Good morning or good afternoon, ladies and gentlemen; I'm here. Next slide, please. Welcome to our results conference for the first half and second quarter of this year. And I'm here with my colleagues, Stephen Toor, our Chief Commercial Officer; Anurag Relan, our Chief Medical Officer; and Jeroen Wakkerman, our Chief Financial Officer who will -- we will collectively guide you through the story. But before I do that, I would like to have this next slide and point you to these forward-looking statement slide. So we will be making forward-looking statements in this presentation. And as you well know, these are based upon our -- upon future expectations that are based on our current expectations and assumptions, and may involve known and unknown risks and uncertainties. As you well know, the results eventually could differ materially from what we have expressed or implied in our statements. Next slide, please. And then, you can immediately move on to slide number 5, please. So what we're doing is -- this is the strategy that we've been embarked on for quite some time now. We're building this leading global rare disease biopharma company. And we do that on the basis of two strong pillars. The first one on the left, of course, is RUCONEST, which has now been on the market for close to 10 years in the US, and of course, deliver sales mainly from the market. And we were very pleased to see that RUCONEST continues to grow very significantly, both in context of the comparison to the previous quarters -- in the second quarter, 23% versus last year; and both when you compare the first half to last year's first half, 16% which is, I would say, a very strong performance, which we're very pleased with. And Stephen will,…

Thank you, Sijmen. Good morning and good afternoon, everybody. We go to the next slide. So I will, as Sijmen said, take you through the RUCONEST and the Joenja performance so far, and then on the last slide for me, before handing over to Anurag, just give you a little update on our medium-term expectations of why we're so confident in the business. So looking at this slide and specifically RUCONEST, you can see, I think in those first four boxes at the top, that the consistent strength in performance really relates to the unique attributes of RUCONEST in the patient population we serve that Sijmen alluded to that more severely affected group. RUCONEST is, as you know, the only recombinant C1 esterase inhibitor in those key core benefits of 97% of patients whose attack is resolved in a single dose. And that sustained effect over a period of days is really why those patients really can abandon other products that don't work as well, find a home with RUCONEST. And it's what drives our continued strong performance. And that performance over a decade now has made RUCONEST the most prescribed -- sorry, the second most prescribed acute therapy in the US, and of course, still posting solid results. In the first half of 2024 -- sorry, yes, first half '24, new patient enrollments have continued to strengthen over and above last year. And in that first half, we actually enrolled 170 new patients, and that represents an increase of 18% over the first half of '23. That, as you would expect, is driven by an increase in new prescribers as well as maintaining our existing ones. And our sales teams added another 36 new prescribers in the first half of '24, taking the totals at over 760, which is…

Thanks, Steve. Now, we can jump to the next slide and we can review some first -- some detail about Joenja. So Joenja is FDA approved to treat activated PI3K delta syndrome, or APDS, in adult and pediatric patients who are 12 years of age and older. APDS, as Steve mentioned, is a rare, serious genetic disease caused by hyperactivity in this PI3K pathway; and it's associated with early mortality, often due to lymphoma. And it's also important to note that APDS is progressive. So experts state that early treatment is important. Joenja is a PI3K delta inhibitor which regulates this hyperactive signaling pathway that's found in APDS patients. The FDA approval last year was based on a randomized pivotal study as well as an open-label, long-term extension study. And Joenja treats the root cause of APDS by correcting the underlying immune defect, thereby addressing both the immune deficiency and immune dysregulation that's found in APDS patients. The safety of Joenja was evaluated in this placebo-controlled study as well as the long-term study that is just wrapping up, in fact. And no drug related serious adverse events or study withdrawals were seen in these trials. And on the next slide, we can see some of our patient finding efforts. Specifically, we are supporting numerous activities to raise the awareness of APDS and share data about leniolisib and Joenja. So on the left, you can see the medical education types of things that we engage with and the organizations that we're working with to raise this type of awareness. But also importantly, we have several efforts to help patients get an accurate diagnosis. The first of which is genetic testing. We have a sponsored, no-cost genetic testing program. We have support from genetic counselors and, as Steve mentioned, we're also working…

