Q1 2022 Pharming Group NV Earnings Call
As we expect for 2022 over 2021.
And there is a very important part in time for the company here because we are having a near term inflection point in our hands with the possible launch of our in licensed compound Lenny ownership from Q to Q1 2023 to treat the orphan disease, a pds and that will of course be a significant.
Different company after that because then we will be actually having two products in multiple geographies.
Im not really going to rely on one product that is mainly deriving sales from one geography, the United States.
This is a.
The rare disease.
Estimated according to the literature.
Some 1350 patients as you can see from this slide and we have started at a relatively modest level with finding these patients and we have already some 400 patient set.
Defied that could be become eligible for the treatment with linearity ship once approved of course.
We will indeed.
Further intensify the search for these patients going forward for the rest of the remainder of the year.
And we have this.
Establishing especially as commercial infrastructure in the U S and Europe that is why we are quite confident that we can make a commercial success out of out of millennials.
And of course, we are looking as we well know from previous calls we are intensively looking for additional late stage opportunities to in license and further bolster our pipeline.
We also work on early stage R&D projects and most notably we have last year in licensed a potentially curative gene therapy candidate for Herritarrok Angioedema, OTR 105 from Orchard Therapeutics, and we will of course update you towards the future as and when important milestones have been achieved in the program.
So we of course are also working on further lifecycle management opportunities for our compounds that we have in house, which include linearly Sip, which may be possible to actually do actually be developed in additional indications other than a pts and last but not least we have this experienced leadership team and a strong balance sheet.
<unk> can actually support our growth strategy.
We'll come to that later, but it is good to say here that the current development plans in the current portfolio that we have requires no additional financing. So how does it look like a please turn to slide number five fees how does it look like.
In terms of our strategy for growth on the left hand side you see on this slide number five the commercialization of <unk>, which is obviously ongoing and is supporting this business and ISI was saying before the launch of lineage <unk> will totally transfer our company transform our company from this one protocol.
One geography company to this multiple geographies and multiple product company, which is very very important step forward and also we expect to be included.
We expect expect to be including Japan, where we have no presence, yet and Japan, obviously as we know is the second biggest pharmaceutical markets in the world.
Then you see on the near term expansion of our portfolio as I was already alluding to that we are actively hunting very intensely for additional in licensing opportunities for rare disease compounds that are in late stage development. So that we can actually establish a launch agenda.
Beginning next year with linear ownership and following with additional compounds to be in license in the subsequent years thereafter, but also in addition, we can continue to work on the development of C. One inhibitor.
I was saying linear obviously for additional indications and then theres interesting aspect here to being entrant entering into this primary immune deficiency domain and starting a systematic patient finding exercise.
Offering genetic testing, we have access to a growing amount of genetic testing.
From those patients with primary immune deficiency patients that undergo testing for Aps. So we could actually start seeing and of course. This will continue to go on that towards the future. We can start seeing patterns of certain mutations that are there and therefore, we can have a for the long term a more direct its business development efforts slash R&D.
<unk> to actually address more of these primary immune deficiencies, because we believe and it's of course, a known fact that worries.
About 400 genetic defects there that actually you will drive that actually our underlying these primary immune deficiency. So we think there's a lot more to be done there and to help patients that are currently without any treatment as our patients in Aps that are waiting for their new ownership.
In addition to that of course I was already alluding to we working on OTR 105 are.
Our in licensed compound for the.
<unk>.
The potential curative treatment of Ara, Terry Angioedema and lost Middle East. We also have offer glucose days, a new version of enzyme replacement therapy for Pompe disease, which is still a big unmet medical need.
I'd like to go to slide number six and you may actually go immediately to slide number seven because I will be briefly discussing here.
<unk> in the heritage Angioedema markets as we have discussed before <unk> of course approved for the acute treatment.
Hello, Terry Angioedema attacks and that means that in this.
Speaking about mainly the U S market, where a prophylactic treatments have become more and more popular over the last couple of years because the good news for those patients as a prophylactic treatments has significantly improved efficacy wise and convenient swaps.
However means still that old prophylactic treatments are still not totally watertight.
