Brilliant. Thank you all for coming. It's day two of the Jefferies New York Healthcare Conference. My name is Ben Jackson. I work in the European Biopharma research team, and it's a pleasure today to be joined by the team from Pharming. We've got Fabrice Chouraqui, CEO. He's going to talk to us a little bit today through the whole portfolio. A lot going on, a lot of exciting things to come later this year as well. Look, let's dive right in with the questions. You've obviously held the position now for just over a year. Why don't you tell us what you see as the most exciting opportunities going forward from today and what we should be looking out for? Thank you, Ben. Good morning, everyone. That's true, I've been now in this CEO role at Pharming for a bit more than a year. Extremely excited. I joined Pharming as I saw an opportunity to apply a 5X, 10X mindset to a commercial-stage company. I must say, I'm extremely excited advancing the companies to a global leader in rare disease. Along the way, obviously, having the durability of our legacy drug, RUCONEST, being recognized, as well as the quality of our pipeline for which we have actually a number of value inflection point in the very near term. There's always an opposite. There's always challenges and struggles. What, over the last 12 months plus, have you noticed have been the biggest challenges for you, and how are you overcoming those in the role? It's true, there is a never dull moment in biotech. Actually, interestingly, to be very candid, what surprised me the most is having the durability of RUCONEST recognized. This drug has been on the market now for more than 10 years. It has carved out a very specific sub-segment of the market. I had thought that the uniqueness of RUCONEST and the differentiated profile would be better appreciated. Again, more work to do. That's clear. Look, let's start there on RUCONEST then. Despite the launch of a couple of competitors last year, holding up pretty well. Obviously, in 1 Q, we saw a little bit of a decline, which can be explained in part, I'm sure, if you touch on it. Do you want to just talk us through how you see that demand shaping out for the rest of the year? What is the feedback that you're getting from the physicians that either are trying patients on a different drug and coming back or thinking about staying with RUCONEST as well? RUCONEST is a drug that has a very differentiated value proposition for a subcategory of HAE patient. It is a drug that has a very unique mechanism of action, acting on all cascade of the etiology of HAE. It's an IV drug. As such, it's a drug that, over the years, have carved out a sub-segment of the market, and it's being used mostly by patients who have failed other treatments. If you are an HAE patient, you're not using RUCONEST because options were laid out for you on a table by your doctor and you picked RUCONEST. You are using RUCONEST because you need the efficacy, the reliability of RUCONEST because you have a more severe form of the disease. You have more frequent relapse, you have more severe relapse, and you need actually the efficacy of RUCONEST profile. As such, we haven't seen a lot of patients trying actually. Obviously, it is natural when the first oral in an injectable market is being launched that some patients would want to try the drug. We've seen some patients wanting to try the drug. At the same time, we've already seen some of these patients coming back to RUCONEST. More importantly, we are still seeing actually new patients starting RUCONEST, and even new doctors, 10 years on, actually starting using RUCONEST. I think that's a good illustration of the differentiated profile of the drug and the fact that doctors see a specific position for RUCONEST in this evolving treatment paradigm. That's very clear. I think that covers well the competitive angle, the volume side of things. We also saw a little bit of a headwind from exiting the ex-US markets. That's fine. That's fairly explainable. The other part of the picture is also inventories and where that goes. Could you just remind us how that plays out through the year in a typical cycle, just to set expectations going forward? It is true that we've seen an inventory drawdown in the first quarter. I expect things to get, by and large, back to normal. There are seasonality. We've seen inventory drawdown in every years in the past, except last year which obviously magnified actually the year-on-year change. The market is digesting not one, but actually three new treatment in HAE, if I consider as well the prophylactic treatment. If, again, This inventory, the effect of inventory will be by and large managed in Q2. Some remaining effects actually will be managed in the rest of the year. I'm not worried about this. Very fair. When we think about this year, I think at 1Q, we were talking about low single-digit growth for the drug this year. How sustainable do you think that is over the mid and long term? Then when you're making strategic decisions in your seat, how do you plan for what you need to do with the other assets or portfolio to compensate for that, perhaps? No. Thank you for the question. Clearly, we see RUCONEST as a durable cash engine. It is not the growth driver. Joenja, leniolisib, is the growth driver. We have many growth catalysts, commercial growth catalysts. We have a pipeline catalyst. When it comes to RUCONEST, it's about reinforcing the unique positioning of RUCONEST for these difficult-to-treat patients. Future drugs are unlikely to address their needs. As such, there may be opportunity to continue to grow the drug since the market is becoming more dynamic. Some patients failing oral may be considered