Good morning and welcome to day three of the Jefferies London Healthcare Conference. My name is Lucy Codrington. I'm one of the European Pharma and Biotech Analysts based in London, and it's my absolute pleasure to have Fabrice Chouraqui, the CEO of Pharming. We're having a fireside today. If anyone would like to answer a question, please raise your hands. Otherwise, Fabrice, why don't we kick off with a short introduction to Pharming in case there's anyone that's new to the name? Very good. Yeah. So I'm Fabrice Chouraqui. I'm the new CEO of Pharming. I joined the company about nine months ago. Pharming is a Dutch-based company that has the vast majority of its business in the U.S.. The company has been around for now a couple of decades, took off the ground in the past 15 years on the back of a protein recombinant platform that yielded a great drug called RUCONEST. Over the past four years, the company evolved dramatically, moving from a one-asset pharma company to becoming a high-growth biotech with two assets on the market, each growing double digit and a high-value pipeline with two programs with a revenue potential for each of them of $1 billion. Really exciting business. I joined the company because I saw a lot of value to unlock and an opportunity to really apply a 10x mindset to a commercial-stage biotech. Great. Okay. You already covered my first question, so I'll move swiftly on. Just quickly, you reported very strong third quarter results a couple of weeks ago, and you raised your revenue guidance again. What contributed to these strong results? I mean, what contributed to the strong results is the continued high growth of our two assets, RUCONEST, a drug which has been on the market for 10 years in HAE, specifically in the on-demand market of HAE, and Joenja. RUCONEST has grown 29% in Q4 year-on-year and Joenja 35%. Joenja is a drug at the early stage of its life cycle. It's a drug indicated for a rare immune condition called APDS, Activated PI3K Delta Syndrome. The combination of those two assets clearly is fueling high growth. Great. Let's start with RUCONEST then. Perhaps it should have been maybe thought of as a legacy drug, but actually it's still, as you said, still growing double digits. Perhaps you could talk us through the role that RUCONEST plays in the HAE market. What are the patients that are typically using it? We've obviously this year seen the launch of an alternative oral option. Why would your typical RUCONEST patients not be tempted to switch for a more convenient oral? RUCONEST has a very distinctive value proposition in the on-demand market. As a consequence of this very distinctive value proposition, it serves the need of severe patients. The vast majority, the immense majority of the RUCONEST patients are patients who, first of all, are not controlled on prophylactic treatment, have failed other on-demand treatments. Because they are patients with more frequent crises, more severe crises, they need actually higher efficacy options. They need treatment which is more reliable, which has a faster onset of action. That is what RUCONEST can provide them. That is because of two things. The mode of action of RUCONEST, it is the only drug that targets the root cause of HAE by replacing the missing or the deficient protein. It is also a drug that has a specific mode of administration. It is an IV drug. It's being self-administered by the patient. As such, it provides the fast onset of action and specifically the reliability that those patients need because we're talking about very severe patients. If they are not able to control their crisis, this means that often they'll end up in Okay. As that, I mean, outlined why current patients would stay on RUCONEST, as that competitor launch continues into next year, how should we be thinking about RUCONEST's position and its growth? Are we thinking about a maintained market share, or actually do you think there's scope for continued growth? As I have just said, RUCONEST is treating a very specific subsegment of the HA market, those hard-to-treat patients. Because of its very distinctive value proposition, I see RUCONEST remaining a cornerstone treatment for those patients. The reason is simple, I do not see any treatment, whether they are new prophylactic treatment or new on-demand treatment, that will be game-changing, that actually will be able to serve those patients either equally or better, far, far from it. I expect to see RUCONEST continue to grow in the HAE market. Obviously, the drug has been on the market for 10 years. Our goal is not necessarily to grow the drug at any cost. It's about to really encore its very, very specific positioning, ensure that we find the right tipping point in terms of level of investment to maintain double-digit growth whilst using the proceeds and the cash that we're generating to invest in high-growth assets in our portfolio and pipeline. Very clear. Okay. So then almost all of RUCONEST's sales are in the U.S.. Why do you think RUCONEST never replicated its success outside of the U.S.? What was the rationale behind your recent decision to exit non-U.S. markets? It is true that RUCONEST has been a very significant success in the U.S. and continues to be. In the rest of the world, I mean, there are many explanations for why we did not see the same. First and foremost, I think for those of you who know the HA market globally, you know that the HAE market is well underdeveloped globally, actually even in well-developed countries. That is actually one of the big reasons. We made the decision earlier this year to withdraw RUCONEST from a number of countries where it was commercialized because it was not financially sustainable. When the drug was launched a decade ago, it was introduced in many countries, but we never put any commercial efforts behind. At a time where we have a number of growth catalysts in our portfolio and in our pipeline, we feel that being disciplined, having the company rightly focused, channeling capital on what matters most would make great sense. Just to give you an idea, we have less than 1.5% of RUCONEST sales coming from all these countries. You can easily understand that