Thank you very much, Anurag. And good morning, good afternoon, everybody. I am starting off with the financial highlights of the second quarter 2024 versus last year. You see that we grew our revenues by 35%, and that was driven by volume of both RUCONEST and Joenja. RUCONEST went up by 23%. Gross profit was fairly stable at 89%. So the margin was at 89%. So the gross profit basically grew in line with our revenues. OpEx development. The OpEx went from $65.8 million last year in the period to $70.1 million, and that is reflecting a continued investment in Joenja, both in the US and EU, rest of the world; and also investment in compliance, IT, HR-related areas. And that is to support the growth of the company. We see an operating profit that we adjusted. So this is not the reported operating profit, but adjusted for a few one-offs that we had last year, and we had a milestone payment of $10.5 million. So that was a cost, but we also had a gain on the Priority Review Voucher that you may remember. But basically, on a like-for-like basis, we see that the operating profit gap narrowed from $5.3 million last year to $3.1 million this year. And the net profit was around $1.3 million last year, minus $1.2 million, so a loss this year. And please be aware for the analysts that in the finance result, we had a gain of $3.4 million this year versus a loss of $1.8 million, and that gain is related to the convertible bonds and a reclassification of the derivative to equity issuance, technical adjustment that you are to be aware of. And the overall cash and marketable securities, they went down by almost $42 million. The biggest driver of that…

Thank you, Jeroen, and thank you very much. So yes, you have just heard from Jeroen that we are sticking to our total revenue guidance for this year between $280 million and $295 million. Obviously, there were these quarterly fluctuations which are typical. Then you heard about our patient finding efforts on all fronts for Joenja and a number of increasing patients that we have identified and especially the expectations that we currently have on the current base on the basis of current initial results from that small trial with regards to the VUS validation efforts, which amount to about 20% of those 1,200 patients that we expect to become available over the coming year, and of course, will drive significantly the growth of Joenja in the US in '25 and onwards. The leniolisib sales, you have heard that there is a great interest and that's obviously, these Named Patient Programs have a lot of administrative procedures and are, of course, not always very fast, but we know that there's quite a few patients in the pipeline waiting for clearance to enter into the program all over the world. The clinical trials you heard Anurag talk about for the regulatory filings for Japan, the second biggest market in the world. And of course, the pediatric label expansion which will make, again, a bonus of at least 25% of additional patients available for therapy for Joenja. You heard about regulatory approval progress, especially in the United Kingdom, where we expect at the end of fourth quarter of this year to hear back from the regulators. And of course, that we are confident that we will be able to speak to EMA again following the submission in January '26 of the remaining -- last remaining question by EMA and the confirmation, of course, that EMA confirmed the clinical benefits and safety of leniolisib makes us very confident as we're looking forward to also getting into the European markets in 2026. Then you heard about the initiation of that Phase 2 clinical trial for that second indication which Anurag described for PIDs with immune dysregulation and our plans for the third that have been submitted to the regulators and where we're expecting feedback and hopefully, starting a trial in the not too distant future as well. So in other words, we will then start developing two subsequent indications for Joenja. And last but not least, the continued focus on potential acquisitions and in-licensing of clinical stage opportunities in rare diseases to further build our portfolio and diversify the company further. And let me tell you, we have a lot of activity going on there, but we are, of course, very precise in terms of what kind of opportunities we actually will bolt on to our company. So that concludes the call, and I would like to now go back to the operator and ask and open the floor for questions. Thank you.

[Operator Instructions]. The first question comes from the line of Sushila Hernandez from Van Lanschot Kempen.

