Francois Nader - President and Chief Executive Officer
NPS Pharmaceuticals, Inc. (NPSP) Leerink Swann Rare Disease Roundtable Conference Call October 2, 2013 11:10 AM ET
So we’re pleased to have Francois Nader, President and CEO at NPS Pharmaceuticals, which we’ve been watching execute very well lately on a number of their programs. So, we’re excited to have an update from NPS now.
And maybe we can start, Francois with a -- just an update on Gattex and the rollout. It’s done very well relative to initial expectations. I think you’ve done a great job building the market, like the most successful orphan drug companies do, finding patients that can benefit.
Can you talk a little bit about the adoption patterns that you’re seeing and what types of patients are going on to drug, and how physicians are getting experience? Can we address the sound, okay? So, what sorts of adoption patterns are you seeing with Gattex at the outset of the launch?
So, the launch of Gattex is going very well. So, I am very pleased with the outcome of all of our efforts preparing the market, and now we’re out in month eighth, I believe, just completed month eighth of the launch. And there are interesting characteristics of this launch.
First, we have, I believe one of first of only products that was launched with 1,000 patients identified at launch, and that was exceedingly important in the rare disease business, knowing that our total market in the U.S. is between 3,000 to 5,000 patients. So we had, depending how you count it between 20% and 30% of those target patients already identified at launch, very helpful.
The second element that we saw over the last eight months that is very encouraging is the steady build-up of demand. The demand has been there. What we did not see was this bolus effect that you tend to see in the first week or month after launch. On the contrary, what we have seen in this very steady build-up and the demand has been very regular, if you will, which is very encouraging.
The third element that is somehow unique was there was a concern shared by many here about reimbursement, and so far reimbursement has been for all practical purposes a non-issue in the sense that our team has been able to demonstrate the value of Gattex to the payers.
And when we look at short-bowel syndrome and we look at Gattex, this is one of those rare disease products that is not additive to the system, okay? Because we reduced parenteral nutrition; we reduce clinical care and other costly healthcare activities.
The other element that is very encouraging is the fact that our NPS Advantage, the Care coordinator, this one-on-one interaction with the patient has worked beautifully well. And if I can pinpoint one of the two or three items that made our success, it's really the effectiveness of our Care coordinator and the fact that very early in the process they have established this one-on-one communication interaction with the patients and with the payers.
And knowing that we have patients that are debilitated, that are sick, old, for many of them, and frankly the last thing they want to do is to deal with insurers. We are playing this role for them with them, and that has been very successful.
Last but certainly not least, and this is correlated to the NPS Advantage are these anecdotes. I mean, I cannot think of one day that passes by without having a patient or a caregiver or a healthcare provider calling us and thanking us for Gattex, thanking us for the benefits that the patient is experiencing with Gattex, which frankly at the end of the day is very fulfilling, very rewarding.
So our 17 years of hard work is paying off not only by the success of the launch, but I would say more importantly by the reaction of the patients and the healthcare providers.
That’s interesting. How is Gattex being used in the market relative to how it’s used in the clinic? In your first Phase III, I remember you had -- well both Phase IIIs had a very defined algorithm or protocol for calculating urine output and guiding the amount of parenteral nutrition reductions which were allowed. But then, in the second Phase III it was more open and there were less parameters, I think the ceiling or the floor was removed.
And then, now in the real -- a lot of patients in the real world have been afraid to push the down button on the pump, because they haven’t had any tool to allow them to wean themselves off of PN, even though that was sort of the goal of most people in the market. So now that Gattex is that tool that's available, how are you -- based on all these anecdotes, what you are hearing and what is your sense of the collective response in the marketplace?
Well, as we all know, when you develop a Phase III protocol, you have to be in a way strict in defining good inclusion, exclusion criterias, so that the results are clear, clean, and definitive. Clinical practice is entirely different in the sense that clinical practice is really driven by two main factors; one, the label and second the physician's discretion. At the end of the day, the physician is the ultimate decision maker.