for RUCONEST. We may see actually potentially an increased source of business here. Overall, it's making RUCONEST this durable cash engine for the company. Ensuring that we rechannel our focus, our capital towards growing Joenja, which has a billion-dollar revenue potential, progressing our pipeline, expanding our pipeline. That's the goal. Let's touch on that point. I think there's a lot going on with Joenja, not just this year, but for the years to come as well. Lots of growth opportunities. Do you want to just highlight in what order do you see these growth opportunities coming forward from today, and how important is each of those in context? We'll dive a little bit more into each as we go. Absolutely. It's true that there are a number of growth catalysts. If you think of it, in APDS, the first indication, the unique indication today we expect pediatric expansion in the U.S. We are launching the drugs in new markets. It was launched in the U.K. successfully a year ago. We just received approval in Japan for both adult and pediatric. We just received approval for adult in Europe a couple of weeks ago. The geo expansion will fuel the growth. There is also a growth opportunity linked to the reclassification of so-called VUS patients as APDS patients. These are patients who had a test. The test was inconclusive, there's been some very interesting data published almost a year ago by a team at Columbia University showing that a number of those patients could be reclassified. The genetic test labs asked for more data, they are working actually to provide more data to allow those reclassification. Pediatric, geo expansion, VUS could really lead to hundreds of millions of revenues for APDS. The real game changer, it's what's coming with the development of leniolisib in higher prevalence PIDs. Genetic PIDs with immune dysregulation, CVID with immune regulation. Here we're talking about moving the addressable patient population from about 1.5 per million to 40 per million. It's a 30 times increase. Here, this is a game changer that will propel the brand to potentially the first billion-dollar drug for Pharming. It's very clear. Let's stick on that point. It does feel like the phase IIs coming up will be very informative to know what kind of confidence we can build in that. Why don't you just touch on what we've seen today so far that builds that confidence as a recent presentation as well, and what gives you that kind of excitement that this could be the next stage, the next step for Joenja in terms of expanding? For sure. There is a strong scientific rationale to study leniolisib in those higher prevalent PIDs. These genetic PIDs with immune dysregulation and CVID are primary immunodeficiencies, as is APDS. We're talking about three adjacent disease under the same umbrella. They share very similar clinical phenotypes which are linked to immune dysregulation, lymphoproliferation, cytopenia, end-organ damages. We see the same across all of those indications. Leniolisib has proven its efficacy in APDS. We also see the same biology. For those genetic PIDs, there is a clear link and documented link between the gene dysfunction and PI3K elevation. For CVID, we see the same type of B and T-cell abnormality. Both, I would say from a clinical standpoint and from a biological standpoint, a very strong rationale. To paraphrase what a world-renowned physician said at our R&D Day a few months ago, one can think about APDS as a kind of clinical proof of concept for these two trials. We're clearly excited, specifically as we have real-world evidence. The drug has been now on the market for three years. On top of it, recently, there was a poster that was presented at CIS, illustrating actually the experience with leniolisib for a cohort of six patients that have been included in our access program. These are six patients that were not included in the CV trial but were included in our access program. The clinicians shared actually their findings. We are seeing that there is actually no disease progression. We are seeing improvement of clinical manifestation across the board, improvement of the immune profile. This is very encouraging. Again, it's not a trial. This is observational finding, but very encouraging ahead of a trial readout. That's clear. I guess when the trial readout comes, what to you does success look like in those phase IIs? Success is obviously a positive benefit-risk profile. It is about improvement on the clinical manifestation of immune dysregulation, so lymphoproliferation, specifically splenomegaly. It is about cytopenia. It is about end of organ damages. It is also improvements in some mechanistic biomarkers related to B and T cell abnormality and inflammatory cytokines. These are actually the type of endpoints that we'll be looking at, which, by the way, are very similar to what we've studied with APDS. That's clear. I guess we always have to think one step ahead in the industry, and that's obviously thinking about what could a phase III look like for both indications. Can you remind us what you've said about that so far? Is it a bit too early to tell before you've got the data? It is indeed. We'd like to see the data. We'll probably engage with the FDA to see what would be the fastest path forward if the data is positive, obviously, to bring the drug to these patients. Our base case that we'll do a phase III. It's important to bear in mind that these are two open label trials. There may be a path forward for some genetically defined patients. Again, to be seen. I don't want to speculate. One step at a time. Let's look at the data. Let's engage with the FDA, and let's ensure that we can find a quick path forward through