I have a few, if I may. On leniolisib. So it's a first in APDS that you're about to start the Phase 2 studies in PIDs with immune dysregulation and now looking into a third indication. Could you share a bit more on what prompts you going after this third indication? And are you expecting that this would expand addressable patient population significantly? And also on Joenja -- or sorry, on the VUS validation studies, could you elaborate on the 12,000 patients identified in the US? How did you find these patients? And how many of these patients would you expect to be diagnosed with APDS and refer to enter treatment? And then lastly, with the MHRA decision near, how many patients have you already identified in the UK and similar to the US, can we expect a large portion of these patients to get on paid therapy in the first half year of the launch?

Sushila, I'm happily handing over to Anurag to answer your -- to start answering your question. Anurag?

Sushila, so with respect to your first question about the Phase 2 study that we're starting. So that one is in patients who have one of the several genes. So the examples I gave were PTEN, CTLA4 or ALPS-FAS. These are genes that are known to be linked through hyperactive signaling and these patients have an immune deficiency as well as an immune dysregulation or the dysregulation of often times a predominant feature in these patients. So that's the first indication that we're pursuing outside of APDS. In addition to that, we are looking at another indication, which is, in fact, even larger. So yes, to your question that this will expand the potential population. But we're looking at this second indication, which also has immune dysregulation and is a subset of primary immune deficiency, and it has features again similar to APDS. So I think that's something you can see across these three potential indications is that they all have these features of immune dysregulation or autoimmunity and auto inflammation. So on the second question was about the US resolution efforts and how we expect that to evolve? So what we've identified already is 1,200 patients. So what does that mean? That means that these are patients who are -- have had a genetic test usually as a part of a primary immune deficiency panel. And again, these are patients that have genetic testing most of which that we were not involved with. So they had this genetic testing perform. The result came back with a result that showed either in one of those two genes, this VUS result. And we are aware of this through these databases that we have access to at large genetic testing companies in the US. And we expect that over time, we'll be able to eventually resolve these in the US. Now we know again, historically, looking at other genes, that's about 20% of the VUSs get resolved and converted into disease causing or what's called likely pathogenic or pathogenic. The other data point that we have is that we recently tested 25 of these US patients, and we had what's called functional testing performed in these patients, and we found a similar number, so about 20%, five out of the 25 were then upgraded or reclassified into APDS. So when you start doing some simple math, you see that this could significantly increase the population of APDS patients in the US.

Thanks, Anurag. And with regards to your question about the UK, Sushila, we have currently 11 patients on early access therapy in the UK. There's 61 patients identified, of which 37 are over 12 years of age. So there's already a quite an interesting population in the UK available. And of course, we expect that once we had the reimbursement, which will take some time and will be somewhere, I suppose, normally in the first half next year, those first patients will go on paid therapy pretty quickly. And of course, by that time, we will have also clarified the VUSs. And of course, there will be in the UK also patients with the VUSs. So they will be additionally coming potentially on to therapy as well. So that's the sort of numbers for the UK that we currently see. And of course, we continue to seek for patients in the UK, but there's already one per million identified, as you can see from 61 out of a population of roughly 60 million in the UK. I hope that answers your question, Sushila.

Yes. And if I may ask one other question. Could you provide an update on your BD efforts? Have you brought in your search?

Yes, we will broaden. We have basically a lot more incoming and also we reached out a lot more because we have now a Chief Business Officer on board since the last quarter of last year. That has led to quite a few interactions, which quite actually even resulted in some non-binding offers that we issued. But of course, when you then look into the diligence following your non-binding offer, you sometimes find stuff that you were not expecting. And of course, it is also sometimes possible that the other party does not necessarily in the end, want to conclude the deal because it takes 2 to 10 as you know. But yes, there's been a lot of activity and we're virtually all the time assessing an asset under due diligence as we speak. So there's quite a lot of intensity here going on.

We will now take our next question. And the next question comes from the line of Jeff Jones from Oppenheimer.

