Prosensa: Luc Dochez. Developing therapies for neuromuscular diseases

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Video title: Prosensa: Luc Dochez. Developing therapies for neuromuscular diseases
Released on: January 09, 2013. © PharmaTelevision Ltd
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In this episode of PharmaTelevision News Review, filmed at BioEurope 2012 in Hamburg, Germany, Fintan Walton talks to Luc Dochez the CBO and Senior VP Business Development at Prosensa
Summary about Prosensa and its focus on Duchenne Muscular Dystrophy
Fintan Walton:
Hello and welcome to PharmaTelevision News Review here at BioEurope in Hamburg, in 2012. On this show I have Luc Dochez, who is the CBO or Chief Business Officer at a company called Prosensa based in Holland, welcome.
Luc Dochez:
Thank you Fintan. Thank you, happy to be here.
Fintan Walton:
Good, Luc we've talked before, but give us a reminder of what Prosensa is all about and particularly in the area of Duchenne Muscular Dystrophy?
Luc Dochez:
That's already a good start indeed, we are focused on Duchenne Muscular Dystrophy, so Prosensa is a rare disease company focused to help patients with rare genetic diseases using our RNA modulation platform and the basic focus, the biggest focus of the company is truly on Duchenne Muscular Dystrophy where we use a technique called exon skipping where we try to correct the genetic mistake at the RNA level and then we hope to reproduce the dystrophin which is the missing protein and by doing that we have help the Duchenne patients and we can hopefully stabilize the disease or prevent any progression of this muscle disease which is very devastating for families in that.
Exon skipping technology and its personalized approach
Fintan Walton:
So basically the technology corrects the mistake that the body is making with the production of the particular protein?
Luc Dochez:
That's a very good summary, and what it does is actually does it at the RNA level, so really using RNAtherapeutics and not at the gene level which gives you the flexibility to change dosing and optimize those regimens as well.
Fintan Walton:
Right, so the underlying basis to the disease is this exon mistake, that's made by the body, but there is also this variance across different patients groups?
Luc Dochez:
Absolutely, the technique requires a very personalized approach, because depending on the exact deletion that causes the problem you need to have a different product to correct it again at the right level, so depending on the genotype we need a different product, so it's all the same disease but all different genetic causes which all require different products. So in contrary to what people were doing before, saying having one medicine and then try to leverage that in many diseases, we really have one disease and we need to develop many, many products to help all the patients that would benefit from this therapy.
Collaboration agreement with GlaxoSmithKline
Fintan Walton:
Right, and the first and your lead product is in, done in the collaboration with GlaxoSmithKline [PharmaDeals ID = 33998]?
Luc Dochez:
Fintan Walton:
Could you tell us something about that alliance?
Luc Dochez:
Yes, we signed the collaboration with GlaxoSmithKline in 2009 [PharmaDeals ID = 33998] and we have progressed a lot since then, so the lead asset is now in Phase III development, where actually requirement has been recently finalized, and so we expect to have data from that by the end of next year. But next to that the other programs are also progressing well and part of them are in collaboration with GlaxoSmithKline with significant part of the Duchenne asset of the products that we need to help these patients is completely retained by us, because that's what we want to do as a company is really help these patients partly by doing that in partnership, but also by bringing it to the patients all the way ourself.
Financial and clinical development progress in Prosensa
Fintan Walton:
Right, and of course it's important for companies like yourselves to be well financed, you have recently raised some money, could you tell us a little bit about that?
Luc Dochez:
Yes, we did a new financing round actually at the beginning of this year where we further strengthened our syndicate. We already had a quite esteemed European investors syndicate with Abingworth, Life Sciences Partners, Gimv and Idinvest, based out of France, but we were very happy and actually proud that we could add New Enterprise Associates out of the US to that syndicate and so we did a 23 million Euro financing round in the beginning of this year and certainly the fact that an esteemed investor as NEA was making their first investment as a life sciences company in Europe and then picking us as their first choice really is something we are very proud of and then we feel it's very important for us as a company.
