Prosensa: Luc Dochez. Using their RNA modulating platform to help patients with Duchenne's, Myotonic Dystrophy and Huntington’s.




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Video title: Prosensa: Luc Dochez. Using their RNA modulating platform to help patients with Duchenne's, Myotonic Dystrophy and Huntington’s.
Released on: December 14, 2011. © PharmaTelevision Ltd
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In this episode of PharmaTelevision News Review, Fintan Walton talks to Luc Dochez, Chief Business Officer and SVP Business Development of Prosensa.
Focus on duchenne muscular dystrophy
Fintan Walton:
Hello and welcome to PharmaTelevision News Review here at BIO-Europe in Duesseldorf in 2011. On this show I have Luc Dochez, who is a Senior Vice President of Business Development and Chief Business Officer at a company called Prosensa based in Holland, welcome to the show.
Luc Dochez:
Thank you.
Fintan Walton:
Luc, you've been on the show before and you've introduced Prosensa to our audience may be you can just give us a reminder of Prosensa and what you are focused on?
Luc Dochez:
Well happy to do that. So Prosensa is a company focused on rare diseases specifically working on RNA modulation platform and we try to help patients with genetic disorders, rare genetic disorders for whom there is no current treatment available and our main focus is on Duchenne Muscular Dystrophy where we have a lead program now in partnership with GlaxoSmithKline in Phase III development [PharmaDeals ID = 33998], but we have many more compounds following that lead compound as I can explain later in the show how that exactly works.
Prosensa's technology and approach with exon skipping
Fintan Walton:
Right, so when you come back to the underlying technology and your specific approach it is to do with exon skipping?
Luc Dochez:
Correct, so that's in the Duchenne Muscular Dystrophy this is where we use RNA modulation through exon skipping what we there try to do is actually correct the genetic disorders, so what happens in Duchenne Muscular Dystrophy is that these boys don't produce dystrophin which is a very necessary muscle protein that kind of keeps the muscle strong and so if you don't have that you run in all the muscle problems that characterize duchenne patients. So what we try to do is to restore the reading frame that kind of then leads to the expression of the dystrophin protein, so in a duchenne patient you miss a couple of actions in the dystrophin gene which is the longest gene in the human body and then by taking out another action you can kind of correct the puzzle and make it work again and that's how we try to do this with Prosensa and this is also why we have to develop multiple compounds all targeting different subpopulation of duchenne boys, because depending on which action you miss you need to skip another action to make the puzzle fit again.
Deal with GSK
Fintan Walton:
Sure, okay and with your collaboration with GSK you've recently announced there some milestone payments?
Luc Dochez:
Yes correct, so what we announced is we gave an update on how the collaboration is going and I think it was very positive news that based on the encouraging data we have seen so far we achieved good milestones in some of other programs on the programs on which they have an option and we've been able to move one other compound further into clinical development into Phase I/II and so that was good news of course, we've also jointly decided to accelerate the development of some of the other compounds and so the third and the fourth compound all compounds targeting different selective sub groups of duchenne patients will be moved into the clinical in the first half of 2012 obviously you know if things continue to go as they have been going so.
Fintan Walton:
And in terms of the payments that are made?
Luc Dochez:
Yes again the milestones could lead up to another 27 million pounds, but the exact split hasn't been disclosed, but I would say it helps us to continue to stay in a relatively healthy financial situation which is obviously a good place to be as a biotech company.
Grant from Dutch government
Fintan Walton:
Absolutely, and so the other piece of news we've noticed was that you've received a grant from the Dutch government, could you describe what that grant is about and what you're focused on?
Luc Dochez:
Absolutely, next to Duchenne Muscular Dystrophy we also wanted to expand into other areas where we can use the RNA modulation platform and so we've been focusing on Myotonic Dystrophy which is another rare Myotonic disease very bad for the patients as well as Huntington's disease which is probably a better known disease to some of the people that would watch the show, but in both areas we're trying to progress that next to the duchenne, I would say franchise that we're building and for the Myotonic Dystrophy we've received a grant of up to 5 million Euro from the Dutch government [PharmaDeals ID = 44338] which is obviously on the one hand a nice validation, because you don't get 5 million like that and on the other hand it definitely helps us now to put more means to work to also push this development forward, so we are trying hard to also make something available to patients one day there, it's still a long way to go as we are only in the preclinical stage at this moment.
Prosensa's ambition and focus on speciality rare disease space
Fintan Walton:
Right, so obviously those are two important events that have happened just very recently, could you just give us some indication of then where Prosensa is going in terms of its overall product development?