Still a significant portion of patients and if you read the published the results from clinical trials. It is up to almost 50% of patients five zero percent of patients that are still suffering from breakthrough attacks under prophylaxis breakthrough attacks and varying frequency.
But it is a still a big unmet.
That need and is exactly where <unk> fits in because the paradigm shifts away from prophylaxis from so you wanted the ambition back in the days, which was the original prophylactic treatment.
Therapy to bradykinin Conakry inhibition it exactly why is very rational to give patients.
One of the better as breakthrough medication in case, they have breakthrough attacks.
And this is exactly what we see here, whereas traditionally we were serving only the right hand segment here. The severe end of the market because we are a late entrant and we offer a protein replacement therapy very highly dose and people started to actually use this started to use our compound because they couldnt get anywhere else at the steel still the core.
Our business, but we see an increasing number of patients being treated.
We'll be at a lower frequency, obviously than if you are severely affected.
<unk> and.
An increasing number of patients treating their breakthrough attacks with with <unk> and as it represents that middle.
Part of the of the of the slide towards the left the little dots.
So in other words, we see a consistent increase of physicians prescribing <unk>, we see ecosystem increase of patients using <unk>.
Underlying underpinning part of the growth that we have that we have in the U S market.
And also on the next slide number eight you can see that of course illustrated what I said just before is that thanks to the increasing efficacy and convenience of prophylaxis, you've seen a gradual increase of the percentage of patients that are actually using prophylactic treatments, but as you can.
Also see during 2021 that seems to be more or less stabilized at around 70%, whereas the.
The acute segment.
Has almost.
Stabilized at around a 30%, having said that is very difficult to market.
Muddle this markets because as I was just saying before all patients that are using prophylaxis will have if they of course adhere to that.
To that treatment plan will have breakthrough medication at hand at all times, because herritarrok entity is a very unpredictable disease and breakthrough attacks can happen at any time. That's why it is very difficult to actually segment. This market in two parts like that because it still means that all of these patients will have almost all of them will have indeed.
The acute treatments enhance even if they are using prophylaxis, so far the heritage angioedema market and as I was already alluding to earlier as this is a very historical moment for our company because we have for the second time in our history, we have positive pivotal data for our new compound we can bring to the market.
I would like to invite.
My colleague Dr. <unk> <unk>, our Chief Medical officer to take you through the latest presentation.
On the <unk>.
<unk> and <unk> over to you and over to slide number nine I suggest.
Thank you Simon what I'd like to do today is quickly review the pathophysiology of Etfs and then share with you some of the recent data that we.
<unk> presented on lineal is intriguing.
Yes patients from our clinical studies.
Please go to slide 10, we can see the pathophysiology of PDF and on the left side you can see how a hyperactive <unk> K delta partly due to the generic effect that these patients have.
Thanks to this abnormal development of their immune system and specifically the <unk> cells.
And due to that abnormal development. These patients with Atvs end up with a whole slew of symptoms any that are consistent with.
With other primary immune deficiency. So first of all of course, they get recurrent infections with a prominent feature of the <unk> patients.
We call them for proliferation and abnormal.
Development of fees.
The local site and that leads to our patent off a piece of swelling of the world large spleen delivers and just abnormal lymphoid tissues throughout the body.
Also have problems with auto immunity.
And due to the recurrent infection because they have problems in their lungs, specifically something called bronchiectasis and a whole slew of other problems, including Gi and even neuro developmental problems.
And down on the left you see one of the other serious consequences that these patients. Unfortunately frequently developed which is a lymphoma and this is due to stock.
Thought to be due to that abnormal proliferation uncheck.
All information that these patients have.
On slide 11, and slide 12 excuse me.
I want to walk you through the.
Studies with lineal.
So on.
On the left side you can see part one which was a dose finding study in the first completed some time ago and these were six patients that were treated with three different dose levels of <unk>.
Based on that study.
Dose of 70 milligrams twice daily with selected for part two which was the placebo control.
And on the bottom right you can see is the open label extension study patients from part one and part two we're able to then enroll in the open label extension study, which allowed us to collect long term efficacy and safety data on the use of language today, we're going to be focusing on part two but.
Year moves on we will be receiving more data on the open label extension study and be able to share that as well because I think this will be another important part of that.
