Q2 2023 Pharming Group NV Earnings Call

Operator: Good morning or good afternoon all, and welcome to Pharming's H1 2023 results call. My name is Adam, and I'll be your operator for today. If you'd like to ask a question at the Q&A portion of today's call, you may do so by pressing star followed by 1 on your telephone keypad. I will now hand the floor over to Sijmen de Vries to begin. Sijmen, please go ahead when you are ready.

Good morning, and welcome to <unk> first half 2023 results my.

My name is not doom and opening of a pinch it for today.

You'd like to ask a question during the Q&A portion of today's call you may do so by pressing star one on your telephone keypad.

Now hand, the floor talking to freeze to begin to Simon. Please go ahead when you're ready.

Sijmen de Vries: Thank you very much, Adam. Good morning or good afternoon, ladies and gentlemen. Welcome to our Q2 and H1 2023 Financial Results Call. Next slide, please. I'm here with my three colleagues, Dr. Anurag Relan, our Chief Medical Officer, Stephen Toor, our Chief Commercial Officer, and Jeroen Wakkerman, our Chief Financial Officer, to take you through the highlights of these results and, of course, to answer all your questions that you may have afterwards. Before I do that, of course, I would like to point you to the next slide. Well, the slide that says something about forward-looking statements, because we will be making some forward-looking statements, of course, today that are based upon our current plans, belief, market circumstances, et cetera. That may, of course, change towards the future.

Thank you very much Adam and good morning, or good afternoon, ladies and gentlemen, and welcome to our second quarter and first half 2023 financial results call.

Next slide piece I'm here with my my three colleagues Dr. Andrew Allen, our Chief Medical Officer, Stephen <unk>, Our Chief commercial officer, and uterine Buck of months, our Chief Financial Officer to take you through the highlights of these results and of course to answer all your questions that you might have afterwards, but before I do that of course I would.

Like to point you to the next slide slide that says something about forward looking statements because we will be making some forward looking statements of course today that are based upon our current plans beliefs market circumstances et cetera that may of course change to us towards the future.

Sijmen de Vries: Without further ado, I would like to move on to two slides ahead and basically share with you some of the excitement over the last six months that we had, of course. We had a very busy six months and indeed made a lot of progress. First and foremost, we're of course very, very pleased to see that RUCONEST did it again, so to say 20% growth over the Q2 results versus the Q1 is, of course, a very spectacular recovery from what was a one-off weakness in the Q1. That means that if you look at it, we're very confident that we can actually continue to be on track for the low single-digit growth for the entire year. We say that based upon the leading indicators that all point in the right direction.

And then without further Ado I would like to move on to two slides ahead.

Basically yeah.

Share with you some of the excitement over the last six months that we had of course and we had a very busy six months and indeed made a lot of progress and but first and foremost we're of course very very pleased to see that.

<unk> did it again, so to say 20% growth over the second quarter results versus the first quarter is of course a very.

Spectacular recovery from what was a one of a weakness in the first quarter and it means that if you look at it we're very confident that we can actually continue to be on track for the low single digit growth for the for the entire year and we say that based upon the leading indicators at all points in there.

Sijmen de Vries: Of course, Stephen, will talk a little bit more about that later in his part of the presentation. That significant cash flows, of course, that RUCONEST has been generating over the years, not only helped us to back in the days of 2019 in license Joenja from Novartis, but also helped us of course to finance the development of Novartis and of course of Joenja and of course to actually prepare for the successful US launch. That's exactly what happened. The FDA approved the product a couple of days before the PDUFA date, and we were very quickly off to a strong start in the market.

The direction of course, Steven will talk a little bit more about that later in his part of the of the presentation.

That's a significant cash flows with cost at <unk> has been generating over the years not only helped us to back in the days of 2019 in licensed <unk> from Novartis, but also helped us of course to finance the development.

Of Novartis and of course offer Joanne jaw and of course to actually prepare for the successful U S launch and that's exactly what happens in the FDA approved the product a couple of days before the <unk> date, and we were very quickly off too to a strong start in the market and you see NAND and I dare to say that.

Sijmen de Vries: You've seen that and I dare to say that, in the ultra-rare business, having already 43 patients on paid therapy in Q1 that you report results out of 60 that are already in the pipeline as of 30 June, is no mean feat and is a testament to the preparation of our US colleagues. Of course, it beats analyst expectations insofar that I've seen analyst reports predicting Q1 sales for Joenja. We've also made a lot of progress, of course, in the regulatory reviews. We've submitted the file to a number of other territories, Canada, Australia, and Israel, and of course, we made strides forward with the pediatric clinical program. That's important because although the current license for leniolisib already incorporates the 12 years and upwards, so only partly the pediatrics.

No in the ultra rare business, having already 43 patients.

On page therapy in the first quarter that you are that you report results out of 60 day early in the pipeline is a 30 to June is no mean feat and it's a testament to the preparation of our out of our U S colleagues and of course, it beats our analyst expectation seems afforded I've seen analysts' reports that predicting first.

Order sales set for Gilenya.

We've also made a lot of progress of course in the regulator who refuse.

Submitted.

The file to a number of other territories, Canada, Australia, and Israel and of course, you made strides forward with the pediatric clinical program of course, and that's important because the current although the current license for a let Joanne you're already incorporates the.

The 12 years and upwards, so already partly the pediatrics.

Sijmen de Vries: There is a big unmet medical need to be fulfilled in those children younger than 12 years. We're working very hard to actually get that pediatric program done so that we can actually submit the file for the extension towards the younger children. Then, of course, the third pillar there on that slide is, of course, as important, if not more for the, let's say, for the future significant inflection point that we believe that we can achieve with leniolisib. That is the second indication. We, as you may have read already, have been already submitting our plans, and have initiated discussions with the FDA on our proposed development program for the second indication. Anurag will come back to that a little bit later.

There is a big unmet medical needs to be feel feels in dose children younger than 12 years, and we're working very hard to actually get that pediatric program done. So that we can actually submit the file for the extension we see it towards the younger children.

And of course.

Third pillar there on that slide is of course as important if not more for the let's say it for the future significant inflection point that we believe that we can achieve with linear ownership that is the second indication and we as you may have read already has been our already submitting our plans and have initiated discussions.

With the FDA on how on our proposed development program for the second indication and Iraq will come back to that a little bit later.

Sijmen de Vries: Of course, last but not least, we continue to look intensively for additional in-licensing opportunities for rare disease assets, that can actually further leverage our commercialization structure that we have in place. Next slide, please. There you see a visual of the pipeline that has now changed. Of course, we're still under review in the European Union and the UK. The pediatric trial has started. Leniolisib Japan, we're on the brink of starting that trial that we agreed with the Japanese authorities to do. Anurag will come a little bit later on Japan. Of course, you see here that we made progress, as I've just alluded to, by submitting the files towards the Canadian, Australian, and Israeli authorities, so just to increase the footprint for Leniolisib or Joenja, as it's called later, in the geographical footprint. Of course, we also made great strides forward.

Of course last but not least we continue to look intensively for for in licensing additional in licensing opportunities for rare disease assets.

That can actually further leverage our commercialization and commercialization.

The structure that we have in place in next slide please they see a visual.

Of the pipeline that has now changed of course Theres still of course under review with the <unk> in the European Union and the U K.

The pediatric trial is of course has started.

Lineal, except Japan.

On the brink of starting that said that trial that we agreed with the Japanese authorities to do in Iraq will come a little bit later on Japan and of course, you see it have you made progress as I just alluded to by submitting the file towards.

The Canadian.

Australia and in the Israeli authorities, such as to increase the footprint, Florida millennia ownership or Joanne just gold later in our geographical footprint.

And then of course.

We also made great strides forward next slide please with our with our leadership strengthening of our leadership. We are very pleased to have found Dr. Richard Peters.

Sijmen de Vries: Next slide, please. With our leadership, strengthening of our leadership, we're very pleased to have found Dr. Richard Peters as new Chairman-elect to succeed Paul Sekhri, who came to his maximum term as allowed under Dutch law as Chairman of our company. As you can see from the outline on Dr. Peters, he's got an impressive track record, a lot of experience in the healthcare industry, but also in academia, but especially in the rare diseases business with very successful companies such as Genzyme or Sanofi Genzyme, as it's called nowadays. He's been CEO of two Nasdaq-listed biotech companies. As you can see, he held positions in earlier successful companies such as Amgen and Sanofi. Of course, he is a medical doctor by education and has actually also served as an editor for very prestigious journals.

As the new chairman elect to succeed Paul Sekhri, who came to its maximum term as allowed under Dutch law as chairman of our company.

As you can see from from.

The outline on Dr. Peters, He's got an impressive track record a lot of experience in the.

And the health care industry, but also in academia, but especially in the rare diseases business and we are very successful companies such as.

Genzyme or Sanofi Genzyme as Kohl's nowadays he's been CEO of two NASDAQ listed biotech companies as you can see he held positions in in early of successful companies such as Amgen.

And Sanofi and of course, he has a medical doctor by education and it has as actually also served as an editor for Fei.

Sijmen de Vries: We're very pleased that Richard was happy to join us as our Chairman elect. Of course, we will organize an extraordinary general meeting of shareholders in the not-too-distant future, to actually get him elected. Then we're very pleased as well to, and especially in the context of our growth strategy and our ambition to actually in-license or acquire additional late-stage assets for rare diseases, that Alexander Breitenbach has agreed to join us as our Chief Business Officer, who will be tasked with the development and execution of our growth strategy and our future plans. He's a very experienced individual as well at several senior positions, most recently as Chief Business Officer and Chief Development Officer at ACM Biolabs, a Swiss, Basel-based company, but also a very distinguished and long career in Roche in Basel, in the business development group.

Very prestigious journals. So we're very pleased that Richard.

It was.

Happy to join Us as our chairman elect and of course, we will organize an extraordinary general meeting of shareholders in the not too distant future.

To actually get him elected and then we're very pleased as well too and it's especially in the context of our growth strategy and our ambition to actually.

In license or acquire additional late stage assets for rare diseases that Alexander Breitenbach has agreed to join us.

