Hans Preusting: uniQure, introducing Glybera the first gene therapy approved in the Western world




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Video title: Hans Preusting: uniQure, introducing Glybera the first gene therapy approved in the Western world
Released on: November 01, 2012. © PharmaTelevision Ltd
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In this episode of PharmaTelevision NewsReview filmed at BioPharm America in Boston, Fintan Walton talks to Hans Preusting, VP of Business Development at uniQure
uniQure's product and technology in gene therapy
Fintan Walton:
Hello and welcome to PharmaTelevision News Review here at BioPharm America, in Boston in 2012. On this show I have Hans Preusting, who is Vice President of Business Development at a Dutch company called uniQure, welcome. So Hans, uniQure is a new company but its origins go back to the late 1990's, could you tell us a little bit more about uniQure?
Hans Preusting:
Yes, uniQure was founded as Amsterdam Molecular Therapeutics (AMT) in 1998 in Amsterdam. It's a spin out of the Amsterdam Medical Centre, Academic Medical Centre and it was founded by a group of professors who had a strong background in lipid science and their aim was to develop a gene therapy product for a rare disorder called lipoprotein lipase deficiency, and they thought the best way to do this was founding a small company and develop this product. When the company progressed, of course they needed more money to develop the product, and in 2007 AMT went public on the Amsterdam Stock Exchange. Finally in 2009 Glybera was filed for a registration in Europe and of course it's a lengthy process, it takes a while before the European regulators come back to you. In the summer of 2011 we got a non-approval which was followed by a second non-approval in October of 2011, and as a result of that the stock price went down significantly and we were running out of cash, and the question was how do we fund the company because our investors really believed in the platform and in the company and in the product and they were very interested to bring the company forward. And so we looked into different avenues to finance the company again and actually the only one forward was to found a new company, a private company that could be capitalized with venture capital. Therefore the new company was founded and was called uniQure, that happened in 2012 in April and basically what we did is we transferred all the assets from AMT to uniQure and that's why this is a young company with already a product for approval, but there is a long history behind it.
Fintan Walton:
Right, now obviously the whole area of gene therapy has had a, has been a rocky road for many, many years and it's quite unique and quite significant that uniQure has been able to get a product to market?
Hans Preusting:
Yes, I think so, yes.
Fintan Walton:
So I mean obviously what is it about the technology that was developed, that allowed it to be successful?
Hans Preusting:
Yes, first of all we use a specific transport vehicle to deliver the gene to the specific target tissue, and that delivery vehicle is a virus and that is a very harmless virus. It's nonpathogenic, it does not integrate its DNA in the host-cell and it can be used for, excellently for gene therapy and it has been used in many, many clinical trials, not only by uniQure, AMT but many different academic groups and companies.
Fintan Walton:
And that vector is?
Hans Preusting:
It's called AAV, adeno-associated virus.
Fintan Walton:
Okay.
Hans Preusting:
So we use that virus to carry the gene to the nucleus of a target cell and from there the human body can basically make the protein again that it is missing or that has a missing activity because of a mutation. Because this is a safe therapy and because it is a long lasting effect, because basically you deliver the product once, the body starts to produce the protein for a long period of time, so for patients it has a huge benefit. So why could we produce this, you know, in the end to a marketed product, first of all one of the issues in gene therapy was manufacturing of the vector, manufacturing of this virus in sufficient quantities to allow basically larger clinical trials and also in the end of course a marketed product. uniQure mastered this basically, this challenge, this technology challenge, to develop a production platform for AAV virus vectors and by doing this we basically jumped over that hurdle where a lot of other companies and academic groups had issues with and they've had fantastic results in first clinical trials, but had difficulties to go to the next stage. We were able to go the next stage by doing this and I think this is one of the success factors of uniQure.
Uniqueness and effectiveness of Glybera in the treatment of lipoprotein lipase deficiency
Fintan Walton:
Okay, could you tell us a little bit about the actual disease itself and what, how it affects the body, and where this actual gene therapy fits into overcoming that disease?