The data package.
Our review will be how these patients do over the long term.
So if we go to slide.
The next slide we can see the demographics for these patients and we can see is that the patients first of all our young patient population with a median age around 20 years with.
Nearly 40% of patients below the age of 18.
And you see the other demographics there on the slide showing similar frequency across.
When you also have and placebo treatment arms.
And on the bottom left you can see in the overall population again.
Population of patients that are quite ill.
A history of numerous.
Complications from Atvs, including ones that we just talked about will improve proliferation infections.
<unk> issues.
Cytopenia, which are the autoimmune.
Got it.
And then again the issues related to the enteropathy.
On the bottom right you can see some of the other.
Each manifestations that these patient trial, including neurological symptoms.
And then.
Paul proportion of these patients were even tried with off label therapy, using a drug cultural elements.
Our next slide we see the first of the co primary endpoints presented and this is a measure of the lymph node swelling and you can clearly see that the lineal foot patients had a significant decrease over placebo.
Obviously statistically significant.
And when we looked at what we call the SPD or some of product diameters and the index lesion, we can see that the endpoint was met.
Statistically significant manner.
On the next slide you can see the distribution of the patients.
Across the two treatment groups. So the lending Elisa patients here are shown in blue and the placebo patients are shown in gray and we see that the millennials with patient.
Obviously, a significant decrease.
As we saw on the previous slide, but we can see the significant change from baseline in there and the size of these so called index lesions.
Cross patients and you can see the placebo response as well so most of the placebo patients had little change or some of you have had a significant increase in the size of their <unk>.
Explanations of these lymph nodes and there were of course, some other legal questions that placebo response. During this 12 week period, but if we can see a clear separation of the two treatment groups.
In the aggregate when we look at the.
Like we saw on the previous slide, but even on a patient by patient basis.
A clear separation across these.
The two treatment groups.
And the other co primary endpoint in the study was the proportion of naive b cells and here again, we see that the <unk> treated patients had a significant increase in the proportion of naive T cells versus placebo treated patients and naive T cells as a reminder, our b cells can.
Develop and mature properly and respond to infections. So these are important part of the maturation process and.
In these patients you see on the right part of the slides that they have a relatively low proportion.
Of these T cells at baseline, but soon after treatment with Lenovo that number increases it stabilizes and stays at that level, whereas we see almost no change here.
<unk>.
In the naive b cell proportion in the placebo treated patients so that we have both the.
Co primary endpoints being nothing a statistically significant manner with a clear separation on the.
Efficacy lineal across these endpoints.
On the next slide we can see some of the other manifestations what I mentioned.
<unk> patients traveling and that is again because of this abnormal proliferation.
And kind of a large screens on deliveries and we see that even in the course of this 12 week study, we see significant changes in <unk> treated patients compared to placebo treated patients the placebo treated patients.
On the right panel you see had a slight increase in the size of their spleen, and liver and NBA Street millennials, who treated patients had a significant decrease in again these were statistically significant changes observed in.
In this 12 week study so quite quite impressive even with short duration, we see.
Improvements in these measures of proliferation.
Actually the macro stuff and then they can see that.
Gains in the livers.
Getting smaller.
On slide 22, we can see that when you all's it was generally well tolerated.
Safety profile across the different grades of adverse events was similar between lending relative to placebo.
Study there were no deaths reported there was no discontinuation due to any adverse events and none of the serious adverse events were thought to be related to study treatment.
And you see even the frequency was lower millennial listen treated arm.
As compared to the placebo treated patients.
And lastly, with the <unk>.
I've shown before but I think it's quite impressive.
This is now thinking back to that those first six patients those patients recall they went on into the open label extension study and so this is solve their long term data. This is actually data out to two years.
Actually have data now and we are analyzing this data for these patients out to five.
Six years, because these patients have been on therapy.
Since the beginning of the development program and you see here, they're IGF levels.
In these patients.
This abnormal development of their b cells. They frequently have high levels of IGN and you can see that in the six patients when they were treated with <unk>.
GM levels dropped and some of these patients weren't able to enroll in the study immediately and you see that if those gas lines those patients had to discontinue appropriate period of time and then the dosage interruption.
