leukaemia

That CombiGene is on the right path was confirmed by the fact that we were awarded a “Seal of Excellence” by Horizon 2020, the EU’s largest-ever research and innovation programme. The Seal of Excellence is a mark of quality for excellent innovations that are promising investments. Soon after we received this stamp of approval, we were also awarded 500,000 kronor in funding by Vinnova. The funds will be applied to further development of our business plan. The main focus will be on our epilepsy project, but we will also investigate the possibilities for broadening our research through additional projects. Jan Nilsson, CEO

EDITORIAL STAFF Contact: Legally responsible publisher: Jan Nilsson Production: Wiberg & Co Reklambyrå. Text: Michael Vallinder English translation: Mark Wilcox CombiGene AB (publ) Medicon Village, SE-223 81 Lund, Sweden The company is listed on AktieTorget. Please read our press releases and newsletters, available on our website.

ALL is the most common form of leukaemia among children under the age of fifteen and accounts for about 90 percent of all leukaemia cases.

▪ Novartis’s treatment for acute lymphoblastic leukaemia (ALL) is the first gene-therapy product to be approved for the US market and therefore represents a historic milestone for gene therapy as a whole. Several treatments are nearing market approval, while interesting possibilities for new treatment methods are announced at regular intervals. August 30th 2017 is a historic day for gene therapy. That was the day when the FDA (the Federal Drug Administration in the USA) approved the first gene therapy treatment for the US market. By approving Novaratis’s Kymriah therapy for certain paediatric and young adult patients with a form of acute lymphoblastic leukaemia the administration

is demonstrating its confidence in the safety and efficacy of a gene -therapy product. This is most likely the first of a series of approved gene therapies that can be expected in the coming years, in both the USA and Europe. ALL is the most common form of leukaemia among children under the age of fifteen and accounts for about 90 percent of all leukaemia cases. A clinical study among patients who no longer responded to other treatment options showed that 82 percent of those treated with Kymriah were fully or partially cured of the disease. The rate of survival after 12 months was 79 percent. Another gene-therapy product with a good probability of approval for use in

the USA within the relatively near future (2018) is Luxturna from Spark Therapeutics. Luxturna has been developed for treating vision loss caused by certain types of gene mutations. Although it is considerably farther away from market approval, this is another very exciting project that clearly demonstrates the potential of gene therapy. At the University of Queensland in Australia researchers are working on a new treatment for severe allergies. In test models the research team has succeeded in “switching off” the harmful immune response that causes allergic reactions. Other conditions that could potentially be treated include chronic asthma, peanut allergies and severe allergic reactions to bee stings.

CombiGene has been awarded the Seal of Excellence by Horizon 2020 and 500,000 kronor by Vinnova. With research funding amounting to €80 billion over seven year (2014 to 2020), Horizon 2020 is the EU’s largest-ever research and innovation programme. The programme was established to promote more scientific breakthroughs and discoveries and take them from the lab to the market. In

July CombiGene was awarded a Seal of Excellence by Horizon 2020, a mark of quality for first-class innovations that are promising investments. Thanks to Horizon 2020’s Seal of Excellence, CombiGene also received 500,000 kronor from Vinnova. The funds will be used to produce a definitive business

plan with focus on the company’s epilepsy project and which will also address a broader development programme. The new business plan will then provide a basis for an application for additional funding via Horizon 2020 to support continued research and development of the epilepsy project

Horizon 2020 was established to promote more scientific breakthroughs and discoveries and take them from the lab to the market. In July CombiGene was awarded a Seal of Excellence.

This newsletter has been produced by CombiGene AB, September 2017



BIO 2017 in San Diego – epicentre of future therapies ▪ With more than 16,000 delegates from 74 countries, 41,000-plus meetings, over 3,500 participating organi­zations and companies, and 1,800 exhibitors, BIO 2017 in San Diego is the world’s biggest partnering event. CombiGene was there and two of the 16,000 delegates were CombiGene’s chairman, Arne Ferstad, and the company’s CEO, Jan Nilsson. Ingeneious managed to locate Arne Ferstad on Sweden’s west coast for a discussion about San Diego and the future of gene therapy.

Let’s begin with your impressions from the meeting in San Diego. The San Diego partnering event is fantastic in many ways. The high level of activity and positive energy are very inspiring. Just being there gives you a sense of the magnitude of the research and development in the pharmaceuticals field that is now under way worldwide. It is also a very well-organized and professional gathering. Since you can effectively and efficiently present yourself and your company in advance and at the same time book meetings with the people you wish to meet, every minute you spend there is devoted to what is most important; namely, establishing contacts that help to drive the development of gene therapy and our project.