As our Chief business Officer, who will be tasked with.

The growth started the development and execution of our growth strategy and our future plans and he's a very experienced individuals well at several senior positions most recently as.

Chief Business Officer, and Chief Development Officer at ACM Biosciences, our Swiss <unk> Basel based a company, but also a very distinguished and long career in Roche in Basel.

In that business and the business development business development group. So we're very pleased to have strengthened our team with these at least two.

Sijmen de Vries: We're very pleased to have strengthened our team with these two extremely experienced and talented individuals. Next slide, please. The next one, please. At what it actually means and what it brings and what we need and what is our bread and butter. That's our very strong rare disease commercial infrastructure. We have what it takes to actually be successful at this. We've already proven that, and we're about to prove that again with Joenja going forward. We have these dedicated sales forces in the US, European Union, and also we have people in the Middle East and North Africa calling on these immunologists and academic hospitals. We have medical affairs teams.

Extremely experienced and talented individuals.

Next slide please let's just look at Sep.

And then the next one please at what does it actually means and what it brings and what we need and what is our bread and butter, that's our very strong rare disease commercial infrastructure.

We have what it takes to actually be successful in this we've already proven that in.

And we're about to proved it again with <unk> going forward. So we have these dedicated sales forces in the U S.

European Union and also we have people in the Middle East and North Africa, calling on these immunologist and academic hospitals, we have medical affairs team and medical affairs steep. So that's of course very important, especially in rare diseases. As you have a very strong medical affairs team at include medical Directors Medical Science liaisons molecular geneticist and publication spur.

Sijmen de Vries: That's of course very important, especially in rare diseases, that you have a very strong medical affairs team that include medical directors, medical science liaisons, molecular geneticists, and publication specialists. Very big medical teams that are absolutely essential for your success in commercialization of rare diseases. Of course, as market access becomes more and more important, we have built up our market access teams over the last few years on both sides of the oceans. They include national account directors in the US, health economic research specialists and directors, both sides of the ocean, to actually work with the authorities to get the product to reimbursement. You've seen how quickly we got the product reimbursed in the US. I think we have an excellent team in place there. There's patient support teams that help services in the US, reimbursement managers, patient care managers.

List very big very big Big medical teams that are.

Absolutely essential for your success in.

Commercialization of Grand diseases.

Then of course.

As market access becomes more and more important.

<unk> built up our market access teams over the last few years on both sides of the oceans. They include National account directors in the U S Health economic research.

Our specialist and directors both sides of the ocean.

Actually work with the authorities to get the product to reimbursement than you've seen Oh.

How quickly we got the product reimbursed in the U S. So I think we have an excellent team in place there and then that patient support teams set up services in the U S reimbursement managers patient care managers, we have a third party nurse force to take care of our patients are patients and clinical educators and then last one at least we're extremely act.

Sijmen de Vries: We have a third-party nurse force to take care of our patients and clinical educators. Then last but not least, we're extremely active with everybody, including our sales force teams, to be at conferences. There's a lot of disease education ongoing, also directly to patients that we actually facilitate. We support the patient organizations and all. Basically, it's an extremely well-oiled team, and that has shown that, for instance, with the launch of leniolisib, they know what they have to do given the fact that we got it immediately reimbursed and found and identified those patients in a new disease. The disease was not even discovered 10 years ago. As you can see on the next slide, we're really ready to leverage all of this for Joenja, as and when Joenja gets approved, of course.

With everybody, including our sales force teams to be at conferences, and there's a lot of education disease education ongoing.

Directly to patients.

We hear that we actually facilitate we support patient organizations and all so basically it's an extremely.

Well oil to team and it has shown that for instance, with the launch of linear ownership. They know what they have to do given the fact that we got it immediately reimburse and found and identified those patients in a new disease diseases, not even discover 10 years ago.

And as you can see on the next slide.

We're really.

Ready to leverage all of this Florida for Joe and John .

As and when <unk> gets approved of course.

Sijmen de Vries: As you heard earlier, we've also now decided to expand to Australia, Japan, of course, and Canada that are not yet on this map because it's just the established business. One more slide from my end here about the durable commercial asset that RUCONEST represents. It's quite rare, I would say, that a product that's already 9 years in the US market is still growing and is still getting more and more meaningful. Why is it getting more meaningful, and why is it growing? It has this very special place. It's the only recombinant protein replacement therapy that is available in the hereditary angioedema market.

As you heard earlier, we've also now decided to expand to Australia, Japan of course, and and kind of that that are not yet on this map because it's just the established business.

Okay, and then one more slide from my end here about the durable commercial asset that <unk> represents it's quite rare I would say that a product that's already nine years in the U S market.

It's still growing and is still getting more and more meaningful why is it getting more meaningful and why is it growing because it has this very special place.

It's the only recombinant protein replacement therapy that is available in the heritage angioedema market and that's really important because although there's a big trends has been going on towards the prophylactic treatments for dairy and Judy My Undoes prophylactic treatments have become better there's also a the notion and the the.

Sijmen de Vries: That's very important because although there is a big trend has been going towards prophylactic treatments for hereditary angioedema, and those prophylactic treatments have become better, there's also the notion and the facts that almost the half of these patients suffer from so-called breakthrough attacks under their prophylactic therapy. That's when it becomes important to have your breakthrough medication at hand at all times. This is actually where RUCONEST is now beginning to make inroads and being used for breakthrough medication. That is a very important aspect because it means that more and more patients are discovering that it is the right drug to have for your breakthrough medication, and that more and more physicians are actually discovering that RUCONEST is the right choice for their patients as breakthrough medication.

<unk> that almost half of these patients suffer from so called breakthrough attacks.

That prophylactic therapy, that's when it becomes important to have your breakthrough medication.

Hand at all times and this is actually <unk> is now beginning and beginning to make inroads and being used for breakthrough medication and that is a very important aspect because it means that more and more patients are discovering that it is due right.

Drug to have for your breakthrough medical breakthrough medication and at more and more physicians are actually discovery that <unk> is the right choice for their patients at breakthrough medication and yes, <unk> is an IV drug that is self administered but we also know that they have.

Sijmen de Vries: Yes, RUCONEST is an IV drug that is self-administered, but we also know that the absolute vast majority, almost all patients are very, very confident and very well trained to actually do these self-injections. They're perfectly happy with that because they can rely on the product. You can see that on the slide here that has an incredible good track record with regards to efficacy and that they can basically count on to stop that breakthrough attack as it becomes clear that they're getting a breakthrough attack. That's why we think that RUCONEST has this very special spot. We know that RUCONEST has this very special place in the market and will continue to have this very special place in the market going forward.

Absolute vast majority almost all patients are very very confident and very well trained.

So actually do do self duty self injections and they're perfectly happy with us because they can rely on our products and you can see that on the slide here that has.

Accretable good track record with regards to efficacy and.

And that can basically count on to stop that said that breakthrough attack as its as it becomes clear that getting a breakthrough attack. That's why we think that weakness has expiry specials, but we know that <unk> has this very special place in the market and we'll continue to have this very special place in the markets going forward.

Sijmen de Vries: I would like to switch over to APDS, and I switch over to now ask for Anurag to present the Joenja story. Anurag, over to you.

Then I would like to switch over to <unk> and a switchover to know aspirin interact to present, the digital angio story and rack over to you.

Anurag Relan: Thanks, Sijmen. On the next slide, we can see a little bit of information about APDS, which is a rare, serious, and progressive primary immune deficiency, and as you heard from Sijmen, just first described about 10 years ago. We think that if APDS affects about 1.5 patients per million based on literature estimates and our own patient-finding efforts. I'll talk a little bit about those patient-finding efforts in a moment. With those efforts, we've identified now more than 640 patients in key global markets already. We think that represents a portion of the total of 1,500 patients or so that are out there with APDS. The signs and symptoms of APDS vary widely. As with many rare diseases, there's also a significant delay to diagnosis because of these varying symptoms and the rarity of the disease.

Thanks Simon.

On the next slide we can see a little bit of information about <unk>, which is a rare serious and progressive primary immune deficiency and as you've heard from Simon just first described about 10 years ago.

We think that if a pds effects about one 5 million $1 five patients per million based on literature estimates and our own patient finding efforts and I'll talk a little bit about those patient finding efforts in a moment, but with those efforts. We've identified now more than 640 patients in key global markets already.

We think that that represents.

A portion of the total of 500 patients or so that are out there with a pds.

The signs and symptoms of <unk> vary widely as with many rare diseases. It's also a signal.

Have been delayed to diagnosis because of these varying symptoms and the rarity of the disease.

Anurag Relan: These delays result in significant morbidity and mortality for these patients, it's something that we're really trying to address with our own patient-finding efforts and making Joenja available, of course. The treatments for APDS up till now have been focused on symptom management. Really when we think about the problems that these patients face, so with infections, so giving them antibiotics or giving them immune globulin replacement therapy, really not addressing the root cause, however. A genetic test, however, is a very simple way to make the diagnosis of APDS, we'll talk a little bit about some of the things that we're doing on this front too in a moment. On the next slide, we can see that Joenja was now approved by FDA earlier this year. Joenja modulates this hyperactive pathway, as a result, allows the immune system now to develop properly.

These delays result in significant morbidity and mortality for these patients and it's something that we're really trying to address with our own patient finding efforts in making gilenya available of course.

The treatments for Atvs up till now has been focused on symptom management. So really when we think about the problems that these patients face, though with infections, so giving them antibiotics or giving them.

<unk> globulin replacement therapy really not addressing the root cause however.

The genetic test however is a very simple way to make the diagnosis of a PDF and we'll talk a little bit about some of the things that we're doing on this front to in a moment.

On the next slide we can see that <unk> engine was now approved by FDA earlier this year to Angel modulates. This hyperactive pathway and as a result allows the immune system now can develop properly this development of the immune system.