So, in the clinical trial, just to -- just mention the two points that you did, which were the fact that patients should have been on parenteral nutrition for at least two years, if I remember correctly. And then, the reduction of parenteral nutrition was limited to 30% per month.
Well in practicality, the label said that Gattex is indicated for patients with short-bowel syndrome that are PN dependent. The label did not define, and we are very pleased with it, what PN dependent really means, and it was left up to the patient and the physician to define the PN dependency.
What we see now in practice is the fact that as we’ve seen it in the (inaudible), it was very important for us to make sure that the patient is in the driver’s seat with their physician. And what I mean by that is the patient is ultimately the one who decides on the reduction of parenteral nutrition because they have to live with parenteral nutrition day in, day out, and they manage their parenteral nutrition.
And it’s very rewarding to see the anecdotes coming back to us of patients who are already weaned off, knowing that the drug has been launched only for eight months, and not every patient has been on Gattex for eight months, yet we’re starting to hear anecdotes of patients who are already weaned.
We’re also hearing anecdotes of patients who have already significantly reduced their parenteral nutrition. So, all this is extremely encouraging, yet I cannot answer the question about the profile of the patients because this is something that we don’t collect here. We’ll be doing this once the registry is up and going, and this is expected to be before the end of this year.
Okay. And do you think that you can get all of the identified 1,000 unique patients that are in your database on to Gattex over time, and what about the 3,000 to 5,000, patients, how did you arrive at that and what is your view of the market over time?
Well, I will convey you to wish Eric Pauwels, our Chief Commercial Officer, and you have to secure those 1,000 patients. I wish it could happen, I don’t know if it will, but at the same time, we also -- we're also very happy with the rest, which is we identified the 1,000 patients, and when I look now at the scripts, interestingly enough the scripts coming from those 1,000 patients represent about 30% of the total, which is very good because it means that our field force was successful and is successful in identifying and creating demand from the rest 70%, and that’s very interesting.
I would have been concerned, for example, if 60% or 70% of the demand would have come from those 1,000 patients. So it’s very well balanced. But also the numbers are confirming that the total target population is between 3,000 to 5,000, okay? So, nice demand created on the balance 70% and frankly confirmation that the market is indeed between 3,000 to 5,000 patients.
Awareness about a year ago was lower than what we would have expected amongst physicians, although the patients seem to be pretty aware of what’s been coming down the pipe for a time, and they’re very engaged. How have you noticed the adoption patterns by center? You’ve identified centers of experience; are they all using the drug, are people starting to want to become centers of experience now that there is a tool to help people?
And this is like a true orphan disease where it happens after surgery, and these patients, once the surgeon is done with them are sort of left to their own devices often, unless there happens to be someone in the community that cares for these types of people which is rare. So how are you noticing diffusion, product diffusion is evolving now?
So we identified about 6,000 target physicians as being our total target space if you will, and then we pared down those 6,000 potential prescribers into about [1,000] (ph) accounts. Of those 1,000 accounts that we’re targeting are dividing in two groups. Again, simplification here. There is probably anywhere between 60-70 centers-of-excellence, and you have about the rest, which are in general large GI practices.
The good news now that we are in eighth month into the launch is we’re able to tap into the centers of excellence and the large GI practices as well. And for me, one of the metrics for success is the fact that many of the patients that we currently have are coming from the large practices and not from the center of excellence.
And this is counter intuitive, because one would expect that most of the patients would come from the centers of excellence because many of them actually were clinical sites. The reason I am happy that we have a lot of patients coming from somewhere else is the fact again, it's another metric that shows that our field organization is doing -- they are doing their job and they are doing a terrific job really at mining if you will the rest.
Now needless to say as well that the large centers of excellence are extremely important for us because they host a larger number of patients. Interestingly enough, what we are noticing is whether it is a large clinical practice or a center of excellence, there is a practical logistical limit to how many patients they can enrol in any given time because these are relatively small units and they hit the bottleneck really quickly.