the right study design. Yeah. When we think about commercial potential as well, obviously that's a big commercial potential, but a little bit further away. There are things driving growth in the coming months and years. I think one of the most talked about ones that we have on our side is how much Europe in the adult population that you've spoken about could contribute to growth this year and could contribute to growth next year. Could you remind us how you think about launch cadence within Europe? Okay. You've got to secure reimbursement and go through those processes. What are the first countries you're going to go into, how should we think about the contribution to 2026 and 2027? Thank you for this question. It's true that we're using Joenja as a platform for expanding our outreach, and we do that in a targeted way. To win in rare disease, you have to execute flawlessly, and you have to do that also very efficiently. We've identified eight countries outside of the U.S. where we believe that because of the nature of the healthcare systems, we can build a sustainable business for Joenja, but also a platform that we can leverage for the launch of future assets. These are the U.K., the top four E.U. countries, Canada, Japan, and Australia. We've launched the drug in the U.K. a year ago. It's doing extremely well. It was an important milestone because it was the first time we were relaunching an asset outside of the U.S. We've now got approval in Japan, where we intend to launch the drug in August. We are now negotiating the price. In Europe, it's going to be a staged launch because of the sequence and the time it takes to get reimbursement. That's going to start with Germany in July. Then, as we will negotiate with the national authorities reimbursement, we'll launch in the other markets. A staged launch, which is not bad actually from a P&L management perspective. So we don't impact the P&L. We can break even quickly and move to, and then shift our focus onto another countries, as the previous countries are becoming profitable. Yeah. I guess the U.S. is also still something that you're building upon, and obviously the release this morning identified that you've had the refiling accepted with the PDUFA date now into October, I believe. Can you remind us how much you think that could contribute to this year in terms of the children potential of that? Then for the lower doses that are also going to be resubmitted, when do you expect to resubmit those, and when could they therefore begin contributing too? It's true that we were very disappointed to receive a CRL in January for the pediatric indication. We were extremely encouraged by the quality of the interaction with the FDA a few weeks later. That led to the resubmission of the SNDA for the high pediatric doses on the day we received the minutes. We didn't lose any time. We are working on the submission of the SNDA for the lowest dose in the summer. We expect some pediatric sales this year. It is included in our guidance. There may be an upside if the approval comes earlier, but today the goal date, as indicated by the FDA, is October 24th for the high dose. Obviously, for the low dose, that will depend on the timing of the submission that will take place this summer. It's clear. When I sat here and spoke to you this time last year, I think we actually started with VUS in this whole discussion. It's now the last one that we're talking about, which is an interesting thing because there's obviously lots more growth opportunities we can think about. Can you remind us what kind of the opportunity in VUS is, and what are the next steps to actually begin crystallizing that and realizing that in a revenue recognition standpoint? No, you're right actually mentioning that now we are about to unlock many more opportunities that we ever thought for Joenja. VUS is still one of them. We were very impressed by the quality of the data that were published by the team at Columbia University in Cell in June last year. One could have expected that this data would lead to the reclassification of some of these VUS patients as APDS. Genetic lab need actually a bit more data, better controls, and so a bit more work is required. This work is ongoing at the present time. It's a bit too soon to tell exactly when the data is going to reproduce. Clearly, the team at Columbia University is very eager to ensure that their work is translating into patient outcomes, and so they're really taking that at heart. I did visit them a few weeks ago. I think we have a duty to ensure that those patients who've had an inconclusive test, that at least some of them can have an answer about their conditions, and potentially access to treatment. That's good. Lots of different growth drivers for Joenja, which is the exciting thing. Then there's even more to talk about that we don't often talk about in today's age, and that's the Abliva acquisition. Obviously picked up a program there for primary mitochondrial diseases. What was it when you saw this program that you thought, "Good fit, this is where we should go now"? What are the next steps for crystallizing that value? I think this acquisition is a good illustration of the capability that we've developed in rare disease and our ability to identify opportunities, assess them, and then engage at the right time. This asset is a great complement to our pipeline. It's a great stepping stone in our ambition to make Pharming a global leader in rare disease. We decided to acquire Abliva because their lead assets, napizimone in PMD, could really address a very significant unmet medical need. There are 15,000 patients in the U.S. alone, an additional 15,000 in the top five EU