Anurag Relan: This development of the immune system, when it occurs improperly in these APDS patients, leads to all of the problems that we see in these patients with autoimmune phenomena, as well as all of the infection-related problems that these patients face. With Joenja, we can try to balance this pathway and to support this immune function in a proper manner. Next slide. Here's a highlight of some of the data on Joenja. You can see in the top left the indication statement that Joenja is approved in the US to treat patients who are 12 years and older with APDS. This was on the basis of a randomized placebo-controlled study, which showed that Joenja met both co-primary endpoints, as well as meeting the several secondary endpoints and exploratory parameters as well.

And when it occurs in properly and these atvs patients leads to all the problems that we see in these patients with autoimmune phenomenon as well as all of the infection related problems that these patients face with <unk>, we can <unk>.

To balance this pathway and to support this immune function in a proper manner.

Thanks, a lot.

And here's a highlight of some of the data on Gilenya.

You can see in the top left the indication statement that joins US is approved in the U S to treat patients who are 12 years and older with Atvs and this was on the basis of a randomized placebo controlled study, which showed that <unk> met both co primary endpoints as well as meeting.

Several secondary endpoints and exploratory parameters as well from a safety standpoint, there were no drug related serious adverse events or withdrawals due to gilenya in the study.

Anurag Relan: From a safety standpoint, there were no drug-related serious adverse events or withdrawals due to Joenja in the study. More importantly, what we saw was that when patients took Joenja on a long-term basis in the open-label study, we saw many other downstream clinical benefits, including reductions and discontinuation in the use of IVIG or immune globulin replacement therapy, as well as reduction in infection rates. These results were consistent with what we saw in the randomized double-blind placebo-controlled part of the study. We continue to collect further data, and we'll report this data on lymphadenopathy as well as some of the biomarkers that were collected in the study, including markers of abnormal B-cell development, as well as the production of some of the antibodies that we see are elevated in untreated APDS patients.

But more importantly, what we saw was that when patients took Joanne is on a long term basis and the open label study we saw many other downstream clinical benefits, including reductions in discontinuation in the use of IV <unk> or immune globulin replacement therapy as well as reduction in infection rates.

And these results were consistent with what we saw in the randomized double blind placebo controlled part of the study and we continue to collect further data and we will report this data on lymphadenopathy as well as some of the Biomarkers that were collected in the study, including markers of abnormal b cell development as well as the production of some.

The antibodies that we see are elevated in untreated patients.

Anurag Relan: As you've seen from our data so far, we're off to a good start with the patients being able to start Joenja very quickly in the US, Steve will report on this progress further shortly. Next slide, please. What are we doing now to look for APDS patients? When we think about APDS, it's really part of a larger pool of patients with what's called inborn errors of immunity or sometimes also called primary immune deficiency. Through those efforts, we've, as I said, identified more than 640 patients worldwide with APDS, among which there are 200 identified in the US.

And as you've seen from our data so far.

We're off to a good start with the patients.

Patients being able to start to enter very quickly in the U S and Steve will report on this progress further shortly.

Next slide please.

And what are we doing now look for Atvs patients. So when we think about Atvs, it's really part of a larger pool of patients with what's called inborn errors of immunity or sometimes also called primary immune deficiency.

And through those efforts, we as I said identified more than 640 patients worldwide.

With Aps.

Among which there are 200 identified in the U S. Those efforts you can see in the box on the left are really focused around education testing patients.

Anurag Relan: Those efforts you can see in the box on the left are really focused around education, testing patients, family testing, multiple medical affairs activities, including raising awareness about the disease, raising awareness about the condition itself, and really working with patients and clinicians to help identify patients. We also know, as with most rare diseases, there's a large pool of undiagnosed APDS patients. We're out there with our medical and commercial teams, trying to identify these patients. We know that many of these patients are seen by immunologists, but we also know that they're also being seen by other providers where they don't even have a diagnosis yet of a primary immune deficiency or an inborn error of immunity. We're making available in the US a comprehensive genetic testing program to help get those undiagnosed patients the proper diagnosis.

Family testing multiple medical affairs activities, including raising awareness about the disease raising awareness about.

The condition itself and really working with patients and clinicians to help identify patients.

But we also know as with most rare disease, there's a large pool of undiagnosed <unk> patients. So we're out there with our medical and commercial teams trying to identify these patients. We know that many of these patients are seen by Immunologists, but we also know that there are also being seen.

By other providers, where they don't even have a diagnosis yet of a primary immune deficiency or an inborn error of immunity.

And we're making available in the U S. A comprehensive genetic testing program to help.

Get those undiagnosed patients.

Proper diagnosis.

Anurag Relan: As a result of this genetic testing and the wider spread use of genetic testing, we're also encountering many patients in the third box here on the right. These are patients who have a result that is inconclusive, and this is called a VUS or a variant of uncertain significance. What that means is that they have a variant in one of the two genes that leads to APDS, but that variant has not been described previously. There's not much information in the literature about it, or there is no information in the literature about it. We're doing a number of efforts now to help those patients, which there are a significant portion of, that have this inconclusive result, that have a primary immune deficiency, that have already had a genetic test, but have a result that doesn't give them a final diagnosis.

And as a result of this genetic testing and the wider spread use of genetic testing. We're also encountered many patients in the third box here on the right and these are patients who have a result that is inconclusive and this is called a V U S. Whereas variants of uncertain significance and what that means is that they have.

A variant in one of the two genes that leads to a pds, but that Varian has not been described previously there's not much information in the literature about it or there is no information on the literature about it so.

Doing a number of efforts now to help those patients, which there are a significant portion of that have this inconclusive result that have a primary immune deficiency that I've already pattern genetic test, but have a result that doesn't give them a final diagnosis and we're doing a number of things that you see there, including looking through the litter.

Anurag Relan: We're doing a number of things that you see there, including looking through the literature, going to other clinical laboratories and seeing what testing may have been done. We're also embarking on functional testing, both in the US as well as worldwide, and importantly, family testing to try to see if we can figure out if this variant is present in other family members, and if those family members also have the condition or not. As we move forward, I expect that we're going to find a significant number of patients in this population of patients who currently are sort of in limbo with this variant of uncertain significance diagnosis currently. Next slide.

Sure.

To other clinical laboratories, and seeing but testing may have been done. We are also embarking on functional testing both in the U S as well as worldwide and then importantly family testing to try to see if we can figure out.

This variant is present in other family members and if those family members also have the condition or not and as we move forward I expect that we're going to find a significant number of patients in this population of patients who currently are sort of in limbo with this variants of uncertain significance diagnosis Kurt.

Next slide.

Anurag Relan: Thinking ahead beyond the FDA, as you heard from Sijmen, we are continuing to work with the European regulators, and we expect an opinion from the CHMP later this year with a potential approval subject to a positive opinion two months later. If we think about the UK, if the CHMP issues its positive opinion, we can file in the UK very soon thereafter with a potential approval also two months later. The Japanese clinical study is open for enrollment, and I expect the first patient to be enrolled and treated there in this quarter. As you heard from Sijmen, we've also made progress with filings in Canada, Australia, and Israel.

And then thinking ahead beyond the the FDA.

You've heard from Simon we are continuing to work with the European regulators and we expect an opinion from the <unk> later this year with a potential approval subject to a positive opinion two months later.

And then if we think about the U K.

See HMP issues, it's positive opinion, we can file in the U K.

Very soon thereafter with a potential approval also two months later the Japanese clinical study is open for enrollment and I expect the first patient to be enrolled and treated there in this quarter.

And as you heard from Simon We've also made progress with filings in Canada, Australia and Israel.

Anurag Relan: We've launched our named patient program to make Joenja available in certain markets. We announced earlier this year that the pediatric patients are able to enroll in the first pediatric study, which is in the age group of four to 11 years old. As Sijmen mentioned, we're also making significant progress in looking at other indications for leniolisib. I expect to be able to talk about this in greater detail later this year toward the end of Q3, beginning of Q4, as we go through some of the regulatory discussions about our clinical development plans. Also later this year, we're going to be starting our second pediatric study. This is in the youngest age group.

Launched our named patient program to make <unk> available in certain markets and we announced earlier this year that the pediatric patients are able to enroll in the first pediatric study, which is in the age group of four to 11 years old.

And as Simon mentioned, we're also making significant progress there looking at other indications for lineal Wilson and I expect to be able to talk about this in greater detail later this year towards the end of the third quarter beginning of fourth quarter as we go through some of the regulatory discussions about our clinical development plans.

Also later this year, we're going to be starting our second pediatric study and so this isn't the youngest age group. These are again.

Anurag Relan: These are, again, patients who are age one to six years old, I expect to be able to start that study in the Q3 of this year. Next slide. Just a quick update on where we are with EMA and the progress that we've made so far. As you know, we submitted our responses to the CHMP Day 120 list of questions, and just a couple of weeks ago, we received further questions, what's called List of Outstanding Issues as part of the Day 180 procedure. We also understand now from CHMP, and we're quite pleased with this, is that the CHMP will consult an ad hoc expert group.

Patients, who are aged one to six years old and I expect to be able to start that study.

In the third quarter of this year.

Next slide.

And just a quick update on where we are with EMA and the progress that we've made so far so as you know we submitted our responses to the <unk> de 120 list of questions and just a couple of weeks ago, we received.

Further questions, what's called list of outstanding issues as part of the day 180 procedure.

We also understand now from CHP and we're quite pleased with this is that the <unk> will consult and AD hoc expert group and they've acknowledged that.

Anurag Relan: They've acknowledged that the condition is rare and there's an unmet need here. They'd like to seek further guidance from an ad hoc expert group of clinicians to talk a little bit about APDS and the leniolisib trial results to put them in context. This will be a closed meeting that will involve Pharming representatives, where we'll have some leniolisib investigators, as well as APDS patients. As I said earlier, we're expecting the CHMP opinion in the Q4 of this year, and subject to a positive opinion, an approval 2 months later. Next slide. It's over to Steve.

Condition is rare and there is an unmet need here and they'd like to seek further guidance from an AD hoc expert group of clinicians to talk a little bit about <unk> and the millennials and trial results to put them in context, and this will be a closed meeting that will involve farming representatives, we will have some millennials to investigators as well as a PDF.