Okay, so we’ve seen adoption from centers of excellence and adoption from large clinical practices practically has the same rate which is in a way counter intuitive, but that’s the reality of the market, yet the centers of excellence per unit per account would have a much deeper reservoir because they have a larger number of patients, which might explain in a way the linearity if you will of the demand that we are seeing.
In other words, we did not have the bolus and the demand is building up in a very much linear way which is very good because this means that we are here for the long haul.
Yeah. What do you expect the trend of discontinuations to resemble, will that be as steady, as people who decide that they are not benefiting enough to justify the daily therapy or just for whatever reason, fatigue, as is human nature, after sometime, do you expect that to be steady and linear too or after a year do you think we’ll start to see a bolus, any thoughts on that?
I mean if I look at the first eight months, the surprise that we had internally, to be candid with you, is the higher rate of compliance and adherence. I mean at our last earnings call, we said it’s about 95%, and today it's still in the same range in comparison to the 70% to 80% that we guided on, which was frankly not a number that we picked up from some ad but these 70%, 80% was actually the clinical program compliance and adherence.
So interestingly enough and contrary to what happens usually we are finding the adherence compliance in real clinical practice is higher than in clinical trials. So we did a little bit further trying to understand why and we came up with at least two explanations. The first one is contrary to the clinical trial where patients would see that investigator or the clinical trial monitor or the nurse every other week in the beginning and then every month or every three months. Here they have access to the clinical coordinator literally anytime of the day or night.
So our clinical coordinator, our Care coordinators are only one call away and this goes both ways So these patients are monitored and are supported literally on a daily basis and interestingly, not as a consequence, of when they face an adverse event or something that bothers them, it's an immediately reaction -- interaction with the care coordinator that makes the huge difference, rather than a patient saying I am giving up and I am out of here.
The second element that is extremely important is when you look at analogues, one of the pre-determinants of compliance and the adherence is the out of pocket. Contrary to the traditional belief it’s not the cost of the drug, contrary to the traditional belief it's not the culture, it’s really literary the out of pocket and we defect or eliminated this by limiting the out of pocket to $10 per month.
So if a patient tends to save money by extending their monthly supply through 45 days of the month actually they are gaining what $2, $5, $3, it’s not meaningful for them.
The third element which is important is for a reason that we cannot fully quantify and explain scientifically, when patients are put on Gattex they start feeling good, they start feeling better, okay and it’s very difficult for us to quantify the why but it’s the reality of the feedback we are getting from the patients.
Interestingly enough when they [subject] that and before parenteral nutrition starts to go up again they start to feel not as good. So there is also this feeling good factor that plays in favor of patients sticking on Gattex and continuing to take their daily dose.
Speaking of symptomatic benefit, you’ve got another program which Natpara has been associated with, some symptomatic benefits in a difficult disease that results from surgery, the thyroid gland or the parathyroid, -- and/or parathyroid gland. So when we'll learn some more about your views of the Natpara opportunity and what you’re doing to prepare for launch?
Well, we hope to file Natpara in the U.S. before the end of the year, so fourth quarter and technically the fourth quarter started yesterday. So we’re actively working, assuming that the FDA will be open for business because as we know for the moment, the FDA does not receive new applications. So hopefully this issue will be solved in time for us to file.
But also we are planning to organize a Natpara day after we file. In this Natpara day we will give an overview of Natpara clinically but also -- hypoparathyroidism clinically but also the clinical development program and we will give more specifics on our pre-launch activities and the market potential.
What we said though is we believe based on the data we currently have the global market potential for Natpara will exceed Gattex.
Okay. Well, we look forward to hear some more and we didn’t get a chance to talk about the rest of the world opportunity but that also is expected to be as big or bigger in the U.S.
For Gattex or what we call Revestive ex-U.S., we believe that the opportunity will be bigger than the U.S. opportunity.
Okay, great. Well, thank you for the update Francois and keep up the great work.
We’ll do. Thank you very much.
[No formal Q&A for this event]
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