Rare dedication

In order to gain a better picture on this aspect of the pharma industry, NGP caught up with the company’s COO Gregg Lapointe.

NGP. Firstly, could you give us some idea of some of the medicines you currently have in development to combat rare diseases?

GL. One we have been working on for a while is for a disease called cystinosis. This affects somewhere around 250-300 patients in the US and another 250-300 in the EU, so it’s an extremely small patient population. Cystinosis itself is a disease where a certain amino acid accumulates on the major organs of the children who are affected by this genetic disorder. Fortunately there’s a form of therapy that assists these patients with the accumulation of the amino acid on their organs. In past years many of these children would have kidney failure at the age of nine and now they can delay that for some years but what’s happened as the children have grown up and become a little older, we now see the accumulation of these amino acids on their eyes, which we refer to as crystals. So we have a product in development that we hope to submit an MDA to the FDA sometime in the first half of next year, which can deal with the accumulation of these crystals on their eyes and get rid of them.

One we are most excited about is a project for premature babies where for very low birth weight babies, typically less than 1250 grams in necrotising entrocolitis, so we refer to that as ‘neck’. And it’s a gastrointestinal disorder that’s often fatal for these very small babies, premature babies and there’s currently, other than massive amount of antibiotics, there’s very little that can be done. We think in the US and in the EU there’s over 10,000 babies a year that die of this disease because they don’t respond necessarily to some of the current therapies and in addition, some of those children that actually do respond, we now know, after many years, as they grow up, they present later in life higher incidence of asthma, allergies, slower development. It’s a very debilitating disease and we’re really excited about the idea of using a pro-biotic we call a live bio-therapeutic to treat these babies.

You may be familiar with some of the interesting things research is going on with pro-biotics ¬– live bacteria to restore sort of the gut flora and some belief in these children is that because they’re born premature they’re not properly colonised at birth, either through the vaginal canal or because when they develop ‘neck’ they’re not being fed the same way with breast milk that they don’t develop an appropriate gut flora and that is a contributor to this disease. So it’s very early stage for us. We have very good scientific rationale and we expect over the next couple of years to develop some clinical programs that will look at treating these preterm babies with a very unique combination of bacteria that we’ve developed, internally. We’re really excited about that because it could have a very significant impact on these babies.

The third one that we’re working on is actually a late stage project, partnered with a company called Gentium, an Italian-based research company, for the treatment of a very rare disease called venous occlusive disease or VOD. Of the patients that are diagnosed with severe VOD, which occurs typically after a bone marrow stem cell transplant, 80 percent of patients will die within one hundred days and there’s no therapy. Gentium is currently enrolling patients for a pivotal Phase III trials and we hope that the drug ultimately will prove to be very successful for these patients.

NGP. Researching therapies for rare diseases does bring us a unique set of challenges. What are the biggest research issues and how are currently dealing with them?

GL. One of the first ones is the lack of patients – some 200,000. This may sound like a lot of patients but it actually really isn’t in the overall scheme of healthcare. Some of the big blockbuster drugs that have been launched worldwide can affect millions and millions of patients. In some of the projects we’re looking at we actually refer to it as ‘orphan’ or ‘ultra orphan’ because we’re looking at areas where there may only be 500 patients that we can identify in the world. The first challenge for us is that the patients are very dispersed because there are so few of them, and there’s typically been very little research done in the area over many, many years.

Of course the development of medicines for these small patient populations is very costly. I think there’s a study that says $800 million – I can assure you we don’t spend $800 million developing a drug for rare disease but on a per patient basis our costs are significantly higher than developing any other. So I would say it’s those things, it’s the lack of research, it’s the cost per patient to bring the drugs to market and manufacturing’s always an issue. None of these challenges we can’t overcome with a little bit of hard work and consistency but it is a challenge, no question.

NGP. How do you deal with aspects such as marketing those products and how to you make sure the cost of the prescription isn’t prohibitive?

GL. That’s a really good question because it’s an issue these days for sure. When you’re developing a drug for a small patient population you think about sales and marketing from a Big Pharma context where I know some of the companies out there might have 4000 or 10,000 or 13,000 medical sales professionals calling on thousands and thousands of physicians. In many cases some of what they’re marketing may have equivalent drugs in other areas so it really is an exercise in sales and marketing.

In our case, none of the medicines that we have been developing, when you think about these being life saving, we don’t really think it’s sales and marketing in that context. We have a drug called Carnator injection for patients undergoing hemodialysis, and we have six people on that sales and marketing team. It’s very small because really what we try and do is ensure that physicians and patients are aware that the medicine is available and they’re aware of how they can access it in cases where survival or mortality is one of the endpoints.

It is extremely important that the drug gets out there quickly so they’re aware of how to can access it, because these are medicines for rare disease so they are often highly priced so you want to be certain that patients have all the support they can have in terms of reimbursement assistance, co-pay assistance. We have extensive compassionate use programs, so that’s a big part of what our sort of sales and marketing effort, if that’s what you want to call it. It is really making sure everyone’s aware of it and how to dose it properly, because it’s much more a medical education process than a traditional sales and marketing process.

NGP. In terms of getting your products to those who needs them do the product benefits speak for themselves? Is it a matter of education and getting the information out there?

GL. I think all of us would agree, we wish drug development was free and we wish all of us could work on this for free. That’s a great thought but it’s not the reality. Having said that, in this area where you’re developing drugs for small patient populations and the cost of these medicines and therapies can be quite high, we also believe that we have a responsibility to get out there and talk to patients directly and to explain to them and be very open and transparent about what it costs and why this is necessary. We try to attend as many patient conferences as we can.