Patients and as I said earlier, we're expecting the <unk> opinion in the fourth quarter of this year and subject to a positive opinion.

And approval two months later.

That's fine.

Over to Steve.

Stephen Toor: Thank you, Anurag. Good morning and good afternoon, everybody. I'm going to provide you with a short update on RUCONEST progress in Q2 in H1 and also the Joenja launch in Q2 of this year. Next slide, please. As Sijmen touched upon already, the disruptions we saw in Q1 were HAE market-wide, and they also affected our competitors. As indicated in the Q1 call, they were transitory. In Q2, RUCONEST performed well. The leading revenue indicators, including growth in unique prescribers, new patient enrollments, and vial ship to patients, were all strong. In fact, new enrollments have hit 70+ for both Q1 and Q2, despite the Q1 market issues. This really clearly reflects the underlying demand in the HAE market and for RUCONEST specifically.

Thank you Eric Good morning, and good afternoon, everybody I'm going to provide you with a short update on <unk> progress in Q2 in the first half and also joined the launch in Q2. This year next slide please.

So starting with <unk>.

The disruption we saw in the first quarter were HIV market wide and they also affected our competitiveness.

And as indicated in the Q1 call they were transitory in.

In the second quarter recognize performed well the leading revenue indicators, including growth in unique prescribers, new patient enrollments and <unk> shipped to patients with <unk> in fact, new enrollments with its 70 plus for both Q1 and Q2, despite the Q1 market issues.

And this really clearly reflects the underlying demand in the HIV market and for <unk> specifically.

Stephen Toor: In Q2, RUCONEST revenues globally increased by 20% compared to Q1, and furthermore, we saw a 2% increase in revenues when comparing the Q2s of 2023 and 2022. Given the strong bounce back in Q2, which we did signal in the Q1 call, we're maintaining our outlook of low double-digit revenue growth for the rest of 2023. Next slide, please. This slide shows the number of unique prescribers continues to grow in the US. 687 represents 62% of the HAE prescribing community and is still growing nine years post-launch, as Sijmen mentioned earlier. That's despite the significant changes to the market throughout those years.

In the second quarter <unk> revenues globally increased by 20% compared to the first quarter. Furthermore, we saw a 2% increase in revenues when comparing the second quarters of 2023 and 2022.

Given the strong bounce back in the second quarter, which we did signal in the Q1 call. We are maintaining our outlook of low single digit revenue growth for the rest of 2023.

Next slide please.

Okay.

This slide shows the number of unique prescribers continues to grow in the U S 697 represents 62% the prescribing community.

And it's still growing nine years post launch as Simon mentioned earlier and Thats. Despite the significant changes to the market throughout those years.

Stephen Toor: This clearly shows that despite the prophylactic launches and the genericization of Firazyr, there remains both a place and need in the treatment armamentarium for a C1 esterase inhibitor and an enduring need for RUCONEST. We expect to continue growing the unique prescriber base in the coming months and years, and with it, of course, the base of patients that benefits from RUCONEST. Next slide, please. Let's turn now to the Joenja launch. As you would expect, Pharming has brought its A game and all its rare disease commercialization experience to bear in the US, which, as with all companies, is a must-win market. We have 54 salespeople and leaders comprised of the existing RUCONEST team, where approximately 30% of APDS patients reside. The Joenja institutional team, which joined us in August of last year.

This clearly shows that despite the prophylactic launches on the generalization. If there is there remains remained sorry, both the place and need in the treatment armamentarium for cone esterase inhibitor and an enduring need for weakness.

We expect to continue growing the unique prescriber base in the coming months and years and with of course, the base of patients with benefits to reconnect.

Next slide please.

So, let's turn to ask the Joe and Joe.

As you would expect farmers bought its a game and all of this rare disease commercialization experience to bear in the U S which is.

As with all companies must win market.

54 sales people to lead is comprised of the existing brick and his team were approximately 30% of <unk> patients reside.

And then the Joe and your institutional team, which joined US in August of last year. They focus on the centers of excellence to which the other 70% of <unk> patients either already are being treated all will be referred to.

Stephen Toor: They focus on the centers of excellence to which the other 70% of APDS patients either already are being treated or will be referred to. These two teams, along with other colleagues that Sijmen already outlined in an earlier slide, are tasked with both identifying patients and ensuring HCPs have all the information and education they need to confidently prescribe Joenja. We also have, amongst others, clinical educators to drive family mapping and testing. I think Anurag outlined how important that is. Just to reiterate, APDS is not the same with dominant condition. There's a 50/50 chance the siblings and other family members are also going to have APDS. Family testing is critical for both them and their welfare and the long-term growth of Joenja, which as we know is a progressive disease. Okay, next slide, please.

These two teams along with other colleagues as Simon already outlined in earlier slides are tasked with both identifying patients and ensuring hcp's up all the information and education, they need to confidently prescribe Joe and Joe.

We also have amongst others clinic clinical educators to drive family mapping and testing.

Mike outlined how important that is and just to reiterate <unk> is notwithstanding dominant condition.

So there is a 50 50 chance with siblings and other family members are also going to have a PBX and soft family testing is critical for both them and their welfare and the long term growth of Joe and Jim.

Which as we know is a progressive disease.

Okay next slide please.

Stephen Toor: Just briefly before I pass over to Jeroen, a couple of things around the value proposition. As Anurag already shared, and we should just remind ourselves, this is the only indicated option for APDS patients, is a precision medication. That's important. When a patient tests positive and a physician prescribes, both the physician and the payer know they're prescribing a treatment that will actually work on modifying the root cause of the disease. Hence, at Pharming, we knew, therefore, that we were launching a product that physicians and their underserved patients would need access to and access to quickly. Let's briefly look at the impact on a patient. Next slide, please. This is John. He's a 20-something-year-old man who's been dealing with APDS since he was 11 years old.

Just briefly before we.

Before I pass it over to you Rune couple of things around the value proposition.

Is that a I already shared and we should just remind ourselves. This is the only indicated.

Option <unk> patients is a precision medication that's important when a physician.

When a patient test positive and a physician prescribes both the physician and the payer node that prescribing a treatment that will actually work on modifying the root cause of the disease.

Hence we use it for that we will launching a product with physicians in the underserved patients would need access to an excess too quickly. So lets briefly look at the impact on our patient mix what please.

So this is John is a 20 something year old man, who has been dealing with IPD S&P was 11 years old.

Stephen Toor: Like most patients, he's been in and out of hospital, was having difficulty developing social skills, keeping friends, and was basically willing to try anything to remove the stress and the burden of taking 11 pills in the morning and 9 pills at night to try and manage his symptoms. Joenja, of course, has removed that heavy pill burden and is treating the underlying cause of John's APDS. Since being prescribed Joenja, John's life has already changed significantly for the better. His lymph nodes have decreased in size, and I think you can see in the thought bubbles his quote around that. His perception is they're actually gone as opposed to just being reduced in size. Importantly, he's starting to think about his future and what he wants to do in life.

Most patients who has been in out of hospital was having difficulty developing social skills keeping friends.

And with basically willing to try anything to relieve the stress and the burden of taken 11 pills in the morning of nine <unk> at night to try and manage these symptoms.

Gilenya courses remove that heavy pill burden that is treating the underlying cause of Jones Aps.

Since being prescribed Joe and Jay Johns life has already changed significantly for the better is lymph node the decreased in size and I think you can see the two bubbles you know his quote around that.

The specifics that are actually gone as opposed to just being reduced in size.

And importantly, he starting to think about the future and what he wants to do in life and as you see here that that includes actually go to college something he couldnt have been previously.

Stephen Toor: As you see here, that includes actually going to college, something he couldn't have envisaged previously. That's basically the promise that Joenja can and will continue to deliver for our patients, and it's what will continue to fuel this launch phase, and also our future growth. Next slide, please. As we discussed in the last call, the preparation for this launch was very, very rigorous and thorough. As expected, we're off to a very good start. Our first fully reimbursed commercial shipments of Joenja occurred within 2 weeks of FDA approval, which is very quick and certainly unusual. To date, we've enrolled 60 APDS patients and shipped to 43 of them on payer-approved product, with the remaining 17 to the end of the H1 in process.

That's basically the promised that joined you can and we will continue to deliver for our patients and it's what will continue to fuel.

This law this launch phase and also our future growth next slide please.

So.

As we discussed in the last call.

Preparation for this launch was very very rigorous and thorough and unexpected we're off to a very good start are.

Our first fully reimbursed commercial shipments of Joe Andrew occurred within two weeks of FDA approval, which is very quick.

Certainly unusual and to date, we have enrolled 68 patients and shipped to <unk> 43 of them on payer approved products with the remaining 17 to the end of the first half in process.

Stephen Toor: With respect to market access, the teams are doing outstanding work, we continue to make good progress partnering with the national and regional payers, including state Medicaid programs, to prepare for clinical review and coverage policy development, many of which are now rolling out. To date, no patient has been denied access to Joenja. As you can imagine, we're very pleased with the Q1 of post-launch progress and expect to build on our early success in the coming months to both get eligible patients on therapy and grow the patient funnel for future patients. That will continue the brand growth and of course, lay the foundations for Pharming's continued success into 2024. Now I'd like to hand over to Jeroen, who will cover the financials.

With respect to market access the teams are doing outstanding work and we continue to make good progress partnering with the national and regional payers, including state Medicaid programs.

If the clinical review and coverage policy development, many of which are now rolling out and to date no patient has been denied access to Joe and Jim.

So as you can imagine we're very pleased with the first quarter of post launch progress and expect to build on our early success in the coming months to both get eligible patients on therapy and grow the patient funnel for future patients.

That will continue to brand growth and of course lay the foundation for <unk> continued success into 2024.

So now I'd like to hand over to you Rune, who will cover the financials.