Hans Preusting:
Yes, so Glybera is the name for a lipoprotein lipase gene-therapy vector for patients with lipoprotein lipase deficiency, and the deficiency means that these patients have a mutation in that specific gene which does not allow them to make a lipoprotein lipase enzyme which is active, they make the enzyme but it's not active and as a result of that they are not able to degrade fats from a dietary intake. So that the fats accumulate basically in the blood stream and that has a negative effect, you get large fat particles basically in the blood stream and they may block small veins, for example in the pancreas, and a result of that they get pancreatitis attacks which could be lethal and have also has negative effects for the patient.
Fintan Walton:
Sure.
Hans Preusting:
So a lot of companies develop enzyme replacement therapies, so you could think, okay if we produce a lipase and we give to patient the lipase enzyme it will work, but in this case that doesn't work, the lipase is rapidly cleared from the body and therefore this technology doesn't work and therefore gene therapy where you give basically the patient the right gene again, they make their own lipase, was the only way forward for this disease and that's what was developed right now and this can be used for many different genetic diseases and therefore we can use this platform now for a develop of very interesting and new products.
Fintan Walton:
Where does the gene normally, where does this normally expressed?
Hans Preusting:
It is expressed for example in muscle cells and Glybera is also administered into muscle cells, for example in the leg.
Fintan Walton:
Okay, so is it actually targeted to the muscle cells or when you do the gene therapy can the vector get into all your cells and they are just turned on in the correct, in the correct tissue?
Hans Preusting:
So what we do is we do a targeted delivery, meaning we deliver the gene into the muscle and therefore it will be expressed in the muscle cell. So if you do, for example for other products we do an IV injection and then the vector travels, for example to the liver and with that liver specific promoter which basically switches on the gene and starts expression of the protein you get only expression in liver, but for Glybera this is a muscle administration and a muscle expression and from there from the muscle cell the protein will travel to basically to the endothelium of blood vessels.
Marketing and sales strategy for Glybera
Fintan Walton:
Very interesting. So Hans, where does a company like uniQure go, obviously you've got this particular product, now so obviously for this product to be used you need to engage with clinicians, who can then administer this gene therapy, so how does that actually work, do you obviously produce the product, you sell it directly to specific clinicians, Tell me how you do that.
Hans Preusting:
Yes, so this is a unique product because what we will do is we will make basically products patient specific, because we dose the product based on the body weight, so that means once a patient is identified we will hear from the hospital where the patient is, what the body weight is, we make a patient specific pack for transportation to the hospital. It's a cold transportation, it's at minus 20, we deliver it to the hospital and there the patient can be treated. We envision to do this ourselves, the whole marketing and sales, because it's a relatively small market, it's a rare disorder, an orphan product as it is called, and we expect patient numbers in Europe, of around, let's say a thousand or less, and therefore you need, you don't need a very large sales force to get this to the patients. It is more important to identify patients and have a specialized group of people that is able to bring this to the patient and also discuss this with doctors that they aware that this product exists, because you know gene therapy is new, so it's a different way of marketing and sales than for a regular drug.
Fintan Walton:
Right, sure. And I presume then the patients come to a specific clinic in Holland?
Hans Preusting:
No they come, for example, you know there are lipid clinics in all of European countries, so usually, I think they will go to a local hospital and they are treated over there, they don't have to come to the Netherlands, we ship the product from our facility in the Netherlands to the European country.
Fintan Walton:
And for a clinician to do the actual therapy is relatively straight forward?
Hans Preusting:
It's relatively straight forward, I mean it's a muscle administration, so it's an injection in the muscle, and it's not one injection it's a series of injections which will take around two to three hours as the full administration and then the patient basically can go home.
Benefits and outcomes of Glybera in patient treatments
Fintan Walton:
Right, and obviously this is still early days, of those patients that have been treated what have been the outcome for those particular patients?
Hans Preusting:
Well, the quality of life of these patients has significantly improved, why is that? you know the ticking time bomb of getting a pancreatitis is a nasty one and that time bomb has been taken away basically by giving them Glybera. So that is I think the major one, we know from patients that they feel much better, they have much more energy, so this is a significant effect on their life and also on their social life, because you know patients who were not able to do, to eat anything else than a very strict diet, and of course they still have to adhere to a diet, but it you know for them this has a huge change on their quality of life.
Products in pipeline
Fintan Walton:
Right, and then going back to uniQure itself, what else do you have in your pipeline, are you going to stick to gene therapy, is that your area of specialization, tell us about the other programs you want to bring forward?