And you see their IGF levels increase but once those patients resumed.
And.
All of them were able to resume you see goes.
Dash lines in the solid line now and those IGN levels decreasing again, so I think.
Quite a dramatic showing here of the response.
IGF levels, two lineal feet across these six patients, but I think probably the most important part of this is that these patients were able to.
Many cases.
<unk>.
The use of IV supplementation therapy. So this is IV or subcutaneous replacement therapy.
Because of their immune deficiency, they were actually able to stop this.
Because there are b cells are now normally functioning so I think.
This is another quite a dramatic showing of the effective lineal have now and a longer term setting and.
As I said earlier, we're going to have data on the patients. So now this will be.
<unk> 35, or 37 patients that we will have data on across.
This longer term endpoint, so we will have.
This data presented throughout the course of this year at some scientific symposia and meetings.
And I look forward to sharing that with you.
Okay.
And here the presentation on <unk> touching on something else on slide 24 talking about.
The launch preparations in terms of finding patients.
Again, <unk> is a relatively recently defined disease back only in 2013 with the genetic mutation identified.
Doing quite a bit of work now to help educate physicians about Aps and also build out a network of doctors, who treat these patients.
Sure.
<unk> in primary immune deficiency, we're also doing some.
AI work to help identify patients based on the symptom profile at Aps patients out and then again quite a bit of education all centered around the navigate atvs program that allows for.
No cost genetic testing for patients that may have <unk> so that.
Theres no barrier for these patients to obtain a genetic diagnosis that allows them to have a specific treatment option with potentially available to them in the future.
And I will turn it back over to you Simon to walk us through some of the milestones that are coming up for the <unk> program.
Thank you very much <unk> and yes, please slide number 20.
The AUC the upcoming milestones that unrecognized team are working very hard on and other commercial teams. The commercial teams as well of course to prepare themselves for launches.
Here at <unk>, we're working towards the first of all the FDA filing of the of the.
Compounds followed after the summer by the EMA and British filings then.
<unk> studies are very high on the agenda to start later on during the second half of this year.
As you know.
There is already patients included up to until the age of 12. So we're looking at the below <unk> and there will be two pediatric studies starting in this perspective.
Im.
As I was alluding to earlier, we're very excited about the prospect of making the move to Japan with this compound.
We've had incoming requests from Japanese opinion leaders to please come to that to come and do a small trial, we had discussions with the Japanese authorities and they are all very cooperative and we anticipate to be able to do this year started out small Japanese clinical trial is required for Japanese authorities to take this under review and then.
Of course, we're anticipating the big moment, assuming this accelerated review by the FDA, which calls for an eight month review cycle that we will be able to launch the product.
I'll get it up to <unk> data in the first quarter of 'twenty three.
Quickly followed by the by the U S launch and then of course, the Imam because it was filed later will follow on later on with.
Within an approval and then subsequently to that in the second half of next year, we anticipate the launches in the EU countries to be rolled out across the EU. So we're very excited about this prospect as I was already you're saying this is the roads. The route map two.
Totally transforming the company to us multiple company in multiple geographies.
Take place during next year for which we are of course investing very heavily speaking about investments. This is probably a good moment to switch over to our chief Financial Officer uterine walk among we're sitting next to me here and we May actually go to slide number 21.
Thank you very much indeed.
On the next slide you will see the key.
Quarterly development of revenues from <unk> over the last two years and we're very pleased to say that we increase the revenue in Q1 2022 by 7% to 46.6 million U S dollars.
And out of that.
May not be a surprise to you most of it is from the U S revenues $45 3 million out of the $46 6 million of revenues was from the U S.
That meant an increase in the country of 7% in revenue growth.
And the increase.
What we saw in the in the number of patients was a driver of it.
And it was slightly offset by tighter inventory management.
Large specialty pharmacies.
And as you see.
The EU and the rest of worlds are still small.
It was another strong quarter for <unk> and the rest of the world, but that was largely due to phasing of orders.
The gross profit increased by 8% to 41 7 million.
And that was obviously, mainly due to growth in revenues, but also the margin obviously increase with this growth number in that.