Looking at CombiGene, specifically, what were your objectives when you went to San Diego and how well did you succeed? Quite simply, we went to San Diego to see and be seen, to listen and be heard. And we succeeded. During our time in San Diego we met seven big pharma companies, a biotech firm, ten CROs (Contract Research Organizations), four companies from the financial sector and representatives from the Epilepsy Foundation of America. These contacts reflect CombiGene’s current situation precisely. We wish to build and maintain relations with major players in the pharma industry in order to be able to enter into fruitful partnerships in the long term. We still have a lot of research and develop­ment ahead of us, and it is not inconceivable that, in future, we may have to outsource some of it to one or more contract research organizations. Like all development companies, Combi­Gene will need more capital before we can achieve a positive cash flow; therefore, it is very important that we create a name for CombiGene in the capital market as well. Last but certainly not least, CombiGene is working to make life better for patients throughout the world. Therefore, it was particularly beneficial to meet with representatives from the Epilepsy Foundation of America.

What response did you get in your meetings? I can say, without exaggerating, that the response was very good. Jan Nilsson, CombiGene’s CEO, did a fantastic job of explaining our operations and our

“San Diego confirmed what we already knew. CombiGene has a very promising technology platform with great development potential for a range of neurological diseases.”

promising technology. We believe that everyone we spoke with will follow the development of our epilepsy project with great interest. And those who understand the science behind our work expressed their interest in very positive terms with respect to the level of innovation of our research and development.

Looking at gene therapy from an R&D perspective, what can be said about the level of activity? Internationally, belief in gene therapy is at a very high level. This is exemplified by, among other things, the large number of clinical studies that are now under way throughout the world. According to the most recent figures, 504 clinical studies are now being conducted in the gene therapy field. I don’t know how many preclinical studies are now being done, but it is safe to assume that there are considerably more. Current clinical studies number 184 phase-I studies, 286 phase-II studies and 34 phase-III studies. The emphasis is in the area of oncology, but cardiovascular diseases and diseases related to the central nervous system are also very common. As far as I know there are currently no clinical studies concerning gene therapy treatment of epilepsy.

This newsletter has been produced by CombiGene AB, September 2017

Every pharmaceutical development project has a financial side and a commercial side. What is happening in these areas? Looking at the financial side, we can start by saying that all pharmaceutical development costs a lot of money. There are two main reasons for this. The first is that we are talking about extremely advanced science. Serious research takes time and therefore costs a lot of money. The second is the patient safety perspective. Obviously, it is crucial that, before a medical product can be launched on the market, it must be proven to be both safe and have the intended effect. That is why clinical studies are so extensive and subject to rigorous controls and regulations. Therefore, companies, financial institutions or private individuals never invest in pharmaceutical development on the basis of rash decisions. Everyone is aware of both the challenges and the opportunities. Considering this, it is really quite incredible that gene-therapy-related research and development attracted investment capital amounting to 1.18 billion US dollars in the second quarter of 2017 alone. In other words, many investors are aware of the great opportunities gene therapy has to offer.

As for the commercial side, gene therapy is now in the starting phase. A range of new medicines are pending approval by the relevant authorities and it will be interesting to follow their commercial development. Major efforts are being invested in several areas related to remuneration systems and regulatory issues. I am convinced that the real potential of gene therapy will be realized within the not too distant future.

To conclude, in your view, what is the outlook for CombiGene? San Diego confirmed what we already knew. CombiGene has a very promising technology platform with great develop­ ment potential for a range of neurological diseases. Our know-how and our area of specialization align well with the overall landscape of the industry. CombiGene still has a long journey ahead before we can launch a drug but, thus far, we have done everything according to our original plan. If we keep working in a focused and professional manner, I am convinced that CombiGene will continue to deliver fantastic results.


CombiGene’s epilepsy project – long-term study

Read all of our news in one place We would like to keep you well informed about what is happening in the company.

CombiGene is now conducting a longterm study for the company’s candidate drug CG01. The long-term study, led by Professor Merab Kokaia, is a decisive preclinical proof-of-concept-study which, thanks to the fact that the test models approximate human epilepsy as closely as possible, can be expected to produce reliable results. The study was initiated in the second quarter of 2017 and is expected to be completed during the first half of 2018.

Therefore, we are now launching CombiGene’s digital newsletter to allow you to read all of our news first-hand. Subscribe via our website, where you can register the e-mail address to which you wish the newsletter to be sent. Use the link “Subscribe to our newsletter” at the top-right corner of the page. The subscription is free of charge and you can cancel it whenever you please. Read “More from the company” and our Press Releases at:

Sign up here! anmalan-nyhetsbrev/

Merab Kokaia and Master’s student Rasmus Vighagen.

Everything you can do that I can’t Epilepsy that cannot be controlled entails very considerable limitations in both the work life and the social life of the sufferer. We hope to change this. Our world-leading gene therapy research offers the potential for effective treatment for millions of people worldwide. People who have no other options.

The gene therapy explorer CombiGene’s objective is to develop treatment methods that can improve the quality of life for millions of people throughout the world.

Ingeneious nr3 2017 en  
Ingeneious nr3 2017 en