Jeroen Wakkerman: Yeah. Thank you very much, Steve. Good morning, good afternoon, everybody. Moving to the next slide. On the financial highlights for Q2 2022, our revenues grew to EUR 54.9 million, which is a growth of 9% versus last year. The EUR 54.9 million consists of EUR 3.8 million of Joenja sales, so the Q1 that we obviously report that Joenja sales, at EUR 51.1 million from RUCONEST. That is a growth of 2% from last year's Q2, 20% even from Q1. A very strong recovery as Sijmen and Steve mentioned before as well from Q1. Gross profits grew to EUR 49.2 million, which is +7%, which is roughly in line with the growth in revenues. Operating costs increased by EUR 23.4 million. That's on a number of items, mostly marketing and sales. I'll give you some more detail later.

Yes. Thank you very much Steve good morning, good afternoon, everybody.

Moving to the next slides on the financial highlights for the second quarter of 'twenty two.

Our revenues grew to $54 9 million, which is a growth of 9% versus last year.

And the $54 9 million consists of $3 8 million of joined yourselves set a first quarter that we obviously report that.

<unk> sales of $51 1 million from <unk> and that is a growth of 2% from from last year's second quarter, 20% even from from Q1, So a very strong recovery.

Simon and and Steve mentioned before as well.

From Q1 gross profit grew to $49 2 million, which is plus 7%, which is roughly in line with the growth in revenues operating cost increased by 23.4.

$4 million and Thats on a on a number of items that most of the marketing and sales, but I'll give you some more detail later.

Jeroen Wakkerman: Also important to understand for Q2 is that we had in what we call other income, so that's between gross profit and operating costs in the P&L. We had an income of $21.1 million for the sale of the priority review voucher to Novartis, which was as per the agreed contract in 2019. Also good maybe to tell you that in the last year's Q2, we also had some big other income that was connected to the BioConnection transaction, as you may remember, which brought us in at EUR 12.8 million. To cut a long story short, the increase in other income from this year to last year was EUR 8 million. On the operating profit, EUR 5.3 million. That's a decline, and that's roughly in line with the increase in the operating costs.

Also important to understand for the second quarter is that we had and what we call. Other income set us between gross profit and operating cost in the P&L.

And income of $21 $1 million for the sale of the priority review voucher to Novartis, which was as per the agreement.

Great contracts in 2019.

And also maybe to tell you that in the last last year's second quarter. We also have some big other income that was connected to the buyer connections transaction as you may remember, which brought us in at $12 8 million to cut a long story short the increase in other income from this year to last year.

Was $8 million.

Then on the operating profit $5 3 million.

Klein and that's roughly in line with the increase in the in the operating costs and the net profit went from 57 to $1 3 million. So a decline of $14 4 million.

Jeroen Wakkerman: The net profits went from EUR 15.7 million to EUR 1.3 million, so a decline of EUR 14.4 million. That is, by the way, an improvement from Q1, where we had a net loss in the quarter. If we look at the H1 results on the next slide. Total revenue grew by 1% to EUR 97.4 million. The gross profit, EUR 87.6 million with a gross margin of 90%, was a good gross profit income. Operating cost, EUR 118.5 million from EUR 82.2 million last year. That's an increase of EUR 36.2 million. More detail about that later. The operating profit was EUR -8.4 million, so an operating loss, so to say. The net loss has been EUR 10.9 million so far in the year. If we look at the revenue breakdown for the last few quarters on the next slide.

And that is by the way.

An improvement from Q1, where we had a loss and net loss in the quarter.

And if we look at the first half results on the next slides.

Total revenue grew by 1% to $97 four.

The gross profits $87 six with a gross margin of 90% was a good gross profit income.

Operating cost $119 5 million from $82 2 million last year. So that's an increase of $36 2 million more detail about that later.

The operating profit was $8 4 million negative shown operating below so to say.

<unk> net loss was has been 10.9 million so far in the year.

If we then look at the revenue breakdown.

Or for the last few quarters on the next slide.

Jeroen Wakkerman: You see indeed the enormous growth from Q1. Well, the drop obviously was from the reimbursement issues that were apparent in the whole HAE market. We showed a strong recovery of 20% in Q2 to EUR 51.1 million, and you see the EUR 3.8 million sales from Joenja for the first time in our history. Moving to the next slide on the costs. Because they went up significantly, just a bit of color on where we spent the money. In Q2 we had EUR 10 million of milestone payment to be paid to Novartis, related to Joenja. Basically over time, you see a substantial increase in marketing and sales to support the launch of Joenja. Stephen just mentioned that the launch preparation was rigorous and thorough, and that was also what you see in the money that we spend on Joenja.

You see indeed.

Enormous growth from from Q1.

While the drop obviously was from the reimbursement issues that were apparent in EMEA.

Market.

But we showed a strong recovery of 20% in the second quarter to $51 1 million.

And you see the $3 8 million sales from from Georgia for the first time in our history.

Moving to the next slide on the costs.

Because they went up.

Difficult lead just a bit of color on where we spend the money.

So in the second quarter, we have.

$10 million of milestone payment to be paid to novartis related to gilenya.

And we had a.

Basically over time Youll see a substantial increase in increase in marketing and sales to support the launch of <unk>. So Steve just mentioned that the launch preparation was rigorous and thorough and that's also what you see in the money that we spend on the ongoing Jim.

Jeroen Wakkerman: You see that a fairly stable development of G&A cost and an increase in R&D in Q2 of this year of EUR 5.3 million, which is largely related to the work we are doing on approvals in several international markets like Europe, Canada, Australia, and Israel, and a buildup of our medical departments both in the US and in Europe. Apart from the cost categories, another way of looking at the increase in cost in H1 of this year, that was EUR +36 million, is that we spent EUR 10.5 million in total on the milestones. We spent EUR 7 million of the EUR 36 million increase on leniolisib out-of-pocket expenses, and think about R&D costs, but also marketing, market access, and amortization costs of the license. We added EUR 16 million of payroll and general expenses.

You see that as a fairly stable development of G&A costs and an increase in R&D in the second quarter of this year of $5 3 million.

Which is largely related to the work we're doing on the approvals in several international markets like Europe , Canada, Australia and Israel.

And a buildup of our.

Medical departments, both in the U S and in Europe .

So apart from the cost categories another way of looking at the.

The increase in cost in the first half of this year that was plus $36 million is that so we spent $10 5 million in total on the milestones we spend $7 million of the 36 increase on Linea <unk> pocket expenses, I think about R&D costs, but also marketing market access.

<unk> and amortization cost of the license and.

And we added $16 million of payroll and general expenses.

Jeroen Wakkerman: Most of it is in payroll because we increased the number of people since last year from 319 at the end of the H1 last year to 383 now. An increase of 64 FTEs, and that is across the board, most of them in marketing and sales. Across the board, we're growing the company as an investment in future growth. A short overview on the next slide of the cash flow over the last quarter. The cash and cash equivalents increased by EUR 8 million to EUR 192.4 million, and that was mostly related to the sale of the priority review voucher, which is considered a cash flow from investing activities. That was the overview of the financial highlights. Going to the outlook for this year on the next slide.

Most of it is in payroll because we increased the number of people since last year from.

319 at the end of the second half last at the end of the first half last year to $3 83, now so an increase of 64 ftes and that is across the board most of them in marketing and sales.

But across the board with growing the company.

As an investment in future growth.

Yeah.

But a short overview on the next slide of the cash flow over the last last quarter it increased by the cash and.

Cash equivalents increased by $8 million to $192 4 million and that was mostly related to <unk>.

The sale of the priority review voucher, which is considered a cash flow from investing activities.

Okay.

So that was the overview of the financial highlights then going to the outlook for for this year on the next slide.

We.

Jeroen Wakkerman: Driven by the strong recovery, we continue to guide on low single-digit growth for RUCONEST revenues for the full year. Joenja was approved at the end of March by the FDA, and we've been commercializing the product since April of this year. We expect a CHMP opinion for the EU in Q4 of this year and the marketing authorization subject to that positive outcome two months later for Europe. In the UK, we will be filing leniolisib with the MHRA following the European Commission decision reliance procedure. Again, that is subject to the positive outcome of the EMA review. We continue to invest in operating costs to accelerate the growth of Joenja and RUCONEST. Further details on the plans to develop additional indications for leniolisib will be provided later this year.

Driven by the strong recovery, we continue to.

Two guides on low single digit growth for <unk> revenues for the full year.

<unk> was approved at the end of March by the FDA and we've been commercializing the production's in April this year.

We expect a CHP.

Opinion for the EU in the fourth quarter of this year.

Marketing authorization.

Object to that positive outcome.

Two months later for Europe .

In the U K, we will be filing lending ownership with the MH or <unk>. Following the European Commission decision reliance procedure and again that is subject to the positive outcome of the EMA review.

We continue to invest.

We invest in operating costs to accelerate the growth of <unk> and <unk>.

<unk>.

And <unk>.

Further details on the plans to develop.

Additional indications for <unk> will be provided later this year.

Jeroen Wakkerman: Last but not least, we continue to look at investments on acquisitions and in-licensing of mid- to late-stage opportunities in rare diseases to build the company further. With that, I want to move to the next slide and give back to the Operator to operate the Q&A. Please feel free to ask any question. Thank you.

And last but not least we continue to.

If you look at investments on acquisitions and in licensing of mid to late stage opportunities in rare diseases to build the company further.

With that I want to move to the next slide and get back to the operator to operate the Q&A and please feel free to ask any question.

Thank you.

Operator: Thank you. As a reminder, if you'd like to ask a question today, please press star followed by one on your telephone keypad now. When preparing to ask your question, please ensure your headset's fully plugged in and unmuted locally. Star followed by one to ask a question. Thank you. As a reminder, if you'd like to ask a question today, please press star followed by one on your telephone keypad now. When preparing to ask your question, please ensure your headset's fully plugged in and unmuted locally. Star followed by one to ask a question. Thank you. As a reminder, if you'd like to ask a question today, please press star followed by one on your telephone keypad now. When preparing to ask your question, please ensure your headset's fully plugged in and unmuted locally.

Thank you as a reminder, if you'd like to ask a question today. Please press star followed by one on your telephone keypad now.

The parents ask a question. Please ensure your headsets fairly plugged in and on mutual likely star followed by one truck.

Thank you <unk>.