Right. Congratulations on the quarter, gentlemen. Just two from us. You spoke to it to a degree early on. But with -- for RUCONEST with the anticipated launch of a competing product or competing products in '25 and beyond., what do you see the impact to be? And how are you planning your response? And then for leniolisib in Europe or in the EU specifically, can you provide any additional detail on the work that needs to be done in completing your definition of regulatory starting materials? And when would you anticipate being able to respond to the CHMP? Sijmen, I think you almost said January of '26. And in that case, how does that impact your potential approval time line?

Jeff, thanks. So let me just first answer your second question about Europe. Yes, we are -- we have already initiated that work, and we have a precise time line, that's why we agreed with the European authorities to actually grant us the extension until '26 -- January '26, that is when we plan to hand in the response. We precisely do what they want. So that is also very clear. And then in that case, we expect that probably towards the end of the first quarter, there could be an opinion, which we are quite confident because we have received clinical -- the confirmation of clinical benefit and safety of the product. And when we have resolved that, we are quite confident about the opinion being positive. So all that said, then it takes two months for the European -- for the European Commission to confirm that. So in other words, you could expect that we entered the European -- the first European market, that's probably Germany in, let's say, the end of the second quarter, beginning of the third quarter of '26. That's basically the time line at this point in time for entering the European markets. And then your first question was about -- yes, had a competition for RUCONEST, thank you. Yes. Well, the slide I showed about the hereditary angioedema market is there showing that basically RUCONEST is protein replacement therapy. And Stephen alluded to the response rates, consistent response rates on RUCONEST. And Sebetralstat is a product that works on that bradykinin/kallikrein pathway, and has, of course, good results. So it's good news for patients that there is new therapeutic options available. However, we're also aware that some Sebetralstat was tested in a patient population that is generally responsive to Icatibant or Firazyr. And…

The next question comes from the line of Alistair Campbell from Royal Bank of Canada.

I just really wanted to talk a little bit about Joenja. You've obviously got quite a significant pool of diagnosed patients in the US. Can you talk about the pathway from getting a diagnosed patients to be basically enrolled? What are the key hurdles you have to overcome and then how onerous is that process?

Yes. So I hand over to Steve in the [indiscernible].

It is, as you would expect, can be quite convoluted, but we try to make that as smooth as possible through our own patient services. So essentially, the first part of the process is obviously the patient coming in. That can take some years sometimes, although we're calling all those key centers of excellence. Then once the symptom is reviewed and the patient is worked up, it's the genetic test and if they get that positive test, then they immediately can go into our enrollment program where we'll start to work with their payer on getting them approved. And we've seen a mixed picture there with the payers. But what I will say is that our approval rate is at 98%. And that 2% is they're in NDC blocks. So they will eventually come on stream. They just take a little longer to actually pull through the entire market access or managed care system. So that's the kind of simplified version, which is patient comes in, patient gets diagnosed. They work with outpatient services and then land on therapy. What can sometimes slow things down, and that's why you see a bolus in the first part of the launch and then slower. But nevertheless, still growth as we move forward before those bigger inflection points that Anurag referred to with the US population and the pediatrics, is we're now into those groups where there may be other complications. So perhaps they're on chemotherapy or for whatever reason there are other comorbidities that are being managed. So they remain within our diagnosed pool, and we monitor and we worked already with the doctor and when we can the patient tell them the right time they can move on to therapy. So I think that's the high-level overview of what happens with managed care. That's where we are today with the existing pool of patients before we get those next big boluses in '25, '26 and beyond. Is that okay Alister? Was there any other questions on that?

Yes, maybe just -- can I just follow-up on that? So in terms of those like the -- you've got -- let's say, pool of 50 diagnosed patients over 12. So you have some of these hurdles to maybe sort of get them on therapy. Do you have any kind of sense of the pace at which you see them sort of come on to therapy ahead of the boluses to come? I'm just trying to get a sense of the temper we should be thinking about in terms of patient adds before some of those big boluses you've pointed come through?