countries, including the U.K. The mechanism of action of this asset is well-characterized, which is important. On top of it, the asset is in a registrational phase. The protocol and the endpoint have been pre-approved by FDA, and the program has undergone a futility analysis, and both endpoint have cleared futility. For all that reason, we found that there was a very significant business opportunity. We're talking about potentially a first-in-class drug that could retransform the lives of these patients who have no treatment option today. It is as de-risked as possible for an asset at this stage. Clearly a great fit with our pipeline, great expectations. We intend to complete the enrollment of this trial by the end of the year, for readout towards the end of next year. That's clear. You've talked about the interim analysis of the registrational trial, but can you remind us how this trial is designed to actually position the drug in the market itself? The same question that I asked earlier, which is difficult to answer, but what does success look like for you guys? No, I think that's a very important question because obviously it's great to have unmet needs. Having the right trial design and the right endpoint to address those unmet needs is absolutely paramount. Unfortunately, I think we've seen a number of drug candidates not reaching the patients, probably not because they were not good, but because trial were not designed correctly. Here, I was personally very impressed with what the Abliva team did. They've developed very significant capabilities and knowledge in this field. They've learned as well from some failures in the past. There are two endpoints in the trial. Two endpoints that are reflecting two critical manifestation of the disease. Those endpoints have been, as I said, validated by FDA, and they've been published as well. Those endpoints are addressing two key aspects of the disease. The energy production through chronic fatigue and muscle weakness through a sit-to-stand test. In the past, when there's been attempt actually to crack this disease, companies have used only one endpoint, kind of composite endpoint, which was a 60-second walk test which was supposed to address series of things like ataxia, muscle weakness, body imbalance at the same time. Here we have two very distinctive endpoint. We only need one of them, by the way, for success. Obviously, we will strive to and we hope the two to show up positively. We are very excited, I would say, by the prospect. Now it's really about execution, ensuring that we have the right patients on the trial, and looking forward for the readout. That's good. If we jump ahead a couple of years and pretend in our minds that it's all successful or all's gone well, how well is the commercial framework that you have today already set up for a launch of this drug? How much more investment needs to go into that? Sure. I really joined Pharming because I saw a unique opportunity to leverage the amazing work that had been done in the past 10, 15 years and re-advance the company to a leading global rare disease company. I was very impressed by the capability platform that we had built in clinical development, in commercial, in supply chain. We already, as a biotech, took two drugs to the finish line. That's very meaningful. I think that illustrate and validate these capabilities. From a commercial perspective, I think we have the backbone infrastructure. Specifically, the know-how, infrastructure and know-how, specifically when it comes to access, when it comes to patient services. These are complex skills to develop. It's not only having the number of the people, the FTEs, it is really about the knowhow and also the ability to do that efficiently on a small scale, on small patient population, which is even more complex. I think Abliva will fit very well with the capability that we have built. I do not expect us to need to scale tremendously. We will need obviously a few more sales representatives. In this field, we are working on it, even if you need to add 15 or 20 sales rep, this is actually very limited additional investment. There may be a bit of marketing, but again, the backbone infrastructure is here. That is what we intend to leverage as we expand our pipeline and portfolio is really building a scalable infrastructure that with which we can replicate the success that we have had with our assets so far. Yeah. That's very fair. I guess the final question that it all comes down to is a little bit on capital allocation. What is important for you at the moment, and is additional M&A and BD still on the cards after a pretty good track record so far for the company on what they've been completing? I think we have a lot in the near term coming, commercial catalyst, pipeline catalyst. As we intend to build a global leading rare disease company, it is important to continue to work at pace to expand our pipeline, have more breadth, de-risk as well, and potentially look for opportunities to licensing drugs that may be closer to commercialization as well. We are looking at these opportunities. It is always easy, I would say, to acquire assets. I think the difficulty is to make sure that you acquire high-quality assets at the right price. We have developed very significant capabilities in that regard, so I am very impressed with the work being done by the team at Pharming, and that is very encouraging for the future. It is not about haste, but it is about, again, building at pace, and that is what we intend to do. I think that's a really good note to leave it on. Thank you so much for joining me, Fabrice. Thank you all for joining as well. Thank you. Enjoy the rest of the conference.