Pharming Group N.V.

Good day and thank you for standing by. Welcome to the Farming Group NV 3Q 2024 results conference call and webcast. At this time all participants are in a listen only mode. After the speaker's presentation there will be a question and answer session. To ask a question during the session you will need to press star 1 and 1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question please press star 1 and 1 again. Please be advised today's conference is being recorded. I'd now like to hand the conference over to your first speaker today, Simon de Vries. Please go ahead.

Thank you very much and good morning. Good afternoon, ladies and gentlemen. Welcome to our results call. I'm happy I'm here with the, let me introduce my colleagues here. Stephen Thor, Chief Commercial Officer. Anne-Marie Brown, our Chief Medical Officer. And Jeroen Wakkeman, our Chief Financial Officer. And we will take you to the results and obviously answer all your questions. So before I do that, I'd like to have the next slide and point you to the forward-looking statements, because during this presentation, we will be making the usual forward-looking statements, which, of course, are expressions of our expectations, which can differ significantly, of course, from future results. So having said that, I would like to start with the next slide. And you see a slide of my face there. And you're seeing, no doubt, the announcement that after 16 years here at the helm, which is actually Precisely next week, I have informed the board of directors that I will not be available for re-election. The company is in great shape today, and I'm reaching the beautiful age of 65 next week, so it would be a good moment to basically hand over the helm to a successor who can continue to build a company as we have been over the last few years into this rare disease company that we are building as we speak with a great pipeline and with a global geographical presence. And having said that, I would like them to know to the next slide because they see how we are going to build that. Ruconest, obviously, on the slide number five, please. On the next, Ruconest is, of course, the pillar of the company, has been the pillar for a long time, and you see the results of Ruconest continues to grow, more prescribers, More patients are using Rucanast in this increasingly competitive air terry angioedema market, resulting in a plus in the three-quarter results of more than almost $64 million and almost $173 million for nine months in 2023. So in other words, Ruconest delivers significant cash flows from which we can actually build that company and build that portfolio and build out our commercial presence with, of course, Joangia, Laniolisip, the first and only FDA-approved treatment for APDS, a new ultra-rare immune disorder, where Anurag Ghannam will speak later on about. And you see the revenues are marching quite nicely. for an ultra-rare that launched to actually make $32 million in the first nine months of this year, which is, of course, an incredible growth versus last year is no mean feat. And of course, it's a new disease, it's ultra-rare, so we'll talk a little bit later about, by the way, how we find these patients and about what's going on with regards to the further exploitation of additional markets beyond the currently approved UK, US, and Israel. and, of course, the extension of the label with our pediatric patients. And then on the right-hand side, of course, we're very excited that we have been able to start a phase two in a way bigger indication for another primary immunodeficiency for lenialisept. And Anurag Rahman will speak to that later on as well. And last but not least, as you well know, we continue to be focusing on extending the portfolio further with an emphasis on in-licensing or acquiring clinical stage with proof of concept, clinical proof of concept opportunities and other rare diseases to basically further leverage our commercialization presence in the US, UK, Europe, and also the future markets like Japan and Australia. And when you read the next slide, you see the pipeline depicted in a visual. This is beginning to look like something, you know, we are in these markets with Ruknes and Joenja, and obviously you see there the graphic depiction of the further rollout of Laniolisip for APDS. And on the bottom, you see the Phase II stage, where Laniolisip is for the subsequent indication, the PIDs with immune dysregulation. So we're beginning to build that pipeline, and we're, of course, working very hard to extend it with additional assets that actually build that portfolio further and leverage our commercialization infrastructure and ability to do clinical trials and get regulatory approvals further. And before I hand over to Steven to talk about commercial results, I would like to show you one thing. That's the next slide. That is where you see an enormous, incredible number of products that are available for hereditary angioedema patients. So it's really good news for hereditary angioedema patients in the US, mainly we're talking about here, that there are so many products available. There's a lot of prophylactic therapies available here And there's a lot of, there is, of course, acute products available as well. There's, of course, one outstanding product, and that is Ruconest. That's the explanation why Ruconest is so strong, and Ruconest continues to be so strong, and will, in the future, continue to be so strong. Ruconest is a high-dose protein replacement therapy. And all the products that you see there that are being on the market and the main ones that are being used for the treatment of acute attacks for hereditary angiotema and breakthrough attacks are the ones that act on the calocrinine-independent pathway, bradykinin-calocrinine pathway. And a good example of that is Firzir Icatibab, which is by far the biggest volume of product used for the treatment of acute attacks. There is drawbacks associated with this approach. These products are not IV, which Brukinest is, and of course all our patients are very confident to do IV self-injections. But these products are, for instance, subcutaneous. And there is new, there's potential news on the horizon, oral opportunities that are being actually reviewed by the FDA and are in clinical stage to actually address the acute attacks. However, these oral opportunities are tested in patients that are actually responsive to Ferroxir. And that is a big, big difference here in terms of patients that we're serving with Ruconest. Every patient that uses Ruconest doesn't use Ruconest because they can, but because they must, i.e. they are not responding to Ferroxir EcatiBand therapy. Hence why you can see there's a clear segmentation in the market where Ruconest serves its own segment on the severe end of the market. And that is, of course, in relative terms, patients-wise, a relatively modest segment, but it is a segment where patients are very severely afflicted with the disease. A lot of the so-called type 3 patients, which are the more severely affected, and that have recently been developed and has recently been acknowledged and discovered, and where more and more are coming, that's exactly where Rucanest, with its unique mode of action, i.e. blocks all the pathways serves the purpose and will continue to serve the purpose and serves that unique segment of patients. That is why we are confident and we see the results of that after all those years in the market, more than 10 years in the U.S. market, by Rucanest continued and continues and will continue to be the mainstay under our company, delivering those cash flows which enable us to confidently develop the further growth potential of the company towards the future. Having said that, I would now like to hand over to Stephen to take us through the commercial aspects of the business and the results. Stephen, over to you, please.