from a supply chain perspective, from an administrative perspective, it is actually extremely burdensome to maintain the commercialization. We are and we will be working, obviously, with patient associations, patients, authorities to ensure continuity of treatment. If patients need to access RUCONEST after its withdrawal, we have compulsion-induced programs. That should not be a problem. Very clear. Okay. Let's move on to Joenja then. As you mentioned, this has been on the market for coming up to two years for adults with APDS in the U.S.. Over the next couple of years, there's a number of opportunities to drive further growth and expand the addressable patient population. Perhaps you could talk us through each of those and the timing we expect each of those to occur. Absolutely. Joenja is a high-growth drug at the very beginning of its life cycle. It may well become the first billion-dollar drug for Pharming. It is today commercialized in its first indication, APDS, standing for Activated PI3K Delta Syndrome. An indication which is characterized by its biology, which has actually a lot of upside because actually you can put a lot of things in this. It is also developed in other primary immunodeficiencies, which are adjacent indications, very similar indications, but with much larger prevalence. We have two trials ongoing that will read out in the second half of 2026. Coming back to APDS, the first indication here, our current source of business, we've seen clearly a nice uptake. We've seen actually an uptake accelerating this year with more adult APDS patients being identified, more and more adult APDS patients being put on drug. Just to give you an idea, in the first nine months of the year, we had more new patients on the drug than the entire last year. Something really accelerating. We have identified more APDS patients than we did last year. That is actually those patients going to the funnel. We have a number of new growth opportunities that we are about to unlock that will further accelerate that growth. First is the expansion of the indication to the pediatric population. We filed that indication. We just received accelerated review from FDA, and we expect to receive this expansion of the label by the end of January. This is a very meaningful opportunity. I mean, the pediatric patient population is about a third of the APDS population. It is going to add an additional 30% of the potential. We have already identified a significant number of pediatric patients, about 54. We have about a third of them already on drugs. Clearly, something that is very material and that is going to fuel and accelerate the growth of the drug next year. There is another growth opportunity that we are working to unlock. This is the reclassification of VUS patients. Some of you may be familiar with that term. VUS stands for Variant of Uncertain Significance. These are patients who have had a genetic test, but the test came unconclusive. It came unconclusive because this patient carries a variant that until now has never been documented in the literature. There was a paper published in Cell in June that shows that scientists have identified an additional 100 new variants, which are linked actually to a hyperactivation of Pi3K Delta syndrome, Pi3K Delta pathway. As such, actually the carriers of those new variants will be reclassified over time as APDS patients. This is a significant opportunity. We expect about 250 patients over time to be reclassified as APDS. That opportunity alone will increase the APDS potential by 50%. That is very significant. As you can see, we're going to be moving very soon from sourcing patients from just one source of business, the adult segment, to unlocking three sources of business that are going to come on top of one another, having the adults, having the pediatric, and having those VUS patients. Clearly, very excited with the prospect of Joenja in APDS. As I mentioned earlier, obviously, this is not the end. This is just the beginning since we have those two new indications that we are exploring, which have much larger prevalence, up to 20 times larger, and therefore clearly a major pipeline milestone in the second half of next year. Great. That was a very helpful summary. Let's just go back to the VUS opportunity then. You've had the Cell publication. Perhaps you could talk us through then what the process is to getting these patients reclassified. How can Pharming influence it, if at all? What do you expect the timeframe might be if you have any visibility on it? Absolutely. I'm glad actually you are highlighting this opportunity. The process is the following. Again, these are patients for whom the doctor thought they had APDS because they took a test. The test came unconclusive. Now there is the new data published. As we speak, the test labs are actually engaging with the authors of that paper to better understand the data with the goal to incorporate the data in their dataset and then to reclassify those patients. Once they have incorporated those new variants in their dataset, they'll be able to find patients that have had a test and that are the carriers of those new variants. That should trigger a letter or a phone call to the doctors that commissioned the test. They'll tell the doctors that the patient that got tested six months, a year ago, at that time, there was no evidence of a correlation with the Pi3K Delta pathway. Now there is because of the new data, and the patient should be reclassified as APDS. The doctors will probably actually reach out to the patients since these are patients which are desperate in need of a diagnostic. These are patients that have been actually looking for a diagnosis about their condition for years. We expect this to be actually very, very meaningful for the treating physician as well as for patients. That is going to happen over time. Obviously, for the reason that you can imagine, we cannot obviously do the work on behalf of the genetic test labs. We have to stay at arm's length. We expect that actually those conversations will lead to this reclassification as quickly as possible. We will keep you closely posted as soon as we have more information