That’s one of the unique things we love about what we do – we actually get to meet the patients, to meet their families, we understand in very personal terms what the disease means for the patient and we work very hard on these relationships. They then come to understand what it is that were trying to do and why it is extremely important that you approach rare diseases with a sustainable economic model, not a charitable one because in the end the charitable one won’t create the same number of therapies, and if we keep doing that and keep communicating that, patients do understand. It’s not easy, we have to be prepared to go out there and explain things and sometimes that’s hard. It’s hard to stand up in front of patients and explain why a therapy can cost so much money but you have to go out there and you have to do it and we work very, very hard at that and the key thing is to link very closely with the patient foundations. It’s not just at the point of launching a medicine but throughout the entire process. It’s open communication that is extremely important.

NGP. Your background is in business and finance operations. What was it that attracted to you such a technical scientific field from a personal standpoint and what sort of benefits do you think this business perspective brings to Sigma Tau?

GL. Well I will say, in defence of guys like you and I, sometimes being the dumbest guy in the room is a great asset because you’ll ask questions that many others may not think of because they take a lot of things for granted.
Most of the people in this industry that I’ve come in contact with are unquestionably brilliant scientists and researchers, extremely passionate and brilliant and have been doing this for a long time. Sometimes a little outside thought process can be very beneficial and I think that’s what I’m like. I might be the COO but I’m not the most important guy on this rare disease team by a large margin. We have people here who’ve been doing this for a long time. We have nurses, we have physicians, we have scientists, we have so many capable people who know that any disease that we look at, any rare disease that we consider, will understand so much more about it than I ever will. Having said that, I think that my background lends itself to kind of ensuring that we stay focussed on developing a business approach to this, a commercial sensibility that can ensure we can keep doing this all the time.

One of the analogies I always use is that when you get on a plane. Before the plane takes off they always show you that if an oxygen mask drops you should always help yourself before you help everyone around you and that makes sense to all of us. If you’re not around, you can’t help anybody. My approach to looking at rare diseases is the same thing – hey, we need to move off this charitable model, it needs to be a sustainable economic model, because if it is, we can keep doing it. Sometimes you have to get scientists to kind of understand that the science is extremely important but the sustainability of what we’re doing is just as important. I think my background lends that. I love being part of this, not as a scientist but the fact that I can bring maybe some outside perspective to it, that I can ask what would seem like dumb questions because I simply don’t know any better and sometimes people go ‘hey wait a second, maybe that’s not so dumb, maybe that can work’.

I always say I could be counting beans at Starbucks or doing a lot of other things us finance guys can do, I just think the opportunity to be part of his company, and to be party of leading this charge, it’s a gift, I mean it’s a divine gift for me. I could never do anything in my life that’d be more enjoyable than what I’m doing right now and if I can be part of the team, not the most important part but part of bringing leadership, direction, focus, a big part of my job in here in this, this industry, in particular rare diseases, there’s a lot of speed bumps in the road and some of them are pretty big. You go down a path, you get very excited about something, and as you probably know, the vast majority of what we look at from a research perspective and development perspective fails and if you let yourself, you can get pretty depressed about it. A big part of my job is keeping everyone focussed; you know what I’m saying?

Failure is to be expected. I have this motto that you learn more in defeat than in victory because when you lose you tend to be more retrospective about why, so a big part of my job is keeping everyone focussed and motivated and keeping everyone outside in front of the patients to understand that there’s a real person out there that needs these medicines and when we stay focussed on that we tend to do okay. So I kind of bring all those qualitative, non-scientific things to the table and I think it’s important, again not the most important but it’s fun to be part of this team.

NGP. So finally, what does the future look like for products of rare diseases, what’s your take on what the market is going to be doing over the next few years?

GL. I think you’re going to see a lot more activity and you’ll probably notice that many of the large pharma companies are now not necessarily getting into rare diseases. I think you’re going to see over time a lot more niche or targeted therapies from all pharma and that we’re very excited about because that’s a pathway to more companies becoming more and more interested in rare diseases and the last thing we want to do at Sigma-Tau Pharmaceuticals is to own this model. There’s six thousand rare diseases, there are 5700 of them looking for therapies. We would be very successful over the next few years if we could potentially launch five new therapies or new medicines for those patients, so we need a lot of help in this area and we’re really glad to see more and more companies taking a targeted approach to developing medicines rather than kind of large market or blockbuster approach.

So for us, the changes that are going on in the industry, ultimately we hope, will produce more medicines for smaller patient populations and I’m glad to see it. We think the future’s tremendous. There’s still a large number of very debilitating and rare diseases out there that need therapies, we’re very excited to partner. You know one of the things I’m always saying is how can we be in so many therapeutic areas and I think when you’re in this area of rare disease it’s a partnership, a model, it’s a facilitating model. There’s a lot of research going on out there in rare diseases it’s just that it’s spread all over the world, it can be one or two scientists who spent many years working on one rare disease, in a lab, in academia or somewhere else and what they’re looking for is someone to help them and what we try and bring is we facilitate. We will partner by bringing money, we’ll partner by bringing clinical development expertise, so perhaps someone’s already gone through the seed or the early stage research and to get to the next level. The true development of medicine is that we can bring those skills, we can bring the commercialisation skills, distribution, manufacturing, medical education, pricing to try and facilitate because there are so many different areas that we can’t target only one particular therapeutic area. There are experts out there that know those therapeutic areas better than we ever will so we like to talk about the model a lot because hopefully a lot of other companies will look at what we’re doing and say ‘hey, you know maybe that can work, maybe that’s a great model’. Hopefully, more and more of them can take up the charge.