If you'd like to ask a question today. Please press star followed by one on your telephone keypad now from the parent to ask a question. Please ensure your headsets fairly plugged in and on mutual likely.

Followed by one truck.

Thank you as a reminder, if you'd like to ask a question today. Please press <unk>.

<unk> followed by one on your telephone keypad now.

To ask a question. Please ensure your headsets fairly plugged in it.

Jeroen Wakkerman: If that happens within the months, the second month will be.

If that happens within the months the second recording has stopped.

Operator: The recording has stopped.

Jeroen Wakkerman: will be a commercial pack. If that procedure is not yet finished, there will be another month supply in the form of a bridging pack provided. To answer your question, yes, they are all having access to therapy once they are enrolled. Hope that is clear to you.

It will be a commercial back.

And if that's a procedure is not yet finished then they will.

Another months supply are important in the form of a bridging Pak provided so to answer your question, yes, they're all having access to therapy winter months are enrolled.

It's clear to you.

Christian Glennie: Yeah. That's good. Thanks. In terms of the identified patient numbers-

That's great. Thanks, and then in terms of the patients identified patient numbers. This meeting is being recorded.

Operator: This meeting is being recorded.

Christian Glennie: You've added 140 in terms of 640 versus the 500 previously reported. I noticed that the US has stayed the same. Just where those extra 140 patients come from, is that mostly these new international markets, Canada, Australia, Japan, or where are they from?

You said you've added.

You've added 140.

<unk> hundred 40 vessels 500 previously reported.

The U S stay the same just where those extra 140 patients come from is that mostly these new international markets.

China, Japan.

Jeroen Wakkerman: Could you comment on that, Anurag? Is that possible?

I think from could you comment on that Iraq.

Hello.

Anurag Relan: Sure. Hi, Christian. We're continuing to find patients in the US, but we're also finding patients in other markets. That does include Japan, Australia, and other markets where we intend to commercialize first.

Sure Hi, Christian So we are finding patient we're continuing to find patients in the U S. But we're also finding patients in other markets. So that does include Japan, Australia, and other markets, where we intend to commercialize the first.

Christian Glennie: Okay. Thank you. Then in terms of the You've got 200 patients in the US identified. If you do your 1.5 per million in the US, there's potentially about 500 or so out there. That's already a 40% diagnosis rate. Where do you think that diagnosis rate could go over the next few years in terms of identifying patients?

Okay. Thank you and then and then in terms of the.

You've got 200 patients in the U S identified.

One five.

<unk> in the U S. That's potentially about 500.

Also out there that's already a 40% diagnosis rate.

Where do you where do you think that.

That create that diagnosis rate could go over the next few years.

Five patients.

Sijmen de Vries: Would you like to answer that as well, Anurag?

Would you like to answer is I'm not sure.

Anurag Relan: Sure. We definitely see this trending up. I think, when we think about markets in Europe and some other countries across the world where there are more centralized healthcare systems, we can see that the prevalence there already exceeds one per million. In the US, of course, we have a more distributed and less centralized system. The diagnosis of these patients is, and the care for these patients is, a little bit more fragmented. We expect that over time, that diagnosis rate will go up as there's increasing awareness of the disease, but that there's also an available therapy available for these patients. I think that we expect that to increase and at the minimum approach, what we see in some other countries, but likely increase beyond that. That's why we think we're being reasonable about an estimate of 1.5 per million.

Sure. So we definitely see this trending up I think when we think about.

When we think about markets in Europe , and some other countries across the world, where there are more centralized health care system. We can see that the prevalence there already exceeds one per $1 million in the U S of course, we have more distributed in la.

Less centralized system. So the the diagnosis of these patients is in the care for these patients is a little bit more fragmented and so we expect that over time the diagnosis rate will go up as there's increasing awareness of the disease.

There is also an available therapy available for these patients. So I think that we expect that to increase and at the minimum approach what we see in some other countries, but likely increase beyond that and that's why we think we're being reasonable about an estimate of $1 five per million as I mentioned earlier Theres a significant.

Anurag Relan: As I mentioned earlier, there's a significant population of patients that we found beyond the 200 in the US, for example, that have these variants of uncertain significance or what are termed VUSs. I think that's another pool of patients who have symptoms of a primary immune deficiency, have had a genetic test, oftentimes that's well before we've been involved in the picture even, but they still don't have a clear diagnosis. I think this will likely lead to an increase in the number of diagnosed patients over time.

<unk> population of patients that we found beyond the 200 in the U S. For example that have this.

That have these variants of uncertain significance order term to the U S and I think that that will that's another pool of patients who have symptoms of a primary immune deficiency have added genetic test oftentimes that's well before we've been involved in the picture even.

But they still don't have a clear diagnosis and I think this will likely lead to an increase in the number of diagnosed patients over time.

Christian Glennie: Sorry, as a quick follow-up to that, is it your understanding that those patients would also be appropriate for Joenja, even if they haven't got formally defined genetic mutations that are driving APDS currently, or understood to drive it?

Sorry, I had a quick follow up to that then is it.

Starting that those patients would also be appropriate for <unk>, even if they have gotten sort of formally defined.

Genetic mutations that are driving EPS currently.

Well on the set to drive it yes.

Anurag Relan: Yes.

Christian Glennie: Right.

Anurag Relan: That's correct. They would be appropriate for Joenja because they would have APDS if that's confirmed. Right now, oftentimes they have symptoms and clinical manifestations of APDS. They have variants or mutations in one of the two genes that leads to APDS. The question is, are those mutations, are those variants disease-causing? That's part of the effort that we're helping clinicians gather information, get access to further testing, and help them resolve that question for those patients. Because they're also, like I said earlier, sitting in limbo and don't have a clear diagnosis yet. If they get a clear diagnosis of APDS, then those patients would be potentially eligible for treatment with Joenja.

Bryan that's correct that they would be appropriate for Georgia, because they would have a PDF. If that is confirmed right now they have a oftentimes they have.

Symptoms and clinical manifestations of AP DFS.

And they have variance so.

Christian Glennie: Thank you. One final one, if I can, then I'll get back in the queue. Is it possible to give any rough sort of guidance for Joenja for the year, whether that's in terms of patient numbers or revenues? Obviously, you've got 60 patients in the program already, $3.8 million revenue in Q2. The 60 patients is already a 40% penetration of the 150 patients in the US that are over 12. Any sense for penetration rates or patient numbers by the end of the year would be great.

Hundred and 50 patients in the U S that are over 12.

Said any any sense for penetration rates will patient numbers by the end of the year would.

It would be great.

Sijmen de Vries: Yeah, I understand that, Christian, but it's early days, right? Yes, we're off to a good start. We would like to see a bit more development of the numbers. Obviously, the numbers are ticking up all the time, right? We'll keep you updated on a quarterly basis for now, until such time that we get a little bit more feel for the market and the development going forward, then we will start giving some different guidance. For now, no, not yet. Have to be a little bit more patient for that. We will eventually, of course, do that. We'll keep you on a quarterly basis updated on the number of patients that are on therapy and are enrolled. Okay?

Yeah, I understand that the Christian but you know.

Early days rights, Yes, we are off to a good start and we would like to see a bit more development of numbers. Obviously the numbers are taking up all the time right and we'll keep you updated on a quarterly basis for now until such time that we can live with more you know feel for the market and the development going forward and then we will.

Start you know, giving some different guidance. So for now no not yet I have to be a little bit more patient for that but we will eventually of course, we'll have a we'll keep you on a quarterly basis updated on the number of patients that are on therapy N. R enrolls okay.

Christian Glennie: Yeah, appreciate it. Thank you.

Yep I appreciate it thank you.

Sijmen de Vries: Thank you.

Okay.

Operator: The next question comes from Joe Pantginis from H.C. Wainwright. Joey, your line is open. Please go ahead.

The next question comes from Joy Pan Genius from Hey, So you went right trying to hold on a second please go ahead.

Joe Pantginis: Hey, guys. Thanks for all the details. Appreciate it. A couple questions on the initial dynamics of the Joenja launch. I guess, and of course prefacing it by this is all early still, what is the general time it takes or approximate time it takes to get drugs to patients once they're identified? Second, when you look at these numbers, the 60 and the 43, how many patients have gotten the drug or are on the drug? Of the revenue number that you posted today, which, like you said, beat overall expectations, how much of that, if any, is based on just getting drug into the inventory channel? Thanks.

Hey, guys. Thanks for all the details that I appreciate it so a couple of questions on the initial dynamics of the <unk> launch so I guess.

Course, perfect thing if I. This is all early still.

What is the general time it takes for our approximate time it takes to get drugs to patients want there once they're identified and then second when you look at these numbers the 60 in a 43, how many patients.

Have gotten the drug around the drug.

Of the revenue number that you posted today, which you know like you said beat overall expectation you know how much of that if any is based on just getting dragged into the inventory channel. Thanks.

Sijmen de Vries: Okay. That's an excellent question, probably for Steve to go into a little bit more detail. Steve, could you pick that one?

Okay. That's an excellent question probably for Steve to go into a bunch of <unk>.

A bit more details please could you pick that one.

Stephen Toor: Yep, sure. Thanks, Sijmen. Morning, Joe.

<unk> good morning, John .

Joe Pantginis: Morning, Steve.

Stephen Toor: The first thing is, as Sijmen alluded to, once the patient's enrolled, we get the starter pack out. They're on therapy pretty quickly. In terms of the time it takes to get approval for patients, it's typically within so far between four and six weeks, which is pretty quick. We've seen one or two is a little quicker, and one or two is a little more. Think of it in a four to six-week period. We're not having to bridge too many patients beyond the starter pack. In terms of stocking, it's almost zero. I mean, we put some in right at the very start. We were able to process patients very quickly after launch, which means we burned through that very quickly. The way in which our partner works, PANTHERx Rare, means we're basically practicing just-in-time delivery.

Uhm.

First thing.

That is kind of alluded to once so patient and rode we get started <unk> and so they don't <unk>, they don't therapy pretty quickly.

And then in terms of the time it takes to get drug Ah sorry to get approval for patients.