on timing. Great. You also launched Joenja in the U.K. this year and then should have launches next year in hopefully Europe and Japan. How's the initial physician engagement and uptake been in the U.K.? What read across is there from that to your potential launches next year? The launch in the U.K. is going extremely well. There's a lot of excitement. There's been even actually late press coverage about the value of the treatment. We're hearing that some patients now are no longer considered for stem cell transplant because they're being put on Joenja. Clearly, the community is extremely excited by the prospect of Joenja in this indication. We expect to receive EMA approval in the first half of next year. We are using Joenja to expand geographically. We have selected eight countries outside of the U.S. where we believe that we can build an ex-U.S. presence with Joenja, and that could pave the way actually for a larger footprint for Pharming. This is a targeted expansion plan. Things actually are going well. We expect regulatory approval in Japan next year. Obviously, as important as the regulatory approval, what's absolutely essential is also to ensure that we get the right price in all these markets, a price that recognizes the value of the drug. Okay. Let's move on to your Abliva acquisition for KL1333 for primary mitochondrial disease. Perhaps you can talk us through what you found to be attractive about that asset and how it fits within Pharming. Yeah, this asset is a great addition to our pipeline and to our vision to make Pharming a leading rare disease company. We got extremely interested in this asset for three reasons mainly. I mean, this is a company that we've been following, Abliva, for quite some time. We decided actually to acquire them because first, the mechanism of action of KL1333 is very well characterized. The patient population is very well identified. We're talking about 15,000 patients in the U.S. and an additional 15,000 in the top five EU countries. Those patients are treated in the center of excellence. Last but not least, the drug is in registrational phase II trials. The protocol as well as the endpoints have been pre-approved by FDA. The program has undergone a positive futility analysis at the end of last year, which obviously largely de-risked or further de-risked this registrational trial. That drove actually our interest and the acquisition. Okay. You mentioned the phase II pivotal trial. What would a successful outcome look like in this trial? Does it differentiate at all to any competitors in the space? PMD, Primary Mitochondrial Disease, as I mentioned, it's a significant rare disease. This asset has more than a billion-dollar potential. In the trial, it's a disease which is characterized by extreme fatigue and muscle weakness. These are the two primary endpoints in the trial. The trial will be successful on either of these endpoints. We only need one of these endpoints actually to be significant, statistically significant for the trial to be successful. Fair to say that the futility analysis that I just mentioned earlier, during that analysis, actually the two endpoints were deemed non-futile, which actually is very encouraging. Okay. How then, assuming the trial is successful, how does your existing commercial setup provide synergies for how you would need to potentially launch KL1333? Do you need any additional investment in order to launch it? I've been very impressed when I joined Pharming by the level of capabilities in clinical development and supply chain and commercial and market access patient services. It's very impressive. Having run Novartis in the U.S. and knowing what's the bar to commercialize efficiently specialty care drugs, I was very impressed by the level of the capabilities that the company has developed over the years. We have actually this capability platform that will allow us to commercialize this new asset. We will probably need a few customer-facing reps, but that is actually a very small fraction of the commercial investments. As you probably know, commercializing a drug in the U.S., it's complex. We have already that infrastructure. We'll be able to leverage that infrastructure fully for the commercialization and the fast uptake of the drug. There'll be some more investment, but it's going to be limited given the existing infrastructure. Okay. So you have achieved impressive growth in operating profit and cash flows in the last few quarters. Do you think this can continue into 2026 and perhaps beyond? Clearly, the company is enjoying a great momentum. It is driven by the growth of our commercial assets. As I've just explained, there are a number of growth catalysts to be unlocked in the very short term that's going to continue to fuel that growth. Building a high-performing company is about obviously driving the top line, but ensuring optimal capital allocation. That is also fueling the operating profits and the cash we generate from operations. We have signaled the market that this is actually one of our clear priorities. We announced, for instance, a few weeks ago that we have cut. It is not like a plan. We have cut 20% of our G&A headcounts because, I mean, just because we feel that this capital would be much more productive in other areas given the number of growth catalysts that we have. The goal is not to manage the company through the bottom line. I mean, we would destroy value given the number of commercial catalysts and pipeline inflection points. The goal is to be disciplined, to channel the capital where it can generate the greatest ROI and return the growth into value. That is a focus. That is why I'm extremely pleased that we were able to attract a new CFO, Kenneth Lynard, who comes with a background of value creation, having been the CFO of two P-backed companies lately. It'll be of great help to me and the leadership team, again, to manage the P&L optimally so we can really drive the growth and the value creation in the near and long term. Perfect. With that, we've run down the clock. I'd like to thank Fabrice for his time and the audience for attending. With that, we'll move on to the next session. Thank you, Lucy.