Trophos: Focus on Orphan drug and fundingFintan Walton:
Hello and welcome to PharmaTelevision News Review here at BIO-Europe in Milan, in spring 2011. On this show I have Damian Marron, who is CEO of a company called Trophos which is based in Marseille in France, welcome to the show.
Damian Marron:
Thank you Fintan.
Fintan Walton:
Damian, could you tell us the history of Trophos?
Damian Marron:
Yes, Trophos is a spin out from the University of Marseille in late '99, 2000 set by founding scientist and a couple of local entrepreneurs and then taken on by VC's afterwards.
Fintan Walton:
Okay, now today the company is recognized as a specialty orphan focused company, orphan drug focus company it's a private company so tell us how the company is actually funded?
Damian Marron:
Yes sure, as I just mentioned it was started by the entrepreneurs taken on by VC's but from very early days with the focus particularly on orphan both neuro diseases funding was provided by a French patient association called the AFM who run a telephone in France and over the years we've established a lot of consortium networks and over the last couple years we've actually when that become leaders of two large EU Framework 7 consortium both bringing in grants of Euro 6 million.
Fintan Walton:
Okay, so you've got a mixture of obviously funding from equity based funding and I suppose non-dilutive funding?
Damian Marron:
Yes and I think that's very important it's enabled us to make the investors money go a lot further obviously the cycle for investors in VC's and further return on their investment in biotech companies can be quite difficult it can be quite hard them to keep on doing complete rounds and be able to bring in non-dilutive funding obviously allows us to build value over and above we could utilize.
Fintan Walton:
Now being the type of company you are has that allowed you to get that type of funding, I mean could a normal biotech company get the same sort of funding or is it a reflection of your own focus as an orphan drug company?
Damian Marron:
I would say yes and no, I think certainly the orphan focus of with working with patient association I think other companies who are in the these very rare orphan diseases some of these terrible diseases will test in the way you can work with patient associations. It is true that you do have to be a small or medium sized enterprise today able to participate in the new consortium, so it's open up to a certain level I think most important is that you are doing scientifically innovative work in areas of real need, so yes being in orphan area does help again I think.
Program on lead compound OlesoximeFintan Walton:
Sure, so tell us then about your specific programs?
Damian Marron:
Yes, we have two major programs in the new consortium, one is a program that our lead compound Olesoxime that's undergoing a pivotal Phase III trial at the moment as part of a consortium called Mitotarget one of the FP7 consortium and so the trial is being run in five countries in Europe, it's a Euro 6 million grant funding EU paying a lot of the cost towards the trial and also for translational research as well around the mechanism of action of our compound and neurodegenerative diseases more widely.
Partnership with AFMFintan Walton:
Okay, so in addition to that option purchase agreement there is also other collaborative agreements anyways is that right?
Damian Marron:
Well actually with Actelion we have a second agreement we've also signed a research collaboration agreement with them at the same time which is to allow them to use our underlying research platform to test their compounds in and of course we are of a if we would call it partnership major partnership is with the French Patient Association AFM [
PharmaDeals ID = 32798] with whom we partner on the development of our lead compound again Olesoxime but for a different orphan neurological disease , spinal muscular atrophy .
Fintan Walton:
Okay, so going back to this option purchase agreement, what is the trigger is there a specific milestone that has to take place before they purchase?
Damian Marron:
Yes, I mean there is a date trigger which is the end of 2012, but the real trigger for it are the Phase III results from our trial in ALS that is ongoing and those are expected in Q4 of this year.
Future plansFintan Walton:
Okay, so obviously you know one of the things that I often ask when I have companies what's the future, I suppose hopefully the future is very clear to you?
Damian Marron:
Well that you know the future is if the project works we all know the risks and the project is not working it is injury, if the project works yes we very much expect that Actelion will exercise that option and Trophos will become part of Actelion and the Trophos technology will continue to grow through that way and the new product will be available for ALS patients. We've also been very careful though to make sure that should for whatever reason Actelion not to exercise its option project failure or whatever other reason that we have the means to carry on with Trophos and to reach other value equation steps as well and we are well funded deep into 2013 even without Actelion raising and taking its option.
Opportunities in orphan drug spaceFintan Walton:
Okay, now just on the topic of taking products through orphan, getting orphan status for products you are obviously European based company most of your clinical trials are here in Europe?
Fintan Walton:
Is that what do you see just looking at it just from a general point of view how much opportunity still exists in that particular field, and how difficult or how easy is it to for a company to move into that particular space?
Damian Marron:
I think an enormous opportunity still there in the orphan field, there are between and you know just to estimate nearly 5000 to 8000 orphan diseases currently there are about 8 - 900 orphan designations in the US and EU but they offer a round about 3 or 400 diseases. So there is still a massive unmet need on the other side of it many of these diseases are ultra-orphan you know 65% of them have fewer than 5000 patients figures like that. The other problem and the other opportunity of course is that the underlying cause its mechanism and many of these diseases are unknown, so a big risk to step into them, but you know a very big reward for everybody involved patients, physicians, companies, investors if one can be successful here.
Difference between personalised medicine and orphan drug areaFintan Walton:
What is the difference between a company moving into personalized medicine and into the area of orphan drug?
Damian Marron:
The two of them do share some commonalities but also some differences, that being said personalized medicine is leading to something which I think could create some problems as we go forward which is slowly slicing of the indications as you get that deep down into little pockets of patients who are characterized by missing a certain gene or having a certain deletional or exon skipping or whatever you know there is going to become a point I do feel particularly where the regulators and the payers are going to be getting worried about how the orphan drug regulations they being used to keep exclusivity, charge very high prices you may have one product it may have 10 orphan indications and they actually be selling an enormous amount which is not really what was the underlying thrust of orphan legislation in the first place, so while there is great opportunity I think we need to be very worried, on the other side of it many orphan diseases as I said the mechanism isn't known so in fact it isn't personalized our approach to ALS I will be quite clear is not personalized it's understanding the mechanism is best to be known and trying to provide some protection against the disease processes.
Fintan Walton:
Right, so in a sense the area of orphan status could be under treat from a regulatory point of view?
Damian Marron:
I think if we don't take care as an industry to treat it with respect If we over milk that cow, yes I think we could create some risks for ourselves down the line.
Fintan Walton:
Damian Marron, thank you very much indeed for coming on the show.
Damian Marron:
Thank you indeed Fintan.