Typically <unk> sofa between four and six weeks, which is pretty quick.

We've sent one or two is real quick here and <unk>, but think of it in four to six week period. So we're not happy to bridge too many patients be <unk>.

In terms of smoking, it's almost zero I mean, we we we put some in bright at the very start Uhm, we were able to we were able to process patients pretty quickly off the launch which means we put him through that very quickly.

The way in which partner works <unk> basically practice and just in time delivery. So for the most part that revenue is generated by demand Bud patient demand.

Stephen Toor: For the most part, that revenue is generated by demand, by patient demand.

Joe Pantginis: That's great to hear. Thanks.

That's great to hear things and then.

Joe Pantginis: Please go ahead.

Joe Pantginis: Oh, yeah, absolutely. I feel I have to ask my next obligatory question that I usually go to. When you look at OTL-105, obviously, you're holding it close to your chest, but I'm curious, with the ongoing preclinical models that you're doing, will we be able to see, or when will we be able to see any of the data coming out of these models?

Oh, Yeah, absolutely I I feel I have to ask my next obligatory question that I, usually go to when you look at Oh T. All 105. So obviously you know, it's you're holding it close to your chest, but I'm curious with the ongoing preclinical models that you're doing well, we'd be able to see or when will be able to see any of the data coming out.

These model.

Sijmen de Vries: Would you like to comment on that, Anurag?

Would you like to comment on that on Iraq.

Anurag Relan: Sure. Good morning, Joe. We're making progress, together with Orchard, in those models. I don't have a firm timeline that I can say that this is going to progress in the next couple of weeks or not. I can say that I expect probably toward later this year, we should be able to provide some further updates on where we are with that program, the data that those preclinical models are generating, and how we plan to move the program forward.

Sure. Good morning, Joe So, we're making progress together with orchard in those models I I don't have a firm timeline that I can say that it's gonna you know.

This is going to progress you know in the next couple of weeks or not but I can't say that I expect preferably towards later this year, we should be able to provide some further updates on where we are with that program. The data that those preclinical models are generating and how we plan to move the program forward.

Joe Pantginis: Great. Appreciate the details and nice to see the strong launch. I'm Joe Pantginis.

Great I appreciate the details and nice to see the strong launch enjoinder.

Anurag Relan: Thanks, Joe.

Thanks, Yeah.

Operator: The next question is from Sushila Hernandez from Kempen. Sushila, your line is open. Please go ahead.

The next question is from Sioux Sheila Hernandez from fee. Okay. So she lay your line is that can please go ahead.

Sushila Hernandez: Yes, thank you for taking my question. Also on Joenja, you mentioned it takes four to six weeks to get approval. Could you elaborate a bit more on the time for identification to enrollment? Could you share a bit more on that? Thank you.

Yes. Thank you for taking my question.

Also enjoying yeah. So you mentioned it takes four to six weeks to get approval, that's congested and I'd like to get more on the same firm identification to enrollment could you hear if it's more than that thank you.

Sijmen de Vries: Steve, would you like to comment on that?

Steve.

Stephen Toor: Yeah. Thanks, Anurag. It's obviously variable, but relatively quick. Once a physician and the patient have that discussion, it's literally governed by the time it takes to complete the form, send it into hub, and then generate the starter pack. It's a day to a week, but really dependent on the speed at which that process happens. It can be very, very quick. Easily a day.

Yeah, I think so and so.

It's always the variable, but relatively quick once once the physician and the patient have that discussion.

<unk> by the time it takes to complete the phone, but then generate the starter pack so I.

I mean, it's the day to a week, but really dependent on the speed at which that process happens, but it can be very very quick these easily [laughter].

Sushila Hernandez: Okay. That's clear. Thank you. Just on the RUCONEST. You mentioned that over 70 quarterly new patients. Where are these patients coming from? Are these treatment-naive patients, or did they switch?

Okay. That's care. Thank you.

And just on the goodness you mentioned and says over 70 currently new patients were already stations coming from already taken the ninth faces alright, Okay switch.

Sijmen de Vries: Again, Steve?

Okay, I'm just <unk> [laughter].

Stephen Toor: For the most part, these will be switch patients. At this point in the evolution of the market, there are very few treatment-naive patients. These are patients who are either not satisfied on their current acute therapy or require, as per the guidelines, a second acute therapy to make sure that they have what they need to deal with any breakthrough attacks.

For the most part these will be switched patients at this point in the evolution of the market. There are very few very few treatment naive patients.

So these are patients who either not satisfied on that kinda cute therapy or required as could the guidelines a second acute therapy check to make sure that I have what I need to deal with any breakthrough attacks.

Sushila Hernandez: That is clear. Thank you. A final question. Could you share progress on your BD activities? How is it progressing?

That's clear. Thank you and then the final question could you hear pro wrestling your BB activities, how it is progressing.

Sijmen de Vries: That's a good one, Sushila. We have a very active team. I think if I remember some numbers, they turned over 150 opportunities over the last 12 months. The vast majority, of course, was quickly disposed of. We have an internal committee, including Anurag and Stephen, who take a next look. Out of that, a much smaller number, of course, comes to the fore, and we do some more analysis on that. We've done a few due diligences over this last year, just last year as well. I think I can say the efficiency of the BD process has significantly improved over the last year. We came a couple of times very close, we're very careful, of course, with the first one. As you know, in business development, it's all or nothing, right?

That's a good one that's for sure.

We have a very active team I think if I recognize I remember some members they turned over 150 opportunities over the last 12 months Uhm endeavor.

<unk> majority of course was quickly.

Disposed off but then we have an internal committee, increasing inquiry, including on a rock and Steven looked I'm, taking next look and then out of that.

Small a much smaller number of course comes to the fore and we do some more analysis on that and we've done a few due diligence is over this this last year. It just last year as well so we're making.

I think.

I can say the efficiency of the BB Pro Bridi process has significantly improved over last year and you know we we can came a couple of times very close, but we're very careful of course.

First one is you know in business development, it's all or nothing right. There's no dealer orders a deal so but yeah, we remain optimistic about being able to to acquire.

Sijmen de Vries: There's no deal or there's a deal. Yeah, we remain optimistic about being able to acquire or in-license, preferably in-license, of course, that's the preferred mode of action, another asset that is in mid to late-stage development so that we can actually plan for another asset to be launched in the not-too-distant future. That's all I can say at the moment, I'm afraid.

Acquire or in license before we in license of course, that's the preferred mode infection and another asset that is in the mid to late stage development. So that we can actually.

Plan for another asset to be launched in the not too distant future.

And that's all I can say at the moment I'm afraid.

Sushila Hernandez: Okay. Thank you. Looking forward to updates on that front as well.

Okay. Thank you looking forward to that if that's on that front as well.

Sijmen de Vries: Thank you.

Thank you.

Operator: As a reminder, that's star followed by one on your telephone keypad to ask a question. The next question comes from Natalia Webster from RBC. Natalia, your line is open. Please go ahead.

As a reminder, that staff led by one on your telephone keypad to ask a question.

And the next question comes from Natalia Webster from RBC totally on your line is open. Please go ahead.

Natalia Webster: Hi there. Thank you for taking my questions. I just have two, one on RUCONEST and one on leniolisib. On RUCONEST, we saw an improvement in Q2, and you reiterated your guidance for the full year. I was wondering if there's any risk of sort of the reimbursement issues that we saw in Q1 reoccurring at all. Also, you mentioned sort of strong market demand and continued growth in unique prescribers, but I was wondering if you're able to provide some color specifically around what you're expecting for H2 and sort of in the near term into 2024.

Hi, there. Thank you for taking my questions and I just have to pay one.

<unk> <unk> <unk>.

He can't any restaurants special.

Yeah.

I was wondering if there's any rescue.

[noise] fascination sent me still in Q1 back hiring until.

And then it'll say you mentioned.

Market continued person unique prescribed just wondering if you're able to find some color specific day around what you're expecting S. H T O N.

And then they can catch textural.

Natalia Webster: Just to-

Natalia Webster: on leniolisib. Do you want to go to that first so I can ask the next one after?

I have an answer that can assist you in your life.

And how did you want to.

Get you that fast.

Sijmen de Vries: Yeah, probably a good idea to let Stephen answer that question first. Is that okay?

Yeah, probably a good idea too that's Steven ask that answered that question first okay.

Stephen Toor: Sure. Firstly, the event that happened in Q1, we're not expecting a repeat of that. It was a very specific market-wide event that was unrelated to Pharming. It affected everybody, and it was rectified towards the end of Q1. My suspicion is that's very much a one-time event, and I don't expect that to occur again in the future. In terms of growth in prescribers, it's actually been very consistent for the last seven or eight years, certainly I've been with Pharming and we've reached a control of RUCONEST. There's still, as I mentioned, another 38% of HAE-specific prescribers in the US who've not yet tried RUCONEST and we continue to call on them as well as our core customers. At this point, I see no reason given the last seven or eight years to not expect that same steady growth as we move forwards.

So firstly the the event that happened in queue <unk>, we're not expecting a repeat of that it was a very specific market a <unk> blanket wide event that was unrelated to it's a farming.

Should everybody and it was rectified towards the end of Q1. My my suspicion is that is very much a one time event and I don't expect that to occur again in the future.

And then it terms of grocery and prescribers, it's actually been very consistent for the last seven or eight years I've been with farming and we've reached a control of the <unk>.

There's still as I mentioned, another 38% of H a specific prescribed as in the USA not yet tried reconnect and we continue to call on them as well as a cool customers.

At this point I I see no reason given the last seven or eight used to it. So don't expect that same steady growth as we move forward.

Natalia Webster: Great. Thank you. Just secondly, on leniolisib, regarding the timing for the CHMP decision. You say that you expect this thing to be for this year. I think you said H2 previously. Just wondering if there's been any delay there or just being a bit more specific.

Great. Thank you and and then for a second <unk>.

Regarding the timing for this H M. P decision you say that you think he for this yet but I think he said H T. Previous based on just wondering if there's been any today.

Okay more specific.