It's -- I would love to give you a specific answer on that, unlike the mass markets I've worked on in the past, these are very low absolute numbers. So for example, we added eight this quarter. I think it was two last quarter, and we have these ones that we're working through today. The good news is we would expect that to keep going. But it just -- it won't be linear. Whereas if we say, cholesterol lowering, you can very easily predict that. With this, I'll give you some example. There was literally one patient who's going to come in, in July because they needed to finish their school year first. But what I can say is that those over 40 patients are close to 50 right now that we're working through, we know every detail there is some pretty much know about where they're at and what the tipping point will be, even down to patients whose 12th birthday will be this year. We're ready in front to go and speak to those physicians and say, okay, the 12th birthday is coming up, are they in the right place now? Are they the right weight, et cetera, et cetera. But the simpler answer is I couldn't give you a good prediction on the pace of which that all those patients will come in and over what time period. Only that we're working very hard on every single one of them on a very regular basis and in a very detailed way.

[Operator Instructions]. And the next question comes from the line of Simon Scholes from First Berlin.

I've got two. The first is on the second indication for leniolisib. I mean I gather that you're expecting the Phase 2 data early next year. Once you get the Phase 2 data, I was just wondering what the further timetable might look like as regards to Phase 3? And then the second question is on approval in Canada and Australia. I mean you're now talking about 2025 for both markets. Can you give us a slightly more precise timing for those markets? I mean is it like to be H1 or H2?

Yes. Maybe, first of all, Simon, I think you're that optimistic here with regards to when this Phase 2 study reports. Obviously, it's an open label study. But I think you should more think about and, Anurag, correct me if I'm wrong here. But by the end of next year is probably a more likely thing that we will have insights and can actually formulate the -- how we are talking about with regards to the following Phase 3 trial. And secondly, we don't necessarily always give detailed information on regulatory interactions because they can be a little bit unpredictable. But you're right, you have seen this -- we are anticipating that we have got regulatory action in 2025 from those and not necessarily in 2024. So basically, yes, that is correct. I hope I answered to those questions.

[Operator Instructions]. As there are no further questions, I would now like to hand back to Sijmen de Vries for any closing remarks.

Thank you very much. And thanks, ladies and ladies and gentlemen, for attending our conference. As you've seen, we've posted some stellar results for both the second quarter and the first half of this year. We illustrated to you that we are very confident that RUCONEST will be an important driver for our financials for the coming years to come. Given the fact, that we serve this very unique patient population despite expected competitive entries. We also showed you that in -- and although, indeed, as you heard, the growth for Joenja is continuing this year, it is not going to be linear from Stephen, but that we have a big bolus of patients expecting to come online in the United States from next year -- beginning of next year onwards because of the expected outcomes of the VUS, which will, of course, being an important growth stimulus for Joenja in '25 and '26. You also heard that we expect that we get a positive discussion with the Europeans in the first quarter of '26 and that we are working very hard on getting our pediatrics and our Japanese trials worked out and so that we can actually submit to the Japanese authorities and enter the second biggest market in the world and have another at least 25% bolus of additional patients under the age of 12 available for commercialization, both in that at that point in time, of course, in the US and the Rest of the World and the European Union where we expect to be on the market by then. And then you heard, of course, how we are going to build a pipeline and a product by starting with a Phase 2 study in -- for PIDs with immune dysregulation as a second indication for Joenja imminently. And we are, as of course, waiting for regulatory feedback for the start of a third indication, also a Phase 2 trial that we expect to start in the not too distant future. And last but not least, about the intensifying efforts to in-license or acquire new opportunities so that we can broaden our portfolio in the rare disease and build this global rare disease company. So thank you very much for being here, and we look forward to updating you on our expectation at the end of October when we have our third quarter results. Thank you very much.

This concludes today's conference call. Thank you for participating. You may now disconnect.