It is a combination of price increases.
Unless discounts that we have to offer so.
Margin improvements.
Going to slide zone.
Please yes.
The operating profit.
<unk> decreased to $2 8 million from $6 3 million last year and that was because of the expected increase in operating expenses.
The operating expense increased to $39 8 million Thats, an increase of about $7 million.
$7 million increase was a combination of several things, but things, but half of it was because of the launch preparations for millennials at all the elements are increased travel activity in.
In the U S.
Post COVID-19.
The phasing of costs.
I think this is doing wrong slice.
Okay eight.
Now.
Thank you.
And a number of initiatives.
Okay.
Operator could you put up the slides.
Please.
Yes.
The financial highlights from Q1 slide three alright, three out of five correct, yes, yes.
That's weird.
Oh, sorry, just just go one back.
We had a technical glitch here. Thank you very much so I know it.
I was saying that the operating profit decrease because of the cost increase and that was all of it was because of the announced increase in cost for millennial ship.
Launch preparation.
Net profits.
<unk> decreased.
Two to $3 5 million and that was caused obviously by the operating expense increase as I just mentioned, but also a decrease in finance income from last year.
Last year, we had a big foreign exchange effect because of the eurodollar.
Volatility.
And we didn't.
<unk> gained as much from that.
This year.
The cash position is still very strong it decreased by $2 2 million, but it is still at $189 7 million.
The positive cash flows from operations amounted to positive $1 6 million and that is obviously, increasing the so including the.
Increased costs from millennials launch preparation.
On the next slides we see.
The summary of this again.
A profit before tax going from 12.8 to $4 two.
That was caused by.
Underlying.
This business gross profit.
$3 million, mainly from the U S.
Increased R&D expenditure because of millennial ship and some brands and that was related to phasing.
The slight increase in G&A expenditure and most of the increase in marketing and sales expenditure is also due to Lenny Ellison financial results from the change was due to foreign exchange.
<unk>.
Last year financial results were $6 6 million now one H and therefore, we come to a profit before tax of $4 2 million.
And on the next slide you see some more detail on the on the cash and cash equivalents again, a decrease of $2 million.
And that is because of the increase in operating cash flows offset by working capital changes.
We invested very little in Q1 that is expected to go up in the next next few quarters.
On the financing cash flows is mainly interest in lease costs.
So again, a strong cash flow strong cash position.
That we will amongst others useful for Gilenya ownership launch preparations.
And with that I would like to go to the next slide please to the outlook and hand over to Simon. Thank.
Thank you very much shoen and yes, the outlook, ladies and gentlemen has been has not changed from the previous.
Outlook that we have.
As we have announced as a result of our full year results. So we continue to guide for single digit growth in the group revenue Smith from <unk>.
Quarterly fluctuations in revenues are to be expected, though.
We still as already stated we are on track for Q1, 'twenty three launch of Florida, <unk> subject of course to regulatory approvals.
The company will continue to invest as you have seen.
<unk> launch preparations.
And the clinical development, obviously for the trials that I've outlined before and this will of course significantly increase as you have seen a significantly.
Impact to profits as we have seen already.
But it is also to be stated and I said it before but the <unk> cash flows will be able to fund. These investments so no additional financing will be needed.
To execute on all these sell these stakes.
We will also continue to invest in potential acquisitions or in licensing of new late stage development opportunities as I alluded to before.
In rare diseases.
And of course, if you think about in licensing, but especially acquisitions. There may be additional financing required which of course can then come from our strong balance sheet access to debt capital.
And access to a eventually equity capital as well certainly in the case of acquisitions.
And last not least we will continue also to be able to focus on the strategic development of the company going forward.
Think about.
The additional indications for instance for a compounded plenty ownership that can be can be can be explored to ours in the future and of course, our early stage compounds will continue to be developed so without that I would like to conclude this part of the of the.
Managing and go off to the next slides and switch open the floor for questions.
So everybody who is attending thank you very much operator, please open the floor for questions.
We will now start the Q&A session, if you'd like to ask a question. Please press star followed by one on your telephone keypad.
If you Michele a question. Please press star two if you have joined US online. Please press the red flag icon.