Hans Preusting:
We are dedicated to AAV gene therapy, so this is adeno-associated virus and we have developed a platform technology. As I said Glybera is the first product out of that but we can use the same technology, the same platform, which is very modular to develop other diseases, and in our pipeline for example we are developing a product for Hemophilia B which is a blood clotting disease, patients bleed, and nowadays they get protein infusions, let's say three times a week. There is now evidence that you know when you give them the gene with the gene therapy, which is one off administration, they make the protein themselves and they don't need the protein administrations anymore on a weekly basis. So that's a huge advantage for patients and also a huge advantage in the end for also for the healthcare system, because you don't need a lifelong treatment anymore with an expensive product. So there gene therapy has a potential to make it win-win, so that's Hemophilia B. We also work on other orphan indications like acute intermittent porphyria, which is also a nasty disease. Also we are working on a lysosomal storage disease called Sanfilippo B which affects some children and if we can help them with this gene therapy that would be an amazing benefit. And then there is a non-orphan disease that we are developing together with a university in the US here and it's for Parkinson's disease. As I said you know we are a small company, so we have a small pipeline, very interesting products, but this can be extended based on the same technology.
Future plans and partnership strategy
Fintan Walton:
So what's the future then for uniQure, you are going to remain a specialized gene therapy company I presume, and are you looking for collaborations with other pharmaceutical companies or other organizations?
Hans Preusting:
Yes, for of course we have to finance the company, so what we are doing here also in Boston at this conference is trying to find partners for our Hemophilia program. I think that's a very interesting program for companies that are active in this space, but also may be companies that want to be active in this space because this is a unique product. So this is a product that is open for partnering, of course we are looking for financing of the company in other ways, equity investments and in the other way of course is Glybera sales which will hopefully ramp up quickly from let's say mid next year onwards.
Fintan Walton:
And you have an application with the FDA for Glybera?
Hans Preusting:
Not yet, this is planned for early next year, we will file also in Canada and in the US.
Fintan Walton:
Hans Preusting, thank you very much indeed for coming on the show.
Hans Preusting:
Thank you very much.
Fintan Walton
Dr Fintan Walton is the Founder and CEO of PharmaVentures . After completing his doctoral research on the genetics of cell proliferation at the University of Michigan(US)and Trinity College (Dublin, Ireland), Dr Walton gained broad commercial experience in biotechnology in management positions at Bass and Celltech plc (1982-1992).
Hans Preusting
VP of Business Development
At the time of this PTV interview Hans Preusting PhD, MBA serves as VP of Business Development at uniQure. Dr. Preusting joined uniQure's predecessor company AMT in August 2006 and is responsible for Business Development. He holds a PhD in Biochemistry and an MBA from Rotterdam School of Management. He has 20 years of experience in product development and manufacturing using fermentation and cell culture techniques. Prior to uniQure, he was at Solvay Pharmaceuticals, DSM and Gist-brocades. Hans Preusting holds two patents and has published over 20 scientific articles.
PharmaVentures
PharmaVentures is a corporate finance and transactions advisory firm that has served hundreds of clients worldwide in relation to their strategic deal making in the pharmaceutical, life science and healthcare sectors. Our key offerings include: Transactions / deal negotiations; Product / technology valuations; Deal term advice; Due diligence & expert reports; Strategy formulation; Alliance management; and Expert opinion for litigation/arbitration cases. PharmaVentures provides the global expertise to ensure our clients generate the highest possible return on investment from all their deal making activities. We have experience of all therapeutic areas and can offer advice on both product and technology commercialisation.
uniQure
uniQure is a world leader in the research and early development of human gene based therapies. Its lead product Glybera for the treatment of lipoprotein lipase deficiency has been recommended for approval by the European Medicines Agency (EMA) in July, 2012. Glybera is the first gene therapy approved in the Western world. uniQure has a product pipeline with several gene therapies: hemophilia B, acute intermittent porphyria, Parkinson's diseaseand Sanfilippo B. uniQure is based in Amsterdam, the Netherlands. The company employs a staff with extensive scientific and industry experience, and is the world leader in manufacturing of AAV-based products on a commercial scale, meeting cGMP requirements, and has extensive experience in the regulatory processes for gene therapies.