Thank you, Simon. Morning, everybody. If you could move to the next slide, please. Thank you. I think Simon did a really nice job of describing there our patient population and why Ruconest is used. And that is, of course, as you can see on this slide, why Ruconest is the second most prescribed acute product in the U.S. market. That talks very much to the features there. It's an IV. It gives a bolus of C1, so it's highly effective. It's very quick-acting. And as Simon said, that's really critical for the patient population we serve that has a more severe course of HAE that doesn't respond well to the alternative therapies. It's also important to remember that in an environment where current or future options may not serve that severely affected population as well, as to why Rucanus will continue to endure and why we continue to be confident in the Rucanus. growth story in the U.S. We also continue to grow our prescriber base, and that's important to mention. We have 57 new prescribers this year, and we're heading towards 800 in total. And that perhaps also – that success is also best demonstrated through our leading and lagging metrics, all of which experienced double-digit growth this year, including new enrollments, which gained momentum. In fact, we had over 100 in Q3, and that's up 25% versus prior year. And all of this translates into around 12% revenue growth for the year so far, which is outperforming last year where we were at 10% revenue growth. So next slide, please. So moving to the Joe Angel launch, we achieved, as you know, a strong initial start to our US launch, and that growth steadied, which was expected as we come towards our next pending big inflection points. Over the 18 months since launch, We found approximately 50% of the known patients, which the prevalence suggests exists, and of those, 25% are pediatric, so they'll begin therapy when we get that indication. Of the eligible patients, the eligible adult patients, we have 93 on paid therapy, and we have five pending already in this quarter, pending insurance authorization, which has been going very, very smoothly. And as we continue to find patients and process the adult patients not on therapy, it's important to note that our next significant growth opportunities or reflection points will be the launch in the UK where Joe and you was recently approved, the potential influx of VUS patients in 2025, and then those pediatric patients I mentioned in 2026. So while we had initial strong growth, which is steadied in that adult population that we can serve, we have those three big inflection points coming, along with global launches beyond that. So for patients outside of the US, and you can see here that it's approaching 900, it's important that many of those are in various of our access programs, over 20% in fact. And as we roll out approvals and launches globally, they'll become eligible for reimbursed treatment. So all of that translates so far to $32 million in sales year to date. And then as the final section of this slide shows, we also continue to drive development of growth opportunities outside of APDS markets with our teams working hard on life cycle management opportunities for Leni-Ovisib, the molecule, outside of APDS. And they have significant potential value to our patients and our customers. So with that, I'd like to hand over to our CMO, Anurag Rowland, to talk more about those opportunities.