I'm sorry to ask a question. Please ensure that your line is on mute to like Helane.
And now first line comes from Jay <unk> at H B.
Please go ahead your line is open.
Hey, guys. Thanks, a lot for taking the question hope everything is well over there and really hi, Joe Hi.
Hey, there.
Three questions are really focused on I guess, what you'd call background activities. So.
For lineage <unk>.
What can you describe with regard to ongoing prelaunch activities and you consider any of those.
Rate limiting at this point and the second question is for <unk> 105.
Do we anticipate any preclinical news from that program.
Over the next 12 months and then the third question is.
How would you describe the maturity of your discussions with regard to potential in licensing.
Okay. Thanks, Joe I'll start with the first one.
Relaunch activities.
I don't think theres any rate limiting steps in there obviously prelaunch activities include regulatory activities to prepare to the.
File for submission.
But also not to forget there is an important element here as well that the market access files for our European markets.
Our in development that's also.
Very intense and.
Most importantly.
I would say be patient finding activities.
<unk> was alluding to already.
They will be a further increase.
And as the logical thing of course as well as your as and when you gain more confidence with regards to their profitability of your file and it is currently the case.
Start intensifying b patient searching activities, both in the U S and in Europe .
See any rate limiting steps there.
At this point in time, so we're going full speed ahead in that respect.
With regards to our OTR 105.
Within 12 months, yes, there could be some updates I expect from OTR 105.
As a as milestones preclinical milestones will be will be achieved.
So, yes, I would say within 12 months, we can actually anticipate some some.
Some news on that as we progress towards getting into IND, enabling studies I would say so what's this space, we will of course update the market as and when there is results.
Now with regards to in license seeing slash acquisition discussions.
From time to time.
We have indeed conversations with with companies that have interesting assets.
For either in licensing or acquisitions.
At this point in time some of these things are quite advanced but of course as you all know.
And Joe There's no news to you either but it means nothing until such time that the ink on paper and that.
It can be a deal can be announced so we have a very I would say efficient business development process.
Also the company because we believe it is in a very very important spear point of our activities as we have developed this commercialization.
Upper office on both sides of the oceans.
And it is a very valuable.
We have and it's very important to key.
No.
Skill set that we have onboard and expertise and therefore it can be leveraged. So we consider this a core competency of our company and therefore, we put a lot of emphasis on this and it is very difficult to say of course as and when a deal for an in licensing or an acquisition can be closed.
Be sure that we have a lot of attention for it I Hope I answer your question Sergio you.
You did I appreciate the color thanks Simon.
Perfect. Thank you very much for your question. Our next question comes from Jeff Stein of Oppenheimer.
Please go ahead.
Great. Thank you for the question and really nice presentation.
Only 719.
No.
Three questions. One is just a couple of follow up.
To the previous question about Joe.
In terms of planning for the line of Mr launch.
On Iraq, and Simon how is reimbursement going to work out I mean are you doing perfectly economic modeling for example in cancer BMT TPN guidelines are very important.
How will reimbursement and preparing for that.
Play into your plans for the launch both in the United States and Europe . So that's number one.
Number two you know in some of our Q all calls recently.
<unk>.
We were told that there is.
Relative tend to also be at risk for this disease.
Are you thinking off potential screening relatives of patients in the future going forward.
You can catch these patients early on.
And then lastly, what's the size of the Japan market.
Or if you were to enter their apartment. Thank you for the question.
Okay, perhaps I shall try first say something about reimbursement for lending ownership.
So yes. We are this is of course, a very important aspect we are developing.
<unk> files in the four months at the various European markets require.
We also.
Of course, I'm thinking of ICD 10 code already.
In the U S.
For this for Aps.
And yes, so we're working basically speaking on all those fronts and important part of the prelaunch preparations and if unrecognized as more core to that feel free with regards to the screening relatives.
Relative to I would like to hand over to <unk> on this respect in a moment and Japanese markets mostly.
As we see box already see that without any systematic search activities. We found six zero 60 patients in France, we therefore have one in $1 million as our minimum incidence for this disease.
Moment.
Then you would say that the Japanese markets would be at least 125 patients that are that are there are I think it's $125 million the population of Japan.