Luc Dochez:
Yes, I think what is very important is that Prosensa really has the ambition to grow into a company that not only discovers and develops products but also we will try to bring these products then to patients ourselves, so we're really focused on building a company and growing the company into a sustainable rare disease player. I think also the partnership with GSK illustrates that, so we have partnered a selected but limited part of the duchenne franchise with have kept un-incumbent rights to bring a significant part of the duchenne products to all these different subpopulations ourselves and I think that is really the underlying ambition and the growth we wanna achieve as a company.
Fintan Walton:
And presumably that fits your model well because it's a really you're basically a truly specialty biopharmaceutical company?
Luc Dochez:
Yes, absolutely we are focused on rare diseases, we've been committed to the rare disease space since the start of the company where duchenne was always I would say the central priority of the company and we will continue to try to leverage that and continue to try to work on that, because finding a cure for patients is really what is all about and so really talking with patients and not just about patients is something we feel very strongly about and this is something we try to achieve and what we try to do at the company.
Future fund raising and growth plans
Fintan Walton:
Sure, now obviously with the cash flow that's coming in from these two events that just happened recently in terms of fund raising obviously you've got shareholders, could you just tell us a little bit about that side of things who your shareholder base is and what are your requirements for further finance going forward?
Luc Dochez:
Yes, happy to do that, so an interesting point about Prosensa is that though we were created in 2002 we only did the first Series A financing in 2007, so in the first five-years we had a lot of support on the government from the government side with grants and loans there, but also from patient advocacy group, so again illustrating the central role that patients have played in the growth of Prosensa and that we will hope that they can continue to play as we are about to announce another grants just kind of giving you a preview on that from a patient advocacy group to accelerate further development of our some of the compounds. Next to that I would say in 2007 we were lucky to attract two of the leading European venture capitalist, so we had Abingworth from the UK, and Life Sciences Partners based in the Netherlands, but having offices also in Munich and then Boston coming to play and kind of they took the lead in Series A round in 2007. In 2008 end of 2008 we did a B round which was again Abingworth, LSP, but then also by AGF which is now called Idinvest as well as Gimv from Belgium. So with that we have a pretty strong European syndicates for now, thanks to the GSK deal we're still in a relatively good cash position and obviously if you wanna realize the ambition of the company to grow into the specialty rare disease company in the future there is gonna be need for other financing rounds and so we will be exploring that in the near future.
Fintan Walton:
Very good, and Luc finally you've been nominated for the Scrip Awards and do you know what the basis for that is?
Luc Dochez:
Well I think people recognized the value that we have and the growth that we have known in the last year or so, I think we have really accelerated the development of the company and achieved some interesting results obviously still a lot to play out in the future, but we are very proud of that nomination and I am happy to tell you that we are also nominated as or shortlisted as one of the most innovative SME companies by EuropaBio. So yes we hope that obviously next to being nominated win one of those but will see already being on the shortlist is a recognition by the community for which we're very thankful and very proud.
Fintan Walton:
Luc, thank you very much indeed for coming on the show.
Luc Dochez:
Thank you Fintan. 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
At the time of recording this PTV interview Luc Dochez works as Chief Business Officer and SVP Business Development of 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
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.
Prosensa
Prosensa is a biopharmaceutical company focused on the discovery, development and commercialization of RNA modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative disorders such as Duchenne Muscular Dystrophy (DMD), Myotonic Dystrophy (DM1), Huntington's Disease (HD) and Spinal Muscular Atrophy (SMA). The company has developed a portfolio of clinical and pre-clinical RNA-based drug candidates. Prosensa's current clinical portfolio is focused on the treatment of Duchenne Muscular Dystrophy . Its lead product, PRO051/GSK2402968, aims at restoring dystrophin expression and improving muscle condition and function in a relatively large subpopulation of duchenne patients and has successfully completed a Phase I/II study. Preparations are ongoing to start enrolling patients in a Phase III study in 2010. PRO044, the company's second product candidate, addresses another subpopulation of duchenne patients and has entered clinical trials in the last quarter of 2009. Prosensa was founded in 2002 and is located in Leiden, The Netherlands. The company works closely together with Leiden University Medical Center (LUMC) and is supported by a consortium of leading biotech investors, including Abingworth, Life Sciences Partners, Gimv, AGF and MedSciences Capital. In October 2009, Prosensa entered into a strategic alliance with GlaxoSmithKline (GSK) to accelerate and broaden the development of its DMD product candidates.