Efficacy of drug Prosensa's lead compound PRO051
Fintan Walton:
Right, the lead clinical trials that your, you've just described, are obviously very important because it basically underscores the proof of concept of whether this a viable therapeutic approach. You've mentioned that the you've now fully recruited for those Phase III clinical trials, how long will it take before you have some announcement to make on the efficacy of the drug?
Luc Dochez:
Yes, it's a good question, it's also a difficult question to say, I think the trial will probably read out by the end of next year, and as the program is licensed to GlaxoSmithKline they will determine when the exact time will be that they can release the data, but I think it's very important and I am sure that by end of next year, end 2013 we will get a good view on the efficacy that we hope to see of course of this diseases, the patients, all deserve a treatment and so we are crossing our fingers, crossing our toes and hope for the best at this.
Progressive deterioration in Duchenne Muscular Dystrophy
Fintan Walton:
Right, of course we should state of course this has got orphan status, in terms of clinical development, but just finally just what sort of clinical end points are you looking for? What sort of development do you expect or improvement that you expect to see for your patients?
Luc Dochez:
Well it's two different questions actually, in terms of the regulatory endpoint there is only one validated well defined endpoint for Duchenne that is accepted by the major regulatory agencies like the FDA, and the EMEA which is the six minute walk test, but it's only a endpoint that will give you an indication that you can indeed preserve the muscle functioning of these boys that suffer from the disease. So typically you expect a boy with Duchenne to between 7 and 10, 12-years old to rapidly decline and lose his ambulation, so every year they can walk 50 to 60 meters less in the six minute walk test, which is exactly what it says that it is, you walk for six minutes and you measure the walking distance, so what we tried to is show that boys that will be on therapy hopefully will not show the decline that boys that were not on therapy will show in these endpoints, having said this is the regulatory endpoint, we feel that with doing that we will be able to preserve the muscle function of the boys which goes much wider than only the ambulation. It will also enable them to keep moving their hands which are typically affected later in the disease progression that is very important to these boys, so even patients that are already in a wheelchair, in our opinion, could benefit from the drug, but this is something we will have to prove over time and it's very difficult to make any definitive statements on that, but it's really to hope to preserve muscle function for these boys and give them as normal of a life as possible.
Dose regimen for PRO051
Fintan Walton:
Right, and is there a repeated dosing regimen as part of the clinical trial?
Luc Dochez:
Well we are still exploring the exact dosing regimen. In the pivotal study it was once a week subcutaneous administration that was given to the patients, but as this is a rare disease and in the orphan space you have to have, you are trying to answer a lot of questions all at the same time, so we are also doing a dose regimen optimization study next to the, the dosing regimens that I just described. So I think also there a lot of answers still to be provided and hopefully that will come in the years ahead of us.
Fintan Walton:
We look forward to that. Luc Dochez, thank you very much indeed for coming on the show.
Luc Dochez:
Thank you.
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).
Luc Dochez
Chief Business Officer and Senior VP Business Development
At the time of this PTV interview Luc Dochez serves as Chief Business Officer and Senior VP Business Development at Prosensa. Luc Dochez, Chief Business Officer and Senior Vice-President Business Development joined Prosensa in November 2008 and is responsible for all business and corporate development activities at the company. He has over 10 years experience in the biotech industry and was directly involved in multiple financing rounds and partnering deals with various biotech companies. Before joining Prosensa, he was a consultant within Arthur D. Little's biotechnology practice, Director of Business Development at Methexis Genomics NV, VP Business Development at TiGenix NV and President of TiGenix Inc. Luc holds a PharmD degree from the University of Leuven (Belgium), a postgraduate degree in Business Economics from the same university and an MBA from Vlerick Management School.
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 commercialization.
Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA-modulating therapeutics correcting gene expression in diseases with significant unmet need, in particular neuromuscular disorders. Prosensa's current focus is on developing treatments for Duchenne muscular dystrophy (DMD) , Myotonic Dystrophy, and Huntington's disease. In 2009 Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa's lead compound (drisapersen), being developed by GSK, is currently in late-stage phase III clinical trials. Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, Gimv, Idinvest Partners, Life Sciences Partners, MedSciences Capital and New Enterprise Associates.