But again.
One of the millions or minimum incidence rate that we think without systematic.
Searching and hand over to <unk>, maybe you could say something about the screening are.
Any of the other things to give more color on <unk>.
Yes, Thanks, Tom.
Absolutely hard times.
The family testing is a big part.
Our search efforts.
The program.
Navigator <unk> genetic testing program no cough program include the provision for family testing.
Patient is identified.
Those numbers are able to get tested also for no charge.
Also doing a lot of education around this front in terms of family testing.
So that is.
Currently and we will continue to be a big part of the search effort.
PDF patients.
Great. Thank you so very much for the question look quantity update.
Thanks, Patrick Thanks.
Perfect. Thank you so much for your question and as a reminder, if you would like to ask a question. Please press star followed by one on your telephone.
You bet.
And our next question comes from Christian Glennie of Stifel. Please go ahead.
Yes, good afternoon gentlemen.
Gentlemen, thanks for taking the question first one on <unk> just wondering.
I'm wondering if you can talk a little bit more about this impact of tighter inventories.
In the U S, whether that's a transit transition or anything or whether that's something a bit more long term.
In the sense that what might sales growth have been if it wasn't sort of correction in the market.
Would you expect a bit more normalization.
Buying and inventory levels through the rest of the year or was this a shift.
Yes, that's always a difficult one that Christian because inventory levels.
Our part of the of the game I suppose so one quarter, you have a little bit of high inventories and the other part.
They are a bit low inventories, but we noticed it this time.
A decrease in inventories because we obviously keep very good track of it because it's only a few of these.
These pharmacies that we're dealing with here.
And I don't think there's any sort of systematic.
Being here, but it was noticeable here.
It was a it was a lowered hence we basically reported it as we should be of course, so there's nothing behind that as I was saying we still are.
Confident about the <unk> business, because we see an increasing number of patients in an increasing number of physicians prescribing. It we see the necessity and everybody seems to be gaining a precedent necessity that it is good to have a C. One inhibitor onboard as breakthrough therapy, when you block the bradykinin <unk> cream in them.
<unk> access by means of your prophylaxis, which was of course.
Those are very long term thing when people have to switch from <unk> inhibition.
And brought it any color at getting an ambitious breakthrough to the other way around that takes a while and it's a slow process, but it continues to go forward as we were predicting I would say back in the days and as we see now developing in the market I think that's all I can say about this at the moment to Christian.
Okay. Thank you.
Turning to Lillian It then.
Yeah.
Couple of things firstly on the patients identified in some of the numbers that I think you said now 400 previously that was at 350, but I cant remember whether that was the start of the year, whether you sort of you added about 50 patients in the cultural or something like that is that about right and then.
That implies about 30% you've found about 30% of the patients in the U S.
And in Europe .
I mean, how high do you think that the diagnosis rate.
Go here.
Well as I was saying before we are already quite confident about the fact that we could see the 1% of the population having these disease.
With further intensification of the of the activities in the market I E. We will be sending more people.
More colleagues into the market to actually start to go into these immunology clinics.
Trying to find these patients that are already being treated.
But that are not diagnosed so in other words, we expect of course that this growth will at <unk>.
<unk> continue or.
Maybe further accelerate but we're certainly going to intensify further activities because we have of course gained more confidence over the last months in millennials you'd be an approvable product and it goes on both sides of the ocean.
I'd say, it's happening in the U S and it's happening in Europe as well, although in Europe , as we know slightly different organized.
Obtaining genetic testing is a little bit easier and often reimbursed.
But there again, we are working with a special especially as centers of excellence.
<unk> immune primary immune deficiencies.
Inefficiencies towards these.
These patients and their families of course, that's an important aspect as well.
The answer to your question.
Thank you and then I just wondered if you had yet done any survey work or market research around.
The 400 OSA patients that you've managed to identify.
And that that physicians I guess that the doctor is what percentage of those.
Would indicate that they would prescribe lenny illicit.
Sort of standard survey what might be.
Yeah no there is.
Our pandemic has something to add obviously, he can but I'll try to answer this first.