Sijmen de Vries: Do you want me answering that, Anurag?

You will be <unk> I don't know.

Anurag Relan: Yeah, I think we're just continuing to work collaboratively with the CHMP and EMA and answering their questions. I think we're just giving some more detail and as we have more detail around the timeline there, that's why we're saying the Q4 now.

Yeah, I think we're just continuing to work collaboratively correct collaboratively with the C. H M P and email and answering their questions and I think we're just getting some more detail as we have more detail around the timeline there that's why I'm using the fourth quarter now.

Natalia Webster: Great. Thank you.

Alright, thank you.

Operator: Next question comes from Hartaj Singh from Oppenheimer. Hartaj, your line is open. Please go ahead.

The next question comes from <unk> from Oppenheimer, sometimes your line as I can and please go ahead.

Asani Jung: Hi, this is Asani Jung for Hartaj. Congratulations on the good quarter for RUCONEST and the nice start for Joenja. I have questions on the APDS patients. How can we think about the growth rate of APDS patient number quarter by quarter? Is seasonality a factor to consider? Thanks.

Hi, this is <unk>.

My account for Hot tubs, congratulations on the corner for nothing nice Diamond dogs.

Okay I'll.

I'll have questions on the P. P. S. I patients how can we think about the glass bedtime ATT application number quarterback whatever.

<unk> a side that your account for that thanks.

Sijmen de Vries: You asked if there's any seasonality in APDS? Is that right?

So you ask if there's any seasonality and in a P. D S.

Alright.

Asani Jung: Yeah, I mean the growth, like how can we think of the growth rate of APDS patient numbers quarter by quarter and would seasonality be a factor to consider down the road?

Yeah.

Like how can we think I'll browse right.

Patient number quarter by quarter.

<unk> nowadays.

A factor to consider.

Sijmen de Vries: Right.

Asani Jung: Thanks.

Sijmen de Vries: Stephen, would you like to comment on that?

So you'd like to comment on that.

Stephen Toor: Thank you, Sijmen. Obviously it's an ultra-rare disease. We had a bonus of patients to start with, but we're still very much in the launch phase. I'd expect steady growth as we move forward and really no seasonality. Obviously in the outer years, you start to see the rate of growth slow as you would do with any launch. Nothing specific in terms of seasonality where we would expect significant troughs or spikes. Does that answer the question?

Thank you so I'm in.

No for rare disease, we were the photos of patience <unk>, we're still through the <unk>, we're still in the very much in the luge face.

Uhm steady growth as we move forward and really no seasonality.

And you have to use you start to see the rate of growth slows you would do with any of those.

Nothing specific in terms of seasonality that do we where we would expect significant drove so spikes does that answer the question.

Asani Jung: Yes. Very helpful. Thank you.

Yes very helpful. Thank you.

Operator: We have a follow-up from Christian Glennie from Stifel. Christian, your line is open. Please go ahead.

We have a follow up from Christian Glennie from stifle Christian your line as I can please go ahead.

Christian Glennie: Hi, thanks. Just I thought it was worth following up on. Obviously, you filed applications, Canada, Australia, Israel, potential approvals next year. What's the commercial strategy there? Is it better to find partners? Is it worth doing it yourself in some of these markets? What should we expect there? Obviously, in Japan, you need to run a trial that's a bit further down the track. What would be the strategy in Japan?

Hi, Thanks.

Thought it was worth pulling up on you know cause he filed applications, Canada, Australia, Israel potential approvals next year.

What what's the what's the commercials trustee. There is is is that you can afford you know is it is it better to find partners. If it if it were thinking of yourself in some of these markets. What should we expect that I don't see in Japan, you need to run a trial misfit further down the track.

Sijmen de Vries: First of all, Christian, before I hand over to Stephen. There's no trial requirements in Australia and in Canada. We have submitted the files. We have received a priority review as well from those authorities. It's a matter of waiting for, first of all, the validation of the regulatory files. We'll go through that, and then we'll await the opinion of those regulatory authorities. It is probably mid-H1 2025. I'll hand over to Stephen to explain how we are approaching the commercialization in those markets.

Yeah, Yeah first of all.

Christian before I hand over to to Steven There's no trial requirements right in Australia and in Canada. We have submitted to <unk>. We have received a priority review as well from those authorities. So it's a matter of waiting for first of all the validation of the vial of the files <unk>.

Files and then of course, we'll go through it off and then a little way to do your opinion of though so the regulatory authorities somewhere and S as alluded to and and some of them.

Probably admit.

The first half of next year, then hand over to Stephen to explain you know how we are approaching the commercialization and dose he knows Marcus.

Stephen Toor: Thank you, Sijmen. Hi, Christian. Certainly, I think to answer what's at the heart of the question, we have no plans to out-license in any of those markets and give away or frankly, the value of the product to other companies. The structure, though, and the go-to-markets with just Joenja would be different from, say, the US or Europe, where we have a pretty large footprint. It would be a light, cost-efficient footprint. In somewhere, for example, like Australia, you would service that market directly with probably a hybrid model, a combination of directly employed Pharming employees and then local distributors and partners who can help us service certain elements of the Australian infrastructure. To keep things really efficient from that market, you would also serve Hong Kong, South Korea, which is the third biggest market in Asia Pacific, and other smaller markets.

Alright, Thank you Sir Bye Bye Christian.

So certainly I think to answer that looks at the home of the questions. We have no plans to out license.

In any of those markets.

Give away or frankly, the the value of the product to other companies.

The scripture that when they go to market with just your engine would be different from say the U S or Europe , where we have pretty large footprints.

<unk>, sorry, a light cost efficient footprint.

Then somewhere for example, like Australia, you would service that market directly with a probably a hybrid model combination of directly employed farming employees.

And then you'd like with distributors and problems you can help us.

So there's certain elements of.

Destroyed and infrastructure.

Keep things really efficient from that market you would also so Hong Kong, South Korea, which is the third biggest smoking <unk> and other smaller markets.

Stephen Toor: The plan is very much for us to keep control of the product, keep control of bringing patients on therapy, and obviously reaping the benefits of that for ourselves and our stakeholders. Does that answer the question, Christian?

The the plan is very much for us to keep control of the product key control of bringing patients on therapy, and obviously reaping the benefits of that.

Ourselves and our stakeholders does that answer the question.

Christian Glennie: Yep. No, that's very clear. Thank you.

Yup, that's that's very clear.

Stephen Toor: Thank you.

Okay.

Operator: As a final reminder, that's star followed by one on your telephone keypad. As we have no further questions, I'll hand back to the management team for any concluding remarks.

That's a fun, Vermont is that staff followed by one on your telephone keypad.

Actually I have no further questions her hand back to the management team for any concluding remarks.

Sijmen de Vries: Thank you very much. Thank you, ladies and gentlemen, for attending our conference. I would like to remind you of some of the elements of the outlook for the remainder of the year. You've heard all the positive developments on the leading indicators, the forward-looking indicators underpinning the sales of RUCONEST, that we remain confident to continue to guide on the low single-digit growth for RUCONEST revenues for this year. You've heard about the steady flow of new patients coming in for Joenja and, of course, the immediately available patients that could be brought to Joenja therapy over the coming quarters. You've heard about the regulatory interactions that we have with the Europeans, the plans to follow up in the United Kingdom, and the submissions, of course, to broaden our footprint with the submissions in Canada and Australia.

Thank you very much. Thank you, ladies and gentlemen for attending a conference and I would like to you know reminds you of.

Some of the elements of the outlook for the remainder of the year you've heard you know all the positive developments on the leading indicators stood a forward looking indicators underpinning and the sales of Brokenness that we remain confident to continue to guide on the low single digit growth for <unk> revenues for the for this year.

You've heard about the the steady flow of a new patients coming in <unk> and of course D. Immediately available patients that could be brought to Georgia therapy over the coming over the coming quarters.

You've heard about the regulatory interactions that we have with the Europeans.

The plans to follow up in the United Kingdom, and the submissions and of course to broaden our footprint towards the submissions in Canada, and Australia and of course that we are continuing to invest in the launch preparations in those markets and of course, the clinical trial plans that we have for the secondhand.

Sijmen de Vries: Of course, that we are continuing to invest in the launch preparations in those markets and the clinical trial plans that we have for the second indications of leniolisib, which we will update you on as and when we have received the feedback from the regulator on our plans that have been submitted to the regulators with regards to both what the new indication is. That indication, by the way, as you already have learned from us, has a bigger patient potential than APDS, and we will reveal that later on during the year.

Vacations are also <unk>, which of course, we will update you are on as as and when we have received the feedback from the regulator on our plans that have been that would have been submitted to regulators with regards to both the what's the new indication is an indication by the way as you already have.

Learn from US has a bigger patient potential than that and <unk> and we will reveal that later on during the year and last one at least you know we hope to be able to come back to you June the remainder of the year of course with.

Sijmen de Vries: Last but not least, we hope to be able to come back to you during the remainder of the year, of course, with in-licensing or acquisition news, because we are very keen and have the capabilities to further leverage our commercialization infrastructure that we have and that we are basically expanding towards the coming years, including markets like Australia, Canada, as you just heard from Stephen, but also Japan, to bring more products to that market to further significantly accelerate the growth of our company going forward. Thank you again for being at our conference, and we look forward to updating you on the next quarterly results somewhere in the last week of October. Thank you very much. Goodbye.

Licensing or acquisition news, because we are raking and have the capabilities of course to further leverage alright commercialization infrastructure that we have and that we are basically expanding towards the coming years, you know, including <unk>, Australia kind of as you're sort from Stephen but also Japan and to bring them.

<unk> predicts without marketers refer to significantly accelerates the growth of our company going forward. So thank you again for being at our conference and we look forward to update you on the next quarterly yourselves set somewhere in the last week of October . Thank you very much goodbye.

Operator: This concludes today's call. Thank you very much for your attendance. You may now disconnect your lines.

This concludes today's cool. Thank you very much for your attendance you may now disconnect your lines.

[music] yeah.

Natalia Webster: You are the last party in the conference.

You are the last party conference.

[music].