One of the things is of course the patients that are eligible for treatment are currently to 12, if the product gets approved from the current adult share of course, there is 12 years and older. So we will have to for the pediatric study we have to wait for the pediatric studies of course for the pediatric population. That's one thing of course, but as far as I know and see.
<unk> from patient narratives and so on.
Patients that are eligible for treatment.
All very interested in getting the treatment as are the doctors because there's simply nothing there in Iraq.
More color on it because he's been so.
Visiting these conferences where.
Amongst others. The data was presented and he picked up a lot more on that backdrop, maybe you can give some color on <unk>. What do you think about the willingness to prescribe slash interest into treatment.
Yeah, absolutely the data has been well received so far from when we presented at Ash.
National conferences, both in the U S as well as in Europe .
And the feedback that we're getting from physicians has been quite positive.
These data are impressive include.
Including some of the long term data that I showed today.
Especially the ability for these patients to potentially be able to stop IGT replacement therapy. This is.
This was something that was viewed quite positively by.
The immunology community. So I think the when they see these data and they see their patients the feedback that we're getting with again.
If lineal feet boots available this would be something that we'll certainly consider for these patients.
Okay. Thank you.
If I can just just finding on the regulatory side with linear.
<unk>.
Is it possible to say at this stage, whether you might.
Thanks to an advisory committee as part of the review process and then is it your understanding that this indication and lending area and the approval would be applicable appropriate for a priority review voucher and if so is that a voucher that which being farming's hands or in Nevada.
Occupancies.
All right stay out and pricing will be industrial.
Maybe I can hand over to Iraq first for the regulatory.
Questions.
Yes, so in terms of the Advisory Committee.
We are just the beginning of the process in terms of we haven't actually filed yet so the FDA will notify us.
After they review the application whether they determine whether an advisory tools necessary. So we've got an FDA capable hands of course, we are preparing.
In case that.
Advisory Committee, if needed we would be ready for that.
But this was this is really fda's decision based on their review of the data that they haven't received final yet.
We don't have any determination on that yet.
And then I think Simon do you want to make a comment about the priority review voucher.
Yes, yes, yes, Cristina we do think it will be a qualifying for Nox and in case, such a priority voucher will be.
We will be granted then obviously that.
It is.
I think let me think about the contract precisely that will be in the hands of Novartis, but we have agreed a certain split of the value of such of such a voucher between Novartis and us.
Commensurate to I think the.
Investment in the compound as and when the compound gets approved so yes. It is there and yes. There is some value in it for farming, but it's an undisclosed amount as you may appreciate.
Okay I appreciate that thank you.
Perfect. Thank you so much for your question question.
And as a final reminder, if you'd like to ask a question. Please press star one on your telephone keypad.
And our next question comes from Simon Coles at first <unk> equity. Please go ahead.
Yes, Hello, Thanks for taking my question.
One.
I was just wondering when we might get the next piece of news flow on the Pompe program.
That's a good question Simon I would say that later on in the year.
<unk> definitely but it's moving more towards the end of the year, we will definitely give some more updates on the on the Pompe program, but again it is not one of the main value drivers as you can appreciate because it's in preclinical right.
And yes, indeed of course.
Yeah. Thank you.
Okay. Thanks very much.
Alright, thank you.
Perfect. Thank you so much.
<unk>.
At this time there are no further questions and I would like to pass back over to Simon for any final remarks.
Thank you very much Victoria, yes, ladies and gentlemen, thanks for attending this as you have seen we.
We deliver on our.
On the first part of the single digit growth expectations that we have for 2021, we also.
F.
Informed you and are executing on increasingly investments.
Ahead of the launch of land you always have which of course, it will transform our company significantly.
It's a multiple product company with multiple markets, where we get our revenues from so we significantly de risk our company going forward, where it also that we are very much hunting for additional late stage assets, because we have the balance sheet to do this and we have access to debt capital and eventually in the case of acquisitions for.
Sure.
For even for equity capital as well.
And last but not least it's very important to note here that executing on our plans on the current portfolio does not require additional financing as you can see so with that said I would like to thank you for your attention and I would like to.
Look forward to updating you again on the progress of the company as and when we have our first half results, which will be in the first week of August if I remember well. Thank you very much and goodbye.
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